Epilepsy, Reflex

Praxis Precision Medicines revealed Phase IIa findings for its experimental PRAX-628 therapy, demonstrating a 100% complete response in the high-dose group of patients with photosensitive epilepsy. The positive readout Tuesday paves the way for Praxis to advance the programme later this year."The strength and consistency of response across both study arms, combined with a continued positive tolerability and safety profile, build on our earlier conviction that PRAX-628 has the potential to be the first precision sodium channel modulator for focal epilepsy patients," said CEO Marcio Souza.The study included 10 epilepsy patients who demonstrated photo paroxysmal response (PPR) during screening, with eight eligible for assessment. PPR studies can reveal abnormal patterns of brain activity in response to flashing or flickering lights. Analysing EEG signatures after intermittent photic stimulation can serve as a gauge for the effectiveness of potential anti-epileptic treatments, according to the company.The study is evaluating two dose levels. Earlier this year, Praxis said a preliminary analysis of the lower 15mg cohort had exceeded its expectations, driving up shares by 23% at the time. On Tuesday, the company said four of the five patients (80%) in that group achieved a complete response, which was defined as the complete elimination of generalised PPR events at any assessment period versus baseline.In the higher 45mg cohort, all three patients (100%) achieved a complete response. The combined response rate was 88% across the two cohorts. In a note prior to the readout, Jefferies analysts said they viewed ≥50% complete/partial suppression as "an important threshold to meet/exceed" in to order to demonstrate proof-of-concept.

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here . Zoom can only go so far. That’s the mindset John Carroll will be bringing to London next week for the first time in several years. If you’re there, consider joining him as he chats with a small crowd of biopharma people — we also have virtual options . Taste of M&A Monday AstraZeneca — specifically its subsidiary Alexion — bought out a struggling gene editing player that just recently overcame a second clinical hold to its only program in human studies. Even at $2.07 per share, the buyout represented a 667% premium for little LogicBio and its best option with dwindling cash. The way Alexion tells it, the rare disease group is much more interested in the biotech’s talent, skillsets and capabilities in genomic medicine than any one project in particular — and all the challenges that LogicBio ran into just means the team is battle tested. A special kind of lab A regarded singer-songwriter, Casey McPherson never imagined going into drug development. Then his daughter, Rose , was diagnosed with an ultra-rare disease that robbed her of the ability to talk, and an intellectual property disagreement prevented Columbia University scientists from tackling her disease. McPherson saw families like his encounter similar barriers and eventually co-founded a first-of-its-kind lab , Everlum Bio , to spark a treatment for Rose — and others like her, Jared Whitlock writes. Biotech founder wins Nobel Carolyn Bertozzi can add “Nobel laureate” to her long list of titles. The Stanford professor and repeat biotech founder shared the Nobel Prize in Chemistry with Morten Meldal of University of Copenhagen and K. Barry Sharpless of Scripps for “elevating chemistry into the era of functionalism.” While Sharpless and Meldal won for their contributions to click chemistry, Bertozzi was credited for taking those findings a step further and finding chemical reactions that can take place within cells — launching a field known as bioorthogonal chemistry. BioMarin layoffs By the time the FDA hands down a decision on its hemophilia A gene therapy, BioMarin will be a different company — literally. The biotech is laying off about 120 employees — 4% of its global workforce — in what it calls an organizational redesign. By simplifying the company structure, it hopes to save $50 million per year. PREMIUM Off-the-shelf CAR-T hits pivotal After calling a lengthy time out on the development race with a clinical hold imposed by the FDA, Allogene finally kicked off a landmark PhII pivotal for its off-the-shelf CAR-T. The single-arm study with 100 patients will test ALLO-501A for large B-cell lymphoma. On top of discussions on dose and CMC, they’re also providing a closeup of the lymphodepletion regimen ALLO-647 . Digital strategy for today’s healthcare Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation. In a Q&A , she talks about how her crew tests, pilots and