US panel accuses China of subsidizing fentanyl production; Nigeria introduces 5-in-1 meningitis vaccine
This week’s news brings a mix of promising advancements and concerning issues.  A majority of opioid overdose deaths in America involve fentanyl. This week, a special House committee released a report that said the Chinese government is directly subsidizing the manufacturing and export of illicit fentanyl and other synthetic narcotics through tax rebates.Nigeria has become the first country to roll out the “revolutionary” new Men5CV vaccine against meningitis that protects against five strains of the bacteria that causes the disease, as opposed to the existing one-strain vaccines.  In regulatory news, the US Food and Drug Administration (FDA) issued India’s Natco Pharma a warning letter after it received inadequate response to its Form 483.In deals, Takeda has signed a potential US$ 1.2 billion deal with Kumquat Biosciences to develop a novel immuno-oncology candidate and AbbVie has partnered with Medincell to develop next-generation long-acting injectable therapies. Meanwhile, reports of a possible takeover of Biogaran, a subsidiary of French pharma giant Servier, has raised concerns over 8,600 production jobs.There were late-stage wins for Intra-Cellular’s Caplyta in treating major depressive disorder, Roche’s Columvi in improving survival in lymphoma patients, and Novartis’ Fabhalta in treating patients with the kidney disease IgA nephropathy (IgAN).Eli Lilly’s popular weight-loss drug Zepbound has cut episodes of irregular breathing associated with sleep apnea in two late-stage trials. And in trial losses, Sage Therapeutics said its investigational oral medicine dalzanemdor did not show statistically significant differences as compared to a placebo in Parkinson’s disease. Sage does not plan to develop dalzanemdor any further in treating Parkinson’s.China subsidizing fentanyl production to fuel US epidemic, says House panelThe Chinese Communist Party (CCP) is directly subsidizing the manufacturing and export of illicit fentanyl materials and other synthetic narcotics through tax rebates, a report by the House’s Select Committee on the CCP revealed. A months-long investigation uncovered a government website  that in turn revealed tax rebates to produce specific fentanyl precursors, so long as companies sold them outside of China. The strong synthetic opioid is commonly used to lace drugs, making them extremely dangerous. A majority of overdose deaths in the US involve fentanyl — the committee said it “kills over 200 Americans daily,” on average.Nigeria makes history by introducing five-in-one meningitis vaccineNigeria has become the first country to roll out the “revolutionary” new Men5CV vaccine against meningitis. The World Health Organization called it a historic move. The new shot protects against five meningitis strains of the meningococcal bacteria, unlike the existing one-strain vaccines. Nigeria is one of the 26 meningitis hyper-endemic countries situated in the African Meningitis Belt. Last year, there was a 50 percent jump in annual meningitis cases reported across Africa. The rollout, funded by Gavi, the vaccine alliance, aims to reach one million people.Takeda inks potential US$ 1.2 bn immuno-oncology deal with Kumquat BiosciencesJapanese drugmaker Takeda has agreed to pay Kumquat Biosciences over US$ $1.2 billion in upfront and milestone payments to develop and commercialize a novel immuno-oncology small molecule inhibitor as a mono- and/or combination-therapy. Kumquat will receive up to US$ 130 million in near-term payments after it concludes phase 1 trial activities.AbbVie partners with Medincell for injectable therapies: French biopharmaceutical company Medincell will use its commercial-stage long-acting injectable technology platform to formulate up to six innovative therapies. It will receive an upfront payment of US$ 35 million and will be eligible for up to US$ 1.9 billion in potential development and commercial milestones, plus royalties on worldwide sales.Possible takeover of Biogaran by Indian company raises concerns over 8,600 jobs French publications Les Échos and Franceinfo reported about the possible takeover of Biogaran, a top generic drugmaker in France. Biogaran, a subsidiary of the pharma giant Servier, has a portfolio of about 900 drugs, and employs subcontractors that represent about 8,600 jobs largely in France and Europe. According to the reports, among the four interested buyers are India’s Torrent Pharmaceuticals and Aurobindo Pharma. This has raised concerns about relocation of production jobs. Biogaran reportedly represents about 32 percent of the French generic market.Intra-Cellular’s Caplyta sees late-stage win for major depressive disorderIntra-Cellular Therapies said a phase 3 study of Caplyta (lumateperone) 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. An atypical antipsychotic of the butyrophenone class, Caplyta got the FDA go-ahead as a once-daily oral treatment for schizophrenia in 2019. In 2021, the approval was expanded to include bipolar depression. However, analysts say MDD’s patient population is about three times larger than bipolar depression and an expanded approval could add at least US$ 1 billion to peak sales.Zepbound reduces sleep apnea severity: Last month, FDA had approved Novo Nordisk’s weight loss drug Wegovy for lowering the risk of a stroke and heart attack in overweight and obese patients who did not have diabetes. And this month, Eli Lilly’s popular weight-loss drug Zepbound (tirzepatide) 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 drug improves survival in lymphoma patients: Roche’s Genentech said Columvi (glofitamab-gxbm), 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. Genentech plans to submit the data to health authorities. An expanded label for Columvi could see it potentially challenge AbbVie and Genmab’s Epkinly (epcoritamab).Novartis lands FDA priority review for Fabhalta in IgAN: Novartis said Fabhalta (iptacopan) reduced proteinuria (protein in the urine) in patients with IgA nephropathy by 38.3 percent in a phase 3 trial. The Swiss drugmaker filed for FDA’s accelerated approval using the data, and the agency has granted it priority review.Sage scraps Parkinson’s dud after mid-stage failure: Sage Therapeutics said its investigational oral medicine dalzanemdor did not show statistically significant differences as compared to a placebo on the primary endpoint in patients with mild cognitive impairment in Parkinson’s disease. Sage does not plan to develop dalzanemdor any further in treating Parkinson’s.Natco hit with FDA warning letter, shipments to US suspendedIndia’s Natco Pharma was hit with a warning letter after an inadequate response to a Form 483. Natco has stopped making products for the US market at the facility in the Indian state of Telangana. FDA said equipment wasn’t being cleaned and maintained properly, which could have led to contamination of the medications being manufactured. Additionally, the company wasn't looking into why some batches of medicine differed from what they were supposed to be, and there were even missing records for some batches.  

