Pipeline Prospector June 2024: FDA approves Merck’s next-gen pneumococcal vaccine, Verona’s COPD therapy
The pharma indices were back in the black in May, and the good streak continued through June with the Nasdaq Biotechnology Index (NBI) gaining 3 percent, the SPDR S&P Biotech ETF (XBI) index up over 3.1 percent and the S&P Biotechnology Select Industry Index (SPSIBI) rising 4.25 percent.The two summer months were similar in more ways than one. In May, Eli Lilly had announced an investment of US$ 5.3 billion to boost the supply of Zepbound and Mounjaro. In June, Novo Nordisk followed suit, investing US$ 4.1 billion to develop a new manufacturing facility to boost the supply of Ozempic and Wegovy.Similarly, the US Food and Drug Administration (FDA) continued to grant vaccine approvals in June after okaying Moderna’s mRNA respiratory syncytial virus (RSV) vaccine in May. Last month, Merck’s next-generation pneumococcal vaccine won an FDA nod, and the agency expanded the use of GSK’s respiratory syncytial virus (RSV) vaccine to include adults aged 50 to 59.However, June was a lackluster month for mergers and acquisitions. The month had to be content with some tie-ups. For instance, Roche signed an up to US$ 1.8 billion deal with Boston-based startup Ascidian Therapeutics to discover and develop novel gene therapies for difficult-to-treat neurological diseases. AbbVie inked a US$ 1.7 billion agreement with China’s FutureGen to bring the latter’s next-generation treatment (FG-M701) for inflammatory bowel disease to market. Takeda signed an option agreement with China’s Ascentage Pharma for an exclusive license to a promising drug for chronic myeloid leukemia and other blood cancers. And Day One Biopharmaceuticals struck a licensing deal with MabCare for a novel antibody drug conjugate (ADC) to treat multiple cancers. The deal’s potential value is US$ 1.2 billion.Access the Pipeline Prospector Dashboard for June 2024 Newsmakers (Free Excel)Merck’s pneumococcal jab wins FDA nod; Moderna’s combo vaccine scores trial winVaccines took centerstage in June. FDA approved Merck’s Capvaxive, a next-generation pneumococcal vaccine designed to protect adults from a broader range of pneumococcus bacteria strains that cause serious illnesses and pneumonia. Capvaxive targets 21 bacterial variations responsible for about 85 percent of invasive pneumococcal disease cases in older adults. Similarly, GSK secured FDA approval for its RSV vaccine for adults aged 50 to 59 years. This makes Arexvy the only RSV shot endorsed for that age group.In a late-stage trial, Moderna’s mRNA-1083, an investigational combination vaccine against influenza and Covid, elicited a higher immune response compared to separate shots in people aged 50 and over. The combo jab generated more antibodies than currently marketed flu vaccines and Moderna’s Spikevax. And the world’s first personalized mRNA cancer vaccine from Moderna has raised hopes for patients with skin cancer, as three-year data from a mid-stage trial showed some benefits on patients who took the vaccine in combination with Merck’s Keytruda.Despite these developments, Moderna’s stock dropped 19 percent in June after it said the efficacy of its RSV vaccine mRESVIA had vaned substantially to 50 percent after 18 months.Access the Pipeline Prospector Dashboard for June 2024 Newsmakers (Free Excel) Verona, Geron score maiden FDA approvals; BMS’ Augtyro bags tumor-agnostic nodTwo companies obtained their first FDA approvals last month. The first one was Verona Pharma (stock up 15 percent) — it received its maiden FDA approval for Ohtuvayre for treating chronic obstructive pulmonary disease (COPD). This is also the first inhaled COPD treatment with a novel mechanism of action.The second company to get its maiden FDA approval was Geron, a commercial-stage biopharmaceutical company. FDA signed off on Geron’s Rytelo for treating transfusion-dependent anemia in patients with low- to intermediate-risk myelodysplastic syndromes (MDS), a group of blood cancers.In June, FDA granted accelerated approvals to at least three drugs. BMS’ Augtyro was granted FDA’s accelerated approval for treating adult and pediatric patients (over 12 years) with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, regardless of where they occur in the body.Ipsen received FDA’s accelerated approval for Iqirvo, a first-in-class, once-daily oral medication for treating primary biliary cholangitis (PBC), a progressive liver disease affecting mostly women.Sarepta Therapeutics’ (stock up 24 percent) Elevidys received accelerated approval for Duchenne muscular dystrophy (DMD) despite failing its primary endpoint in a late-stage trial.Amongst other approvals, Argenx’s Vyvgart Hytrulo was approved to treat chronic inflammatory demyelinating polyneuropathy (CIDP). This is a novel treatment option for this rare and debilitating neuromuscular disorder. And BMS’ Krazati got its second approval for treating colorectal cancer with a specific KRAS mutation.Access the Pipeline Prospector Dashboard for June 2024 Newsmakers (Free Excel) GSK’s failed cancer drug posts trial win; Gilead’s HIV prophylaxis shows efficacyIn late-stage trials, GSK’s multiple myeloma therapy Blenrep cut the risk of disease progression or death by almost half compared to standard-of-care treatments. The once-failed ADC was pulled from the lucrative US market in 2022, but the results could signal a comeback for Blenrep.Intra-Cellular’s antipsychotic drug Caplyta scored another remarkable late-stage win for treating major depressive disorder (MDD). In another late-stage trial, Gilead’s (stock up 6 percent) long-acting injection proved to be more effective in preventing HIV infection in women compared to the daily pill Truvada. This is the first time that an HIV pre-exposure prophylaxis (PrEP) has shown zero infections in a phase 3 trial, the drugmaker said. Dosed just twice a year, Sunlenca could be a game-changer in HIV prevention. Analysts estimate that PrEP-related sales could be over US$ 1.7 billion.In a blow to millions affected by long Covid, Pfizer’s Paxlovid did not appear to improve the symptoms as was hoped. Pfizer’s stock fell 7 percent in June.Access the Pipeline Prospector Dashboard for June 2024 Newsmakers (Free Excel) Our view This was the second month when pharma indices showed an upward trend. It’s been a good first half for the US stock markets. But there are mixed reports on how markets will perform in the second half, given the uncertainties around interest rate cuts and the outcome of the presidential elections in the US slated to be held in November. Though for those tracking biopharma news, there are enough interesting developments to fret over the markets.Access the Pipeline Prospector Dashboard for June 2024 Newsmakers (Free Excel) 

