Pipeline Prospector March 2024: FDA approves pathbreaking NASH drug from Madrigal, two meds for PAH
March was clearly a month of drug approvals, as the US Food and Drug Administration (FDA) went on an overdrive, green-lighting several therapies before the close of the first quarter (Q1 2024). Amongst them was the first drug to treat non-alcoholic steatohepatitis (NASH) and a breakthrough therapy that treats pulmonary arterial hypertension (PAH).However, the buoyancy in drug approvals didn’t play out in the stock markets and most pharma indices witnessed a marginal dip. The Nasdaq Biotechnology index (NBI) fell marginally (-0.43 percent) to 4,429.97 from 4,449. The SPDR S&P Biotech ETF index (XBI) dropped 4.6 percent to 94.89 after ending February at 99.44. And the S&P Biotechnology Select Industry index (SPSIBI) was down by 3.4 percent from 7,662.14 to 7,402.47 in March.In deals, AstraZeneca bought two companies, shoring up its cancer and rare disease pipelines. The Anglo-Swedish drugmaker first bought France’s Amolyt, which focuses on rare endocrine diseases, for a total of US$ 1.05 billion and then acquired next-generation cancer drugmaker Fusion Pharmaceuticals for around US$ 2 billion. AstraZeneca’s stock was up 4.6 percent in March. Novo Nordisk (stock up 4.8 percent) agreed to acquire Cardior Pharmaceuticals for up to US$ 1.1 billion in order to boost its pipeline for cardiovascular diseases.Access the Pipeline Prospector Dashboard for March 2024 Newsmakers (Free Excel)FDA okays first drug for NASH; Wegovy approved as med to reduce heart risksFor quite some time, the drug development field for the liver condition non-alcoholic steatohepatitis (NASH) has been a graveyard for failed programs. In March, the field celebrated a hurrah moment when Madrigal Pharmaceuticals (stock up 6 percent) won the race to have the first NASH treatment approved by the FDA. Madrigal’s oral drug Rezdiffra (resmetirom) treats adults with NASH, a disease that causes histologic liver damage and occurs in patients who are not alcoholics and are often obese or have type 2 diabetes. The approval has opened a multi-billion dollar opportunity for Madrigal.The GLP-1 agonist drugs that are used to treat diabetes and obesity have shown heart-related benefits too in clinical trials. Last month, FDA approved Novo Nordisk’s Wegovy (semaglutide) to reduce the risk of cardiovascular death, heart attack, and stroke in obese or overweight adults with cardiovascular disease (CVD). The move makes Wegovy the first weight-loss medication that is also approved to help prevent life-threatening cardiovascular events. The approval was hailed as a major advancement in public health.Another significant FDA approval was granted to Akebia’s anemia drug, vadadustat. After being turned down in March 2022, Akebia has now been approved to treat anemia caused by chronic kidney disease (CKD) in dialysis patients.Access the Pipeline Prospector Dashboard for March 2024 Newsmakers (Free Excel) FDA okays two meds for PAH; Italfarmaco’s Duvyzat approved for DMDFDA approved a breakthrough therapy from Merck, known as Winrevair (sotatercept-csrk), to treat adults with hypertension that is caused by constriction of the arteries in the lungs, known as pulmonary arterial hypertension (PAH). The agency also approved Johnson & Johnson’s Opsynvi – a single-tablet combination of macitentan and tadalafil – for the chronic treatment of adults with PAH. With this approval, Opsynvi became the only once-daily combination therapy for PAH.Apart from PAH, there was another significant approval for hypertension. FDA okayed Idorsia’s once-daily treatment Tryvio (aprocitentan) in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not able to adequately control their BP on other drugs. Idorsia’s stock was up 50 percent in March. Tryvio is the first oral anti-hypertensive therapy, which works via a new therapeutic pathway, to be approved in almost 40 years.The US agency also approved Italfarmaco Group’s oral medication Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. Duvyzat is the first nonsteroidal drug approved to treat patients with all variants of DMD, a genetic disorder characterized by progressive muscle degeneration.Orchard Therapeutics’ Lenmeldy was also greenlit by the FDA as the first gene therapy in the US for a debilitating and rare pediatric disorder, known as metachromatic leukodystrophy (MLD).Access the Pipeline Prospector Dashboard for March 2024 Newsmakers (Free Excel) Breyanzi okayed for complex leukemia; Tevimbra approved for esophageal cancerAmong cancer treatments, FDA granted an accelerated approval to BMS’ Breyanzi, making it the first and only CAR-T cell therapy for adults with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). BMS’ stock was up 6 percent in March.BeiGene’s PD-1 blocker Tevimbra (tislelizumab) finally got the go ahead from the FDA as a treatment for adult patients with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic chemotherapy that did not include a PD-(L)1 inhibitor.FDA also granted accelerated approval to Takeda’s Iclusig (ponatinib) to be used with chemotherapy for newly diagnosed patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), a type of blood cancer of the bone marrow and blood.Access the Pipeline Prospector Dashboard for March 2024 Newsmakers (Free Excel) Pfizer-Takeda’s Adcetris posts trial win in DLBCL; FDA defers donanemab approvalIn clinical trials, there was some positive news from a phase 3 trial on Pfizer and Takeda’s drug Adcetris, which is known as a standard of care in classical Hodgkin lymphoma. The med has now shown to be efficacious in diffuse large B-cell lymphoma (DLBCL), a common type of non-Hodgkin lymphoma, when used in combination with two other drugs — lenalidomide and rituximab.In a late-stage trial, Novo Nordisk’s broadly used blockbuster diabetes drug Ozempic (semaglutide) slashed the risk of kidney disease progression and death from cardiovascular or kidney complications by 24 percent in diabetic patients with CKD. In negative news from the trials, the much anticipated approval of Eli Lilly’s donanemab scheduled for March was deferred as FDA opted to convene a panel of independent experts to assess the drug’s safety and efficacy. Similarly, Amylyx’s Relyvrio (sodium phenylbutyrate and taurursodiol), a promising investigational treatment for amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, failed to demonstrate it works better than a placebo in a 48-week long trial. ALS is a fatal motor neuron disease characterized by progressive degeneration of nerve cells.Access the Pipeline Prospector Dashboard for March 2024 Newsmakers (Free Excel) Our viewWith so many drug approvals, March was a good month for the biopharma industry. While it may look like the markets didn’t cheer the approvals, all the three indices — the NBI, XBI, and SPSIBI — ended the quarter in the green, growing 2.1 percent, 7.3 percent, and 6.4 percent, respectively, between January 2 and March 28. As we enter the second quarter of 2024, we hope the buoyancy in drug approvals and the indices is maintained.Access the Pipeline Prospector Dashboard for March 2024 Newsmakers (Free Excel) 