develops new strategies needed in today’s consumerized healthcare world. DEALS The question has hung around the air for months now: Where will Nektar , the one-time high-flying biotech once best known for its Bristol Myers Squibb alliance, go after the multibillion-dollar IL-2 program fell apart? PureTech revealed that it’s in talks with Nektar about a possible merger , which could bring two platform technologies together. Getting the M&A wheels turning, Incyte shelled out $70 million in cash to acquire Villaris Therapeutics and its lead candidate auremolimab , a preclinical anti-IL-15Rβ monoclonal antibody being investigated in vitiligo. There are also possibilities of going into other autoimmune and inflammatory indications, areas that Incyte has been targeting with its JAK inhibitor. Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant as part of a 5-company, $3 billion deal, it’s coming back for the whole thing . But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage, as the board demands a better proposal than the current $2.5 billion offer. Sanofi is jumped into the antibody-RNA conjugate space and elevated a little-known private biotech into the spotlight by licensing a preclinical ARC candidate from Gaithersburg, MD-based miRecule that’s designed to treat a genetic muscle disease known as facioscapulohumeral muscular dystrophy. Upfront and near-term milestones add up to about $30 million, while additional biobucks could bring the deal’s total value north of $400 million. With its FDA decision one month away, Provention Bio has already begun its commercial efforts for its type 1 diabetes drug — and Sanofi is signing on as a partner . If teplizumab , Provention’s antibody treatment for delaying the onset of symptomatic type 1 diabetes, gets approved in November, Sanofi will invest $35 million upon approval and get dibs on negotiating rights. The latest addition to Bristol Myers Squibb’s protein degrader lineup comes by way of a Celgene connection. SyntheX will be its partner in a multi-target $550 million deal centered on molecular glues. The small team is focused on making the early-stage drug discovery process “a bit more functional,” and it had been talking to scientists at Celgene for the past few years. Pfizer wasn’t kidding around when it said it’s leaving “very few stones unturned” in dealmaking. The pharma giant is paying about $116 million to scoop up University of Queensland spinout ResApp Health , which is developing a smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds. Are SPACs slowly creeping back? Estrella Biopharma , a Bay Area biotech looking to push CAR-T in solid tumors using tech from Eureka , is going public via a merger with TradeUP Acquisition Corp . And if it all goes as planned, the public version of Estrella — dubbed Estrella Immunopharma — will be worth around $398.5 million. Boehringer Ingelheim wants in on Wnt, and it’s willing to cash out the big bucks if it pans out. With an upfront payment of $12.5 million, it’s licensing a bispecific antibody, plus two backups , from Surrozen to go after retinal disease, as preclinical data showed the candidate could stimulate Wnt signals in the eye and possibly induce retinal vessel growth. Sanofi is enlisting TrialSpark to scout promising programs to in-license or acquire, with a goal of finding six Phase II/Phase III drugs to buy within the next three years. Whereas TrialSpark originally set out to hasten clinical studies for biopharma partners, it has pivoted to internal R&D and begun hunting for programs. Under the pact, Sanofi will take charge of commercialization. PEOPLE Two months after his abrupt exit from J&J , Mathai Mammen is emerging as a top candidate to succeed Michel Vounatsos as Biogen’s next CEO, STAT reported. As it steers past the Aduhelm fiasco into the future of the next-gen Alzheimer’s drug lecanemab , the biotech is looking for someone with more R&D experience, according to STAT. Normally, a former biotech exec going on a board wouldn’t be news — it happens every day for post-buyout CEOs. But it’s a different story when Jeff Marrazzo , the co-founder and ex-CEO of Spark Therapeutics , makes that jump. So John Carroll sat down with him to talk about the next big cause he’s choosing to champion: an epigenetic editing startup called Chroma Medicine . In an effort to preserve cash, cancer immunotherapy startup OncoSec Medical will lay off about 45% of its workforce , or 18 employees. The penny stock and Merck partner is slimming down to complete a Phase II trial for its sole program in melanoma. With the restructuring, OncoSec says it now feels confident it can complete the trial within the next six months. John McHutchison’s