The FDA has approved Scemblix (asciminib), a STAMP inhibitor from Novartis AG, for treating chronic myeloid leukemia (CML) in adults. The approval comes in two forms, including an accelerated approval for adults with Philadelphia chromosome-positive CML in chronic phase (Ph+CML-CP) who already have been treated with two or more tyrosine kinase inhibitors. Continued approval hinges on verification and description of clinical benefit from confirmatory evidence. The second is a full approval for adults with Ph+CML-CP with the T315I mutation. Scemblix is the only FDA-approved treatment that binds to the ABL myristoyl pocket, an allosteric site within the gene sequence BCR/ABL that is found in certain versions of leukemia. It’s referred to as a STAMP inhibitor by Basel, Switzerland-based Novartis as it Specifically Targets the ABL Myristoyl Pocket.

Abbvie’s Vuity eye drop wins FDA nod for presbyopia

FDA clearance of Vuity (pilocarpine HCl ophthalmic solution) 1.25% for the treatment of presbyopia in adults brought relief for an affliction that strikes almost half of the U.S. population. Vuity, from Abbvie Inc., is the first and only eye drop cleared to treat the condition also known as age-related near blurry vision. It works as quickly as 15 minutes and lasts up to six hours.

Merck & Co. inks possible $1B R&D deal with Synthekine as Acceleron merger hits speed bump

Merck & Co. Inc. has signed a potential $1 billion research tie-up with Synthekine Inc. to develop engineered cytokines to fight autoimmune diseases, as its $11.5 billion merger with Acceleron Pharma Inc. hit a regulatory speed bump. Both the Synthekine and Acceleron deals are designed to diversify Kenilworth, N.J.-based Merck’s pipeline as it prepares for biosimilar competitors to its blockbuster cancer immunotherapy, Keytruda (pembrolizumab), toward the end of the decade. But while the Synthekine deal could give Merck, known as MSD outside the U.S., access to a cutting-edge approach to autoimmune diseases, the acquisition of Acceleron and its pipeline of pulmonary and hematology medicines is proving tricky.

Myocarditis concerns derail hopes of speedy EUA for Moderna COVID-19 vaccine in children

An ongoing evaluation of heart muscle inflammation risk, a key concern with mRNA COVID-19 vaccines globally, could delay through January 2022 completion of an FDA review of Moderna Inc.'s vaccine in adolescents 12 to 17 years of age. The side effect, called myocarditis, has been a particular concern with regulators, especially for adolescents and young men. Shares of Moderna (NASDAQ:MRNA) fell as much as 7.1% on the news, which the company said would also delay its filing of a request for emergency use authorization (EUA) of the vaccine for children ages 6 through 11, a group that can soon be vaccinated with Pfizer Inc. and Biontech SE's COVID-19 vaccine following an Oct. 29 EUA award from the U.S. regulator.

The battle against COVID-19 rages on amid development hits and misses

As booster shots become increasingly available and COVID-19 vaccines for young children are introduced into pediatric offices, several potential therapeutics to battle the SARS-CoV-2 virus are lining up for regulatory authorizations. Despite ongoing inoculation campaigns worldwide, vaccine hesitancy and inequities still leave plenty of room for new therapeutic options to help care for those already sick. As of the end of October, BioWorld has tracked a total of 1,014 therapeutics and vaccines that have entered development since the pandemic began, including 768 therapeutics and 246 vaccines. Of those, 32 are no longer studied. According to the World Health Organization, there are currently 246.4 million confirmed cases and nearly 5 million deaths worldwide from COVID-19.

Shanghai Pharma out-licenses global rights for cancer drug in $292M Huya deal

Shanghai Pharmaceutical Holding Co. Ltd. out-licensed global rights of its antitumor small-molecule chemical drug, SPH-6162, to U.S.-based Huya Bioscience International LLC in a deal worth up to $292.5 million, plus a sales commission of 3% to 6%. San Diego-based Huya gained the right to develop, manufacture and commercialize the drug in all areas except for mainland China, Hong Kong, Macau and Taiwan. “This is the first overseas licensing project of our company,” a Shanghai Pharma representative told BioWorld.

Japan’s Aculys Pharma raises $60M in series A for sleep disorder candidate

Aculys Pharma Inc. closed its $60 million series A financing round, with the funds to be used to develop pitolisant (Wakix), a selective histamine H3 receptor antagonist/ inverse agonist, in Japan. The series A financing was led by Softbank Vision Fund 2. Others such as Catalys Pacific LLC, HBM Healthcare Investments Ltd., Global Founders Capital GmBH, Sumitomo Mitsui Trust Investment Co. Ltd. and Anri Investment Fund Ltd. also participated in the round. Fujisawa-shi, Japan-based Aculys inked an exclusive licensing agreement for the clinical development and commercialization in Japan for pitolisant, which was developed by Paris, France-based Bioprojet Pharma SAS. The drug works by selectively binding to the histamine H3 receptor, an autoreceptor located in the presynaptic region of the histamine. This region contains neurons in the brain critical in regulating sleep and wake rhythm.

Court: HRSA 340B letter ‘arbitrary and capricious’

Biopharma scored a bit of a victory in the ongoing 340B war that’s pitting drug companies against the combined forces of hospital groups, contract pharmacies and the U.S. Department of Health and Human Services (HHS). Although not “contrary to law, unconstitutional or violative of notice and process procedures,” a May 17 enforcement letter HHS’ Health Resources and Services Administration (HRSA) sent to Eli Lilly and Co., of Indianapolis, violated the Administrative Procedures Act because it is “arbitrary and capricious,” a federal judge ruled Oct. 29. The HRSA letter ordered Lilly to give the 340B drug discounts to all contract pharmacies or face civil monetary penalties of up to $5,000 for each instance of overcharging. HRSA has sent similar letters to other drug companies clamping down on the expansion of the discounts on outpatient drugs.

DoJ signals more aggressive disclosure policy for corporate misconduct prosecutions

The U.S. Department of Justice has announced a new set of policies dealing with federal prosecutions of corporate misconduct that includes a far-reaching policy on disclosure. Deputy attorney general Lisa Monaco announced Oct. 28 that companies will have to disclose all non-privileged information in connection with alleged misconduct rather than just information regarding those who were “substantially involved,” just one of several changes that promises to raise the stakes for companies in the life sciences.

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