Impressions: 655

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PharmaCompass.com
04 Apr 2024
Pipeline Prospector Feb 2024: Novo’s parent buys Catalent for US$ 16.5 bn, FDA okays Iovance’s cell therapy
February was a good month for the pharma sector, complete with some important deals, successes from clinical trials and healthy fourth quarter (Q4) results announced by some drugmakers. The market cheered the good news — the Nasdaq Biotechnology index gained 1 percent from 4,385.06 to 4,428.56, the SPDR S&P Biotech ETF index spiked 12 percent to 98.42 after ending January at 87.43. The S&P Biotechnology Select Industry Index (SPSIBI) soared 16 percent from 6,815.3 to 7,896.47 in February.The month saw some big acquisitions. Novo Holdings, the parent company of Novo Nordisk, acquired contract development and manufacturing organization (CDMO) Catalent for US$ 16.5 billion. Novo Holdings plans to sell three of Catalent’s “fill-finish” sites to Novo Nordisk for US$ 11 billion. Novo’s game plan hasn’t gone down well with its arch rival in the diabetes and obesity segment Eli Lilly. The Indianapolis-headquartered drugmaker has called for a scrutiny of the deal as it relies on Catalent for some of its diabetes and obesity drugs that compete with Novo’s meds.Both Novo and Lilly have been struggling to meet the demand for their weight loss meds. With the acquisition of this key CDMO, Novo has scored an edge over competition.Meanwhile, Gilead boosted its liver portfolio with the US$ 4.3 billion acquisition of CymaBay Therapeutics. And Novartis announced the acquisition of cancer treatment developer MorphoSys for € 2.7 billion (US$ 2.9 billion). With this buyout, Novartis gets a late-stage, rare bone-marrow cancer treatment — pelabresib — that holds plenty of promise.Several drugmakers announced their Q4 results last month. Notable amongst them was Eli Lilly, as it posted a 28 percent increase in revenues — from US$ 7.3 billion in Q4 2022 to US$ 9.4 billion led by its new products Mounjaro and Zepbound. GSK touted the “exceptional launch of Arexvy”, the world's first RSV vaccine, as it brought in sales of £ 1.2 billion (US$ 1.52 billion) in 2023. And Merck’s Keytruda saw a 19 percent increase in its sales in 2023 — at US$ 25 billion.Access the Pipeline Prospector Dashboard for February 2024 Newsmakers (Free Excel)FDA okays Iovance’s T-cell therapy; Roche’s asthma med okayed for food allergiesThe US Food and Drug Administration (FDA) granted accelerated approval to Iovance Biotherapeutics’ cell therapy — Amtagvi (lifileucel) — for treating the deadliest form of skin cancer. Amtagvi is the first and only one-time, individualized T cell therapy to receive FDA approval for a solid tumor cancer. Iovance hopes to start reporting noteworthy revenue in the second quarter of 2024, despite the fact that Amtagvi comes with a boxed warning (the highest safety warning) for treatment-related mortality. FDA also approved Takeda’s Eohilia as the first oral treatment for an allergic inflammation of the esophagus. Eohilia (budesonide oral suspension) was on course to becoming the first treatment for eosinophilic esophagitis (EoE) until it was rejected by the FDA in December 2021.In January, Sanofi and Regeneron’s jab Dupixent had pipped Eohilia to become the first FDA drug approved for EoE. The chronic condition can make it very hard for patients to consume food. Dupixent (dupilumab) is injected weekly or biweekly and two pens cost US$ 3,803.20. In contrast, Takeda’s drug is taken twice daily and costs US$ 1,875 per month.Roche and Novartis’ asthma drug Xolair (omalizumab) became the first FDA-approved med to help people with food allergies avoid severe reactions following accidental exposure. This opens the drug to around 17 million people in the US whose allergies can potentially be deadly.Access the Pipeline Prospector Dashboard for February 2024 Newsmakers (Free Excel) Boehringer’s obesity candidate, Lilly’s tirzepatide show promise in trials for MASHBoehringer Ingelheim announced promising phase 2 trial data for its experimental obesity drug survodutide in treating metabolic dysfunction-associated steatohepatitis (MASH), a liver disease. The drug is being developed in collaboration with Zealand Pharma. It had received a fast-track designation from the FDA in June 2021. MASH causes histologic liver damage and occurs in patients who are not alcoholics and who are often obese or have type 2 diabetes.The trial met its primary endpoint with survodutide reaching a biopsy-proven improvement in MASH after 48 weeks. Survodutide also met all secondary endpoints, including a statistically significant improvement in liver fibrosis. The companies said this potentially positions it as a “best-in-class” treatment. The drug belongs to the glucagon-like peptide 1 (GLP-1) class of drugs.In another trial, Eli Lilly announced positive results from a phase 2 study of tirzepatide in adults with MASH — 74 percent of participants were cured of MASH and their fibrosis also did not worsen at 52 weeks (as compared to nearly 13 percent for participants in the placebo group).Access the Pipeline Prospector Dashboard for February 2024 Newsmakers (Free Excel) J&J’s autoimmune drug posts trial gains; Sanofi’s frexalimab shows benefit in MSOne of Johnson & Johnson’s most watched experimental drugs — nipocalimab — posted positive results in trials for the treatment of two autoimmune disorders. In a phase 3 study, nipocalimab significantly reduced symptoms of generalized myasthenia gravis (GMG). And, in a phase 2 study, it helped reduce the severity of Sjögren’s disease (SjD). GMG weakens the skeletal muscles and particularly affects control of the limbs, throat, mouth, and eyes. SjD is an immune disorder that is identified by symptoms like dry eyes and dry mouth. J&J expects over US$ 5 billion from this drug. In a phase 2 trial, Sanofi’s frexalimab significantly slowed disease activity in people with relapsing multiple sclerosis (MS). In December last year, the French multinational had named frexalimab as one of three “pipeline-in-a-product” assets with the potential of generating annual peak sales in excess of €5 billion (US$ 5.4 billion). The phase 2 results give credence to those expectations.In trial failures, Gilead (stock down 8 percent) said the FDA has paused all trials of its drug magrolimab in the treatment of two blood cancers – acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) – because of increased risk of patient death (when used in combination with azacitidine plus venetoclax). Magrolimab was the lead candidate of Gilead’s US$ 4.9 billion acquisition of Forty Seven in 2020.Access the Pipeline Prospector Dashboard for February 2024 Newsmakers (Free Excel) Our viewThe year has gotten off to a good start with drugmakers posting healthy Q4 results and the indices showing some positive growth month-on-month. Several drugmakers have entered 2024 with a simplified business model and an increased strategic focus on R&D. Given the favorable demand, we are hopeful of a better year for the industry, as opposed to the volatility of the last two years.Access the Pipeline Prospector Dashboard for February 2024 Newsmakers (Free Excel) 

Impressions: 1702

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PharmaCompass.com
07 Mar 2024
Pipeline Prospector Jan 2024: Vertex’s non-opioid painkiller succeeds in trials;  Sanofi buys Inhibrx for US$ 2.2 bn
The New Year got off to a stable start, with some good news trickling in from clinical trials and Big Pharma announcing a few acquisitions at the annual JP Morgan healthcare meet.The benchmark index S&P 500 has been on a green streak, growing 15.33 percent since October. In comparison, pharma indices underperformed. The Nasdaq Biotechnology index gained 1 percent in January to 4385.06, mirroring the 1 percent it had gained in December 2023. The SPDR S&P Biotech ETF index lost 1 percent to end January at 87.43 after soaring 18 percent to 89.29 in December. And, the S&P Biotechnology Select Industry Index (SPSIBI) dropped 3 percent in January to 6813.77, after spiking 8 percent in December.During the month, Sanofi acquired rare disease drugmaker Inhibrx for about US$ 2.2 billion and J&J acquired Ambrx for US$ 2 billion to enter the antibody-drug conjugates (ADC) space. ADCs are biopharmaceuticals designed as a targeted therapy for treating cancer.Cell therapies have been facing a rough period with narrower funding options that have forced many startups in the biotech sector to shut shop. The field received a further blow when the US Food and Drug Administration (FDA) mandated “black box” warnings — the highest safety warnings — on six major CAR-T cancer therapies over the risk of developing secondary cancer. However, the regulator is also helping out the sector and has finalized guidance for companies and academic researchers working on CAR-T cell therapies.Access the Pipeline Prospector Dashboard for January 2024 Newsmakers (Free Excel)Vertex’s non-opioid painkiller scores trial wins; Lilly’s therapy cures deafnessVertex Pharmaceuticals’ non-opioid painkiller VX-548 met its primary goals in two late-stage trials, marking a major development in a decades-long effort to have an alternative to opioids. The experimental non-opioid painkiller reduced acute, post-surgical pain. It was found to be safe and did the job without the risk of addiction. Vertex plans to file for FDA approval by mid-2024. Analysts estimate VX-548 to bring in peak sales of US$ 5 billion, if approved.There was good news from clinical trials for those with deafness. In an early-to-mid-stage trial, an 11-year-old boy on Eli Lilly’s investigational gene therapy AK-OTOF, could hear within 30 days of a single administration. Similarly, an experimental gene therapy being developed by a Chinese drugmaker Refreshgene Therapeutics also restored hearing in five out of six children with congenital deafness. Both treatments worked on profound deafness caused by mutations of the OTOF (otoferlin) gene. Vera Therapeutics (stock up 138 percent), a clinical-stage biotechnology company, announced positive 72-week data from a phase 2b clinical trial of atacicept in participants with IgA nephropathy (IgAN), also known as Berger’s disease. IgAN is a serious and progressive autoimmune disease of the kidney, for which there remains a high unmet medical need. Vera holds an exclusive worldwide license from Merck KGaA for the development and commercialization of atacicept in all indications.Meanwhile, Regenxbio touted positive interim data from a mid-stage trial for its experimental treatment for wet age-related macular degeneration (wet AMD) that uses a novel delivery, known as suprachoroidal delivery. Regenxbio has developed the drug along with AbbVie.Access the Pipeline Prospector Dashboard for January 2024 Newsmakers (Free Excel) FDA finalizes CAR-T guidance; hands Casgevy second nod for genetic blood disorderFDA has finalized guidance for companies and academic researchers working on CAR-T cell therapies. The guidance enlists the clinical, non-clinical and manufacturing expectations the agency has before a premarket approval (PMA) application is made.Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy has bagged a second FDA approval, this time for transfusion-dependent β-thalassemia, a rare genetic blood disorder that routinely requires blood transfusions.Across the pond, the European Commission granted marketing authorization to Stada and Alvotech’s Uzpruvo, a biosimilar to Johnson & Johnson’s Stelara (ustekinumab). Biosimilar competition in the €2.5 billion (US$ 2.7 billion) ustekinumab market in the EU could significantly expand patient access to this life-changing biologic therapy within gastroenterology, dermatology, and rheumatology, a press statement said. Meanwhile, Stelara sales grew by nearly 12 percent to about US$ 11 billion in 2023 compared to US$ 9.7 billion the year earlier.Access the Pipeline Prospector Dashboard for January 2024 Newsmakers (Free Excel) Sanofi buys Inhibrx for about US$ 2.2 bn; J&J enters ADC space with Ambrx dealSanofi has agreed to buy California-based clinical-stage biopharmaceutical company Inhibrx for about US$ 2.2 billion. The acquisition is aimed at acquiring the biopharma’s mid-stage experimental treatment INBRX-101, which will bolster the French drugmaker’s rare genetic disease portfolio. Inhibrx’s other investigational drugs will be spun off into a different company in which Sanofi will hold an 8 percent stake. INBRX-101 is a potential treatment for alpha-1 antitrypsin deficiency, a rare disease that causes the lung tissue to progressively deteriorate.Meanwhile, Johnson & Johnson announced the acquisition of ADC drug developer Ambrx Biopharma for about US$ 2 billion. This makes J&J the latest drugmaker to bet on ADCs after Pfizer, AbbVie and Merck.Access the Pipeline Prospector Dashboard for January 2024 Newsmakers (Free Excel) Novo sales soar 36% due to obesity drugs; Pfizer ekes out surprise Q4 2023 profit The month saw several drugmakers announce financial results for the full year 2023. Novo Nordisk saw sales rise 36 percent at constant exchange rates (CER) to DKK 232.3 billion (US$ 34.36 billion) in 2023 compared to DKK 177 billion (US$ 26.18 billion) in 2022. Ozempic (semaglutide) clocked sales of DKK 95.7 billion (US$ 13.77 billion) compared to DKK 59.6 billion (US$ 8.57 billion), a 66 percent rise. Novo’s stock was up 13 percent in January.Pfizer’s 2023 revenue fell 42 percent year-on-year to US$ 58.5 billion (from the record US$ 100 billion revenue it had posted in 2022) due to a sharp drop in Comirnaty and Paxlovid sales. Yet, Pfizer managed to eke out a profit of US$ 593 million in Q4, when analysts were expecting a US$ 1.1 billion loss. Keytruda raked in US$ 25 billion in 2023, a 19 percent increase over the US$ 21 billion posted in 2022, helping Merck achieve sales of US$ 60.1 billion.Access the Pipeline Prospector Dashboard for January 2024 Newsmakers (Free Excel) Our viewThe pharma landscape is clearly changing. As financial results for 2023 pour in, we know that our list of top drugmakers is going to look a lot different. We may see further shuffling in 2024, with companies shifting focus to new areas like obesity drugs, ADCs and rare diseases.The operating environment in 2024 faces “continued risk from geopolitical tension, domestic political uncertainty and heated campaign rhetoric, and increasing attention on regulatory enforcement around the world,” says a PwC report on the sector. Given this scenario, companies that are able to reinvent themself this year with new products and new strategies to fight the changing business and regulatory environment will emerge winners.Access the Pipeline Prospector Dashboard for January 2024 Newsmakers (Free Excel) 

