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.  

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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).  

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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: 1154

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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: 798

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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.  

Impressions: 1233

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#Phispers by PharmaCompass
15 Feb 2024
Novo’s parent buys Catalent for US$ 16.5 bn, Lilly calls for scrutiny; Novartis acquires MorphoSys
The Novo Nordisk-Eli Lilly rivalry over obesity drugs got intensified with Novo Nordisk’s parent company acquiring contract development and manufacturing organization (CDMO) Catalent for US$ 16.5 billion. Lilly has called for regulatory scrutiny of the deal as it relies on Catalent for its diabetes and obesity meds.Novartis has acquired German biopharma MorphoSys for US$ 2.9 billion, thereby gaining its hands on a promising rare bone-marrow cancer treatment — pelabresib.In news from clinical trials, Amgen has a promising weight loss drug in the works — MariTide — which could prove more effective than Novo’s Wegovy and Lilly’s Zepbound. In two separate trials, Johnson & Johnson’s experimental drug — nipocalimab — showed promise in treating myasthenia gravis and reducing the severity of Sjögren’s disease (SjD). Both are immune disorders. And FDA has asked Gilead to pause all blood cancer trials of its drug magrolimab due to increased risk of death.Novo’s parent buys Catalent for US$ 16.5 bn; to sell three sites to drugmakerNovo Nordisk’s parent company, the Novo Nordisk Foundation, is acquiring Catalent through its investment arm Novo Holdings for a total of US$ 16.5 billion. Novo Holdings will then sell three of Catalent’s “fill-finish” sites to Novo Nordisk for US$ 11 billion. The deal is expected to allow the Danish drugmaker “to serve significantly more people living with diabetes and obesity.”Novo Nordisk doesn’t expect any antitrust issues with the deal as it is acquiring only three of the roughly 50 sites Catalent operates. These sites are in Bloomington (Indiana, the US), Brussels (Belgium), and Anagni (Italy).Lilly calls for scrutiny: Eli Lilly has called for scrutiny by competition regulators over the US$ 16.5 billion Novo Holdings-Catalent deal. CEO David Ricks has called the deal “unusual”, and the company plans to hold Catalent accountable to its contract. Lilly relies on Catalent to produce some of its diabetes and obesity treatments that compete with Novo’s Ozempic and Wegovy. A report published in The Wall Street Journal said Eli Lilly executives are worried about the deal, which seeks to expand Novo Nordisk’s weight-loss drug production and might hinder their own capability to get drugs to customers. Both Novo and Lilly have been struggling to meet the demand for their weight loss meds, and both have been working on expanding their manufacturing capacities.FDA finds quality lapses, ‘pest’ at Catalent plant: Reuters has obtained an FDA report on Catalent’s Bloomington (Indiana) factory that says it had recorded 194 deviations between October 31, 2021 and October 31, 2023. Catalent had failed to identify the root cause of 171 of those incidents, it added. The agency also found a “pest” on the manufacturing line. FDA had conducted an inspection last year (between October 31 and November 15) that noted five separate observations, including failure to thoroughly review unexplained discrepancies in certain batches.Novartis buys MorphoSys for US$ 2.9 bn, gains promising bone-marrow cancer medNovartis is acquiring cancer treatment developer MorphoSys for € 2.7 billion (US$ 2.9 billion). With this, the Swiss drugmaker’s pipeline gets a rare bone-marrow cancer treatment — pelabresib — that holds plenty of promise. The drug is in late-stage trials.AbbVie raises 2027 sales forecasts for Skyrizi, Rinvoq: AbbVie has lifted its 2027 sales forecast for Skyrizi (risankizumab) and Rinvoq (upadacitinib) by US$ 6 billion to US$ 27 billion. Its 2024 sales forecast for the two immunology drugs is US $16 billion – US$ 10.5 billion for Skyrizi and US$ 5.5 billion for Rinvoq. The drugmaker has been turning to its newer immunology drugs to counter declining sales of its blockbuster arthritis medicine Humira (adalimumab).Amgen’s weight-loss drug shows impressive results in early-stage trialAn early-stage study suggested that Amgen’s experimental weight-loss drug MariTide (maridebart cafraglutide) could have longer-lasting effects than popular glucagon-like peptide-1 medicines, such as Novo Nordisk’s Wegovy and Eli Lilly’s Zepbound.After 12 weeks on the highest MariTide dose, obese patients without diabetes saw a 14.5 percent reduction in body weight. Even patients on the lowest dose lost 7.4 percent weight after just three doses. This compares to Zepbound and Wegovy that showed weight loss of 21 percent and 15 percent, respectively, in trials conducted over a year.J&J’s closely watched autoimmune drug succeeds in mid, late-stage trialsOne of Johnson & Johnson’s most watched experimental drugs saw positive mid and late-stage results in 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. The data further supports J&J’s expectations of over US$ 5 billion for the drug.4DMT posts positive mid-stage data for eye therapy: 4D Molecular Therapeutics released mid-stage interim data that showed its gene therapy, 4D-150, was well tolerated and effective in patients with wet age-related macular degeneration (wet AMD). Due to frequent injections required for the condition, the treatment burden on patients is high. The high-dose version of 4D-150 reduced standard-of-care injections required by patients by 89 percent. Furthermore, 63 percent of the 51 participants were injection-free through 24 weeks.FDA pauses Gilead’s blood cancer drug trials over increased risk of deathGilead 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). The company will discontinue testing the drug for all blood cancers and review its safety in studies concerning the treatment of colon and breast cancers. Magrolimab was the lead candidate of Gilead’s US$ 4.9 billion acquisition of Forty Seven in 2020.New reporting norms for drugmakers: FDA is giving drugmakers and others involved in medicine manufacturing three weeks to comply with new reporting requirements set up to help the agency mitigate possible drug shortages. The obligations are part of the Coronavirus Aid, Relief, and Economic Security (CARES) Act passed in March 2020. On February 5, the agency had published a final guidance on how drugmakers must report the amount of each listed drug manufactured, prepared, propagated, compounded, or processed for commercial distribution.  