Impressions: 714

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#Phispers by PharmaCompass
18 Apr 2024
Vertex buys Alpine Immune Sciences for US$ 4.9 billion; Merck KGaA signs ADC deal with AI biotech Caris
This week’s Phispers comes packed with deals, approvals and trial wins. Vertex Pharmaceuticals has decided to bet big on immunotherapies by buying Alpine Immune Sciences for around US$ 4.9 billion in cash. Merck KGaA has entered into a multi-year partnership with Caris Life Sciences, a next-generation AI TechBio company and precision medicine pioneer, to develop novel antibody drug conjugates (ADCs). In devices business, Johnson & Johnson MedTech has picked up Shockwave Medical for around US$ 13.1 billion.In approvals, the US Food and Drug Administration (FDA) has allowed Johnson & Johnson-Legend’s Carvykti, and Bristol-Myers Squibb-2seventy bio’s Abecma as earlier lines of treatment for blood cancer. FDA has granted AstraZeneca and Daiichi’s oncology med Enhertu a broad (accelerated) approval to treat various cancers that are stimulated by a certain protein.In trials, BMS’ schizophrenia drug KarXT and AstraZeneca’s Imfinzi saw late-stage wins.In regulatory news, India’s Kilitch Healthcare was hit with a scathing warning letter from FDA over alarming conditions at its facility in Mumbai. And Jubilant Generics was issued a Form 483 by the agency.Vertex bolsters its pipeline for autoimmune diseases with US$ 4.9 bn Alpine buyVertex Pharmaceuticals is acquiring Alpine Immune Sciences for US$ 4.9 billion. The deal gives Vertex access to Alpine’s protein-based immunotherapies for autoimmune diseases. Through the acquisition, Vertex gains a promising drug known as povetacicept, which is in mid-stage development to treat IgA nephropathy (IgAN), a serious kidney disease.Merck KGaA inks ADC deal with Caris: Merck KGaA has entered into a multi-year partnership with Texas-based Caris Life Sciences for the discovery and development of first-in-class ADCs for cancer patients. The German drugmaker will provide Caris’ therapeutic research arm an undisclosed upfront payment. Caris will also be eligible to receive milestone payments of up to US$ 1.4 billion along with tiered royalties. Meanwhile the US’ Merck (MSD outside US) also snapped up a biotech startup — Abceutics — that was spun out of the laboratory of the University at Buffalo for about US$ 208 million. Abceutics’ payload-binding selectivity enhancer (PBSE) technology is meant to boost the safety of ADCs.J&J buys Shockwave Medical for US$ 13.1 bn: J&J has agreed to buy Shockwave Medical for about US$ 13.1 billion. The move boosts its cardiac-health-centric medical devices business. Shockwave Medical makes devices that rely on shockwaves to break down calcified plaque within heart vessels, mirroring the methodology used in treating kidney stones.FDA approves Carvykti, Abecma as earlier lines of blood cancer treatmentJ&J and Legend’s Carvykti has become the first and only BCMA-targeted therapy approved by FDA for patients with relapsed or refractory multiple myeloma, who have received just one prior line of treatment. In 2022, the CAR-T therapy was approved by the US agency as a fifth-line option for multiple myeloma patients and now it has become a second-line option. The approval is an important milestone in J&J’s plans to make Carvykti (ciltacabtagene autoleucel) a US$ 5 billion-plus asset at peak yearly sales. It brought in US$ 500 million last year and following the label expansion, analysts predict US$ 950 million in sales this year and as much as US$ 7.6 billion in global peak sales.FDA also greenlit BMS and 2seventy bio’s Abecma (idecabtagene vicleucel) for triple-class exposed relapsed or refractory multiple myeloma after two prior lines of therapy. Both therapies significantly reduced the risk of disease progression or death in their respective phase 3 trials – Carvykti by 59 percent and Abecma by 51 percent.Astra-Daiichi’s Enhertu wins broad approval: AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) 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 alternative options available. Now, with this broad approval, Enhertu has become the first ADC okayed to treat such tumors anywhere in the body.BMS’ schizophrenia drug reduces symptoms without weight gain in phase 3 trialLate-stage interim results have shown that BMS’ schizophrenia drug KarXT (xanomeline-trospium) continued to improve symptoms of the severe mental disease at 52 weeks. More significantly, this was achieved without weight gain, a common side effect associated with other antipsychotics. In fact, most patients (65 percent) experienced reductions in weight over the course of the trial with a mean weight decrease of 2.6 kilograms observed at one year. KarXT was the lead candidate in the New Jersey drugmaker’s US$ 14 billion buyout of Karuna Therapeutics. FDA has given it a Prescription Drug User Fee Act (PDUFA) date of September 26.Diabetes drug may slow impact of Parkinson’s disease:  Patients with Parkinson’s disease who took Sanofi’s diabetes med lixisenatide did not see worsening of motor symptoms after one-year of clinical trial, a paper published in the New England Journal of Medicine said. This development was hailed as a vital advance in the decades-long quest for a drug to slow the impact of Parkinson’s disease.AstraZeneca’s Imfinzi improves survival in phase 3 lung cancer trial: Imfinzi (durvalumab) significantly improved overall survival and progression-free survival for patients with limited-stage small cell lung cancer in a phase 3 trial. This makes the blockbuster drug the first and only immunotherapy to demonstrate survival benefit for this aggressive form of lung cancer in a late-stage trial. Imfinzi had clocked sales of US$ 4.24 billion in 2023.FDA issues warning letter to India’s Kilitch; Jubilant hit with Form 483India’s Kilitch Healthcare was hit with a scathing warning letter from the FDA over alarming conditions at its facility in Mumbai. It was found that staff worked barefoot in sterile rooms, operated machinery without goggles, leaned over opened and filled eye drop bottles and failed to disinfect components that touched the facility walls. FDA found the facility in “disrepair” with peeling paint, stains, and residues. In November last year, Kilitch had recalled 27 eye drops in the US market due to safety concerns.Jubilant slammed with Form 483: India’s Jubilant Generics was issued a Form 483 following an FDA inspection of its Bhagwanpur (Uttarakhand, India) facility between January 25 and February 2. During cleaning checks, FDA found evidence that some medicines may not have met quality standards (out-of-specification drugs). Jubilant Generics failed to identify a root cause of why these drugs failed quality standards. The company also could not take appropriate corrective and preventative actions. Furthermore, it distributed the affected lots in the US market.  