Impressions: 1545

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#PipelineProspector by PharmaCompass
08 Feb 2024
Pipeline Prospector 2023 highlights: Obesity drugs perk up Lilly, Novo sales; ADCs spur dealmaking
The year 2023 was marked by volatility. The good news is that despite factors like inflation, interest rate hikes and the two ongoing wars, pharma indices ended the year in the green. Though growth was nowhere close to S&P 500’s remarkable 24 percent rise, the Nasdaq Biotechnology Index (NBI) gained a modest 3 percent and rose to 4,370.58, while the SPDR S&P Biotech ETF (XBI) grew 6 percent to 89.29 in 2023. And the S&P Biotechnology Select Industry Index (SPSIBI) ended the year higher by 8 percent, at 6,954.64. Many saw 2023 as the first year post the pandemic. Clearly, attention turned away from Covid-19 to handling one of the world’s biggest epidemics – obesity. Novo Nordisk and Eli Lilly outshone other drugmakers in 2023, as their newly launched weight management drugs became immensely popular. Falling under the class of drugs known as glucagon-like peptide 1 (GLP-1) agonists, these drugs were originally launched to improve blood sugar control in those with type 2 diabetes. The year also saw respiratory syncytial virus (RSV) vaccines hitting the markets. Another noteworthy debut was Casgevy, a therapy based on Nobel Prize-winning CRISPR technology that uses molecular “scissors” to trim faulty parts of “sickle” shaped cells that cause the highly debilitating sickle cell disease. The year’s biggest M&A deal was Pfizer’s acquisition of Seagen for US$ 43 billion. As the year drew to a close, we saw significant M&A activity, with Bristol Myers Squibb acquiring Karuna Therapeutics for US$ 14 billion and RayzeBio for US$ 4.1 billion.Access the Pipeline Prospector Dashboard for December 2023 Newsmakers (Free Excel)  Lilly, Novo Nordisk leap ahead with anti-obesity drugs; Roche, Astra join GLP-1 race Eli Lilly saw its sales rise by 17 percent in the first nine months of 2023, at US$ 24.77 billion, on the back of rising sales for Mounjaro (tirzepatide) of US$ 2.96 billion along with other diabetes and weight management drugs during the period. Novo’s sales rose 33 percent in the same period to DKK 166.4 billion (US$ 24.4 billion) from DKK 128.87 billion (US$ 18.9 billion) in the corresponding period of 2022. Wegovy (semaglutide) accounted for sales of DKK 21.73 billion (US$ 3.19 billion) compared to sales of just DKK 3.74 billion (US$ 0.55 billion) in 2022, a rise of 492 percent. Ozempic’s (semaglutide) sales rose to DKK 65.65 billion (US$ 9.63 billion) in the first nine months of 2023, compared to DKK 42.77 billion (US$ 6.03 billion) in 2022, a rise of 58 percent.  The anti-obesity market is projected to increase to US$ 100 billion by 2030. While existing players are busy expanding capacities for their GLP-1 drugs, more players are eager to enter this market to cash in on the opportunity. In December, Roche bought Carmot Therapeutics for US$ 2.7 billion, thereby getting its hand on CT-388, a promising once-a-week injection currently in mid-stage trials. Similarly, AstraZeneca entered into a potential US$ 2 billion deal with Eccogene for the experimental oral drug ECC5004 for obesity, type-2 diabetes, and other cardio-metabolic conditions. Merck’s GLP-1 strategy is to come up with treatments whose benefits go beyond weight loss. Its investigational GLP-1 drug efinopegdutide for non-alcoholic steatohepatitis (NASH) demonstrated compelling weight-loss benefits. This experimental drug has won the FDA’s Fast Track designation for treating NASH. Access the Pipeline Prospector Dashboard for December 2023 Newsmakers (Free Excel) Pfizer enters ADC space with US$ 43 bn Seagen buy; BMS buys Karuna for US$ 14 bn After GLP-1 drugs, antibody-drug conjugates (ADCs) have emerged as another big growth area. ADCs are designed as a targeted therapy for treating disease, and are being widely used for the management or treatment of cancer. Pfizer invested some of the cash it accumulated from its Covid sales to buy out Seagen for US$ 43 billion. The ADC space also saw AbbVie pick up ImmunoGen for US$ 10.1 billion and Merck sign a potential US$ 22 billion deal with Daiichi Sankyo. The other big deals of 2023 include BMS’ acquisition of Karuna Therapeutics for US$ 14 billion and Merck’s buyout of Prometheus and its potential best-in-class candidate to treat ulcerative colitis and Crohn’s disease for US$ 10.8 billion. Similarly, AbbVie acquired neuroscience drugmaker Cerevel Therapeutics for about US$ 8.7 billion in the hope of shoring up its revenues that have been hit by generic competition to its blockbuster Humira (adalimumab). Access the Pipeline Prospector Dashboard for December 2023 Newsmakers (Free Excel)  Pfizer, BioNTech, Moderna post lower revenues due to plummeting Covid-19 sales Pfizer reported a 42 percent drop in revenues in the first nine months of 2023 — at US$ 44.25 billion down from US$ 76.04 billion in the same period of 2022 — owing to disappointing sales of its Covid products. Comirnaty sales dropped 77 percent from US$ 26.48 billion to US$ 5.86 billion, and revenue from Paxlovid was down 73 percent at US$ 4.41 billion, from US$ 17.1 billion in 2022. Pfizer’s Comirnaty partner BioNTech was worse hit, and saw an 82 percent decline in total sales at € 2.34 billion (US$ 2.56 billion) compared to € 13.03 billion (US$ 14.25 billion) in the first three quarters of 2022. Meanwhile, Moderna’s sales plunged 71 percent in the same period to US$ 4.04 billion, compared to US$ 14.18 billion in 2022. Access the Pipeline Prospector Dashboard for December 2023 Newsmakers (Free Excel)  FDA okays gene therapies for sickle cell disease, RSV vaccines from GSK, Pfizer  The year saw the United Kingdom and the US approve the world’s first human gene therapy for sickle-cell disease (SCD). The US Food and Drug Administration (FDA) not only approved CRISPR Therapeutics and Vertex Pharmaceuticals’ Casgevy (exagamglogene autotemcel) for SCD, it also approved bluebird bio’s Lyfgenia (lovotibeglogene autotemcel) for the treatment of SCD in patients aged 12 and older who have a history of vaso-occlusive events (when sickled red blood cells block blood flow to the point that tissues become deprived of oxygen). In May, FDA approved GSK’s RSV vaccine Arexvy for people aged 60 and older. Arexvy is the first RSV vaccine to be approved in the US for the common condition that can be fatal for the elderly. Less than a month later, Pfizer’s RSV vaccine Abrysvo also got approved. In July, FDA approved Sanofi and AstraZeneca’s preventive RSV antibody therapy Beyfortus (nirsevimab-alip) for newborns and infants. RSV is the number one cause of infant hospitalization in the US. Access the Pipeline Prospector Dashboard for December 2023 Newsmakers (Free Excel)  Our view A strong portfolio and drug pipeline can help drugmakers sail through the toughest economic and regulatory environment. While it’s difficult to say how things will pan out in 2024, we do know that several pathbreaking drugs are coming up for approval by the FDA early this year, including Madrigal Pharmaceuticals’ resmetirom (the first treatment for NASH with liver fibrosis), Merck’s sotatercept (a treatment for pulmonary arterial hypertension) and, later, in September, Karuna Therapeutics’ drug to treat schizophrenia. In short, there is much to look forward to in 2024.Access the Pipeline Prospector Dashboard for December 2023 Newsmakers (Free Excel) 