Impressions: 681

https://www.pharmacompass.com/radio-compass-phisper/novo-s-parent-buys-catalent-for-us-16-5-bn-lilly-calls-for-scrutiny-novartis-acquires-morphosys

#Phispers by PharmaCompass
08 Feb 2024
Vertex’s non-opioid painkiller succeeds in late-stage trials, FDA finalizes CAR-T guidance
In Phispers this week, there is news of an experimental non-opioid painkiller from Vertex Pharmaceuticals showing positive results in two late-stage trials. Once approved, it will provide a viable alternative to addictive opioids that have led to thousands of deaths and a crisis in America.In other news from clinical trials, BMS’ Opdivo failed to meet its primary endpoint of disease-free survival in patients with localized renal cell carcinoma who have a high risk of relapse after surgery. A day earlier, Merck’s Keytruda showed remarkable clinical benefit for the very same indication.After mandating a serious “boxed warning” on several CAR-T therapies last week, the United States Food and Drug Administration (FDA) has  finalized a guidance on how sponsors may go about getting such therapies approved.Pfizer managed to surprise markets by eking out a profit of US$ 593 million in the fourth quarter (Q4) of 2023 when analysts were expecting a 10-figure loss.In deals, 2seventy Bio has decided to sell all its preclinical and clinical programs to Regeneron. It will now focus on commercializing its CAR-T therapy Abecma. Meanwhile, Biogen has ended a post-approval study of its Alzheimer’s drug Aduhelm to focus on its other drug for the disease — Leqembi — which bagged FDA approval last year.Vertex’s non-opioid painkiller succeeds in two late-stage trialsVertex 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.The opioid crisis in America has claimed over 645,000 lives since 1999. While opioids work by activating the reward centers in the brain, VX-548 works by blocking pain signals at the source.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.FDA finalizes CAR-T guidance; gene therapies eligible for accelerated approvalCell therapies have been facing a rough period with narrower funding options that have forced many startups in the biotech sector to shut shop. Last week, FDA issued letters to six manufacturers of cancer therapies that use CAR-T technology to add a boxed warning on their label after the agency found a serious risk of developing secondary cancer associated with them. This warning may raise further concerns over CAR-T therapies.But now, FDA has stepped in and 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. FDA also said gene editing therapies are eligible for accelerated approval and detailed the information that should be provided in an investigational new drug (IND) application.Approves faster manufacturing of Yescarta: FDA has approved a new manufacturing process for Gilead-owned Kite Pharma’s Yescarta (axicabtagene ciloleucel). The turnaround time for the therapy has now been reduced to 14 days from 16. This gives it an edge over its competitor, BMS’ Breyanzi (lisocabtagene maraleucel), which targets a turnaround time of 24 days.Regeneron buys 2seventy bio’s pipeline: Cancer cell therapy company 2seventy bio has decided to sell all its preclinical and clinical programs to Regeneron. It will now focus on its sole approved product Abecma (idecabtagene vicleucel). About 160 2seventy bio employees who support the acquired programs will join Regeneron Cell Medicines, a newly formed research and development unit to advance cell therapies and combination approaches in oncology and immunology.      Pfizer ekes out surprise Q4 2023 profit after announcing thousands of job cutsPfizer’s full year results for 2023 were being watched closely, given that its record US$ 100 billion revenue for 2022 wasn’t sustainable with the pandemic receding. The New York-based drugmaker’s 2023 revenue fell 42 percent year-on-year to US$ 58.5 billion due to a sharp drop in Comirnaty and Paxlovid sales. Furthermore, its Q4 2024 revenue of US$ 14.25 billion was lower than Wall Street predictions of US$ 14.42 billion. Yet, Pfizer managed to eke out a profit of US$ 593 million in Q4, when analysts were expecting a US$ 1.1 billion loss.Pfizer has been tightening its belt for sometime now, and had announced over a thousand job cuts on both sides of the Atlantic. The drug behemoth says it’s on track to deliver at least US$ 4 billion in yearly cost savings by the end of 2024, thanks to its “cost realignment program.” That said, Pfizer also saw a record nine molecules being approved by the FDA in 2023. It also won seven novel drug approvals last year, more than double the number of any other company.Novavax to reduce workforce by 12 percent: Another drugmaker facing post-Covid blues is Novavax. It plans to reduce its workforce by 12 percent, bringing its total reduction in workforce since Q1 2023 to 30 percent. The cash-strapped biotech is now concentrating its efforts on an updated Covid vaccine and a Covid and flu combination vaccine.BMS’ Opdivo disappoints, Merck’s Keytruda scores win in kidney cancer trialsBMS has said its PD-1 blocker Opdivo (nivolumab) has failed to meet its primary endpoint of  disease-free survival in a late-stage trial in patients with localized renal cell carcinoma who have a high risk of relapse after surgery. The difference between the Opdivo group and the placebo group in terms of efficacy was not noteworthy and the treatment-related adverse events in the former were more. Interestingly, a day prior to this announcement, its rival Merck’s blockbuster PD-1 inhibitor Keytruda (pembrolizumab) showed remarkable clinical benefit for the very same indication. Opdivo as a subcutaneous form, however, did demonstrate non-inferior efficacy and safety compared to its intravenous formulation.Biogen ends post-approval study on Aduhelm to focus on LeqembiEisai-Biogen’s first drug to treat Alzheimer’s disease, Aduhelm, faced problems soon after it bagged FDA’s accelerated approval in 2021 with questions being raised about the evidence of patient benefit. Biogen abandoned the drug in 2022 after Medicare limited its coverage. Now, Biogen is ending a post-approval study of Aduhelm, and will focus on its other Alzheimer's medicine Leqembi, also developed along with Eisai. Leqembi bagged FDA approval last year, and has the ability to slow cognitive decline in early Alzheimer's patients.  