Impressions: 1011

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#Phispers by PharmaCompass
11 Apr 2024
Genmab buys ProfoundBio for US$ 1.8 bn; FDA okays Vanda’s Fanapt to treat bipolar disorder
April kicked off with Danish drugmaker Genmab buying ProfoundBio for US$ 1.8 billion in cash. ProfoundBio is a clinical stage biotech with three next generation antibody drug conjugates (ADCs) that should boost Genmab’s oncology portfolio.In approvals, the United States Food and Drug Administration (FDA) greenlit Vanda Pharmaceuticals’ Fanapt tablets to treat manic or mixed episodes associated with bipolar-I disorder in adults. The agency also approved a new antibiotic from Basilea Pharmaceutica for three indications. And AstraZeneca’s Voydeya bagged FDA approval as an add-on therapy to the drugmaker’s blockbuster drugs in the treatment of a rare blood disorder, known as paroxysmal nocturnal haemoglobinuria (PNH).The ever-increasing demand for glucagon-like peptide 1 (GLP-1) agonists is causing shortages and the FDA has said four doses of Mounjaro are likely to be in tight supply through April. Incidentally, a study published by Doctors Without Borders/Médecins Sans Frontières (MSF) in the Journal of the American Medical Association (JAMA) Network revealed a global double standard in access to this class of drugs.Meanwhile, Biogen and Eisai have missed their March 2024 target to file a biologics license application (BLA) for a subcutaneous version of their Alzheimer’s disease therapy Leqembi (lecanemab).Genmab buys next-generation ADC developer ProfoundBio for US$ 1.8 bnThe oncology space is witnessing a deal every month. In March, AstraZeneca had bought Fusion Pharmaceuticals for up to US$ 2.4 billion. In February, Novartis had acquired MorphoSys for US$ 2.9 billion and in January, Johnson & Johnson had said it would buy Ambrx Biopharma for about US$ 2 billion. And this month, Denmark’s Genmab has agreed to acquire privately-owned ProfoundBio for US$ 1.8 billion in cash.The Seattle-based clinical-stage biotech has three next-generation ADC candidates and the move will boost Genmab’s oncology portfolio. ProfoundBio had recently received FDA’s fast track designation for its “potential best-in-class” treatment for ovarian cancer, known as Rina-S.AbbVie acquires Landos: AbbVie will acquire Landos Biopharma for up to US$ 212 million in a bid to boost its portfolio of inflammatory and autoimmune diseases. Landos’ lead investigational asset NX-13 is currently in a mid-stage study for ulcerative colitis (UC).Blue Wolf buys seven sites from Recipharm: Sweden’s Recipharm, a contract development and manufacturing organization (CDMO), has said US private equity firm Blue Wolf Capital Partners has committed to acquire seven of its sites, including five sites in Sweden and one each in France and Spain. Recipharm is looking to expand its biologics production capacity to take advantage of the increasing demand for cell and gene therapies. Blue Wolf will use the sites to create a new CDMO.FDA okays Vanda’s Fanapt to treat bipolar disorder; approves antibiotic from BasileaFDA has approved Vanda Pharmaceuticals’ Fanapt (iloperidone) tablets for treating manic or mixed episodes associated with bipolar-I disorder in adults. The atypical antipsychotic agent has been used for the treatment of patients with schizophrenia since its FDA approval in 2009. However, it has been increasingly facing competition in recent quarters. This second approval, for bipolar-I disorder patients who experience manic episodes that last at least seven days, could help revive Fanapt prescriptions. In a late-stage study, iloperidone helped reduce severity of core symptoms of mania when tested across 400 patients.Meanwhile, a US appeals court has allowed Teva Pharmaceutical and Viatris to resurrect challenges to the final US patent on Johnson & Johnson’s blockbuster schizophrenia drug. Invega Sustenna (paliperidone palmitate) raked in US$ 4.1 billion last year including US$ 2.9 billion from the US market. The case will be sent back to a New Jersey federal court — it had ruled against Teva’s challenge of J&J’s patent in 2021.New antibiotic from Basilea: FDA has approved Basilea Pharmaceutica’s Zevtera (ceftobiprole medocaril sodium for injection), 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 three months to less than 18 years old with community-acquired bacterial pneumonia.Astra’s Voydeya given go-ahead as add-on therapy for rare blood diseaseAstraZeneca’s Voydeya (danicopan) has been approved in the US as an add-on therapy to Ultomiris (ravulizumab) or Soliris (eculizumab) for the treatment of extravascular haemolysis (EVH) in adults with paroxysmal nocturnal haemoglobinuria (PNH).PNH is a rare blood disease and EVH is a condition that causes red blood cell destruction outside of the blood vessels. Voydeya is a first-in-class, oral, factor D inhibitor developed as an add-on to Astra’s standard-of-care blockbuster drugs.Mounjaro’s tight supply to continue; ‘GLP-1 drugs unaffordable in poor nations’, says studyFDA’s website now shows four doses of Mounjaro (tirzepatide) are expected to be in tight supply through April due to increased demand. Those include the 7.5, 10, 12.5, and 15 milligram doses. In early February, the agency had said the 10, 12.5 and 15 milligram doses would be in tight supply through early March.Meanwhile, a study by Doctors Without Borders/Médecins Sans Frontières (MSF) has revealed that drug companies are pricing GLP-1 receptor agonists at almost 400 times the level necessary to make a profit. Those include Novo Nordisk’s Ozempic (semaglutide) and Eli Lilly’s Trulicity (dulaglutide), which are now a standard part of treatment in high-income countries. However, in low-income countries, they are unaffordable.“Eli Lilly and Novo Nordisk can in no way supply the world with the amount of these medicines needed to meet global demand, so they must immediately relinquish their stranglehold and allow them to be produced by more manufacturers around the world,” MSF said.Biogen, Eisai fail to submit BLA for subcutaneous Leqembi injectionThe wait for a more convenient version of Biogen-Eisai’s Alzheimer’s disease drug Leqembi has become longer with the two companies missing their own deadline of submitting the biologics license application (BLA) for a subcutaneous version of Leqembi by March. The drug is currently being administered intravenously. The subcutaneous Leqembi is being proposed as a weekly maintenance regimen.Meanwhile, the two companies have filed a supplemental BLA (sBLA) for a monthly intravenous maintenance dose schedule of Leqembi. If approved, this maintenance regimen will be available to patients who complete the biweekly intravenous initiation phase.  

Impressions: 568

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#Phispers by PharmaCompass
04 Apr 2024
Novo buys Cardior Pharma for up to US$ 1.1 bn; FDA okays two meds for pulmonary arterial hypertension
This week’s Phispers has news on drug approvals, particularly meds to treat pulmonary arterial hypertension (PAH), Duchenne muscular dystrophy (DMD), and anemia caused by chronic kidney disease (CKD). The United States Food and Drug Administration (FDA) has approved Merck’s Winrevair and Johnson and Johnson’s combo tablet Opsynvi for treating adults with PAH. The agency has also okayed Italfarmaco’s Duvyzat for the treatment of DMD. And, after rejecting Akebia’s vadadustat in March 2022, the agency has okayed the drug to treat anemia caused by CKD.In deals, Novo Nordisk has agreed to acquire Cardior Pharmaceuticals for up to US$ 1.1 billion in order to boost its cardiovascular diseases pipeline. Blackstone’s life sciences arm is offering Moderna a funding of US$ 750 million to bolster its efforts to develop flu vaccines. And, non-profit Caring Cross is collaborating with Fiocruz, a foundation set up by the Brazilian government, to develop local manufacturing of CAR-T cell and stem cell gene therapies in Brazil, thereby making them affordable.In legal and regulatory updates, a federal judge in the US has given J&J a new chance to contest the scientific evidence linking its talc to cancer. And, FDA’s India office has said that inspectors from state drug control authorities from four Indian states are eligible to be included as observers for relevant inspections in their respective states.Novo strengthens cardio pipeline through US$ 1.1 billion Cardior Pharma buyoutNovo Nordisk has agreed to buy Cardior Pharmaceuticals for up to € 1.03 billion (US$ 1.1 billion), thereby strengthening its drug pipeline for cardiovascular diseases. Cardior’s therapeutic approach targets distinctive non-coding RNAs as a platform for addressing root causes of cardiac dysfunctions with an aim to achieve lasting patient impact, the Danish drugmaker said.Germany-based Cardior’s lead candidate CDR132L is designed to halt and partially reverse cellular pathology by selectively blocking abnormal levels of the microRNA molecule miR-132, potentially leading to long-lasting improvement in heart function. CDR132L is currently being investigated in a phase 2 trial in people with heart failure with reduced ejection fraction (HFrEF) who have previously suffered a heart attack (myocardial infarction).Blackstone offers US$ 750 million funding to Moderna: Blackstone’s life sciences arm is offering funding of US$ 750 million to Moderna for funding its flu vaccines. This tranche of funding would bolster Moderna’s efforts to advance multiple vaccines and help lift its sagging fortunes due to the slump in post-Covid sales. The biotech firm is developing several new vaccines, including shots for cancer and respiratory syncytial virus (RSV), and plans to spend about US$ 4.5 billion on research and development in 2024.Caring Cross, Brazil’s Fiocruz to produce affordable gene therapies: Non-profit Caring Cross is collaborating with Fundação Oswaldo Cruz (Fiocruz), a foundation from the Brazilian Ministry of Health, to develop local manufacturing of CAR-T cell and stem cell gene therapies in Brazil. Caring Cross has developed manufacturing processes that significantly decrease the material cost of making CAR-T cell therapy products. The initial phase of the program will focus on CAR-T cell therapies for leukemia and lymphoma.FDA approves Merck’s Winrevair, J&J’s Opsynvi to treat rare lung diseaseFDA has approved a breakthrough therapy from Merck that treats adults with hypertension that is caused by constriction of the arteries in the lungs, known as pulmonary arterial hypertension (PAH). Merck’s Winrevair (sotatercept-csrk) is the first FDA-approved activin-signaling inhibitor therapy for PAH. It is to be given once every three weeks as a subcutaneous injection. Merck had acquired the drug through its US$ 11.5 billion acquisition of Acceleron Pharma in 2021.The agency has 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 has become the only once-daily, single-tablet combination therapy for PAH.After rejection in 2022, Akebia’s med bags FDA nod for treating anemia in CKDFDA has approved Akebia’s anemia drug, vadadustat, to treat anemia caused by chronic kidney disease (CKD) in dialysis patients. The drug will be available under the brand name Vafseo. In March 2022, FDA had declined to approve vadadustat due to safety concerns as it posed an increased risk of blood clot formations and drug-induced injuries to the liver.Italfarmaco’s Duvyzat approved for DMD: FDA has approved the 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 genetic variants of DMD, the most common hereditary neuromuscular disease. Over time, the muscles deteriorate causing problems with walking. Ultimately, DMD leads to problems with breathing, causing early death. Duvyzat is set to compete with Sarepta Therapeutics’ bestselling medicine Exondys 51, and Sarepta’s other therapies Vyondys and Amondys.J&J gets fresh chance to contest evidence linking its talc to cancerIn the US, a federal judge has ruled that J&J will be given a new chance to contest the scientific evidence linking its talc to ovarian cancer. J&J has repeatedly denied claims that its baby powder and other talc products cause cancer or contain asbestos. The company is facing over 53,000 lawsuits over its talc products. J&J has welcomed the ruling as it said it intends to “shine a light on some of the made-for-court junk science” used in recent trials.FDA allows Indian states to observe its drug inspections: Amid growing concerns over the quality of drugs coming out of India, FDA’s India office has said that inspectors from state drug control authorities from four Indian states are eligible to be included as observers for relevant inspections in their respective states. The four states include Gujarat, Telangana, Goa, and Karnataka, where there are high numbers of FDA-registered facilities. The US agency has shared best practices and inspectional protocols in regulatory forums with the Indian states.  

