Despite the approval of Biogen Inc.’s Qalsody (tofersen) for treating amyotrophic lateral sclerosis (ALS), there are still hurdles for the drug to clear. Because the U.S. FDA granted Qalsody accelerated approval, there are plenty more data to collect in the ongoing confirmatory phase III Atlas study of those who develop ALS symptoms during the trial compared to placebo. Also, Biogen has yet to release the price it plans to charge. Competitor Relyvrio, from Amylyx Pharmaceuticals Inc., has a wholesale acquisition cost of about $158,000 for the first year, then jumps to about $163,000 in the second year. The market size is small, with only about 330 patients in the U.S.

Orbital Therapeutics lands $270M series A round to join the next cycle of RNA innovation

Orbital Therapeutics Inc. raised $270 million in a series A round to fund a big push into the next generation of mRNA-based therapies. The based firm is building out a comprehensive RNA platform from which it will launch programs in oncology, autoimmune disease and indications involving protein replacement approaches. Orbital boasts a stellar lineup of co-founders, including RNA pioneer Drew Weissman, of the University of Pennsylvania, and company chairman John Maraganore, long-time leader of RNAi leader Alnylam Pharmaceuticals Inc. It has a strategic alliance with base editing pioneer Beam Therapeutics Inc., which provides it with exclusive access to the latter firm’s RNA technologies and know-how and its targeted lipid nanoparticle delivery technologies. Beam has received a minority stake in the company.

Pharma groups: EC’s revised regulations could harm industry, innovation

The industry has hit out at the European Commission’s proposals for new pharmaceuticals regulations, saying they risk “sabotaging” life sciences in Europe. “Today’s proposals manage to undermine research and development in Europe while failing to address access to medicines for patients,” said Nathalie Moll, director general of the European Federation of Pharmaceutical Industries and Associations. The largest reform of EU pharma legislation in more than 20 years is intended to make drugs more available, accessible and affordable, while supporting innovation and boosting the competitiveness of the sector. Currently, there is a 10-fold variation in time to access new drugs across the EU. Presenting the proposals, the EU’s vice president, Margaritis Schinas, said it is an “epic” package of measures that will “create a single market for medicines.”

No political divide in frustration over CMS Alzheimer’s coverage 

U.S. Centers for Medicare & Medicare Services (CMS) Administrator Chiquita Brooks-LaSure made her first appearance today before the House Energy and Commerce’s Subcommittee on Health, ostensibly to discuss legislative solutions to increase transparency and competition in health care. But member after member, regardless of political party, demanded answers about why CMS continues to limit access to Eisai Co. Ltd.’s Alzheimer’s drug, Leqembi (lecanemab), to people participating in qualifying clinical trials, especially since another government agency is covering it for all veterans that meet the labeling requirements. Brooks-LaSure said once Leqembi gets full FDA approval, which is expected in early July, Medicare will cover it for the indicated population so long as patients are enrolled in registries. But when those registries come online is a whole other question.

Newco news: Albatroz launches with $3M in seed funding to tackle new target for solid tumors and arthritis

Albatroz Therapeutics Pte Ltd. has secured $3 million in seed funding to develop therapeutic antibodies against a new target that degrades the extracellular matrix, a key contributor to cancer and arthritis. Cancer that has metastasized is notoriously difficult to treat and accounts for most cancer deaths, and Albatroz has secured exclusive rights on a highly specific target that blocks tumor growth and recurrence, including a range of therapeutic antibodies that inhibit that target.

Little support seen for AI as inventor at US PTO

The U.S. Supreme Court has declined to hear a case that tests the notion that artificial intelligence (AI) can be an inventor, a development that may be nothing more than the beginning of the AI-as-inventor story under U.S. law. The Patent and Trademark Office’s (PTO) April 25 webinar on the subject included some remarks that AI could be used to produce a tsunami of potentially duplicative patent applications, but the event demonstrated that there is almost no at-large support for AI-as-inventor, suggesting that the status quo will stand for the time being.

The BioWorld Insider Podcast: Better times ahead for the biopharma sector? Could be, the new numbers say

It used to be a lot easier to find partners. After two years of strong dealmaking, the volume and value of partnerships slid in the first quarter of 2023. M&As also are down compared to prior years. But there are some reasons to be cheerful. Looking at first quarter numbers and surveying the big picture on this BioWorld Insider podcast are Karen Carey, BioWorld’s managing editor and senior data analyst, along with Tim Shannon, a general partner at Canaan. Carey just wrote about biopharma deals and completed M&As in the first quarter. Shannon, who’s been with Canaan since 2009, has a new fund sporting $850 million in new capital, including an oversubscribed $650 million fund for seed and series A financings. They both offer their insights into the first quarter’s numbers and what may lie ahead.

Also in the news

Aldena, Alloy, Alx, Amo, Aphaia, Arbutus, Arrowhead, Artelo, Atea, Atsena, Awakn, Baudax, Bergenbio, Cellectis, Endo, Entera, Epsilogen, Erasca, Evelo, Evommune, Finch, First Wave, GSK, Hillevax, Hipra, Hoth, Immutep, In8bio, Invivyd, Linkinvax, Lyra, Mesoblast, Mezzion, Morphic, Myrtelle, Nanoviricides, NRG, Oculis, Okyo, Omeros, Oxford Biomedica, Parthenon, Provention, Purple, Sable, Samsung Bioepis, Sanofi, Santhera, Slayback, Stoke, Systimmune, Tarsier, TC, Trefoil, Ultimovacs, Vaccinex, Virios, Xencor, Zyversa