Impressions: 742

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#Phispers by PharmaCompass
01 Feb 2024
Six CAR-T therapies to carry boxed warning for secondary cancer; Sanofi buys Inhibrx for about US$ 2.2 billion
The US Food and Drug Administration (FDA) has mandated “black box” warnings on six major CAR-T cancer therapies over the risk of developing secondary cancer.Two experimental gene therapies that work on mutations of the OTOF gene reportedly cured profound deafness in young children.The Biden Administration has urged the US Supreme Court to reverse an August 2023 ruling that curbs access to abortion pill mifepristone by barring telemedicine prescriptions and mail shipments of it.Eli Lilly’s New Jersey plant is back in the news, as the FDA reportedly found “eight separate deficiencies” concerning manufacturing processes and quality controls.Johnson & Johnson’s Stelara raked in over US$ 10 billion last year. Meanwhile, the drugmaker agreed to resolve talc probes in 42 states in the US through a tentative settlement of US$ 700 million.In acquisitions, Sanofi forked out US$ 2.2 billion to buy Inhibrx, a clinical-stage biopharma, to gain access to its experimental treatment for a rare disease.FDA mandates ‘boxed warning’ on six CAR-T therapies over secondary cancer riskFDA has issued letters to six manufacturers of cancer therapies that use CAR-T technology to add a serious warning on their label (i.e. boxed warning), after the agency found a serious risk of developing secondary cancer associated with those therapies.These therapies include Bristol-Myers Squibb’s Abecma and Breyanzi, Janssen Biotech’s Carvykti, Gilead’s Yescarta, Novartis’ Kymriah, and Kite Pharma’s Tecartus.“Boxed warnings” or “black box warnings” are reserved for the highest safety warnings to bring attention to the major risks of a treatment.Amgen’s osteoporosis drug to carry boxed warning: Amgen’s blockbuster drug Prolia to treat bone loss was also hit with a boxed warning requirement as it raises the risk of severe hypocalcemia (low calcium levels) in patients with advanced chronic kidney disease (CKD), especially those on dialysis.Biden urges Supreme Court to undo curbs on major abortion pillIn June 2022, the US Supreme Court had ended its recognition of a constitutional right to abortion by overturning an earlier ruling (of 1973). And later that year, an umbrella group of anti-abortion doctors and activists in the US had claimed that the FDA had unlawfully approved Danco Laboratories’ mifepristone.Now, the Biden Administration has urged the apex court to reverse an August 2023 ruling that curbs access to the pill by barring telemedicine prescriptions and mail shipments of it.The apex court’s ruling in this case is expected by June-end, months before the November presidential elections.The pill had been legal in the US since 2000. The two drug-regimen was approved for use in pregnancies of up to 10 weeks. A patient is first given mifepristone to induce an abortion, followed by misoprostol to empty the uterus. Over the last two decades, the FDA, the American College of Obstetrics and Gynaecologists (ACOG) and other mainstream medical bodies have maintained that both drugs are safe.Since 2016, the FDA had further eased access to the drug by allowing doctors to meet patients via telemedicine, and issue prescriptions over email.Lilly, Refreshgene’s gene therapies cure congenital deafness in clinical trialsThe week brought some good news for those with congenital deafness. According to Reuters, an experimental gene therapy being developed by a Chinese company restored hearing in children with congenital deafness. Five of six young children with profound deafness experienced hearing recovery and improvements in speech recognition six months after being treated with the therapy from Refreshgene Therapeutics. All the children had profound deafness caused by mutations of the OTOF (otoferlin) gene.In another early-to-mid-stage trial of Eli Lilly’s investigational gene therapy AK-OTOF, the hearing of an 11-year-old boy was restored. The boy was born deaf. The boy could hear within 30 days of a single administration of AK-OTOF.After 2021 DOJ probe, fresh manufacturing, quality lapses found at Lilly’s NJ plantEli Lilly’s New Jersey plant, which was subject to a Department of Justice (DOJ) probe back in 2021, has now been found to have several manufacturing problems. According to a Reuters report, the FDA has detected “eight separate deficiencies” at the plant. The issues included problematic tracking of manufacturing processes and quality controls. There were also lapses in the calibration of equipment and the company failed to appropriately maintain facilities and equipment.The Indianapolis-based drugmaker told Reuters the inspection took place in July, after it had asked FDA for “additional flexibility” in the manufacturing process for migraine treatment Emgality (galcanezumab-gnlm). Drugs manufactured at the facility include diabetes medicine Trulicity (dulaglutide), and cancer therapies Cyramza (ramucirumab) and Erbitux (cetuximab). Lilly has said safety, quality, and supply of current or planned products remain unaffected.J&J to pay US$ 700 million to settle talc probe in 42 US statesJohnson & Johnson confirmed it has reached a tentative settlement of US$ 700 million to resolve probes by US states into whether it misled consumers about the safety of its talcum powder. The deal includes 42 states and Washington DC. The settlement does not extend to private plaintiffs’ cases against the company, some of which are expected to go to trial later this year.Stelara crosses US$ 10 billion in 2023 sales: J&J’s blockbuster Stelara (ustekinumab) clocked sales of US$ 10.86 billion in 2023. The psoriasis drug’s sales are expected to drop 3 percent in 2024 to US$ 10.54 billion. The first biosimilar for the drug, Amgen’s Wezlana (ustekinumab-auub), is expected to be launched in 2025.Sanofi buys rare disease drugmaker Inhibrx for about US$ 2.2 billionSanofi 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, known as Inhibrx, in which  Sanofi will hold an 8 percent stake. INBRX-101 is a potential treatment for Alpha-1 Antitrypsin Deficiency (AATD), a rare disease that causes the lung tissue to progressively deteriorate.Meanwhile, there was news that the Walgreens Boots Alliance is looking into the possibility of selling its specialty pharmacy company Shields Health Solutions for over US$ 4 billion.  