Impressions: 995

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#Phispers by PharmaCompass
28 Mar 2024
Madrigal’s Rezdiffra becomes first FDA-approved drug to treat NASH; Astra buys Fusion for US$ 2.4 bn
It was a week of drug approvals, as the US Food and Drug Administration (FDA) okayed several therapies before the close of the first quarter of 2024.For quite some time, the drug development field for the liver condition non-alcoholic steatohepatitis (NASH) has been a graveyard for failed programs. This week, the field celebrated a hurrah moment when Madrigal Pharmaceuticals won the race to have the first NASH treatment approved by the FDA.After nearly 40 years, FDA approved a new therapeutic pathway for hypertension. Idorsia’s Tryvio is a once-daily med for hypertension that is to be used in combination with other BP drugs by people who are not able to adequately control their BP on other drugs.FDA has also granted the chimeric antigen receptor (CAR) T-cell therapy Breyanzi accelerated approval for two types of hard-to-treat blood cancers and its oncology panel has ruled favorably on Johnson & Johnson’s Carvykti and Bristol Myers Squibb’s Abcema.Orchard Therapeutics’ Lenmeldy has secured FDA approval to become the first gene therapy in the US for a rare pediatric disorder – metachromatic leukodystrophy (MLD). The agency also approved Optinose’s Xhance —the first medicine to treat chronic sinusitis.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. Meanwhile, Pfizer said it intends to cut its stake in Haleon from 32 percent to 24 percent.Madrigal’s Rezdiffra becomes first FDA-approved drug for liver disease NASHMadrigal’s oral drug Rezdiffra (resmetirom) has become the first treatment in the US for adults with the common fatty liver disease — NASH. Also known as MASH (metabolic dysfunction-associated steatohepatitis), the disease 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.After a year of Rezdiffra, liver biopsies showed more subjects achieved NASH resolution or an improvement in liver scarring as compared to those on the placebo. The annual wholesale price of Rezdiffra is US$ 47,400. Rezdiffra had received breakthrough therapy designation last April. The American Liver Foundation CEO Lorraine Stiehl called the approval a “game-changing” moment.Astra to buy next-generation cancer drugmaker Fusion for up to US$ 2.4 bnAstraZeneca has agreed to buy clinical-stage biopharma Fusion Pharmaceuticals for US$ 2.4 billion. According to a company statement, the acquisition marks a major step forward for Astra, as it gains foothold into radioconjugates (RCs) — a promising modality in cancer treatment.Boosts rare-disease pipeline with Amolyt buyout: Astra also agreed to buy France’s Amolyt, which focuses on rare endocrine diseases, for a total value of US$ 1.05 billion. Amolyt’s pipeline includes a late-stage asset — eneboparatide (AZP-3601) — with the potential to address a significant unmet need in patients with chronic hypoparathyroidism.Pfizer to slash stake in Haleon to 24 percent: Pfizer, which is currently the largest shareholder in the world’s biggest standalone consumer health company — Haleon, is reducing its stake from 32 percent to 24 percent. Haleon makes household brands like Panadol, Advil, and Sensodyne. The New York-based drug behemoth is set to offload 630 million Haleon shares, which was worth about £ 2.03 billion (US$ 2.58 billion), going by the stock’s closing price on March 15.FDA okays Idorsia’s once-daily antihypertensive drug TryvioFDA has approved Idorsia’s once-daily treatment Tryvio (aprocitentan) for hypertension 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. Tryvio is the first oral anti-hypertensive therapy, which works via a new therapeutic pathway, to be approved in almost 40 years. Idorsia expects millions of patients in the US to benefit from it.BMS’ Breyanzi becomes first CAR-T therapy for hard-to-treat blood cancersFDA has granted 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). Breyanzi is a personalized one-time treatment where a patient’s own white blood cells are engineered to better target and destroy malignant “B cells”. In a mid-stage trial, Breyanzi demonstrated a response in 45 percent of patients and achieved a complete response in 20 percent of patients. This reportedly makes it an effective option for patients who have relapsed after receiving targeted therapies.FDA panel backs expanded use of J&J, BMS’ CAR-T therapies: FDA’s Oncologic Drugs Advisory Committee voted unanimously (11-0) that the benefits of J&J and Legend’s Carvykti outweigh its risk in earlier lines of multiple myeloma treatment. Similarly eight of the committee’s members voted in favor of BMS and 2seventy bio’s Abcema. They said it demonstrated a favorable benefit/risk profile for patients with triple-class exposed relapsed or refractory multiple myeloma.Takeda’s Iclusig wins accelerated nod: FDA has 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. This makes it the only targeted therapy approved as a first line of treatment for the condition in the US.FDA okays Orchard’s Lenmeldy as first gene therapy for rare pediatric disorderOrchard Therapeutics’ Lenmeldy has secured FDA approval to become the first gene therapy in the US for a rare pediatric disorder, known as metachromatic leukodystrophy (MLD). The agency indicated it for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile MLD. The debilitating hereditary disease affects the brain and nervous system and causes loss of cognitive and motor function and early death.FDA okays drug to treat chronic sinusitis: FDA has approved the first medicine to treat chronic sinusitis, which affects about 30 million adults in the US. The nasal spray Xhance (fluticasone propionate) uses an exhalation delivery system to deposit a topical steroid in target areas of inflammation deep in the nose not typically reached by standard nasal sprays, its maker Optinose said.BeiGene’s Tevimbra bags nod for advanced esophageal cancer: BeiGene’s PD-1 blocker Tevimbra (tislelizumab) has finally got the go ahead from the FDA as the 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.  