three years as CEO of Assembly Bio since jumping from Gilead has been marked by tumult and clinical setbacks. Now, as the biotech maintains its goal of developing a functional cure for HBV but shifts its focus on next-generation programs, he’s retiring and handing the keys to Jason Okazaki , who’s just recently been promoted to president and COO. Cyclerion Therapeutics , the spinout tasked with developing the old Ironwood pipeline, hit another speed bump and will make a somewhat significant turn. In conjunction with an announcement that it will slash its staff by 45% , trimming the headcount to about 16 employees, the biotech is pivoting toward mitochondrial diseases while offloading a handful of programs. Bob Duggan’s Summit Therapeutics is running out of moves for its sole clinical-stage candidate. The biotech issued a terse statement that it’s pulling the plug on the only active clinical trial for ridinilazole , which has been through a failed late-stage trial for C. difficile. A pediatric study is being curtailed as Summit says it decided a few days ago to either partner out the therapy or get a buyer — if they can find one. As Severin Schwan gets ready to hand the Roche reins to diagnostics chief Thomas Schinecker , the pharma giant will promote Matt Sause to take Schinecker’s place. Matt Sause , who currently leads Roche’s North American diagnostics business, has served a handful of roles across the company’s diagnostics and pharma units over the past 20 years. Under the weight of a two-year clinical hold on its cancer cell therapy, PharmaCyte appears to be on its last legs. Kenneth Waggoner , its CEO of almost 10 years, is out the door , and the company’s largely reshuffled board enlisted a “business review committee” to look at ways to “realize optimal shareholder value.” As the pharma industry buckles down to reduce its carbon footprint — with some committing to zero emissions as early as 2025 — so is the advertising industry. Ad Net Zero , an initiative based in the UK and Ireland, has tapped former OMD US and BBDO New York CEO John Osborn to lead its extension to the US and spread the message on reducing emissions. AI Eli Lilly is the latest to tap Schrödinger for a small molecule discovery and optimization deal. This one is worth $425 million total and the pharma giant will lead everything once a given program enters the preclinical stage. The partnership was disclosed as part of Schrödinger’s platform day, where it outlined internal programs and path forward. AstraZeneca is doubling down in chronic kidney disease and idiopathic pulmonary fibrosis by selecting one target for each of those via its AI partner BenevolentAI . Since their tie-up in 2019, the pharma giant has selected five targets in total, and it says the new targets are “novel rare variants.” FINANCING Cellarity doesn’t have any lead candidates yet. It doesn’t even have a public pipeline. But it’s still attracting new investors, who are chiming in for a $121 million round . Traversing what it calls “white space,” the Flagship startup takes a non-traditional drug development approach that examines the entire cell system rather than a single target. Out to replicate the university spinout approach that has cemented the biotech creation blueprint for Boston funds, Red Tree Venture Capital nailed down its first fund to bankroll at least a dozen biotechs and medical device startups in California and some other West Coast states for which the capital deployer wants to be the first institutional backer. As Amylyx gets ready to debut its ALS drug in the US — amid scrutiny and criticism of the price it’s set — the biotech is going back to the public market for some cash to fund the production and commercial work. The public offering is expected to fetch Amylyx anywhere from $167 million to $192.2 million . But the biotech also made sure to warn investors that the drug may have to be yanked from the market, alluding to an informal pledge by its co-CEOs. For the better part of its first decade, UC Berkeley spinout Valitor has largely been funded by government grants. That’s changing now as it secured a new CEO and the funds to take its research into the clinic in early 2024. Morningside led the $28 million Series B in support of its mission to upend the treatment of wet age-related macular degeneration. With only two full-time employees, Vergent Bioscience now has $21.5 million to work on a fluorescent imaging agent that would allow surgeons to find, more quickly, solid tumors in patients during surgery. The biotech had originally in-licensed the molecule from Stanford, and it notes the funding will last well into Phase III studies, most likely into 