Impressions: 2093

https://www.pharmacompass.com/radio-compass-phisper/six-car-t-therapies-to-carry-boxed-warning-for-secondary-cancer-sanofi-buys-inhibrx-for-about-us-2-2-billion

#Phispers by PharmaCompass
25 Jan 2024
Lilly to launch Zepbound in more markets this year; Casgevy bags second FDA nod
This week’s Phispers brings you news from the annual World Economic Forum in Davos (Switzerland) where some senior executives of pharmaceutical companies discussed their growth plans for 2024. A top Eli Lilly executive said the company plans to launch Zepbound in several countries this year, and is “optimistic” about the pen injector gaining European approval by mid-2024. And, chairman of Dr. Reddy’s Laboratories said the company is planning to scale up its reliance on artificial intelligence (AI) to drive innovation.The US Food and Drug Administration (FDA) expanded the indications of three important drugs. Less than a month after CRISPR-Vertex’s Casgevy saw its first FDA approval for sickle cell disease, the agency approved it for β-thalassemia, another inherited rare blood disorder. The approval comes two months ahead of its Prescription Drug User Fee Act (PDUFA) date of March 30. Similarly, Takeda’s HyQvia has won a second approval for a rare nerve disorder and Merck’s Keytruda continues to score expanded approvals, this time for a type of advanced cervical cancer (when used in combination with chemotherapy).In deals-related news, there were reports that Novartis is no longer the frontrunner for acquiring Cytokinetics, and has possibly been replaced by Amgen. And, after spinning off Haleon in February 2022, GSK managed to raise an additional US$ 1.24 billion this week by selling more of its stake in this consumer health business.Lilly plans to launch weight loss drug Zepbound in new markets this yearSpeaking at Davos 2024, Ilya Yuffa, president of Lilly International, told Reuters that the company plans to launch its weight-loss drug Zepbound (tirzepatide) in new countries this year. The company is already working on expanding the manufacturing capacity for its diabetes and weight loss drugs.Zepbound saw approvals in the UK, US, and Europe in the last quarter of 2023. While it has been launched in the US market, individual countries in Europe have a say in whether it is okay for their populations.The Indianapolis-based drugmaker, however, is “optimistic” that Zepbound’s pen injector will win European approval in the first half of 2024. As of now, its vials have been approved. After launching its direct-to-customer website — Lilly Direct — in the US in January, Lilly is weighing a direct-to-patient model for individual European markets based on the regulations in each country.Dr. Reddy’s banks on AI to drive innovation: Davos 2024 also saw Dr. Reddy’s Laboratories’ Co-Chairman and Managing Director GV Prasad talk about how the Indian drugmaker is leveraging AI to scale up research and development. He said the company is using “machine learning to understand how scale ups are done,” making its R&D a lot more productive. It’s also being used to gauge how “products will behave in the patient.”The company is looking at AI not just for productivity but also to drive innovation by reducing the amount of experimentation, and to bring about more precision. He said Internet of Things (IoT) has become an integral part of the generic company’s operations.CRISPR-Vertex’s Casgevy bags second FDA approval for genetic blood disorderVertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy has bagged a second FDA approval, this time for transfusion-dependent β-thalassemia (TDT), a rare genetic blood disorder that routinely requires blood transfusions. The approval, which applies to patients over the age of 12 years, comes over two months prior to its PDUFA date of March 30. Analysts estimate peak sales of Casgevy for both indications — β-thalassemia and sickle cell disease — to be around US$ 400 million.Takeda’s HyQvia wins second approval for rare nerve disorder: While Casgevy bagged a second FDA approval in two months, it took Takeda’s HyQvia ten years to bag another approval from the agency. A decade back, HyQvia (immune globulin infusion 10% with recombinant human hyaluronidase) was approved as a treatment for primary immunodeficiency in adults. And this week, it was okayed as a maintenance therapy for chronic inflammatory demyelinating polyneuropathy (CIDP) to prevent the relapse of neuromuscular disability and impairment in adults. CIDP is rare autoimmune disorder wherein the body attacks the fatty coverings that insulate and protect the nerves. Importantly, this subcutaneous infusion therapy requires just one dose a month. With the right training, it can be self-administered, the company said.Keytruda granted expanded approval for cervical cancer: One drug that’s no stranger to getting FDA’s expanded use approvals is Merck’s blockbuster immunotherapy Keytruda, which now has no fewer than 39 approvals in the US. Now, Keytruda (pembrolizumab) administered in combination with chemoradiotherapy has been approved for newly diagnosed patients with a certain kind of advanced cervical cancer, making it the first anti-PD-1-based immunotherapy combination for patients with FIGO 2014 Stage III-IVA cervical cancer.Novartis drops pursuit of Cytokinetics, Amgen maybe new frontrunner, say reportsLast week, there was news that Novartis was in talks to acquire Cytokinetics for well over US$ 10 billion after its experimental heart disease drug — aficamten — pulled off quite a performance and met its main goal in a late-stage trial. This potentially pits aficamten against Bristol Myers Squibb’s Camzyos (mavacamten).But this week, Wall Street Journal has reported that Novartis has backed away from the deal.However, the San Francisco-based biopharma is not short of suitors. According to a report published in Seeking Alpha, the new frontrunner maybe Amgen.GSK sells off another stake in Haleon, raises US$ 1.24 billionGSK has raised £ 978 million ($US 1.24 billion) by selling off more stake in Haleon, the biggest consumer healthcare business in the world with brands like Panadol, Advil and Sensodyne. The British drugmaker offloaded 300 million shares at a discounted price, reducing its stake in Haleon to 4.2 percent. It had originally retained 12.9 percent in Haleon. This is the third time in under a year that GSK has sold a stake in this business.  

