Tolebrutinib

The FDA has pushed back its target decision date for BTK inhibitor tolebrutinib to Dec. 28.\n Sanofi’s long and winding path to get its investigational multiple sclerosis drug over the regulatory finish line has taken another turn with the FDA\'s move to delay its approval decision on the candidate by three months.BTK inhibitor tolebrutinib—which was acquired in Sanofi’s $3.7 billion takeover of Principia Biopharma in 2021—had been on course for an FDA decision by Sept. 28 after Sanofi nabbed a priority review earlier this year. The company is seeking approval for the drug to treat non-relapsing secondary progressive multiple sclerosis (nrSPMS) and to slow disability accumulation independent of relapse activity in adult patients.But the FDA now wants three more months to review the drug’s case after the French Big Pharma sent the U.S. agency “additional analyses during the review,” something that constituted a major amendment to its original NDA. The new decision target date is Dec. 28.In a brief statement, Sanofi said it is “confident in the potential positive impact tolebrutinib can provide and will continue to collaborate closely with the FDA during the review period.”The pharma has long touted the med as a potential blockbuster, but this is another bump in the road for the drug. A year ago, the med fell short in two of three phase 3 trials, denting its prospects and ultimately reducing its therapeutic scope.Tolebrutinib had been evaluated across two forms of the chronic neurological disorder. The Hercules study involved patients with nrSPMS, while two identical phase 3 studies, dubbed Gemini 1 and 2, were focused on relapsing MS. The Hercules study was a success, with tolebrutinib hitting the primary endpoint of delaying progression of disability compared to placebo.But, in the Gemini trials, tolebrutinib failed on the primary endpoint of besting Sanofi’s own approved MS drug Aubagio when it came to reducing relapses over up to 36 months. Trying to find the positives, the company said an analysis of six-month data from those trials showed there had been a “considerable delay” in the onset of disability.Sanofi then decided to specifically focus on the indication of nrSPMS where it saw success in the Hercules trial.That was not the first time tolebrutinib had faced challenges in the clinic. The FDA placed a partial hold on further enrollment on all three of those trials three years ago over what the company described at the time as “a limited number of cases of drug-induced liver injury that have been identified with tolebrutinib exposure.”Sanofi has, however, had better news on the BTK front of late, having grabbed an FDA approval just last month for Wayrilz in immune thrombocytopenia, a drug that also came out of its Principia buyout. 

Plus, news about Sciwind Biosciences, Ipsen, Genfit, Ascletis, AusperBio, Ensoma, Jasper Therapeutics, Verrica, Incyte and Johnson & Johnson: 🏛️ FDA delays decision on Sanofi’s tolebrutinib: The company said that some additional data it submitted qualified as a “major amendment,” leading the FDA to push back the decision by three months. Tolebrutinib is a BTK inhibitor being tested for non-relapsing, secondary progressive multiple sclerosis. The new PDUFA date is Dec. 28. — Max Gelman 💊 Roche’s Genentech licenses Starpharma’s drug delivery technology: The company will pay $5.5 million upfront for access to Starpharma’s DEP platform, which Starpharma will use to develop dendrimer-drug conjugates using Roche’s medicines for certain cancer targets. Starpharma could also get development and commercial milestones of up to $564 million, plus tiered royalties on global sales of any products that emerge from the partnership. Roche would have the exclusive worldwide license. The DEP technology is designed to improve solubility, efficacy, pharmacokinetic control and toxicity profiles of drugs, Starpharma said . — Elizabeth Cairns 🇨🇳 Sciwind Biosciences submits IPO pitch: The company, which has an obesity deal with Verdiva Bio, filed for an IPO on the Hong Kong Stock Exchange, joining several other Chinese drug developers looking to go public as China’s biotech sector surges. Sciwind was founded in 2017. It’s expecting approval next year in China for ecnoglutide, its GLP-1 receptor agonist for obesity and type 2 diabetes. Verdiva Bio in January emerged with $411 million and the