Impressions: 1889

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#PipelineProspector by PharmaCompass
04 Jul 2024
Pipeline Prospector May 2024: J&J inks two deals for eczema drugs; Novo scores trial wins in hemophilia, kidney disease
Pharma indices have rebounded after ending March and April in the red. May saw the Nasdaq Biotechnology Index (NBI) spike 5.6 percent from 4,168.63 to 4,401.1 and the SPDR S&P Biotech ETF (XBI) index rise 5.1 percent from 84.82 to 89.13. The S&P Biotechnology Select Industry Index (SPSIBI) saw a 5.3 percent gain to 6,930.67 from 6,584.4 at the end of April.The month didn’t see very many approvals from the US Food and Drug Administration (FDA). But what May lacked in approvals, it made up in acquisitions, deals, and trial news. Among the noteworthy approvals were Amgen’s (stock up 9 percent) Imdelltra, which won FDA’s accelerated approval to treat adults in the advanced stages of small cell lung cancer (SCLC). The agency okayed Tris Pharma’s Onyda XR, a once-a-day treatment for attention deficit hyperactivity disorder (ADHD) for patients six years and older. This makes it the first liquid non-stimulant ADHD drug approved in the US.Bristol Myers Squibb’s Breyanzi saw two FDA approvals in May. The agency first expanded its use to treat adults with a type of blood cancer known as follicular lymphoma. FDA then okayed it to treat relapsed or refractory mantle cell lymphoma. With five approvals, Breyanzi is the only CAR T cell therapy approved in the US for four distinct subtypes of non-Hodgkin lymphoma, bringing this personalized therapy to the broadest array of patients with B-cell malignancies. And FDA approved Moderna’s (stock up 39 percent) mRESVIA, an mRNA respiratory syncytial virus (RSV) vaccine, to protect adults aged 60 years and older from lower respiratory tract disease caused by RSV infection.Access the Pipeline Prospector Dashboard for May 2024 Newsmakers (Free Excel)J&J pays US$ 2.1 bn for eczema drug assets; Merck to buy EyeBio for up to US$ 3 bnSeeking to address the significant need in atopic dermatitis (eczema), J&J spent US$ 2.1 billion on bispecific antibody candidates. This included acquiring Proteologix for US$ 850 million and rights to Numab Therapeutics' NM26 for US$ 1.25 billion. NM26 is a promising phase 2-ready treatment. Bolstering its ophthalmology portfolio, Merck is acquiring EyeBio for up to US$ 3 billion. The deal includes an upfront payment of US$ 1.3 billion and grants Merck access to EyeBio's lead candidate, Restoret, for diabetic macular edema and neovascular age-related macular degeneration.In a US$ 1.8 billion deal (including US$ 1.15 billion upfront), Biogen acquired Human Immunology Biosciences (HI-Bio) to gain access to felzartamab, a potential cure for various immune-mediated diseases. Expanding its oncology portfolio, Novartis acquired Mariana Oncology for US$ 1 billion upfront and US $750 million in milestone payments. Mariana focuses on developing novel radioligand therapies to treat cancers with high unmet needs. Aiming to become a major player in the pharmaceutical industry, Japanese conglomerate Asahi Kasei offered US$ 1.1 billion to acquire Swedish drugmaker Calliditas Therapeutics.Access the Pipeline Prospector Dashboard for May 2024 Newsmakers (Free Excel) Sanofi bails out Novavax with US$ 1.2 bn deal; Takeda nabs option to license Alzheimer’s therapiesNovavax has received a timely shot in the arm, as Sanofi will pay the former up to US$ 1.2 billion in exchange for a license to commercialize its existing Covid vaccine and develop a combo jab to protect against Covid and influenza. The struggling Maryland biotech’s management, or lack thereof, had come under fire from investors. Sanofi also gets a minority 4.9 percent stake in Novavax. Following this, Novavax raised its 2024 revenue guidance from between US$ 800 million and US$ 1 billion to between US$ 970 million