Impressions: 660

https://www.pharmacompass.com/radio-compass-phisper/lilly-to-launch-zepbound-in-more-markets-this-year-casgevy-bags-second-fda-nod

#Phispers by PharmaCompass
18 Jan 2024
FDA allows Florida to import drugs from Canada; India mandates new GMP standards for drugmakers
In this week’s Phispers, the United States Food and Drug Administration (FDA) has allowed the state of Florida to import prescription drugs from Canada. In the aftermath of deaths in The Gambia, Uzbekistan and Cameroon in 2022 and 2023 that were linked to India-manufactured cough syrups, the Indian government has announced new manufacturing standards for drugmakers.In the ever-expanding obesity market, Eli Lilly has launched a website and Novo Nordisk has announced strategic collaborations with two research biotech firms to come up with the next generation of glucagon-like peptide 1 (GLP-1) products. Meanwhile, Merck has said its GLP-1 strategy is to come up with treatments with benefits beyond weight loss.The M&A space saw yet another acquisition related to antibody-drug conjugates (ADCs) — Johnson & Johnson bought Ambrx Biopharma for about US$ 2 billion. AbbVie has signed two deals with Umoja Biopharma for up to US$ 1.44 billion for novel cancer therapies. And, GSK has snapped up asthma drugmaker Aiolos Bio for US$ 1 billion. Moreover, there is buzz around Novartis looking to buy San Francisco-based Cytokinetics that may result in an over US$ 10 billion deal.Florida becomes first US state to get FDA okay to import drugs from CanadaIn a major policy shift, FDA has okayed Florida’s request to import prescription drugs from Canada, citing a 2021 Joe Biden executive order. The Sunshine State estimates the move could save taxpayers US$ 150 million a year. Drug prices in the US are among the highest in the world and over two times more than that of industrialized nations where government healthcare systems negotiate prices of prescription meds. The US drug industry has relentlessly opposed such imports and has termed the approval granted to Florida as a "reckless decision". This policy measure opens the door for other states to follow suit. In fact, FDA said it is committed to helping them do so.Lilly launches website for ordering medicines, connecting with telehealth firmsEli Lilly has launched a website — LillyDirect — where users can directly order its products including weight loss med Zepbound. LillyDirect will also connect patients with independent telehealth providers. This move comes on the back of companies like WeightWatchers and Ro offering weight loss drugs on similar platforms, and analysts say the “model is ideal for obesity medications and drives uptake.”Novo in strategic deals with two biotechs: Novo Nordisk has announced strategic collaborations with two research biotech firms – Omega Therapeutics and Cellarity Inc – to stay ahead of the curve. The deal could lead to next-generation treatments for people living with obesity and a type of liver disease known as MASH (short for metabolic dysfunction-associated steatohepatitis). Moreover, a study of data from over 1.8 million patients debunked the notion that the Danish drugmaker’s semaglutide-based drugs Ozempic and Wegovy are linked to an increase in suicidal thoughts.Merck’s GLP-1 drugs to focus beyond weight loss: Merck CEO Robert Davis has said its strategy with GLP-1 is to come up with treatments with benefits beyond weight loss as this could make it easier to get reimbursements. The