Impressions: 878

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#Phispers by PharmaCompass
21 Mar 2024
FDA approves Wegovy to reduce cardiovascular risks; delays action on Lilly’s Alzheimer’s drug
In this week’s news, Novo Nordisk’s Wegovy became the first obesity drug cleared by the US Food and Drug Administration (FDA) to lower the risk of cardiovascular death, heart attack, and stroke.There was a fair bit of unfortunate news for patients with neurological disorders. First, FDA delayed the approval of Eli Lilly’s donanemab for Alzheimer’s and called for further scrutiny of its safety and efficacy. Second, Amylyx Pharmaceuticals’ lead candidate Relyvrio bombed in a large, phase 3 clinical trial for amyotrophic lateral sclerosis (ALS), raising questions about its future.The Biotechnology Innovation Organization (BIO), an industry trade group based in Washington, is taking steps to remove China’s WuXi-AppTec from its membership. The staff at FDA has raised doubts over the benefit of giving Johnson & Johnson and Bristol Myers Squibb’s cell therapies, Carvykti and Abecma, to blood cancer patients as an early treatment.Serum Institute of India, which teamed up with AstraZeneca to produce the Covishield vaccine, has repurposed its manufacturing facilities for newer vaccines against malaria and dengue. And, India’s Sun Pharma was forced to recall 55,000 bottles of the gout drug febuxostat from the US market due to microbial contamination in stagnant water in the duct of the manufacturing equipment.Wegovy approved in US to lower risk of serious heart problems in obese adultsFDA has approved Wegovy (semaglutide) to reduce the risk of cardiovascular death, heart attack, and stroke in obese or overweight adults with cardiovascular disease. The move makes Wegovy the first weight loss medication that is also approved to help prevent life-threatening cardiovascular events and is hailed as a major advance for public health. In a trial spanning 17,604 patients, over a mean duration of 33 months, Wegovy lowered the risk of non-fatal heart attack by 28 percent, heart-related death by 15 percent, and non-fatal stroke by seven percent.FDA delays action on Lilly’s closely watched Alzheimer’s drug donanemabThe much anticipated approval of Eli Lilly’s donanemab scheduled for this month has been deferred as FDA has opted to convene a panel of independent experts to assess the drug’s safety and efficacy. The decision took Alzheimer’s experts and Lilly by surprise as the latter said, “it is unusual for an advisory committee to occur after the anticipated FDA action date.” The decision underscores the high stakes and challenging history of developing Alzheimer’s treatments.According to a Lilly press statement, FDA is likely to convene a meeting of its advisory committee that looks into peripheral and central nervous system drugs to discuss its trial that evaluated the efficacy and safety of donanemab in early symptomatic Alzheimer’s disease.Roche’s persistence for Alzheimer’s therapy sees promising results: After two failures to bring an Alzheimer’s drug to the market, Roche touted “best-in-class potential” for trontinemab in treating the disease. Though the antibody is still in early phase 2 trials, Roche came out with promising data that showed trontinemab cleared clumps of amyloid protein from patients’ brains faster than Leqembi and donanemab. Trontinemab’s delivery uses the Swiss pharma’s proprietary “Brainshuttle” platform which allows it to penetrate the blood-brain barrier resulting in more exposure to the brain and broader distribution across the central nervous system.Biotech trade group severs ties with China’s WuXi AppTec over security concernsThe Biotechnology Innovation Organization (BIO), an industry trade group based in Washington, said it is taking steps to remove China’s WuXi-AppTec from its membership. BIO’s new CEO, a former Naval Intelligence Officer, said it was part of “several important steps to reaffirm the organization’s position with regards to national security.” Last week, the Senate’s homeland security committee voted to pass a bill that prohibits federal agencies from having contracts with four Chinese biotechs, including WuXi AppTec.Amylyx’s ALS drug Relyvrio flunks late-stage trial, could get pulled from marketJust under two years ago, Relyvrio (sodium phenylbutyrate and taurursodiol) became one of only three treatments approved by the FDA for ALS, commonly called Lou Gehrig's disease. The agency had done so on the back of results from a phase 2 trial. The debilitating condition robs patients of the ability to walk, talk, and eventually breathe within five years. Now, the results of a 48-week trial in 664 patients are in, and Relyvrio failed to demonstrate that it worked better than a placebo.“We’re deeply saddened, and I’d say it’s been a really, really tough day for the ALS community,” Amylyx Co-CEO Justin Klee said. The Cambridge, Massachusetts-based biotech said it might withdraw Relyvrio from the market and will announce its plans within eight weeks.Acadia to stop trials for its antipsychotic drug: Acadia Pharmaceuticals’ antipsychotic drug Nuplazid (pimavanserin) failed to meaningfully improve negative symptoms of schizophrenia in a phase 3 trial. Those symptoms included “blunted affect, poor socialization, and lack of motivation.” Nuplazid was the first FDA-approved drug to treat psychosis-related delusions and hallucinations experienced by certain patients with Parkinson’s disease. However, previous attempts to expand the drug’s use to treat dementia and Alzheimer’s disease had also resulted in failure.SII shifts focus to malaria, dengue vaccines: Adar Poonawalla, CEO of Serum Institute of India (SII), has said his company has bolstered its manufacturing capabilities ahead of launches of shots against dengue and malaria planned over the next few years. During the pandemic, SII had invested US$ 2 billion to boost production. Now, with the demand for Covid-19 products waning, the world’s largest vaccine maker has repurposed its facilities for newer shots.FDA tells Sun Pharma to recall 55,000 bottles of gout med due to contaminationThe New Jersey unit of Indian drugmaker Sun Pharma is recalling 55,000 bottles of generic gout treatment Febuxostat, according to FDA’s enforcement report. Deviations from current good manufacturing practices (cGMPs) have caused microbial contamination in stagnant water in the duct of the manufacturing equipment. The lots in question were made in Dadra (India) for Memphis-based Northstar Rx LLC.Carvykti, Abecma may not benefit patients with early-stage blood cancerThe staff at FDA has raised doubts over the benefits of giving J&J and Bristol Myers Squibb’s cell therapies, Carvykti and Abecma, to blood cancer patients as an early treatment. Both are chimeric antigen receptor (CAR) T-cell therapies. An FDA advisory committee will vote Friday on whether the two therapies have a favorable benefit-risk ratio in earlier lines of multiple myeloma treatment.  