and US$ 1.17 billion. Novavax’s stock rocketed 200 percent in May.Japanese pharmaceutical giant Takeda made two significant moves. After an upfront payment of US$ 100 million, Takeda now has the exclusive option to secure global rights to AC Immune's Alzheimer's vaccine and immunotherapies targeting amyloid beta for another US$ 2.1 billion. Additionally, Takeda partnered with Shanghai-based Degron Therapeutics in a deal valued at up to US$1.2 billion. This collaboration focuses on discovering and developing novel cancer, neurological, and inflammatory drugs using a promising new approach called "molecular glue degraders."Access the Pipeline Prospector Dashboard for May 2024 Newsmakers (Free Excel) Novo scores late-stage wins in hemophilia, kidney disease; Lilly’s weekly insulin proves to be as effective as daily dosesNovo Nordisk’s Mim8 has hit its primary endpoint in a late-stage trial to treat people with hemophilia A. This paves the way for filing for approval later this year and competing with Roche’s blockbuster Hemlibra. Meanwhile, Ozempic lowered the risk of death in type 2 diabetes patients with chronic kidney disease. A late-stage trial with 3,533 people showed weekly semaglutide injections reduced kidney failure and death due to kidney complications by 24 percent.However, Novo did suffer a setback this month. FDA’s advisory panel voted seven to four that the benefits of Novo’s once-weekly insulin, Awiqli, do not outweigh its risks. The Danish drugmaker is in a race with Lilly to bring to market weekly injections with long-acting insulins, which would lower the treatment burden for diabetes patients. An increased risk of low blood sugar was flagged by the advisors in patients with type 1 diabetes. Novo's sales increased 24 percent to DKK 65.3 billion (US$ 9.4 billion) in the first quarter of 2024.Eli Lilly’s once-weekly insulin jab, efsitora, showed blood sugar reduction consistent with the daily insulins widely used today by patients with type 2 diabetes. Efsitora met its primary endpoints in two phase 3 trials. The Indiana drugmaker said it is investing an additional US$ 5.3 billion at its Lebanon (Indiana, US) manufacturing site to boost the supply of Zepbound and Mounjaro.Access the Pipeline Prospector Dashboard for May 2024 Newsmakers (Free Excel) Novartis tees up two rare kidney disease therapy approvals; Bayer’s menopause drug reduces hot flashes Novartis touted two promising late-stage readouts for rare kidney disease treatments as it tees up two potential approvals in the renal therapy space. The Swiss drugmaker’s experimental drug, atrasentan, reduced proteinuria (elevated protein in the urine) by 36.1 percent in IgA nephropathy patients. Fabhalta achieved a 35.1 percent proteinuria reduction in patients with the ultra-rare kidney disease C3 glomerulopathy (C3G). C3G currently has no approved therapies.Bayer said its non-hormonal drug, elinzanetant, significantly reduced debilitating bursts of body heat (hot flashes) associated with menopause. This tees it up to become only the second non-hormonal drug to do so after Astellas’ Veozah.Access the Pipeline Prospector Dashboard for May 2024 Newsmakers (Free Excel) Our viewFor long, mental health researchers have been captivated by psychoactive ingredients found in cannabis and LSD. However, advocates suffered a setback in early June when an FDA advisory panel voted against the first MDMA treatment for post-traumatic stress disorder (PTSD). Still, June kicked off with the American Society of Clinical Oncology (ASCO) annual meeting in Chicago, which saw tons of developments in treating cancers. It is shaping up to be an interesting month with over a dozen Prescription Drug User Fee Act (PDUFA) dates including for two drugs to treat chronic obstructive pulmonary disease (COPD) – Dupixent and ensifentrine.Access the Pipeline Prospector Dashboard for May 2024 Newsmakers (Free Excel)  