New Jersey-based drugmaker said its investigational GLP-1 drug efinopegdutide for non-alcoholic steatohepatitis (NASH), demonstrated "compelling" weight-loss benefit.Post cough syrup deaths, India introduces new GMP standards for drugmakersShaken by over 141 deaths in The Gambia, Uzbekistan and Cameroon that the World Health Organization (WHO) and other authorities have linked to India-made cough syrups, the Narendra Modi government in India has set new manufacturing standards pharmaceutical companies must satisfy this year. Schedule M of the Drugs and Cosmetics Rules prescribes good manufacturing practices (GMPs) for pharmaceutical products. This schedule was revised to ensure companies “do not place patients at risk due to inadequate safety, quality or efficacy.”Products may be marketed only after testing shows “satisfactory results”. Small companies, many of whom are heavily in debt, have asked for an extension after it was found that less than a quarter of 8,500 small drug factories in India’s US$ 50 billion pharma industry met international drug manufacturing standards set by the WHO. The move “would bring our GMP recommendations at par with global standards, especially to those of [WHO],” said the health ministry.Novartis in talks to buy Cytokinetics for over US$ 10 billion, reports WSJLast month, results from a late-stage study of Cytokinetics’ experimental heart disease drug — aficamten — revealed that the drug had met its main goal, making it a potential competitor to Bristol Myers Squibb’s Camzyos (mavacamten). And now, drugmakers are reportedly lining up to buy the company. According to a report published in the Wall Street Journal, Novartis is poised to beat AstraZeneca and Johnson & Johnson and acquire Cytokinetics for well over US$ 10 billion. However, Novartis CEO Vasant Narasimhan is holding his cards close and declined to comment on the news report. He said the Swiss drugmaker’s strategy is to “focus on sub US$ 5 billion assets.J&J boosts cancer pipeline, enters ADC space with US$ 2 bn Ambrx buyoutOne of the major deals announced at the annual J.P. Morgan Healthcare Conference was Johnson & Johnson’s 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. Ambrx’s portfolio includes experimental drugs such as its lead candidate ARX517 for metastatic castration-resistant prostate cancer (mCRPC) and ARX788 for metastatic HER2+ breast cancer.AbbVie inks CAR-T deals with Umoja: AbbVie has signed two in-situ CAR-T deals with Seattle-based Umoja Biopharma for up to US$ 1.44 billion plus tiered royalties. Umoja’s VivoVec platform takes a novel approach that enables T cells in the body to manufacture their own cancer-fighting CAR-T cells in vivo, i.e. within the patient’s body. Traditionally, CAR-T approaches involve extracting the patients’ cells or using donor cells, which are then modified and delivered back to the patients. This new approach could potentially eliminate numerous challenges.GSK buys Aiolos for up to US$ 1.4 billion: GSK has snapped up asthma drugmaker Aiolos Bio for US$ 1 billion and its mid-stage-ready, long-acting antibody AIO-001 which could redefine the treatment of certain respiratory and inflammatory conditions. Aiolos is also entitled to US$ 400 million in milestone payments. 

Impressions: 1068

https://www.pharmacompass.com/radio-compass-phisper/fda-allows-florida-to-import-drugs-from-canada-india-mandates-new-gmp-standards-for-drugmakers

#Phispers by PharmaCompass
11 Jan 2024