2024 and 2025. A generally gloomy public market hasn’t stopped biotechs with big clinical plans from raking in the cash — either through stock offerings or loans. The oncolytic virus experts at Replimune are taking the latter route, inking a deal to borrow $200 million from Hercules Capital over the next five years as it works through clinical readouts. Despite Covid woes, Valneva lined up $100 million in a share offering to push forward its Pfizer -partnered Lyme disease vaccine and a jab for chikungunya that awaits an FDA decision. The capital injection is nearly equal to the amount Pfizer paid to nab equity in the company earlier this summer as part of the duo’s vaccine tie-up. CELL/GENE TX Nested Therapeutics believes it’s found a way to unlock new aspects of precision oncology. And with KRAS pioneer Kevan Shokat as a pioneer, the Versant-backed startup scored $125 million to move two lead programs into the clinic. By screening protein structures, it wants to better understand driver mutations in certain cancers and zero in on them. At a 12-month deadline for their partnership, Pfizer elected to only move forward with one of two capsids out of Voyager Therapeutics for use in its AAV gene therapies. The selection of the capsid, which targets a rare neurologic disease, triggers a $10 million payment but also leaves a second capsid for a cardiac target on the table. Kite Pharma just secured FDA green light to kick off operations at a new manufacturing campus in Oceanside, CA, where it will make retroviral vectors, a key ingredient for its CAR-T cell therapies. The facility has been in the works for about four years, and Kite is looking at automating part of the manufacturing process so that it can become more efficient. Faced with the shortage of lentiviral vectors worldwide, Legend Biotech and J&J will be doubling their investment in Legend’s New Jersey manufacturing center, investing a total of $500 million to manufacture their own. The move is designed to support a ramp-up in production of the partners’ BCMA-directed CAR-T Carvykti , approved in February for multiple myeloma. PHARMA When the CMS restricted its coverage of Biogen’s controversial Alzheimer’s drug Aduhelm , it also lumped all amyloid-targeted monoclonal antibodies with it. But a new bipartisan House bill, if it passes, would seek to separate the follow-on lecanemab and other amyloid-targeted drugs with data coming soon from Aduhelm. And it would restrict CMS from making the same sort of class-wide decisions moving forward. When new drugs win approval in the US, the FDA often tasks sponsors with conducting postmarket studies as a condition of the OKs. But a new JAMA study shows that for the 135 new drugs approved from 2013 to 2016, the majority of pharma companies who had certain commitments were late in meeting them. Pharmacy benefit managers have become a thorn in the side of the pharma and insurance industries in recent years, and just a couple of months after the Federal Trade Commission signaled it would investigate unlawful PBM practices, FTC chair Lina Khan is looking to turn up the heat even more. Speaking at a conference, Khan pledged to use all tools in investigating the pharma middlemen, Paul Schloesser reports from Kansas City, MO. Amgen opened the doors to its second-largest research site with lab and office space right next to Genentech’s 1 DNA Way in South San Francisco. It’s looking to fill the building with 650 staffers working to discover drugs to treat cancer, inflammatory diseases and cardiometabolic disorders — and it’s got some built in perks to attract top talent. Almost two years after pushing a third drug to market, Alnylam notched an expanded label for Oxlumo in primary hyperoxaluria type 1. The rare genetic disease, in which the body is unable to regulate the overproduction of oxalate, can spur the development of kidney stones and play a role in later-stage kidney disease. In the already buzzy obesity drug market, Eli Lilly is the latest newsmaker, nabbing a fast-track designation and rolling review from the FDA. While Lilly’s tirzepatide study in obesity initially read out positive results in April, it’s initiating an FDA filing as it awaits results from a second Phase III study. What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the new Biohaven post- Pfizer takeover. Regrouping following the $11.6 billion sale of its migraine drugs, Coric brought in a couple more experts to help build an independent company with a renewed focus on neuroscience and rare diseases. CORONAVIRUS Amid already plunging use of Merck’s Covid-19 antiviral Lagevrio , the preprint of a real world study of Lagevrio conducted in the UK suggests the