Pieris slammed with FDA hold; Biogen adds PhIV for Spinraza
→ Boston-based Pieris $PIRS has been forced to hit the brakes on a slate of Phase I studies for its 4-1BB/HER2 fusion protein drug PRS-343 after the FDA hit the company with a partial hold. The biotech can continue to dose patients already in the studies, but can’t add patients until after it conducts “an additional in-use and compatibility study of PRS-343 with various infusion materials under specific conditions to confirm suitability of PRS-343 for administration in clinical settings.” The biotech says no adverse events were cited and still plans to launch their Phase II program for the drug later this year — provided the FDA can be satisfied. “We also remain on track to present comprehensive data from both the monotherapy and atezolizumab combination phase 1 studies at a medical conference later this year,” says CEO Stephen Yoder. Their stock was down about 6% in morning trading Tuesday.
→ Biogen wants to see exactly what benefits its big SMA blockbuster Spinraza can have on patients who don’t respond sufficiently well to Novartis’ gene therapy rival Zolgensma. The big biotech says the Phase IV study was inspired by the stories of some patients who went on Spinraza after the gene therapy failed to prove a once-and-done treatment. “Available data now show that some patients in the long-term study of Zolgensma have moved on to treatment with Spinraza. We believe that, for certain patients, motor neurons may be insufficiently treated by this gene therapy, and we plan to initiate this study to understand the extent to which Spinraza may potentially improve outcomes,” said Maha Radhakrishnan, the CMO at Biogen.
→ Myokardia is jumping on board Fulcrum‘s discovery platform in search of new drugs to treat genetic cardiomyopathies. The deal starts with a payment of $12.5 million, with a little more than $300 million on the table for the first drug and up to $150 million more for each additional program. “We have been impressed by Fulcrum’s ability to discover new biology around genetic muscle disorders,” noted Myokardia CSO Robert McDowell.
→ UPMC Enterprises, Casdin Capital and Dolby Family Ventures have stepped up with an extra $20 million to add to Cerevance‘s latest round, bringing the total to $65 million. Foresite Capital also chipped in some extra cash on the round, which already included GV (formerly Google Ventures), Bill Gates, Takeda Ventures, Inc., the Dementia Discovery Fund and Lightstone Ventures. The biotech is working on a pipeline of drugs aimed at a range of CNS diseases.
→ Germany’s Tubulis has raised €10.7 million in Series A cash to back its work on antibody-drug conjugates. BioMedPartners and High-Tech Gründerfonds co-led the launch round, with contributions from some individual investors, the founders as well as Seventure Partners, coparion, Bayern Kapital, and Occident. “Tubulis’ objective is to use our dual platform approach to generate uniquely matched and disease-specific ADCs that combine selective antibodies with effective payloads,” said Dominik Schumacher, CEO and co-founder of Tubulis.
→ In more emerging-from-stealth news, Bethesda, MD-based Gain Therapeutics reports that the company bagged a $10 million Series B for their work targeting “novel allosteric binding sites on enzymes for the treatment of rare genetic and CNS diseases.” They’re setting up 2 IND-enabling studies for their lead programs. “Our proprietary and patented platform offers the ability to discover and validate previously unidentified non-classical binding sites on enzymes that can be regulated by our novel first-in-class drug candidates to restore (or gain) enzyme function in rare and devastating genetic diseases,” said Khalid Islam, the founder and chairman of Gain.
→ A collaboration involving pharma giants and a trio of tech transfer offices at top UK universities has out-licensed a gene therapy to Deerfield, which plans to hand it to researchers at their newly launched Cure building in the heart of New York City. The program was initially developed at University College London, which is part of the Apollo Therapeutics alliance.
→ There has been a slew of terms set for this week’s biotech IPOs. The oncology biotech iTeos plans to raise around $151 million by pricing shares at $16 to $18. Renaissance Capital is also tracking a $75 million IPO from Inozyme. Annexon, meanwhile, is scouting for $150 million to back its work in degenerative diseases.