Privately held Dyno Therapeutics Inc. has added another notch to its adeno-associated virus (AAV) vectors development portfolio in a deal with the Roche Group that includes $50 million up front and ultimately could top $1 billion. Dyno will help in developing next-generation AAV vectors, optimized by artificial intelligence, to target neurological diseases. This is the second time around with Roche. In October 2020, Dyno entered a $1.8 billion deal for gene therapies to treat CNS and liver-based indications for Roche and Spark Therapeutics Inc., which is a member of the Roche Group.

Biopharma deals hit $50B in Q3, pushing 2024 deal value up 14.5% from 2023

Biopharma deals and M&A activity in 2024 continued to surge past the last two years, with deal value in the first three quarters jumping 14.5% year-over-year. The total climbed from $130.38 billion through 3Q23 to an impressive $149.24 billion so far in 2024, the highest value in the first nine months of a year, according to BioWorld’s records. Q3 alone saw $49.81 billion in deals, following a strong Q2 at $55.26 billion. Meanwhile, M&As skyrocketed 75.5% in 2024, hitting $98.02 billion, up from $55.82 billion during the first nine months of last year.

Newco news: Hope for endometriosis as HMI-115 reduces pelvic pain by 50%

Hope Medicine Inc. reported positive interim results for monoclonal antibody HMI-115 in a phase II endometriosis trial that saw the mean non-menstrual pelvic pain score reduced by 50%. “HMI-115 is a prolactin receptor blocker, and we're using it to treat endometriosis and some other diseases. It is a first-in-class new mechanism to treat endometriosis,” Hope Medicine CEO Nathan Chen told BioWorld.

Hypoparathyroidism space percolates as Septerna hikes IPO terms

Septerna Inc. raised the terms of its proposed IPO, intending to raise $275 million by offering 15.3 million shares at $18 each. The firm had previously filed to offer 10.9 million shares at a range of $15-$17. Septerna is developing orally available small molecules taking aim at G protein-coupled receptors. SEP-786, a small-molecule parathyroid hormone 1 receptor agonist, is enrolling patients in a phase I study testing the compound against hypoparathyroidism – an indication where a handful of developers large and small are busy.

ESGCT 2024: Steps forward in gene and cell therapies for brain tumors

Scientists from different laboratories around the world have presented the latest advances in research into malignant brain tumors at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), which is being held Oct. 22 to 25 in Rome. This type of cancer, which includes gliomas, poses a challenge for the design of effective therapies. In addition to the difficulty of eliminating it, there is the obstacle of crossing the blood-brain barrier and avoiding adverse effects if using viral vectors for gene editing.

Marinus tanks on ganaxolone miss; all development ends

For a company that was running out of money, a missed phase III endpoint for its only development product knelled a death blow for Marinus Pharmaceuticals Inc., tanking its stock by more than 80% in midday trading. The company will no longer develop oral ganaxolone for seizures associated with tuberous sclerosis complex, or for any other indication, as it reduces its workforce and explores strategic alternatives. Ganaxolone, a positive allosteric modulator of GABAA receptors, works by regulating brain activity, specifically modulating the synaptic and extrasynaptic GABAA receptors. Shares (NASDAQ:MRNS) lingered around 29 cents, down $1.40, on Oct. 24, following news of the failed phase III and Marinus’ engagement of Barclays as an advisor to review its next steps.

The BioWorld Insider podcast: A quantum leap into the future of drug development

In the newest BioWorld Insider podcast, Victoria Lipinska, the America's lead for Quantum Innovation Centers at IBM Quantum, talks about the future of drug development using quantum computing. “The new technology is a completely different branch of computing as opposed to what we know right now, and it's meant to complement what we know, not to really replace it,” she said. Quantum computing could lead to more efficient drug discovery by identifying promising compounds faster, understanding their effects at the molecular level, and then reducing the need for costly or time-consuming lab experiments. Lipinska is one of the more than 100 experts who will evaluate the future of health care at the upcoming 2024 Biofuture conference. Each year, a group of trailblazers, disruptors and forward-thinking executives converge to evaluate and forecast the future of health care. This year, BioWorld is a sponsor of the Oct. 28-30 event in New York. If you attend, you'll have the chance to hear panels and join workshops and fireside chats with key opinion leaders like Lipinska.

Also in the news

Akamis, Alkeus, Alpha-9 Oncology, Alto Neuroscience, Amyra, Be Bio, Bristol Myers Squibb, Bryn, Calidi, Cyclacel, DBV, Duhn, Eli Lilly, Evotec, Ikarovec, Janssen, Lexicon, Lumos, March, Nurix, Ocuphire, OM Pharma, OPM, Opus Genetics, Otsuka, Pfizer, PMV, Proqr, Redwire, Repare, Revolution Medicines, SK Bioscience, Smart Immune, Tela, Transcode, Vivus