Impressions: 1860

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#Phispers by PharmaCompass
14 Mar 2024
Court dismisses Astra’s lawsuit over IRA price talks; Ozempic shows benefits to kidney, heart in diabetics with CKD
This week’s Phispers brings updates on the Inflation Regulation Act (IRA) in the US. A federal district court judge rejected AstraZeneca’s claims that the drug price negotiations under IRA violate the Fifth Amendment of the US Constitution. The negotiation program currently foresees adding 10 to 20 drugs a year through 2029, and Biden plans to increase this to 50.Novo Nordisk’s diabetes med Ozempic reduced the risk of kidney disease progression and death from cardiovascular or kidney complications by 24 percent in diabetic patients with chronic kidney disease (CKD). Additionally, in the first clinical trial of Ozempic in people with HIV and a type of liver condition, participants (on average) experienced a 31 percent reduction in liver fat.The World Bank, UN and the Gates Foundation have come together and pledged nearly US$ 600 million to eradicate cervical cancer. Cancer-causing benzene has now been found in acne products. And, the WHO has warned of growing resistance to GSK’s HIV drug — dolutegravir.Judge rejects Astra’s lawsuit calling Medicare price talks unconstitutionalA federal judge has rejected AstraZeneca’s claims that the Inflation Reduction Act’s (IRA) drug price negotiations are unconstitutional. The Anglo-Swedish drugmaker’s blockbuster drug Farxiga (dapagliflozin) for CKD, type 2 diabetes, and heart failure was among the 10 treatments chosen for price negotiations.The US Medicare health insurance program covers 66 million Americans mostly over 65 years. Last August, the Biden government had allowed Medicare to negotiate the prices of 10 drugs with their manufacturers, via the IRA. The list included drugs like Stelara, Farxiga, Imbruvica, Eliquis, and Entresto. Astra had contended that this violated the Fifth Amendment of the US Constitution, which states private property shall not “be taken for public use, without just compensation.”Drugmakers respond with counter offers: All the manufacturers of the 10 drugs have responded with counter offers after the United States Department of Health and Human Service (HHS) sent them initial offers on February 1.  With negotiations now underway, the HHS was “glad the drug companies are coming to the table.”‘Medicare should negotiate for at least 50 drugs’: The US President Joe Biden will call on Congress to expand the number of drugs subject to annual Medicare price negotiations. The program currently foresees adding 10 to 20 drugs a year through 2029, and Biden will propose increasing this to at least 50, a White House press statement said.Ozempic cuts risk of CKD progression, cardiac death in late-stage trialNovo 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. About 40 percent of people with type 2 diabetes have CKD. Confident about the efficacy, the drugmaker has decided to stop the trial a year ahead of schedule. Novo will file for expanded approvals for Ozempic in the US and EU this year.Reduces severity of liver disease in people with HIV: Ozempic also showed benefits in HIV. The drug was found to be safe and it reduced the amount of fat in the liver by 31 percent in people with HIV and a liver disease, known as metabolic dysfunction-associated steatotic liver disease (MASLD). In the first clinical trial of semaglutide in people with HIV for MASLD, participants experienced a 31 percent reduction in liver fat on average, with 29 percent of participants experiencing a complete resolution of MASLD (i.e. their liver fat decreased to 5 percent or less).Akero’s drug shows promising long-term benefits for MASH patientsAkero Therapeutics’ experimental drug efruxifermin significantly reduced scarring after 96 weeks while treating patients with metabolic dysfunction-associated steatohepatitis (MASH), a liver disease. MASH causes histologic liver damage and occurs in patients who are not alcoholics and are often obese or have type 2 diabetes. In fact, the condition is the next target of obesity drugs. In the mid-stage study, biopsies showed 75 percent of the patients experienced a reduction in liver scarring by at least one stage on the higher dose and 46 percent did so on the lower dose. The condition has no approved drugs in the US and affects over 17 million people in the country.Cancer-causing benzene now found in acne products like Clearasil, ProactivHigh levels of benzene, a known carcinogen, have now been found in topical acne treatments, Valisure said. The Connecticut-based company has issued a citizen petition calling on the FDA to “recall and suspend sales of benzoyl peroxide from the US market.” Unacceptably high levels of benzene were found in acne products including popular household brands like Clearasil, Proactiv, and PanOxyl, as well as products from Estée Lauder, Walmart, Target, Walgreens, and Clinique. According to Valisure, this was because benzoyl peroxide decomposed into benzene over time when used with substances in acne medication.WHO warns of growing resistance to GSK’s HIV drug — dolutegravirThe World Health Organization (WHO) had recommended GSK’s Tivicay (dolutegravir) back in 2018 as a preferred treatment for the virus. But now, the organization has cited surveys from four countries to suggest that resistance to the GSK drug has exceeded levels observed in clinical trials.Meanwhile, GSK has said a new investigational formulation of cabotegravir, known as cabotegravir ultra long-acting (CAB-ULA), can be administered at intervals of at least four months, based on results from a phase 1 trial. The current version of the HIV prevention and treatment drug (cabotegravir) requires dosing every two months. The British drugmaker’s HIV-focused unit, ViiV Healthcare, plans to conduct large-scale trials later in 2024.World Bank, Gates Foundation, UN pledge US US$ 600 mn to end cervical cancerAt the first-ever meet of the Global Cervical Cancer Elimination Forum in Colombia, global health donors have pledged nearly US$ 600 million to rid the world of cervical cancer. Those donors included the World Bank (US$ 400 million), the Bill & Melinda Gates Foundation (US$ 180 million), and the UN children's agency UNICEF (US$ 10 million).  