Impressions: 1777

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#PipelineProspector by PharmaCompass
11 Jan 2024
Pipeline Prospector Nov 2023: Lilly, Novo post sharp rise in Q3 sales; AbbVie buys ImmunoGen for US$ 10.1 billion
Through much of 2023, markets remained volatile, with pharma indices managing to inch up only in some months. November bucked the trend — the Nasdaq Biotechnology Index (NBI) rose by 4 percent at 3,902.97 after an 8 percent drop in October. The SPDR S&P Biotech ETF index (XBI) spiked 12 percent to 75.52, compared to a drop of 12 percent in October. And the S&P Biotechnology Select Industry Index (SPSIBI) gained 12 percent to 5,882.09 after plunging 9 percent in October.The indices mirrored the bullish activity witnessed in the US stock markets, with the S&P 500 rebounding 8.92 percent in November. Importantly, healthcare stocks outperformed all 11 major sectors of the S&P 500.The month witnessed another significant deal in the antibody-drug conjugates (ADCs) space after Pfizer acquired Seagen in March for US$ 43 billion. As November drew to a close, AbbVie picked up ImmunoGen for US$ 10.1 billion, including its first-in-class ADC, Elahere, approved for platinum-resistant ovarian cancer (wherein patients relapse within six months of receiving platinum-based chemotherapy). ADCs are fast-growing, innovative biopharmaceutical products that target cancer cells, while protecting the healthy cells.In other big news, the United Kingdom became the first country to grant regulatory approval to a medical treatment involving the revolutionary CRISPR gene editing tool. And the month also saw several drugmakers announce their third quarter (Q3) results.Access the Pipeline Prospector Dashboard for November 2023 Newsmakers (Free Excel) Astra joins obesity med jamboree with Eccogene deal; Lilly, Novo ride GLP-1 waveNovember was a good month for Eli Lilly (stock up by 7 percent) as both the US Food and Drug Administration (FDA) and the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) approved its weight-loss injection Zepbound (tirzepatide). Moreover, the drugmaker’s Q3 revenue increased by an impressive 37 percent, driven by the growth of its GLP-1 diabetes treatment Mounjaro (tirzepatide) and breast cancer drug Verzenio (abemaciclib).The other drugmaker that rode on the popularity of GLP-1 meds is Novo Nordisk (up by 4 percent). The Danish drugmaker posted a rise in sales of 29 percent in the first nine months of 2023. Its diabetes and obesity care meds saw a gain of 36 percent, bringing in DKK 153.8 billion (US$ 20.6 billion) for Novo in the first nine months of 2023. Out of this, the GLP-1 meds (Saxenda, Ozempic, Rybelsus, Wegovy) contributed DKK 30.4 billion (or US$ 4.1 billion). The company also plans to expand its manufacturing capacities with an investment of over DKK 42 billion (US$ 6 billion) in Kalundborg (Denmark), and an additional investment of DKK 16 billion (US$ 2.3 billion) at its Chartres (France) facility to capitalize on the burgeoning market for weight loss drugs. Its weight loss med Wegovy has also shown heart protection benefits in a phase 3 trial.Not wanting to miss out on the bourgeoning market for weight loss drugs (estimated to reach an annual market size of US$ 100 billion by 2030), AstraZeneca (up by 2 percent) has entered into an exclusive license agreement worth US$ 2 billion with Chinese biopharma Eccogene for its investigational treatment ECC5004 for obesity, type-2 diabetes, and other cardio-metabolic conditions. The company has reported a 5 percent rise in its Q3 revenues (at US$ 11 billion), fueled by good performances in oncology, cardiovascular renal management, and rare diseases.Access the Pipeline Prospector Dashboard for November 2023 Newsmakers (Free Excel)  GSK’s RSV jab nears US$ 1 bn in sales; Novartis, Sanofi post positive trial updatesLaunched in early May 2023, GSK’s (stock up by 3 percent) RSV vaccine, Arexvy, has made an impressive debut. GSK’s Q3 results show that its total sales increased by 10 percent, fueled by a 33 percent surge in vaccine sales. This includes £0.8 billion (US$ 0.97 billion) from sales of its shingles vaccine (Shingrix) and £0.7 billion (US$ 0.85 billion) from sales of Arexvy.  In its first year of launch, Arexvy sales are poised to cross £1 billion (US$ 1.26 billion).In crucial phase 3 trials, Novartis’ (up by 4 percent) highly selective, oral Bruton’s tyrosine kinase (BTK) inhibitor remibrutinib showed clinically significant results in treating chronic spontaneous urticaria (CSU). Currently, H1-antihistamines are the only recourse for people plagued by chronic hives.Sanofi (up by 2 percent) also made trial gains as its blockbuster drug Dupixent (dupilumab) showed “overwhelming positive efficacy” in a second, large phase 3 trial undertaken to test it as a cure for chronic obstructive pulmonary disease (COPD). Sanofi now plans to seek FDA approval to expand its use for COPD. If approved, Dupixent will be the first biologic to treat the disease.Access the Pipeline Prospector Dashboard for November 2023 Newsmakers (Free Excel)  UK okays CRISPR therapy for thalassemia, Madrigal’s NASH drug posts trial gainsIn a world first, the UK’s MHRA has given the go-ahead for CRISPR Therapeutics (up by 61 percent) and Vertex Pharmaceuticals’ (down by 4 percent) gene therapy Casgevy for patients aged 12 and above to cure the genetic conditions — β-thalassemia and sickle-cell disease (SCD). This renders it the first treatment ever that uses the gene-editing tool CRISPR, whose inventors won the 2020 Nobel Prize.Large-cap Alnylam (up by 8 percent) achieved robust Q3 commercial results, reporting US$ 313 million in revenue, driven by the successful launch of Amvuttra, a rare heart disease drug.Mid-cap Madrigal (up by 53 percent) announced positive data from a late-stage trial demonstrating broad treatment effects of resmetirom on nonalcoholic steatohepatitis (NASH) with liver fibrosis. It is the only investigational therapy for NASH that has achieved both fibrosis improvement and NASH resolution primary endpoints in a phase 3 trial. Among small-cap companies, FDA approved SpringWorks Therapeutics’ (up by 42 percent) Ogsiveo (nirogacestat) for adults suffering from rare desmoid tumors, rendering it the first approved treatment for the non-cancerous condition.Among losers, Bayer (down by 22 percent) scrapped a significant late-stage trial assessing the efficacy of a new anti-clotting drug, asundexian, compared to Eliquis (apixaban) in patients with atrial fibrillation at risk of a stroke.  The drugmaker had hoped asundexian would generate revenues in excess of €5 billion (US$ 5.5 billion) and compensate for the drop in revenues of its blood thinner Xarelto, set to lose protection from key European patents in 2026. Bayer also voluntarily recalled a single lot of its cancer drug Vitrakvi due to microbial contamination.Access the Pipeline Prospector Dashboard for November 2023 Newsmakers (Free Excel)  Our viewIn the US, the Federal Reserve left interest rates unchanged in its meeting in November. This has raised investor expectations, and hopes of inflation cooling down this winter.However, given the rise in bankruptcy filings, layoffs, weaker availability of finance to small and medium-sized businesses and larger concerns of a slowdown in the global economy, we would exercise caution before putting inflationary and other economic concerns to rest.Access the Pipeline Prospector Dashboard for November 2023 Newsmakers (Free Excel)  