antiviral does not reduce “already low” numbers of hospitalizations or deaths in mostly vaccinated adults. But patient-reported outcomes were better on the Lagevrio arm. Merck maintains its plans of filing for a full NDA. While AstraZeneca’s Covid-19 pre-exposure prophylactic Evusheld has managed to remain relevant for immunocompromised and other patients through new Omicron subvariants, one of the new strains may render it almost obsolete, the FDA warned. A spokesperson acknowledged that the drug is unlikely to be active against BA.4.6 but said the subvariant isn’t expected to become a dominant strain. Eiger BioPharmaceuticals is dropping plans to submit an emergency use application to the FDA for its repurposed Covid-19 drug after the agency turned down its request for a pre-EUA meeting. Citing concerns about the conduct of a Phase III study, regulators said they will not consider an EUA application. Moderna refused to disclose its mRNA vaccine technology to China during negotiations to sell its Covid-19 shot in the country, according to a new report from the Financial Times. Ultimately, Moderna, which has been fiercely protective of its intellectual property, declined to hand over its recipe and backed out of those talks due to “safety and commercial concerns.” LAW Eli Lilly and Teva are set for a court battle over their migraine drug rivalry after a federal appeals court refused to invalidate three of Teva’s patents for its migraine treatment Ajovy , while also declining to issue a summary judgment in favor of either company. Teva filed suit against Lilly back in 2018, alleging that the company infringed upon nine patents with Emgality . As Illumina continues to fight for its $7.1 billion Grail buyout, the American Antitrust Institute and William Baer , former director of the FTC’s Bureau of Competition and assistant attorney general for DOJ’s antitrust division, filed a request to submit an amicus brief that about a proposed fix that they say carry “important implications for the future of merger law and sound antitrust enforcement.” R&D The FDA might have approved a new ALS drug, but that doesn’t mean R&D in the space gets any easier. Clene Nanomedicine reported that its ALS program, which uses gold nanocrystals to try to catalyze intracellular reactions, did not achieve its Phase II primary or secondary endpoints. It’s now looking for “potential strategic partners.” GSK claimed a win for what it calls the largest head-to-head trial pitting a PD-1 against Merck’s best-selling Keytruda in a type of lung cancer, as Jemperli met the primary endpoint of objective response rate (even though the trial was not powered for superiority). In a separate positive move, GSK says it’s moving both arms of the COSTAR Lung trial into Phase III to test Jemperli as well as the TIM-3 inhibitor cobolimab . Talzenna , the low-profile PARP inhibitor Pfizer scored in its $14 billion buyout of Medivation in 2016, hit the primary endpoint in a Phase III study. Pfizer said a combo of Talzenna and Xtandi beat Xtandi alone in PFS for patients with metastatic castration-resistant prostate cancer. But details are slim and the numbers will have to wait. In the wake of serious side effects, KalVista is closing down a Phase II trial for one of its experimental drugs. Multiple patients in every treatment group saw unsafe, elevated levels of certain liver enzymes after taking its oral plasma kallikrein inhibitor for hereditary angioedema, the company reported. Aldeyra touted some positive data out of the first part of a Phase III study in a rare eye disease with no approved therapies. But going forward with the study in a similar fashion will be “difficult, if not impossible,” something it will need to hash out with the FDA in an upcoming meeting. The same drug is being prepared for a filing in another indication. After being forced to delay two Phase IIa trials and blaming CMC issues on a Phase Ib miss, Eliem Therapeutics believes it’s now in the clear . New early-stage studies ruled out CMC as the reason for the foiled study, and the company will no longer pursue photosensitive epilepsy — where low exposure to its drug was first observed. It will however move forward in other indications. Adamis Pharmaceuticals is slowly tumbling down, and the biotech is looking at all its options . After a Phase II/III trial failure last month sent the penny stock player’s shares even further down, the company said it is examining options to get the best value for its investors. Alternatives include anything from a partnership to a sale of its two commercial products to M&A — and bankruptcy in the worst-case scenario. Danish biotech Scandion Oncology’s lead candidate failed a Phase II trial in metastatic colorectal cancer, sending shares into a tailspin. Neither the primary endpoint on tumor reduction nor the secondary endpoints on progression-free survival and stable disease were met. The flop comes after both the COO and CEO stepped down from their positions. #AAO22 J&J’s Janssen will take its gene therapy for geographic atrophy into a Phase II study later this year. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses. Kyle LaHucik reports from Chicago. Ahead of the launch of its pivotal trial, Aura Biosciences detailed some interim data from an ongoing Phase II trial of an eye cancer drug that the biotech hopes will reduce the need for radio therapies that can blind patients with ocular tumors. At the interim look, its drug kept choroidal melanoma in check while nearly all patients retained their vision. MARKETINGRX Pfizer and BioNTech are collaborating with Marvel to celebrate “everyday” people getting Covid-19 vaccines in a custom comic book. In the “Everyday Heroes” digital comic book, a grandfather who is waiting with his family at a clinic for Covid-19 vaccinations tells the story of how the Avengers defeat Ultron, a leading villain, much the same way vaccines fight viruses. There are some odd-looking purple creatures lurking around the halls of AstraZeneca lately. The “Phil” character cutouts — which are also showing up online — are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of the company’s newest awareness effort to help people understand eosinophilic asthma. The goal is to highlight the annoyance and difficulty of uncontrolled asthma in a lighthearted way. With its first drug launch in the books, Insmed is now focusing on building out a road map for upcoming products – with an eye on consumer marketing strategies. For CEO Will Lewis , that means tapping consumer insights as early as possible and developing products and packaging that are intuitive and easy to use. Beth Snyder Bulik explains. In an effort to get people talking about cardiovascular disease, Novartis’ new “From the Heart” video series talks to researchers, patients, public health officials and advocates. The interviewer is Marie-France Tschudin , Novartis’ president and chief commercial officer, who opens the series bringing to light shocking statistics and challenging viewers to confront them. As Leo Pharma aims to take on heavyweight champ Dupixent in atopic dermatitis, it’s launching “AD Days Around the World,” an awareness campaign documenting real patient stories across Europe. In short video clips, they discuss the challenges of living with AD, the most common form of eczema. Now that Fred Hutchinson Cancer Research Center and Seattle Cancer Care Alliance are under one umbrella following a merger, the new Fred Hutch debuted a rebrand highlighting the newly unified research and clinical care programs with a new brand identity featuring a multicolor logo and advertising campaign. Healthcare agency veteran Leo Tarkovsky joined Fingerpaint Group as chief commercial officer to oversee its growing portfolio of pharma and healthcare agencies and communications companies. Tarkovsky came to Fingerpaint from WPP , where he was EVP for global healthcare growth over the past year, but he’s enticed by the chance to work at a smaller, growing agency. Servier has dropped billions of dollars over the past several years with the goal of becoming a top player in hard-to-treat cancers. Now it’s setting a deadline for 2030 — and giving its brand a makeover. The company changed up its logo , adding a star and “moved by you” slogan. The “r” at the end of the logo curves into a “smile” representing “hope for patients.” When Kaile Zagger took the helm of UC Davis spinout Evolve Biosystems several months ago, the company billed itself as a probiotic maker. However, she believes the company’s Evivo supplement designed to help infants develop a healthy gut microbiome is “so much more” — and that, she said, calls for a rebrand . Meet Infinant Health , where nature is a key theme. Novartis is turning to a winning NFL coach to talk about Leqvio and the struggles of high cholesterol — including his own; Pfizer is selling branded merchandise — specifically “Science Will Win” T-shirts, hoodies, water bottles and even golf balls and a golf umbrella — online; A study finds oncology digital marketing to be on the rise in Europe; don’t miss out anything in the pharma marketing world. Check out the MarketingRx roundup . FDA+ The FDA’s Center for Drug Evaluation and Research dropped its briefing documents spelling out why Covis’ controversial preterm birth drug Makena should be pulled from the market — and it isn’t mincing