Exsilio Therapeutics emerged from stealth mode today with $82 million from a series A financing. The Orbimed-seeded startup plans to use naturally occurring, genetic elements to integrate therapeutic genes at a defined location in the genome, making it safer than random integration, which can cause tumor formation. The genetic information will be delivered via an mRNA in a lipid nanoparticle, which should allow for repeat dosing, a challenge for current gene therapies delivered via viruses.

Fosun Pharma to buy out Henlius in $691.7M merger deal

Shanghai Fosun Pharmaceutical Co. Ltd announced it will buy out partner Shanghai Henlius Biotech Inc. for up to HKD$5.4 billion (US$691.7 million), according to a joint announcement on the Hong Kong Stock Exchange. Under the agreement, Fosun will pay HKD$24.60 per Henlius share and ¥$22.44 per unlisted share. Henlius shares were trading at HKD$18.84 per share before the stock went into a trading halt prior to the announcement. The offer represents a 36% premium to its last closing price.

Innovent’s mazdutide reduces liver fat content in obese patients

Innovent Biologics Inc.’s glucagon-like peptide-1 receptor and glucagon receptor dual agonist, mazdutide, saw 80% reduction in liver fat content in patients with more than 10% liver fat content in the phase III Glory-1 obesity trial. Innovent said in January 2024 that mazdutide met the primary endpoints and all key secondary endpoints in its first phase III trial in Chinese adults who were overweight or obese, and the mazdutide NDA for weight management is under review by China’s National Medical Products Administration. Mazdutide not only induced robust weight loss in adults with obesity or overweight, but also reduced liver fat content and multiple cardiometabolic risk factors.

Six months out, new EU rules still raising feasibility concerns

The industry is again raising concerns that new EU health technology assessment rules coming into force on Jan. 12, 2025, will have the opposite of the desired effect and slow down access to innovative therapies. The legislation introduces joint clinical assessments (JCAs) of new therapies vs. current treatments, with the aim of providing a common, single input for national cost-effectiveness assessments of new drugs. Every EU member state has a different approach to deciding if a drug gets listed on its national formulary, but it is hoped that having an EU-level JCA in hand at the same time as a drug gets EMA approval will speed up reimbursement, pricing and access. But when the European Commission published the rules last month on how JCAs will be scoped and conducted, there was disquiet across the board, from companies, medical colleges and societies, and patients’ groups.

China’s Ascentage drafts confidential IPO for dual listing in US

Suzhou, China-headquartered Ascentage Pharma Group Corp. Ltd. has filed plans for a confidential IPO to the U.S. SEC for a potential dual listing on both the U.S. and Hong Kong stock exchanges. News of the U.S. IPO came just a few days after Ascentage drew a $75 million equity investment from Osaka, Japan-headquartered Takeda Pharmaceutical Co. Ltd. with the issuance of about 24.3 million shares at a purchase price of HK$24.09 (US$3.08) per share. Ascentage said it received all proceeds by June 20.

Device patents still listed, FTC embraces chance to expand review

Even though the U.S. FTC recently claimed a court victory in its campaign to shut down the listing of device patents for drugs in the FDA’s Orange Book, 80% of the listings targeted in the commission’s first round of warning letters remain in place more than seven months later. Those warning letters, sent to 10 drug companies in November, were the FTC’s first attempt to enforce a newly adopted policy that considers “improper” Orange Book listings as an antitrust violation that extends the innovator’s monopoly by delaying generic competition. Meanwhile, the FTC is welcoming an opportunity to expand its authority over patent settlements through a new rule being proposed by the Patent and Trademark Office (PTO) that would require parties to file all pre-institution patent settlement agreements with the PTO.

Botanix doesn’t break a sweat: FDA approves axillary hyperhidrosis NDA

The U.S. FDA has approved the NDA for Sofdra (sofpironium), for treating excessive underarm sweating, from Botanix Pharmaceuticals Ltd. The approval was based on two pivotal phase III studies. Sofdra is, according to Botanix, the first and only new chemical entity approved by the FDA for treating primary axillary hyperhidrosis. Botanix bought sofpironium bromide from a previous developer, Brickell Biotech Inc., in May 2022. Brickell was to receive up-front and potential near-term regulatory milestone payments of up to $9 million from Botanix, and be eligible to receive additional success-based regulatory and sales milestone payments of up to $168 million and tiered earn out payments ranging from high-single digits to mid-teens on net sales of the topical gel.

US FDA issues CRL for Abbvie’s Parkinson’s disease drug, ABBV-951

After receiving a complete response letter (CRL) in 2023 related to device issues, North Chicago-based Abbvie Inc. received another CRL for ABBV-951 (foscarbidopa/foslevodopa) for treating motor fluctuations in advanced Parkinson’s disease, this time for issues at a third-party manufacturer. Observations cited by the FDA’s inspection do not relate to ABBV-951 or any other Abbvie drugs. “The CRL does not identify any issues related to the safety, efficacy or labeling of ABBV-951, including the device,” Abbvie said in a statement, adding that the company was not requested to conduct additional trials. The company is working with the FDA to resolve the issues.

Novo CEO joins growing list of Rx execs bowing to HELP Committee

Novo Nordisk A/S’ CEO Lars Jørgensen is set to be the next executive in the U.S. Senate Health, Education, Labor and Pensions (HELP) Committee’s pharma parade of shame. HELP Chair Bernie Sanders (I-Vt.) announced yesterday that Jørgensen will testify before the committee Sept. 24 about his company’s U.S. pricing of its blockbuster semaglutide drugs, Ozempic and Wegovy. The announcement came a week after Sanders, without explanation, canceled a June 18 meeting in which HELP members would have been asked to vote on investigating Novo Nordisk’s U.S. pricing and issuing a subpoena to force Doug Langa, the company’s executive vice president of North America operations and president of Novo Nordisk Inc., to testify before the committee.

Also in the news

Actym, Alnylam, Altimmune, Alumis, Amylyx, Ani, Arvinas, Avalo, Biodexa, Biogen, Biontech, Bristol Myers Squibb, Chromadex, Curevac, Cypris, Dualitybio, Effector, Eli Lilly, Engitix, Entrada, Epilepsygtx, Evommune, Frontier, G1, GSK, Maat, Moma, Mannkind, Matinas, Moberg, Mustang, Neurobo, Novavax, Novo Nordisk, Oncoinvent, Oragenics, Poltreg, Rani, Regulus, Revelation, Rezolute, Roche, Sellas, Seroba, Shape, Sun, Takeda, Tiumbio, Twostep, Valneva