Impressions: 1857

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#PipelineProspector by PharmaCompass
06 Jun 2024
Pipeline Prospector April 2024: Indices dip amid muted Q1 results; Vertex acquires Alpine Immune for US$ 4.9 bn
Pharma indices had begun to recede in March. Their red streak accelerated in April with the Nasdaq Biotechnology Index (NBI) falling 6 percent from 4429.97 in March to 4162.33 last month and the SPDR S&P Biotech ETF (XBI) index tripping 10 percent from 94.34 to 84.62. Similarly, the S&P Biotechnology Select Industry Index (SPSIBI) was down 11 percent from 7402.50 to 6584.40 in April.This muted performance was also reflected in the first quarter (Q1) results. Biogen, Biomarin, Roche, Gilead, Merck, Sanofi, BMS and Johnson & Johnson announced their Q1 results in April. While Roche and Biogen reported a drop in revenues, BMS posted a loss in Q1, even though its revenues increased. And Sanofi reported a 14.7 percent drop in operating income due to competition from generics and currency fluctuations. BioMarin, Gilead and Merck reported a 9 percent, 5 percent and 9 percent increase, respectively, in their Q1 revenues over Q1 2023.There were a handful of deals announced last month, the biggest being Vertex Pharmaceuticals’ acquisition of Alpine Immune Sciences for US$ 4.9 billion. The deal gives Vertex access to Alpine’s protein-based immunotherapies for autoimmune diseases.Japanese drugmaker Ono Pharmaceutical agreed to acquire Massachusetts-based Deciphera Pharmaceuticals for US$ 2.4 billion. The acquisition gives Ono access to Deciphera's oncology pipeline. Denmark’s Genmab agreed to acquire privately-owned ProfoundBio for US$ 1.8 billion. ProfoundBio has three next-generation antibody drug conjugate (ADC) candidates in its pipeline that will now go to Genmab.Access the Pipeline Prospector Dashboard for April 2024 Newsmakers (Free Excel)FDA okays ImmunityBio’s Anktiva, Day One’s Ojemda for bladder, brain cancerLike March, April too saw several significant drug approvals, especially in oncology. ImmunityBio (its stock was up 50 percent in April) won its first US Food and Drug Administration (FDA) approval as the agency greenlit Anktiva as part of a combination therapy to treat a type of bladder cancer. With this approval, Anktiva will compete with Merck’s Keytruda.AstraZeneca (stock up 12 percent) and Daiichi Sankyo’s Enhertu has been granted accelerated approval in the US to treat adult patients with HER2-positive solid tumors that have spread or cannot be surgically removed. These patients have undergone prior treatment and have no satisfactory alternatives available to them. J&J and Legend’s Carvykti has become the first and only BCMA-targeted therapy approved by the FDA for patients with relapsed or refractory multiple myeloma, who have received just one prior line of treatment. The approval is an important milestone in J&J’s plans to make Carvykti a US$ 5 billion-plus asset at peak yearly sales.Day One Biopharmaceuticals’ Ojemda has been granted FDA’s accelerated approval to treat certain types of pediatric brain cancer. The pan-RAF kinase inhibitor has been okayed for patients six months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This is the first FDA approval of a systemic therapy for treating what is the most common form of childhood brain tumor.Access the Pipeline Prospector Dashboard for April 2024 Newsmakers (Free Excel) Basilea’s antibiotic bags FDA nod; Vanda’s Fanapt approved for bipolar disorderThe agency also approved Vanda Pharmaceuticals’ (stock up 25 percent) Fanapt tablets for treating manic or mixed episodes associated with bipolar-1 disorder in adults. The atypical antipsychotic agent has been used for the treatment of patients with schizophrenia since its FDA approval in 2009. This second approval for bipolar-1 disorder patients (who experience manic episodes that last at least seven days) could help revive Fanapt prescriptions.FDA has also approved Basilea Pharmaceutica’s Zevtera, an antibiotic for bacterial infections including multidrug-resistant strains. The US agency has okayed it for three conditions – treatment of adults with Staphylococcus aureus bacteremia, including those with right-sided