rights to ecnoglutide and other assets, including an amylin candidate, outside of China and South Korea. — Kyle LaHucik ✔️ Ipsen’s aesthetic product smooths glabellar lines: In a Phase 2 trial, patients treated with IPN10200 had less severe glabellar lines at four weeks compared with placebo. A “substantial majority” of patients had a clinically significant response, defined as a score of “none” or “mild,” at six months compared with placebo and Dysport, a form of botulinum A marketed by Galderma, according to Ipsen. The trial will continue to investigate IPN10200 in forehead or lateral canthal lines, and Ipsen is also planning to move the product into Phase 3 trials. — Elizabeth Cairns 🏥 Genfit stops lead liver failure program: The company attributed the discontinuation following a serious case of peritonitis, or inflammation of the belly lining, in a Phase 2 clinical trial. The French biotech was studying VS-01 in certain patients with acute-on-chronic liver failure. Its independent data monitoring committee said the trial could continue with additional data and monitoring. However, Genfit decided to discontinue the UNVEIL-IT trial as well as a proof-of-concept study assessing the experimental drug in certain people with hepatic encephalopathy or liver failure. Genfit said it would continue preclinical work on the drug in urea cycle disorder. Genfit, which is listed on the Nasdaq and Euronext, saw its shares fall around 10% Monday morning. — Lei Lei Wu 💉 Ascletis’ weight loss shot shows early promise: In a small trial in obesity patients in the US, a single 30 mg dose of Ascletis’s ASC47 plus four 0.5 mg weekly doses of Novo Nordisk’s Wegovy led to a 56.2% greater relative drop in body weight at day 29 versus the same doses of Wegovy alone. However, a single 60 mg dose of ASC47, an adipose-targeted thyroid hormone receptor beta agonist, plus the same doses of Wegovy, only managed a 15.1% bigger reduction. Only around six to nine patients were enrolled in each dose arm. The gastrointestinal tolerability was better with the combo than with Wegovy alone, and there were no discontinuations. — Elizabeth Cairns 💸 AusperBio collects $63M Series B2: With the new funding , the company has raised about $186 million since December . The San Francisco and Hangzhou biotech’s AHB-137 is in pivotal testing for hepatitis B. It said the funds will go toward ongoing clinical trials, an antisense oligonucleotide platform, preclinical work and commercial manufacturing partnerships. Its investors include Qiming Venture Partners and HanKang Capital. Most of its management team comes from Gilead, which markets medicines in hepatitis B. — Kyle LaHucik 💵 Ensoma gets $53M, starts a Phase 1/2 trial: The Boston biotech raised the money from existing investors, including Gilead, 5AM Ventures and Catalio Capital Management. Ensoma also began enrolling patients with X-linked chronic granulomatous disease in a Phase 1/2 trial of EN-374, an in vivo hematopoietic stem cell therapy. The company is led by Sanofi and Genzyme veteran Jim Burns . Back in early 2023, it disclosed an $85 million Series B and an acquisition of Twelve Bio. — Kyle LaHucik 💰 Jasper Therapeutics’ $30M offering: The biotech is developing briquilimab as a treatment for mast cell-driven diseases. It’s selling about 11.7 million shares at $2.43 apiece. — Jaimy Lee 🇯🇵 Verrica to get $10M milestone: The payment is due from Torii Pharmaceutical after Ycanth received approval in Japan to treat molluscum contagiosum, a viral skin infection. Shionogi recently acquired Torii. — Jaimy Lee 🩺 Incyte’s Opzelura is approved for younger kids with eczema: The FDA approved Opzelura, a Janus kinase (JAK) inhibitor that comes in the form of a cream, to treat mild-to-moderate eczema in children 2 years old and older whose disease is not well controlled with certain other therapies. Opzelura was previously approved for those 12 years old and older with eczema and patients with nonsegmental vitiligo. — Lei Lei Wu 💉 FDA approves Johnson & Johnson’s fully injectable Tremfya regimen: The IL-23 blocker previously required an IV induction regimen to begin treatment. J&J said Friday that the regulator approved the subcutaneous induction regimen for Tremfya for adults with moderately to severely active ulcerative colitis, enabling patients to start the therapy with a self-administered injection. — Lei Lei Wu