words. Ahead of an advisory committee meeting, CDER decried “false hopes” and risks of the continued marketing of Makena in the absence of evidence that it really works. Thanks to the passing of PDUFA VII, the FDA released eagerly-awaited details behind the two industry-friendly pilot programs. The first is a pilot program, dubbed STAR, to shorten review times for supplemental apps aimed at unmet medical needs, while the second will advance the development of new rare disease endpoints via additional meetings. Pfizer , GSK , Janssen , Regeneron , Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience. The Big Pharma heavyweights made clear they aren’t happy with the lack of specificity. As abortion bans take effect nationwide, doctors and advocates are keeping the pressure on the FDA over concerns that the Supreme Court’s Dobbs decision leaves a drug used off-label for miscarriage treatment vulnerable to restrictions. In a new petition, they requested that regulators encourage Danco Laboratories to file an application for its pregnancy termination pill Mifeprex in miscarriage management. Otsuka subsidiary Taiho Oncology nabbed an accelerated approval for Lytgobi ( futibatinib ), its new kinase inhibitor to treat adults with a type of previously treated bile duct cancer, known as metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements. In 2020, the FDA rejected Mesoblast’s remestemcel-L , or Ryoncil , its lead candidate for pediatric acute graft-versus-host disease that didn’t respond to steroids. Responding to one of the concerns spelled out by regulators, the Australian biotech says it sent in an optimized potency assay to ensure consistency of its stem cell therapy, setting the biotech up to resubmit its BLA in the near future. The FDA+ roundup is making a return this week to note the biggest stories that you probably missed: The FDA is convening an adcomm to review Cytokinetics’ heart drug; New generic drug guidance seeks to reduce facility-related delays for regulatory review; and the FDA recently proposed a new rulemaking to amend its regulations to modernize, simplify, and enhance the current system for overseeing human research. MANUFACTURING BioNTech signed what’s called a “Letter of Intent” with the state of Victoria and a part of Australia agreeing on a partnership on mRNA therapies and vaccines. The proposed partnership includes plans to build a “research and innovation” center, working on projects and backing Australia’s mRNA efforts to move more into preclinical and clinical development, on top of a product manufacturing facility. In a boost to its Asia Pacific regional headquarters in Singapore, Merck opened a new secondary packaging facility and broke ground on a separate facility that will manufacture next-generation inhalers. The two manufacturing sites are part of a $500 million investment to expand regional production of medicines and vaccines such as Keytruda and Gardasil . After winding down its only clinical trial and laying off a sizable group of staffers, PACT Pharma sold a lab in South San Francisco to AmplifyBio , an Ohio-based contract research organization specializing in cell and gene therapy development. PACT, whose goal has been to create personalized cancer therapies, will also transfer some personnel and offer access to technology platforms that can be applied on “product characterization.” Roche has broken ground on a 56,000-square-foot production facility at its 63-acre campus in the town of Branchburg, NJ, according to the local news website mycentraljersey.com. The facility will be used to manufacture Roche’s polymerase chain reaction that is used in diagnostic tests, per the report, and is expected to house hundreds. Only a few months after expanding its facility in Holly Springs, NC, vaccine maker CSL Seqirus is putting the new location to use with its latest, $30.1 million contract. HHS’s BARDA chose CSL Seqirus to produce an H5N8 A/Astrakhan virus vaccine candidate for a Phase II study which is slated to start in Q2 of next year. A growing gene therapy company named Myrtelle is teaming up with Forge Biologics to make Myr-201 , a gene therapy indicated for monogenic hearing loss. Forge will provide plasmid manufacturing, plus cGMP AAV process development and scaling manufacturing services at its manufacturing site in Columbus, OH. To date, only two gonorrhea vaccines have made it into human trials, with neither proving successful. Intravacc and Therapyx hope to make the third — and Intravacc, a Dutch CDMO, received a grant from NIAID for up to $14.6 million to push their vaccine candidate toward the clinic. The vaccine consists of IL-12 capsules inside an outer membrane vesicle, and