Impressions: 2214

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#PipelineProspector by PharmaCompass
07 Dec 2023
Pipeline Prospector Oct 2023: Disappointing Q3 results, attacks on Israel, Gaza drag pharma indices down further
October was a gloomy month that saw Palestinian militant group Hamas attack Israel in the first week, resulting in the killing of hundreds of innocent people. Israel retaliated, formally declaring a war on Hamas. Another raging war, after the ongoing Russia-Ukraine conflict, doesn’t augur well for the planet, let alone humanity, economies and industries.The pharma indices reacted to the bloodshed, as also to the disappointing third quarter (Q3) results posted by some drugmakers. The Nasdaq Biotechnology Index (NBI) was down 8 percent, at US$ 3,637.05 while the SPDR S&P Biotech ETF (XBI) was down 12 percent, at US$ 64.12, and the S&P Biotechnology Select Industry Index (SPSIBI) was down by 9 percent, at US$ 4,991.31.On the upside, the month saw several large deals. The biggest deal of the month was Roche’s acquisition of Telavant from Roivant for US$ 7.1 billion. BMS announced the acquisition of Mirati Therapeutics for US$ 4.8 billion. Through this acquisition, BMS will gain access to the US Food and Drug Administration (FDA) approved lung cancer drug Krazati (adagrasib). And Eli Lilly expanded its oncology capabilities by acquiring Point Biopharma for US$ 1.4 billion.Access the Pipeline Prospector Dashboard for October 2023 Newsmakers (Free Excel)Sanofi, Takeda cut 2023 profit forecasts; AbbVie, BMS post drop in Q3 salesSeveral drugmakers announced their Q3 2023 results last month. Sanofi (stock down by 15 percent in October) reported a 4.1 percent drop in Q3 sales, at €11.9 billion (US$ 12.5 billion). What was worse, the French drugmaker lost €20 billion (US$ 21 billion) in market capitalization soon after announcing these results as it had abandoned its 2025 profit target of 32 percent. Instead of chasing the lofty target, Sanofi has decided to focus on “long-term profitability”, and increase spending on immunology and inflammation drug development. Sales of Sanofi’s multiple sclerosis drug Aubagio dropped by a whopping 60.5 percent to €199 million (US$ 210.2 million) primarily due to competition from generics in the US market. Takeda (down by 7 percent) slashed its full-year profit forecast by 36 percent to 225 billion yen (US$ 1.5 billion) as it contends with disappointments in its development pipeline and the loss of patent protection in key products.BMS (down by 10 percent) reported Q3 revenue of US$ 11 billion, a 2 percent decrease from the same quarter last year, mainly due to 41 percent decline in sales of its cancer drug Revlimid.AbbVie (down by 5 percent) reported Q3 net revenues of US$ 13.92 billion, marking a 6 percent decrease over Q3 2022. This drop is attributed in part to the launch of Humira biosimilars, which have caused a 36.2 percent reduction in the immunology drug’s net revenues. Additionally, AbbVie’s blood cancer drug Imbruvica’s net revenues have dropped by 20 percent.Access the Pipeline Prospector Dashboard for October 2023 Newsmakers (Free Excel) Low Covid sales hit Pfizer, BioNTech, Roche stocks; Pfizer’s Q3 sales dip by 42%Pfizer (down by 9 percent) saw a 42 percent decline in its Q3 revenues — at US$ 13.23 billion — primarily due to lower sales of its Covid products. While sales of its mRNA vaccine Comirnaty fell by 70 percent (at US$ 1.3 billion), its Covid drug Paxlovid saw a 97 percent drop in sales over Q3 2022.Pfizer has significantly reduced its full-year revenue forecast and also plans to save US$ 3.5 billion through job cuts and cost reductions. Its partner for Comirnaty, BioNTech, is yet to announce its Q3 results, but its stock fell by 13 percent during October.Roche’s Q3 sales also slipped by 3 percent to CHF 14.3 billion (US$ 15.9 billion) owing to a slump in Covid sales. The Swiss drugmaker has predicted a decline in 2023 sales of Covid products at about CHF 4.5 billion (US$ 4.99 billion). Its previous estimate was CHF 5 billion (US$ 5.5 billion). Roche’s stock was down by 6 percent last month.Access the Pipeline Prospector Dashboard for October 2023 Newsmakers (Free Excel) Merck, Novo raise 2023 guidance; Ozempic, Wegovy boost Novo salesAmong the gainers were mega-cap drugmakers Merck (stock up by 1 percent in October) and Novo Nordisk (up by 4 percent). Merck has reported higher-than-expected third-quarter results on strong demand for its Covid-19 treatment, Lagevrio (molnupiravir), cancer med Keytruda and HPV vaccine Gardasil. As a result, Merck has revised its 2023 sales upwards, in the range of US$ 59.7 billion to US$ 60.2 billion (from US$ 58.6 billion to US$ 59.6 billion).Novo Nordisk’s popular diabetes med Ozempic and weight loss med Wegovy are boosting the company’s sales. Ergo, the company expects full-year sales to grow between 32 and 38 percent at constant exchange rates. Moreover, Novo has acquired ocedurenone, a drug in late-stage trial, from KBP Biosciences for up to US$ 1.3 billion. Ocedurenone is an oral treatment for uncontrolled hypertension with potential applications in cardiovascular and kidney disease.Access the Pipeline Prospector Dashboard for October 2023 Newsmakers (Free Excel) Astra, Ventyx stocks hit by disappointing trial data; Lilly, Vertex post gainsAstraZeneca’s stock was hit by disappointing results from a late-stage trial of its experimental lung cancer drug — datopotamab deruxtecan. Astra’s stock was down by 6 percent in October and its partner in developing the drug, Daiichi Sankyo’s stock fell by 4 percent.Similarly, Ventyx Biosciences (down by 56 percent) announced positive results from a phase 2 trial  in patients with moderate-to-severely active ulcerative colitis, but investors were unimpressed due to doubts about whether the candidate has an edge over rival Pfizer’s etrasimod and BMS’ Zeposia. Akero Therapeutics’ (down by 76 percent) nonalcoholic steatohepatitis (NASH) candidate, efruxifermin, failed mid-stage trial as it didn’t show any significant benefit for patients with cirrhosis caused by NASH, the most advanced and life-threatening stage of the fatty liver disease. Similarly, Sarepta Therapeutics’ treatment for Duchenne muscular dystrophy (a muscle-wasting disease) — Elevidys — failed a phase 3 study, taking its stock down by 44 percent in October.In a positive clinical trial news, Lilly’s Crohn’s disease experimental drug mirikizumab met the main goals of a phase 3 study. Similarly, Vertex (up by 4 percent) posted encouraging positive clinical trial results of VX-880 to treat type 1 diabetes.Meanwhile, FDA issued a complete response letter (CRL) to Alnylam Pharmaceuticals’ siRNA therapy Onpattro (patisiran) for the treatment of cardiomyopathy of transthyretin-mediated amyloidosis. It stated that Alnylam (down by 15 percent) has not provided enough evidence of the therapy’s benefit in the proposed indication.Access the Pipeline Prospector Dashboard for October 2023 Newsmakers (Free Excel) Our viewThe biopharma industry is eagerly awaiting the approval of several experimental drugs in Q4 2023, including Italfarmaco’s givinostat for Duchenne muscular dystrophy (DMD), and Valneva’s single-shot chikungunya vaccine VLA1553.We are already seeing the market inch upwards. Let’s hope the exciting pipeline and new drug approvals are able to pull pharma indices out of the doldrums.Access the Pipeline Prospector Dashboard for October 2023 Newsmakers (Free Excel) 

