Strategy
ARM’s Cell and Gene Meeting on the Mesa offered optimism that a biopharma financial market recovery is under way, but cell and gene therapies still are seen as risky investments.
Emerging Company Profile: The Maryland-based biotech is working on a functional cure for type 1 diabetes that is similar to Vertex’s VX-880 – but with some key differences.
The US major is investing over $360m in new money into the UK but the country has being losing its lustre as an appealing place to invest since Brexit and needs to offer more to multinationals.
Big pharma executives at ARM’s Cell and Gene Meeting on the Mesa shared some of the practical challenges of bringing one-time treatments to market in hemophilia, cancer and beyond.
The US FDA approved the drug for hemophilia A and B, and while it may struggle to compete in hemophilia A against Roche’s Hemlibra, it has a big convenience advantage in hemophilia B.
Public Company Edition: Upstream grossed $255m and CAMP4 raised $75m in October’s first initial public offerings. In follow-on offerings, Crinetics and Scholar Rock brought in $500m and $300m, respectively. Also, Turnstone cut 60% of its workforce and Gritstone filed for bankruptcy.
The French major and partner Denali's attempts to validate RIPK1 as a promising target have once again foundered as oditrasertib comes up short in a multiple sclerosis trial months after a failure in amyotrophic lateral sclerosis.
The TCR therapy showed strong efficacy and durability in patients with previously treated melanoma. A Phase III pivotal trial is due to start in December.
The biopharma industry posted 22 mergers and acquisitions during the third quarter, according to Evaluate, for a total of $8.2bn. Those tallies marked a second consecutive quarter of decline.
An overall survival benefit for patients in TALAPRO-2 could pave the way for a broader label for the combination in metastatic prostate cancer.
The company is developing the drug as a way to reduce injection burden for wet AMD patients and plans to meet with the US FDA early next year to discuss Phase IIb topline results.
Deal Snapshot: Boehringer Ingelheim will work to develop a cyclin inhibitor that can stop cancer cell growth, using Circle’s macrocycle platform.
But biotechs are still having to price their offerings lower than they had planned.
Indian CMOs, CDMOs and CRDMOs are expanding capacity and making leadership moves ahead of the BIOSECURE Act, anticipating demand from big pharma and budding biotechs. Scrip brings details on leading Indian firms which could be potential go-to suppliers for US companies affected by the proposed Act
Dalzanemdor missed its primary endpoint in a Phase II Parkinson’s study, Sage announced, following April’s Phase II miss in Alzheimer’s. Phase II data in Huntington’s are still expected this year.
A US regulatory filing for the drug/device combination product in earlier stages of bladder cancer remains on track for early 2025.
As broad stock markets finished the third quarter of 2024 on a high, two large biotech companies battled on more than one front. Despite both announcing positive news in the last week of the quarter, neither seemed to be a clear winner over that period.
Judo Bio, incubated for three years by Atlas Venture to deliver siRNA and other genetic medicines to the kidney, came out of stealth mode with $100m and new CEO Rajiv Patni.
Phase III success positions Scholar Rock’s selective myostatin inhibitor for regulatory filings in early 2025. Analysts see a blockbuster opportunity for additive therapy in spinal muscular atrophy.
Starboard Value has reportedly bought a $1bn stake in Pfizer with an ambition to turn the big pharma around. But growth, not cost cuts, are Pfizer’s challenge.