it would be delivered through the nose. With few new details of what needs fixing, Lupin disclosed that the FDA recently sent a warning letter to its Tarapur, India, site. The warning letter follows a Form 483 received in April. The manufacturer says it does not believe the FDA slap will disrupt its drug supplies or the existing revenues from operations of this facility. Dupo, IL-based contract manufacturer Sterling Pharmaceuticals has had a long, tough year. Verrica Pharmaceuticals were slammed with two CRLs that they blamed on Sterling. Now, details of that reinspection are coming to light in a new warning letter published this week by FDA, which raises major concerns about the way the site is designed and how it operates. Months after acquiring Q1 Scientific , a stability storage company located in Ireland, Cambrex says it will be expanding by adding a new storage building in Belgium; MilliporeSigma cut the ribbon on a new 29,000-square-foot manufacturing facility in France that will make drug substances; learn more in the Manufacturing roundup . DON’T MISS Takeda decided to pull its hypoparathyroidism drug from the market entirely, devastating almost 3,000 people with the rare disease who have been anxiously monitoring the situation since 2019, when the injection was recalled due to manufacturing woes. As supply issues remain unresolved, Takeda said it will halt all production by the end of 2024. In a letter to the editor, Ron Cohen , president and CEO of Acorda , responds to an Endpoints report on a JAMA paper “that purports to show that there is no correlation between the price of a drug and its R&D costs.” He takes a few issues with the design of that study . With its Covid-19 monoclonal antibody sidelined in the US since April and plans for full approval no longer in the cards, Vir Biotechnology is putting more eggs into the influenza basket with a hefty-sized contract from the federal government. The up-to-$1 billion contract will bankroll a Phase II testing of its antibody as a pre-exposure prophylaxis, and BARDA has already injected $55 million. In total, Vir could be on tap to create as many as 10 candidates. Three pharma leaders landed on Fortune’s most powerful women in business list for 2022. GSK’s CEO Emma Walmsley leads the group at No. 8, while Pfizer’s chief commercial officer and president Angela Hwang landed at No. 13, and Johnson & Johnson’s EVP and chairman of pharmaceuticals Jennifer Taubert is at No. 36.

Eliem expects to report topline data around the middle of 2024. Eliem Therapeutics’ would-be challenger to Biogen and Sage Therapeutics’ zuranolone is moving into a phase 2a clinical trial. The transatlantic biotech greenlit the study after a pharmacokinetic study cleared up drug exposure concerns raised in another clinical trial. Like zuranolone, Eliem’s ETX-155 is a GABAA receptor positive allosteric modulator that is designed to treat conditions including major depressive (MDD) order. While Eliem has ceded a big head start to Biogen and Sage’s candidate, it is betting that its prospect’s apparent lack of a clinically meaningful food effect and longer half-life will translate into improved efficacy, tolerability, compliance and durability.  Eliem is now preparing for the most rigorous test of its hypothesis yet. In the first quarter of next year, the biotech will start a phase 2a trial of either a 60 mg or 75 mg dose of ETX-155 in MDD. Eliem will decide the dose after assessing the effect of repeat 75 mg doses on healthy volunteers in a phase 1 trial. ETX-155 is moving into phase 2 after coming through a trial designed to clear up a concern identified in another study. Specifically, Eliem saw lower-than-expected drug exposure levels in the three subjects evaluated in a phase 1b photosensitive epilepsy trial, leading it to test single and repeat doses in healthy volunteers to confirm the pharmacokinetic profile.  Eliem found no meaningful difference between the exposures at the 60 mg dose in its pharmacokinetic trial and its earlier phase 1 studies. The biotech said the low exposures seen in its other trial were within “the range of previously reported moderate variability,” adding that it saw no irregularities or differences in the chemistry, manufacturing and controls associated with the drug product and the drug substance. The 60 mg data, and the fact that a single 75 mg shot was well-tolerated, has persuaded Eliem to test the effect of giving the higher dose repeatedly before deciding on which dose to trial in the phase 2a. If the phase 2a starts on time, Eliem expects to report topline data around the middle of 2024. The biotech’s $130 million cash runway is scheduled to run into 2025.