Impressions: 723

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#Phispers by PharmaCompass
07 Mar 2024
India to have BMS’ largest R&D facility after US; its drug plants to face increased FDA inspections
This week’s Phispers has a lot of news from India. First, Bristol Myers Squibb (BMS) inaugurated a US$ 100 million facility in Hyderabad and said it expects to make this facility its largest R&D outfit outside of the US by next year.Second, the country director of the United States Food and Drug Administration (FDA) Sarah McMullen has said the regulator will increase the number of inspections in India this year. Requests have been made for more drug investigators to be stationed in India.Third, Japanese drugmaker Takeda has tied up with India’s Biological E to increase access to its dengue vaccine Qdenga. And fourth, in the cough syrup deaths case, a court in Uzbekistan has sentenced 23 people to prison. The cough syrups were made by India’s Marion Biotech.In other news, New York’s attorney general has urged the FDA to address safety risks associated with the widely used asthma drug Singulair due to its neuropsychiatric side effects, particularly in minors.In trials, Zealand Pharma and Boehringer Ingelheim’s obesity drug has aced its primary and secondary endpoints in a mid-stage trial in treating a liver disease — metabolic dysfunction-associated steatohepatitis (MASH). And Viking Therapeutics’ experimental obesity drug has helped patients lose 15 percent weight in a mid-stage trial.BMS to raise reliance on India for R&D, expands drug substance facility in IrelandBristol Myers Squibb inaugurated a US$ 100 million facility in the Indian city of Hyderabad this week. It expects to make this facility BMS’ largest R&D presence outside of the US by 2025. The unit will use artificial intelligence (AI) and digital technologies to develop drugs, and accelerate the development of cell therapies for autoimmune diseases like multiple sclerosis. It will employ over 1,500 people.Meanwhile, the first drug substance facility of BMS outside of the US, located in Dublin, Ireland, is getting a US$ 400 million boost. The investment will be used to build and design a sterile drug product (SDP) facility. It will also serve as a launch excellence facility for pipeline products. The site will tackle manufacturing across BMS’ oncology, immunology and hematology business. Construction is expected to start in March, with completion expected in 2026.FDA to step up inspections of manufacturing units in India, says ReutersThe country director of the FDA Sarah McMullen told Reuters that the US agency is set to increase the number of inspections of drug manufacturing units in India this year. Requests have been made for more drug investigators to be stationed in the country.The Indian government has been pushing drug manufacturers to implement good manufacturing practices that are current to global standards. However, McMullen said they need to invest more in automation for better compliance and that the country’s drug regulatory body needs more resources.Takeda ties up with India’s Biological E to increase access to its dengue vaccineJapanese drugmaker Takeda has entered into a strategic partnership with Indian drugmaker Biological E to accelerate the access to its dengue vaccine Qdenga. These doses will ultimately be made available for procurement by governments in endemic countries by 2030 to support their national immunization programs. Biological E will ramp up manufacturing capacity to up to 50 million doses a year, in order to accelerate Takeda’s efforts to manufacture 100 million doses a year by 2030. Meanwhile, a Reuters news report said Takeda is in talks with Indian regulators over making Qdenga available in the country.Uzbekistan court serves prison terms to 23 over cough syrup deathsA court in Uzbekistan has sentenced 23 people to prison over the deaths of 68 children linked to contaminated cough syrup. The cough syrup in question was made by Marion Biotech in India and distributed by Quramax Medical in the Central Asian nation. Indian national Singh Raghvendra Pratar, a Quramax director, has received the longest prison sentence of 20 years.The court found the defendants guilty of abuse of office, sale of substandard or counterfeit medicines, forgery, bribery, and tax evasion. The families of the children will receive US$ 80,000 each. The money will be taken from the convicts, the country’s Supreme Court said.Boehringer’s obesity candidate shows promise in mid-stage trial for MASHBoehringer Ingelheim has 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. The drug 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 mellitus.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.Viking’s obesity drug helps patients lose 15 percent weight in mid-stage trialAnother obesity drug showed promise in a mid-stage trial. Viking Therapeutics’ experimental drug helped obese patients lose nearly 15 percent of their body weight after 13 weeks (on average). Moreover, the reduction in weight didn’t plateau, which suggests more weight loss could be achieved by continuing treatment.The news grabbed the attention of Wall Street analysts who said Viking could be in possession of a potential best-in-class agent that threatens the “duopoly” of Novo Nordisk and Eli Lilly.New York attorney urges FDA for stricter warning on asthma drug SingulairThe attorney general for New York has urged FDA to address safety risks associated with a popular asthma and allergy medicine Singulair (montelukast). The attorney said the current warnings on the drug's packaging are insufficient, particularly for children.Thousands of parents and patients have linked the drug to psychiatric problems, including hallucinations, anxiety, and rage. After a hearing in 2019, FDA had mandated a boxed warning, the most stringent drug warning, for Singulair. However, the New York Times found that parents were still unaware of its side effects, which include suicide or suicide attempts.  

Impressions: 1247

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#Phispers by PharmaCompass
29 Feb 2024
FDA okays Iovance’s cell therapy for skin cancer, issues warning letter to China’s Sichuan Deebio
The United States Food and Drug Administration (FDA) has granted accelerated approval to Iovance’s cell therapy — Amtagvi (lifileucel) — for treating adult patients with unresectable or metastatic melanoma (a kind of skin cancer). The agency also expanded the label of Roche and Novartis’ Xolair to help people with food allergies avoid severe reactions following accidental exposure.Following a priority review, FDA approved AstraZeneca’s blockbuster cancer therapy Tagrisso (to be used in combination with chemotherapy) for treating adults with a type of advanced lung cancer.In regulatory news, Chinese API maker Sichuan Deebio was hit with an FDA warning letter after it failed to ensure data integrity and significantly deviated from current good manufacturing practices (cGMPs).When it comes to managing HIV, developing long-acting treatments has become a major focus for drugmakers. In a late-stage trial, GSK’s long-acting injection for HIV, Cabenuva, worked better than daily pills for patients with a challenging history of adherence.Iovance’s groundbreaking T-cell therapy scores FDA’s accelerated approvalFDA has granted accelerated approval to Iovance Biotherapeutics’ cell therapy Amtagvi (lifileucel) for treating the deadliest form of skin cancer. Amtagvi is the first and the only one-time, individualized T cell therapy to receive FDA approval for a solid tumor cancer. It has been greenlit for adult patients with advanced melanoma whose cancer has spread to other parts and is beyond surgery. As part of the therapy, the patient’s own T cells (white blood cells that fight disease) are deployed with tumor-infiltrating lymphocytes (TIL).Amtagvi will come with a boxed warning, the highest safety warnings, for treatment-related mortality. Iovance doesn't see the serious warning impacting sales of Amtagvi, and hopes to start reporting noteworthy revenue in the second quarter of 2024. The mid-cap company expects to list the drug in the US at US$ 515,000 per patient.FDA okays Xolair as first med to prevent severe food allergy reactionsRoche and Novartis’ Xolair (omalizumab) has become the first med approved by the FDA 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. A study sponsored by the National Institutes of Health showed that 68 percent of patients with peanut allergies tolerated half a teaspoon (600 milligrams) of peanut butter compared to around 6 percent in the placebo group. Similar results were seen among participants with milk, egg, tree nuts, and wheat allergies. Xolair is already approved to treat allergy triggered asthma, chronic inflammatory sinus disease with nasal polyps, and chronic hives.Chinese API maker Sichuan Deebio hit with warning letter over cGMP violationsWhen the FDA comes around for an inspection and asks for test results, “I haven't written it yet,” and “it's in my head,” are definitely not the answers the agency is looking for from a quality control (QC) personnel. This, and other failures on the part of Sichuan Deebio, prompted FDA to issue a warning letter to the Chinese API manufacturer this week. The agency has chided Sichuan Deebio for “significant deviations from” cGMPs.FDA found that the company failed to ensure data integrity after a QC leader embarked on a series of lies over whether results had been read and recorded as per established procedures.A Form 483 was issued following the September 2023 inspection. With this warning letter, the FDA has given the company 15 working days to address the deviations and prevent their recurrence. In case the company fails to do that, FDA could withhold approvals and block US imports from the facility, the agency said.FDA approves AstraZeneca’s Tagrisso-chemo combo to treat advanced lung cancerAstraZeneca’s best-selling drug Tagrisso (osimertinib) has been approved by the FDA for the treatment of adult patients with locally advanced or metastatic epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC). The approval follows a priority review by the agency, and is based on results from the Flaura2 phase 3 trial published in The New England Journal of Medicine. The trial showed that the Tagrisso-chemo combo further reduced the risk of disease progression and death by 38 percent. This makes it a critical new treatment option for patients with advanced EGFR-mutated NSCLC.In another late-stage trial, christened Laura, Tagrisso demonstrated a statistically significant and “highly clinically meaningful improvement” in progression-free survival (PFS) for patients with unresectable, stage 3, EGFRm NSCLC after chemoradiotherapy (CRT) compared to placebo after CRT.Sanofi’s frexalimab shows promise in treating relapsing multiple sclerosisIn a phase 2 trial, Sanofi’s frexalimab significantly slowed disease activity in people with relapsing multiple sclerosis (MS), according to a paper published in The New England Journal of Medicine. 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.Fast track designation for Certa’s scleroderma drug: Australia’s Certa Therapeutics won FDA’s fast track designation for its investigational therapy FT011 for the treatment of systemic sclerosis (scleroderma). In a mid-stage study, scleroderma patients saw a 60 percent improvement with FT011 compared with 10 percent in the placebo group.GSK’s HIV injection performs better than pills in late-stage trialGSK said interim results of a late-stage trial showed that its long-acting injection for HIV, Cabenuva (cabotegravir + rilpivirine), worked better than daily pills for some patients. Participants who had a challenging history with adherence when it came to oral antiretroviral treatment, were better treated with the once-a-month regimen. Lack of consistent adherence due to access or other reasons causes many patients to struggle with keeping the virus in check. The British drug giant generated £ 6.44 billion (US$ 8.13 billion) in revenue from sales of HIV meds in 2023.  