Impressions: 1887

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#PipelineProspector by PharmaCompass
02 Nov 2023
Pipeline Prospector Sept 2023: Pharma indices dip further; Merck, BioCardia hit by trial setbacks
Biotech indices underperformed in September. The downward trend witnessed in August got accelerated last month — the Nasdaq Biotechnology Index slipped by 5 percent, at US$ 3,953.65 in September. It was up by 1 percent, at US$ 4,150.80, in August. The SPDR S&P Biotech ETF index was down 10 percent in September, at US$ 73.02. It was down by 4 percent in August, at US$ 80.15. The S&P Biotechnology Select Industry Index (SPSIBI) was down by 10 percent, at US$ 5,694.34, as against a drop of 5 percent, at US$ 6,161.27, in August.Stocks for companies such as Roche, Merck, and Alnylam fell due to news of adverse US Food and Drug Administration (FDA) decisions and failures in clinical trials. Coupled with this was the continued shrinking of demand for Covid vaccines and therapies, and this led to stock market losses for Pfizer (stock down 8 percent), Moderna ( stock down 9 percent) and BioNtech (stock down 10 percent).September was also a lackluster month for deals — no deal above US$ 1 billion got reported. Among the sub-US$ 1 billion deals, Alfasigma’s acquisition of US liver disease drugmaker Intercept Pharmaceuticals for up to US$ 800 million was of significance. This buyout will expand Alfasigma’s range of treatments for liver diseases and digestive system disorders.Access the Pipeline Prospector Dashboard for September 2023 Newsmakers (Free Excel)Delayed FDA decision drags Roche’s stock; Novo hit by quality lapses at US plantIn early September, FDA delayed its decision on the subcutaneous version of Roche’s cancer immunotherapy Tecentriq. Roche needs to update Tecentriq’s manufacturing processes as per FDA’s evolving requirements, and the Swiss drugmaker now expects to receive an approval in 2024. Overall, Roche’s stock was down 8 percent last month. Alnylam’s stock tumbled by 13 percent after FDA’s advisory committee recommended its RNA interference therapy, Onpattro, albeit with caveats. It voted nine to three, saying the benefits of Alnylam’s Onpattro outweigh the risks for patients with transthyretin amyloid cardiomyopathy. However, the committee suggested that the treatment will only be useful to patients who worsen while receiving Pfizer’s rare and fatal heart disease drug, tafamidis. Though patients treated with Onpattro showed improvements in symptoms, Pfizer’s drug has a proven benefit on mortality.In other regulatory news, FDA issued a complete response letter (CRL) to ARS Pharmaceuticals (stock down 50 percent) for Neffy, an epinephrine nasal spray, to treat allergic reactions.Novo Nordisk’s stock fell after reports that back in May, FDA had found lapses in quality control to prevent microbial contamination at its Clayton, North Carolina plant. The Clayton facility produces semaglutide, the active ingredient used in its blockbuster weight loss and diabetes drugs Wegovy and Ozempic. Overall, Novo’s stock was down 2 percent in September.Bristol Myers Squibb faced a new lawsuit filed by Blue Cross and Blue Shield of Louisiana that accused it of using fraudulent patents and other illegal tactics to maintain its monopoly on blockbuster blood cancer drug Pomalyst for years. Its partner 2seventy Bio said in a statement that it anticipates a decline in Abecma sales in the third quarter. “The US revenue for Abecma could be lower than the US$ 470 to US$ 570 million range previously projected,” 2seventy Bio said in a statement. Overall, BMS’ stock fell by 6 percent last month.Access the Pipeline Prospector Dashboard for September 2023 Newsmakers (Free Excel) Merck, Acelyrin, BioCardia stocks take a hit due to failures in clinical trialsA combination of Merck (stock down 5 percent) and Eisai’s (stock down 13 percent) drugs Keytruda and Lenvima witnessed another loss as it failed in a phase 3 clinical trial for non-small cell lung cancer (NSCLC). The combo failed to meet both the primary endpoints of overall survival and progression-free survival in two separate lung cancer studies. Earlier in 2023, the same combo had failed in melanoma, colorectal cancer and head and neck squamous cell carcinoma.Among small-cap companies, Acelyrin’s experimental lead drug izokibep failed to meet the main goal of a mid-to-late stage trial to show statistically significant reduction in abscesses and inflammatory nodules compared to placebo in patients with hidradenitis suppurativa (HS), a chronic inflammatory skin condition. Its stock was down 64 percent in September. Similarly, BioCardia’s stock took a 100 percent dive as the company’s CardiAMP cell therapy failed in a phase 3 heart failure trial.Access the Pipeline Prospector Dashboard for September 2023 Newsmakers (Free Excel) GSK gains post cancer drug approval; AbbVie, Novartis post trial-related gainsCompanies like GSK, AbbVie (stock up 3 percent), Novartis (stock up 3 percent), and Immunovant saw their share prices move upwards during September. GSK  (stock up 6 percent) gained as its oral cancer med, momelotinib, was approved by the FDA. It is the first and only treatment for a type of bone marrow cancer known as myelofibrosis patients with anaemia.There was more good news from GSK — the European Commission authorized GSK’s HIV-focused unit ViiV Healthcare’s cabotegravir, a pre-exposure prophylaxis to reduce the risk of sexually-acquired HIV-1 infection in high-risk adults and adolescents.AbbVie had a good month. One, AbbVie’s autoimmune medicine Skyrizi is poised to give Johnson & Johnson’s (stock down 2 percent) blockbuster arthritis therapy Stelara a run for its money. In a late-stage head-to-head study in Crohn’s disease, Skyrizi matched up to Stelara in terms of inducing clinical remission in patients with active, moderate to severe Crohn’s disease who had failed on traditional anti-TNF agents. Two, AbbVie is set to receive US$ 480.6 million in damages from Takeda over a supply agreement dispute for Lupron, as per a court verdict. Novartis announced data from a late-stage trial that showed how health-related quality of life scores had increased in patients treated with Kisqali, its cancer drug, when used in combination with endocrine therapy (ET) compared to those treated with ET alone. This finding suggested that Kisqali reduced the risk of cancer recurrence without adding to the burden of care.Meanwhile, Novartis confirmed plans for Sandoz spin-off on October 4. Sandoz also announced partnership with Samsung Bioepis to develop and commercialize a Stelara biosimilar.Mid-cap company Immunovant saw its stock surge by 71 percent last month after early-stage data from its experimental antibody IMVT-1402 to treat autoimmune diseases exceeded analysts’ expectations. The drug is set to compete with treatments from companies like J&J and UCB, all of which are in the development stage.Access the Pipeline Prospector Dashboard for September 2023 Newsmakers (Free Excel) Our viewSeptember was the worst month of 2023 with S&P 500 dropping by 5 percent and the Nasdaq falling by 6 percent. Overall, the market was reacting to several concerns in the US economy such as rising interest rates, a slowing down of the housing market and signs that households are tightening their belts.Given these concerns, it’s unlikely that the coming months will be dramatically different for the biopharma industry. Let’s hope the tide turns in 2024.Access the Pipeline Prospector Dashboard for September 2023 Newsmakers (Free Excel)  

Impressions: 1944

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#PipelineProspector by PharmaCompass
05 Oct 2023
Pipeline Prospector Aug 2023: Trial gains, Q2 results push up Lilly, Novo stocks even as indices dip
After a buoyant July, when all the three pharma indices inched up by 2 percent, August proved to be a downer. While the Nasdaq Biotechnology index (NBI) was up 1 percent, at US$ 4,150.80 (against US$ 4,129.98 in July), the SPDR S&P Biotech ETF index was down by 4 percent, at US$ 80.15 (against US$ 84.29 in July) and the S&P Biotechnology Select Industry Index (SPSIBI) was down by 5 percent, at US$ 6,161.27 (against US$ 6,544.43 in July).Many drugmakers, such as Eli Lilly, Novo Nordisk, Amgen, Regeneron, Pfizer and Bayer, announced their Q2 results last month.August was a lackluster month for deals. Only one deal over US$ 1 billion got reported last month — Novo Nordisk’s acquisition of Inversago Pharma, a privately held obesity drugmaker, for up to US$ 1.08 billion. This buyout will broaden Novo’s blockbuster weight loss portfolio.Access the Pipeline Prospector Dashboard for August 2023 Newsmakers (Free Excel) Lilly, Novo, Amgen, Regeneron post stock market gains due to upbeat Q2 resultsSeveral drugmakers posted an increase in Q2 revenues. Eli Lilly (stock up 22 percent in August) posted a 28 percent increase in its revenues for the quarter — at US$ 8.31 billion, up from US$ 6.48 billion in Q2 2022. This growth was driven by drugs such as Mounjaro (a diabetes med) that brought in US$ 979.7 million, Verzenio (a breast cancer drug) whose sales increased 57 percent at US$  926.8 million, Jardiance (a diabetes med) with revenues of US$ 668.3 million and psoriasis therapy Taltz that brought in US$ 703.9 million.Lilly posted some gains in clinical trials as well. Its oncology drug Retevmo (selpercatinib) demonstrated superior progression-free survival in phase 3 clinical trial compared to other approved multikinase inhibitors as an initial treatment for patients with a type of advanced or metastatic Rearranged during Transfection (RET) medullary thyroid cancer.Novo Nordisk (up 15 percent) posted sales growth of 30 percent on a constant exchange rates (CER) basis in the first half of 2023 to DKK 107.66 billion (US$ 15.49 billion). There was much good news on its weight loss med Wegovy (semaglutide 2.4 mg) — it met its primary goal of preventing major cardiovascular events by reporting a 20 percent drop in the risk of cardiovascular death, heart attack, or stroke in overweight and obese patients. In another phase 3 study, Wegovy reduced symptoms and physical limitations in patients with obesity and heart failure with preserved ejection fraction as compared to placebo.Amgen (up 10 percent) reported strong Q2 results, with a 6 percent increase in revenue — at US$  7.0 billion, compared to US$ 6.6 billion in Q2 2022. The growth was driven by its osteoporosis drug Evenity (US$ 281 million), leukemia drug Blincyto (US$ 206 million), cholesterol-lowering medicine Repatha (US$ 424 million) and Humira biosimilar Amjevita (US$  150 million).Another gainer was Regeneron (stock up 12 percent) —  its Q2 revenues increased by 11 percent over Q2 2022 to touch US$ 3.16 billion. Moreover, the company secured two FDA approvals last month. One, its drug Veopoz (pozelimab) bagged approval to treat an ultra rare disease, known as CHAPLE disease, in both adults and pediatric patients. Two, FDA okayed Regeneron’s Eylea HD (aflibercept) injection 8 mg — a higher dose version of Eylea 2 mg. Developed in collaboration with Bayer, Eylea HD is the first and only treatment approved in wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR) for immediate dosing at eight-week and up to 16-week intervals following three initial monthly doses.Gilead (stock up 1 percent) saw its Q2 revenue increase by 5 percent — to US$ 6.6 billion, as opposed to US$ 6.3 billion in Q2 2022. Growth was driven by its HIV drug portfolio that brought in US$  4.6 billion.Access the Pipeline Prospector Dashboard for August 2023 Newsmakers (Free Excel)  Pfizer reports 54% drop in Q2 revenues; BMS, Astra resolve patent lawsuitsAmong the big stock market losers in August were Pfizer (-3 percent), Roche (-5 percent), AstraZeneca (-4 percent) and Bayer (-6 percent). Pfizer reported a 54 percent drop in its Q2 revenues, at US$ 12.73 billion against US$  27.74 billion in Q2 2022, owing to reduced sales of its Covid-19 products Comirnaty and Paxlovid.But negative news ran deeper than just the Q2 results. The drug behemoth removed several phase 1 and mid-stage candidates from its pipeline, including a phase 1 respiratory syncytial virus (RSV) infection candidate, two phase 2 assets including an oral obesity and diabetes candidate and a  pneumococcal vaccine, along with an atopic dermatitis drug. In clinical trials, a heart drug from BridgeBio — acoramidis — showed better results than Pfizer’s approved drug Vyndamax (tafamidis) in transthyretin amyloid cardiomyopathy, a rare heart disease. To make matters worse, GSK sued Pfizer in a US court alleging that Pfizer’s RSV vaccine Abrysvo violates GSK’s patent rights in its rival RSV shot Arexvy.However, there was some good news too — FDA granted accelerated approval to Pfizer’s Elrexfio (elranatamab) for the treatment of relapsed or refractory multiple myeloma and approved its RSV vaccine Abrysvo for use in infants through active immunization of pregnant women.Bayer’s group turnover was down 8.2 percent to €11.04 billion (US$  11.94 billion) in Q2 2023, from US$ 13.6 billion in Q2 2022. The group’s pharmaceutical sales were down from €4.81 billion (US$ 5.16 billion) in Q2 2022 to €4.55 billion (US$ 4.88 billion) in Q2 2023. In March 2022, Bristol Myers Squibb (BMS) had sued AstraZeneca over infringement of Opdivo patents. In January this year, BMS sued Astra yet again for infringing on its Yervoy patents. In three separate lawsuits, BMS had complained that Astra’s drugs, Imfinzi and Imjudo, infringed patents on BMS’ therapies, Opdivo and Yervoy.But last month, BMS and Astra resolved patent infringement lawsuits. According to news reports, AstraZeneca has agreed to pay BMS and Ono Pharmaceutical US$ 510 million to resolve these lawsuits. Astra’s stock was down 4 percent in August.However, there was some positive news for AstraZeneca — the US Centers for Disease Control and Prevention (CDC) advisory committee on immunization practices unanimously recommended the use of Sanofi and Astra’s antibody, nirsevimab, to prevent RSV in infants and toddlers.Access the Pipeline Prospector Dashboard for August 2023 Newsmakers (Free Excel)  Our viewOur Pipeline Prospector in July had ended with the caveat that even though the indices have ended the month on a stronger footing, August could be different. Though this is the second consecutive year when the markets have remained volatile, what gives us hope are the innovative drugs in the pipelines of various drugmakers — both big and small.Access the Pipeline Prospector Dashboard for August 2023 Newsmakers (Free Excel)  