infective endocarditis; adults with acute bacterial skin and skin structure infections; and adult and pediatric patients with community-acquired bacterial pneumonia.Access the Pipeline Prospector Dashboard for April 2024 Newsmakers (Free Excel) BMS’ schizo drug shows benefit sans weight gain; Imfinzi scores in lung cancer trialA common side effect associated with anti-psychotics is weight gain. However, interim results from a late-stage trial have shown that BMS’ schizophrenia drug KarXT continued to improve symptoms of the severe mental disease even at 52 weeks, and without weight gain.In another phase 3 trial, Imfinzi significantly improved overall survival and progression-free survival in patients with limited-stage small cell lung cancer. This makes this blockbuster drug the first and only immunotherapy to demonstrate survival benefit for this aggressive form of lung cancer. Imfinzi had posted sales of US$ 4.24 billion in 2023.Intra-Cellular Therapies said a phase 3 study of its drug Caplyta in people with major depressive disorder (MDD) met both its primary and key secondary endpoints. Given once daily as an adjunctive therapy to antidepressants, Caplyta 42 mg significantly beat the placebo at improving depression severity.Access the Pipeline Prospector Dashboard for April 2024 Newsmakers (Free Excel) Zepbound shows benefit in sleep disorder; Novartis’ Fabhalta reduces proteinuriaEli Lilly’s popular weight-loss drug Zepbound cut irregular breathing episodes associated with a common sleep-related disorder – obstructive sleep apnea (OSA). Treatment with Zepbound reduced the frequency of irregular breathing episodes by as much as 63 percent in adults with OSA and obesity across two late-stage trials. Lilly plans to submit the findings to FDA to expand Zepbound’s use for OSA. Roche’s Genentech said its drug Columvi, in combination with chemotherapy, demonstrated a statistically significant improvement in overall survival for people with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). A late-stage study pitted the Columvi-chemotherapy combination against a rituximab-chemotherapy combination in patients who received at least one prior line of therapy and are not candidates for autologous stem cell transplant. Patients in the Columvi group lived longer.In another late-stage trial, Novartis' drug Fabhalta reduced proteinuria (protein in the urine) in patients with IgA nephropathy by 38.3 percent. The Swiss drugmaker filed for FDA’s accelerated approval using the data, and the agency has granted it priority review.Access the Pipeline Prospector Dashboard for April 2024 Newsmakers (Free Excel) Our viewAt the start of 2024, the US market saw inflation falling and there were talks of interest rate cuts by the Federal Reserve. But few months on, the outlook has darkened a bit with inflation refusing to let off steam and economic output slowing down in the US. This is getting reflected in the pharma indices and the Q1 results of several drugmakers. Though overall, we have faith in the pharma sector’s spirit of innovation and its zest for dealmaking. Despite the macroeconomic challenges, there is hope that the other three quarters of 2024 will look better.Access the Pipeline Prospector Dashboard for April 2024 Newsmakers (Free Excel) 

Impressions: 1425

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#PipelineProspector by PharmaCompass
09 May 2024
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: 1683

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#PipelineProspector by PharmaCompass
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: 2150

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#PipelineProspector by PharmaCompass
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: 1877

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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: 2126

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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: 2517

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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) 

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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