Impressions: 873

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#Phispers by PharmaCompass
22 Feb 2024
Gilead acquires CymaBay for US$ 4.3 billion; Exscientia sacks founder CEO Andrew Hopkins
In this week’s Phispers, Gilead boosted its liver portfolio with the US$ 4.3 billion acquisition of CymaBay Therapeutics. The buyout included a potential blockbuster — seladelpar — being investigated to treat primary biliary cholangitis (PBC), a debilitating liver condition.British biotech Exscientia’s founder and CEO Andrew Hopkins, who was recently named a top AI innovator by Time magazine, has been sacked over his “inappropriate” relationships with two employees.After being threatened with subpoenas, the CEOs of Merck and Johnson & Johnson joined their Bristol Myers Squibb counterpart at a Senate hearing committee on why drug prices in the United States are substantially higher than in other countries. The Big Pharma heads managed to walk away without making any promises to lower drug prices.A federal judge has dismissed the Pharmaceutical Research and Manufacturers of America (PhRMA) lawsuit that had termed drug price negotiations via the Inflation Reduction Act (IRA) “unconstitutional”.Data published ahead of a global conference on novel RSV therapies has shown that the efficacy of Moderna’s mRNA-1345 vaccine dwindled faster than its rival shots by GSK and Pfizer.The US Food and Drug Administration (FDA) has approved Takeda’s Eohilia as the first oral treatment for an allergic inflammation of the esophagus. The Japanese drugmaker had all but given up on the candidate, which it got through its US$ 62 billion acquisition of Shire.Gilead boosts its liver portfolio with US$ 4.3 billion acquisition of CymaBayGilead Sciences has entered into an agreement to acquire CymaBay Therapeutics for US$ 4.3 billion. California-based CymaBay has developed a potential blockbuster liver drug — seladelpar — that analysts estimate could generate revenue of US$ 1.9 billion by 2029. Seladelpar has a Prescription Drug User Fee Act (PDUFA) target date of August 14. Gilead said the treatment complements its existing liver portfolio. Seladelpar is used to treat primary biliary cholangitis (PBC), which causes debilitating fatigue, progression of which can lead to liver-related death.BMS, Merck, J&J’s CEOs grilled by US Senate over high drug pricesThe Senate Committee on Health, Education, Labor, and Pensions (HELP) grilled the CEOs of Bristol Myers Squibb, Merck, and Johnson & Johnson as to why drug prices in the US are considerably higher than in other countries. However, the committee failed to elicit any promises to lower drug prices. The top executives stuck to their guns with Merck’s CEO saying Keytruda (pembrolizumab) is approved in the US for 39 indications compared to just over 20 in Europe and perhaps lesser in Japan. He said that’s because those markets are “unwilling to support innovation.” Generally, the three CEOs argued the extra costs meant that Americans can access cutting-edge medicines months, sometimes years, ahead of the rest of the world.Lawsuit against IRA price negotiations dismissed: A federal judge in Texas has tossed a lawsuit filed by PhRMA and two other organizations that argued the IRA price negotiations violated Eighth Amendment rights (excessive fines) under the US Constitution. The judge found that the plaintiffs lacked standing. This is seen as a victory for the Biden administration given that this was the first time a court has dismissed a price negotiation challenge outright. Eight other such lawsuits have been filed.Exscientia sacks CEO over ‘inappropriate’ relationships with two employeesAI-focused biotech Exscientia has fired its founder and CEO Andrew Hopkins a little over a month after they celebrated him being appointed Commander of the Order of the British Empire. The Oxford drug company said it sacked Hopkins after an investigation found Hopkins had engaged in relationships with two employees that were “inappropriate and inconsistent” with its standards and values. Chief Science Officer Dave Hallett was appointed interim CEO. Hopkins was named one of the top AI innovators in a Time 100 list. His wife is also employed with Exscientia.Efficacy of Moderna’s RSV vaccine dwindled faster than those of GSK, Pfizer, implies dataData published ahead of a global conference on novel RSV therapies showed the efficacy of Moderna’s mRNA-1345 vaccine dwindled faster than its rival shots by GSK and Pfizer. Moderna’s shot, which had an efficacy of 84 percent at 3.3 months, fell to 63 percent by 8.6 months. Meanwhile, GSK’s Arexvy, which had an efficacy of 83 percent in the prevention of RSV, fell to 77 percent at 14 months. Pfizer’s vaccine had an efficacy of 89 percent, which fell to 79 percent in the middle of the second RSV season, experts said.Moderna cautioned against reading too much into the data. “In the absence of head-to-head clinical trials, comparative conclusions regarding the safety and efficacy of mRNA-1345 relative to other RSV vaccines cannot be made,” it said.Takeda’s once-snubbed Eohilia becomes first FDA-approved oral therapy for esophageal conditionIn the US, FDA has 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. Takeda had all but abandoned the drug since. In January, Sanofi and Regeneron’s jab Dupixent pipped Eohilia to become the first FDA drug approved for EoE.The chronic condition is caused by a build up of certain white blood cells in the esophagus lining resulting in inflammation and tissue injury. This 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.Ipsen’s Onivyde okayed as first-line pancreatic cancer treatment: FDA has approved Ipsen’s Onivyde (irinotecan liposome injection) as part of a four-drug regimen, first line of treatment for metastatic pancreatic adenocarcinoma (mPDAC). A late-stage trial showed the regimen Nalirifox was significantly superior to the current standard of care. The Pancreatic Cancer Action Network (PanCAN) welcomed the decision “as it has been more than 10 years since there has been a PDAC-specific approval in the first-line setting.” Pancreatic cancer is hard to treat and has the lowest five-year survival rate of all cancers.First med for severe frostbite approved: FDA has approved Eicos Sciences’ Aurlumyn (iloprost) as the first ever medication to treat severe frostbite. The injection will help prevent amputation of fingers and toes, the agency said.  

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https://www.pharmacompass.com/radio-compass-phisper/gilead-acquires-cymabay-for-us-4-3-bn-exscientia-sacks-founder-ceo-andrew-hopkins

#Phispers by PharmaCompass
15 Feb 2024