Impressions: 2965

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#PipelineProspector by PharmaCompass
07 Sep 2023
Pipeline Prospector July 2023: Indices inch up on better than expected Q2 results
July saw all three biotech indices inch up by 2 percent as several drugmakers announced their second quarter (Q2) results. Overall, most drugmakers posted better than expected Q2 results, with some even raising their guidance for 2023.During July, the Nasdaq Biotechnology Index (NBI) was up 2 percent, at US$ 4,129.98, as opposed to a 0.7 percent drop in June. The SPDR S&P Biotech ETF index was up 2 percent, at US$ 84.29, as opposed to a 1.8 percent drop in June. And the S&P Biotechnology Select Industry Index (SPSIBI) was also up 2 percent, at US$ 6,544.43, as opposed to a 2.9 percent drop in June.The month witnessed four deals of over US$ 1 billion. Towards July-end, Biogen announced the acquisition of Reata Pharmaceuticals for US$ 7.3 billion. Through this acquisition, Biogen will bolster its neuromuscular and rare disease portfolio. The announcement led to a 61 percent jump in Reata’s stock.In mid-July, Eli Lilly bolstered its weight-loss portfolio with the acquisition of New York-headquartered biopharma Versanis Bio for a potential value of US$ 1.93 billion. Novartis announced the acquisition of DTx Pharma for US$ 1.0 billion. And on July 28, AstraZeneca’s rare diseases arm — Alexion —  announced it has agreed to buy Pfizer’s early-stage rare disease gene therapy portfolio for up to US$ 1 billion, plus royalties on sales.Access the Pipeline Prospector Dashboard for July 2023 Newsmakers (Free Excel)AbbVie stock posts gains despite drop in Humira sales; J&J’s Darzalex sales up 22%The last quarter was a good one for most drugmakers, and companies like AbbVie, Novartis and Johnson & Johnson (J&J) managed to beat estimates for both earnings and sales.In the month of July, seven biosimilars to AbbVie’s Humira got launched in the US market. As a result, Humira sales plummeted 25.2 percent during the quarter (at US$ 4.01 billion). However, AbbVie’s Q2 sales, at US$ 13.86 billion (a drop of 5 percent), topped investor expectations. This resulted in a 12 percent increase in AbbVie’s stock in July. There was more good news from the American drugmaker. In Europe, AbbVie received positive Committee for Medicinal Products for Human Use (CHMP) opinion for epcoritamab for the treatment of relapsed/refractory diffuse large B-cell lymphoma. The drug had bagged US Food and Drug Administration (FDA) approval in May 2023.J&J’s sales surged 6.3 percent in Q2, at US$ 25.5 billion, compared to US$ 24.2 billion in Q2 2022. The company’s global pharmaceutical revenue reached US$ 13.7 billion, driven significantly by the success of its oncology drug Darzalex, which posted a 22.4 percent jump in Q2 sales. Danish biotech Genmab received royalties from global Darzalex sales and its stock rose 11 percent during July.Carvykti, J&J’s CAR-T therapy for blood cancer (developed in partnership with Legend Biotech), reported robust Q2 sales of US$ 117 million, compared to US$ 24 million in Q2 2022. While J&J’s stock was up 4 percent, Legend’s stock rose 10 percent in July.Meanwhile, J&J’s Janssen reported positive phase 3 results for Rybrevant (amivantamab-vmjw) used on top of chemotherapy in patients with newly diagnosed NSCLC with EGFR exon20 insertion mutations. The company received FDA’s accelerated approval in 2021. Now, with positive trial results, the company hopes to win full approval and extend the drug to newly diagnosed patients.Access the Pipeline Prospector Dashboard for July 2023 Newsmakers (Free Excel)Oncology drugs boost Astra’s Q2 sales; BMS, Biogen post drop in quarterly revenuesAstraZeneca (stock up 7 percent) reported a total revenue increase of 9 percent at US$ 11.41 billion, compared to US$ 10.77 billion in Q2 2022. The growth was mainly driven by increased demand for oncology medicines such as Tagrisso, Imfinzi and Calquence.Astra-Merck’s Lynparza, in combination with abiraterone and prednisone/prednisolone, bagged conditional approval in Canada to treat BRCA-mutated metastatic castration-resistant prostate cancer.Novartis (stock up 5 percent) announced strong sales growth of 7 percent (at US$ 13.62 billion) as a result of impressive performance of its heart failure drug Entresto, multiple sclerosis drug Kesimpta and cancer drugs Kisqali and Pluvicto. Novartis’ Sandoz division launched the first citrate-free high-concentration formulation of Hyrimoz, a Humira biosimilar. Sandoz also received a positive CHMP opinion for its multiple sclerosis biosimilar natalizumab. If approved, it will be the first-of-its-kind biosimilar in Europe for use in all indications of reference biologic Tysabri.Amid these winners were two losers — BMS and Biogen. BMS (-5 percent) reported Q2 sales of US$ 11.22 billion that declined by 6 percent when compared to the previous year’s sales of US$ 11.88 billion. The decline prompted a cut in BMS’  full-year forecasts. This downturn comes as two of its top drugs — the multiple myeloma treatment Revlimid and the anticoagulant drug Eliquis — face competition from generics.Biogen (-5 percent) posted a 3 percent drop in its Q2 revenue, with sales at US$ 2.45 billion compared to US$ 2.58 billion in Q2 2022. The decline is attributed to a loss in multiple sclerosis revenue, which decreased by 14 percent from last year, reaching US$ 1.20 billion. In positive news, Biogen and Eisai’s Leqembi became the first med for Alzheimer’s to bag FDA’s full approval.Access the Pipeline Prospector Dashboard for July 2023 Newsmakers (Free Excel)Astra-Sanofi’s RSV therapy, GSK’s jab bag approvals; Roche, Caribou post trial gainsIn July, FDA granted approval to Sanofi-AstraZeneca’s respiratory syncytial virus (RSV) antibody therapy, Beyfortus (nirsevimab-alip), for newborns and infants. Moreover, GSK’s RSV vaccine — Arexvy — was approved by the UK health regulator in adults 60 years and older. Similarly, Moderna began its application process in the US, Australia and Europe to get an approval for its mRNA-1345 RSV vaccine in older adults. Despite positive news, Moderna stocks fell 3 percent. In trials, Roche (stock up 3 percent) posted positive results from the trial of its subcutaneous version of Ocrevus, a treatment for multiple sclerosis.  Additionally, Roche’s glofitamab became the first CD20xCD3 T-cell-engaging bispecific antibody to receive approval in Europe for relapsed or refractory diffuse large B-cell lymphoma (most common type of blood cancer). And Caribou Biosciences’ stock rose 100 percent on positive data from a phase 1 trial for its experimental CAR-T cell therapy CB-010 in relapsed or refractory B cell non-Hodgkin lymphoma, a type of blood cancer.Access the Pipeline Prospector Dashboard for July 2023 Newsmakers (Free Excel)Our viewThough pharma indices ended July on a stronger footing due to better-than-expected earnings, August opened with news that Pfizer has posted a whopping 54 percent drop in its Q2 revenues at US$ 12.7 billion, from US$ 27.72 billion in Q2 2022. Similarly, Merck reported a quarterly loss during the last quarter. While there are reasons why the two drugmakers haven’t performed well, and we also know that markets can be volatile, news on inflation easing off in some countries raises hopes that market conditions could improve in a few months.Access the Pipeline Prospector Dashboard for July 2023 Newsmakers (Free Excel) 

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#PipelineProspector by PharmaCompass
03 Aug 2023
Pipeline Prospector June 2023: Lilly, J&J, Merck stocks gain even as indices slip into red
June was a lackluster month for pharma stocks. The Nasdaq Biotechnology Index (NBI) was down 0.7 percent at US$ 4,065.95, against a 4 percent drop in May (at US$ 4054.54). SPDR S&P Biotech ETF index (XBI) was down 1.8 percent at US$ 83.95, against a 2 percent rise in May, and the S&P Biotechnology Select Industry Index (SPSIBI) was down 2.9 percent at US$ 6,409.05, against a 1 percent rise in May. Even though the indices slipped into red, several mega cap drugmakers like Eli Lilly, Johnson & Johnson (J&J) and Sanofi saw their stocks rise.There was a pick-up in dealmaking. Among the bigger deals were Novartis’ (up 4 percent) acquisition of American biotech Chinook Therapeutics (up 56 percent) for up to US$ 3.5 billion, Eli Lilly’s acquisition of Dice Therapeutics (up 47 percent) for around US$ 2.4 billion and Bausch + Lomb’s (up 22 percent) buyout of several eye-care products from Novartis for up to US$ 2.5 billion.Access the Pipeline Prospector Dashboard for June 2023 Newsmakers (Free Excel)Weight loss med boosts Lilly’s stock; concerns over obesity drug drag Pfizer downEli Lilly’s (up 8 percent) stock reached an all-time high after the company set a new bar among weight loss drugs with retatrutide (a GIP, GLP-1 and glucagon receptor triple agonist). In a phase 3 trial, the drug led to an average 24.2 percent reduction in weight over 48 weeks in patients without diabetes. The market has high expectations from this class of obesity drugs. That’s why it was unkind to Pfizer (-3 percent) when it announced it had discontinued the development of its experimental obesity and diabetes drug — lotiglipron (GLP-1 receptor agonist) — due to safety concerns. Pfizer’s market cap took a nearly US$ 12 billion hit on this news. The drug behemoth is shifting focus to its other obesity drug — danuglipron.In approvals, the US Food and Drug Administration (FDA) approved Lilly and Boehringer Ingelheim’s Jardiance for the treatment of type 2 diabetes in children 10 years and older. This is the first and only SGLT2 inhibitor approved for this patient population.Access the Pipeline Prospector Dashboard for June 2023 Newsmakers (Free Excel) J&J gains on new Carvykti data; Merck’s Lynparza scores win in prostate cancer trialJ&J and Legend Biotech filed for expanded use of Carvykti after presenting remarkable data from a phase 3 trial in relapsed and lenalidomide-refractory multiple myeloma. The drug was previously administered after four or more lines of therapy. Now, the companies hope to get approval for those who have received at least one prior line of therapy. With such an approval, Carvytki could get ahead of BMS’ multiple myeloma CAR-T therapy, Abecma, which was approved for fourth-line use in 2021.J&J also gained due to results from a phase 2 combination study of talquetamab and Tecvayli in relapsed or refractory multiple myeloma. Overall, J&J’s stock was up 7 percent in June, while Legend Biotech’s stock went up 11 percent.Another gainer was Merck (stock up 4 percent). Its Lynparza (olaparib) plus abiraterone combination has been approved by the FDA for the treatment of BRCA-mutated metastatic castration-resistant prostate cancer. The approval is based on data from a phase 3 trial, where the drug combo reduced the risk of disease progression or death by 76 percent compared to abiraterone alone in patients. With this approval, Lynparza has become the first poly ADP ribose polymerase (PARP) inhibitor to be approved in combination with a novel hormonal agent.Among mega-cap losers was Roche (-5 percent) whose PD-L1 inhibitor Tecentriq and Exelixis’ Cabometyx not only failed to offer any additional benefit in previously treated kidney cancer but also added toxicity.Among small cap gainers, Black Diamond Therapeutics saw its stock rise 150 percent as it announced phase 1 trial data on BDTX-1535 that showed positive results in non-small cell lung cancer (NSCLC) patients.Access the Pipeline Prospector Dashboard for June 2023 Newsmakers (Free Excel) GSK gains from Zantac settlement; FDA panel backs Astra-Sanofi’s RSV drugGSK (up 6 percent) reached its first legal settlement in litigation against its heartburn medication Zantac (ranitidine). This may set a precedent for thousands of other ranitidine cases set to go to trial next year.In another Zantac lawsuit, an International Chamber of Commerce tribunal rejected Boehringer Ingelheim’s attempt to gain indemnification from Sanofi for future liabilities arising from lawsuits in the US from users of the heartburn drug. Sanofi (up 6 percent) had acquired Zantac through an asset swap with Boehringer in 2016.There were updates on respiratory syncytial virus (RSV) vaccines. The US Centers for Disease Control and Prevention (CDC) recommended that Pfizer’s Abrysvo and GSK’s Arexvy vaccines be used to prevent severe RSV infections in older adults.Similarly, an FDA panel voted unanimously that AstraZeneca (-1 percent) and Sanofi’s nirsevimab has a favorable benefit-risk profile for the prevention of RSV lower respiratory tract disease in newborns and infants. If approved, nirsevimab would be the first preventive option for newborns and infants. The drug is likely to get an FDA nod in the third quarter of this year.FDA has accepted the biologics license applications (BLAs) submitted by Vertex Pharmaceuticals (up 9 percent) and CRISPR Therapeutics for the investigational treatment exagamglogene autotemcel (exa-cel) to treat severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). FDA has granted a priority review for SCD and a standard review for TDT.Access the Pipeline Prospector Dashboard for June 2023 Newsmakers (Free Excel) Doubts over Leqembi hits Biogen stock; Regeneron loses due to rejection of higher-dose Eylea variantAlzheimer’s disease experts in Europe raised doubts over the clinical benefits of Biogen and Eisai’s Leqembi. They said the benefits of Leqembi may not outweigh its potential side effects and the cost to the health system. This led to a 7 percent dip in Biogen’s stock. Last month, an FDA advisory panel unanimously agreed that a late-stage trial of Leqembi had verified the benefit of the treatment for those at an early stage of Alzheimer’s disease. The drug had bagged FDA’s accelerated approval in January this year.Similarly, Regeneron (-3 percent) was down on news that FDA has rejected a higher-dose variant of its blockbuster drug Eylea for the treatment of wet age-related macular degeneration (AMD), along with two other eye conditions common in diabetic patients. And Sarepta (-8 percent) was hit by news that analysts have voiced concerns over upcoming confirmatory trial data for its gene therapy — Elevidys — to treat Duchenne muscular dystrophy (DMD). While Elevidys has received an accelerated approval from the FDA as the first-of-its-kind gene therapy for DMD in children, analysts said the trial may not be enough to secure approval for the drug’s expanded use.Access the Pipeline Prospector Dashboard for June 2023 Newsmakers (Free Excel) Our viewPharma stocks have had a tumultuous ride over the past year. While the Nasdaq Composite Index and S&P 500 have been going up, pharma indices have not seen such a rally due to reasons such as a drop in sales of Covid treatments, the loss of patent protections, and a looming recession.In stock market parlance, this means pharma stocks may be undervalued and are likely to draw investor interest. Pathbreaking research and new drug pipelines should only increase their interest.Access the Pipeline Prospector Dashboard for June 2023 Newsmakers (Free Excel) 

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#PipelineProspector by PharmaCompass
06 Jul 2023