July 10, 2025
Biotech Correspondent

Hello! Today, we talk about a cool experimental enzyme therapy, observe more patent maneuvers over Eylea, and see an 'underdog' startup get a huge seed round to target a common kidney disease.

The need-to-know this morning

  • Soleno Therapeutics pre-announced $31-33 million in Vykat XR sales for the second quarter — beating consensus expectations by a wide margin. The drug was approved in late March to treat Prader-Willi syndrome, a rare genetic disease that causes an insatiable desire to eat. Soleno is also raising $200 million in a follow-on stock sale.
  • AbbVie is paying $700 million upfront to acquire licensing rights to a "trispecific antibody" treatment for cancer developed by Ichnos Global Innovation. The drug, called ISB 2001, targets CD38 and BCMA protein receptors on tumor cells and the CD3 receptor on T cells. A Phase 1 study in multiple myeloma is underway.

patents

Brawl over Eylea gets biosimilar industry's attention

A high-stakes legal fight between Regeneron and Amgen over the blockbuster eye drug Eylea is putting the U.S. patent system under a microscope — and is being closely watched by biosimilar makers.

After Regeneron sued Amgen for patent infringement, Amgen sidestepped the dispute by showing its biosimilar, Pavblu, didn’t need the formulation’s patented buffer — a workaround that enabled an early, at-risk launch. Regeneron quickly responded, hitting Amgen with another lawsuit in a maneuver that some say exemplifies how patent thickets can be abused to delay lower-cost competition.

“By filing continuation patents, rivals can never get product on the market,” one patent law professor told STAT’s Ed Silverman. “Only in the U.S. do we allow this game, this procedural tool.”

With billions in revenue on the line, and legislation like the ETHICS Act and the Affordable Prescriptions for Patients Act stalled in Congress, the outcome could shape how aggressively brand-name drugmakers use the patent system to block biosimlars.

Read more.

rare disease

Ultragenyx stock plummets after trial continuation

Shares of Ultragenyx plunged 33% and Mereo BioPharma shares sank 40% after the companies announced their Phase 3 Orbit trial for setrusumab in osteogenesis imperfecta will continue to final analysis, rather than stopping early for efficacy. The decision, based on a data monitoring committee review, signals that interim data didn’t meet the threshold for early success — though the drug was deemed safe. 

Ultragenyx CEO Emil Kakkis said in a statement that the company had “hoped to be able to stop the study early,” but emphasized confidence in the drug’s potential to reduce fractures and improve mobility by boosting bone mass. Final results from the trials are expected by the end of the year.

financing

An 'underdog' gets huge seed round for common kidney disease therapies

With major players crowding into the kidney disease space, Renasant Bio is hoping its scientific pedigree and a $54.5 million seed round (an unusually large sum for this stage of development) will help it punch above its weight. The startup aims to develop two small-molecule therapies for autosomal dominant polycystic kidney disease, a hereditary condition that causes extreme kidney swelling.

The round is backed by Atlas Ventures, 5AM Ventures, OrbiMed, and Qiming Venture Partners, STATs Allison DeAngelis writes.

Renasant hopes to have drug candidates ready for clinical trials in a couple of years. 

Read more.

rare disease

Experimental enzyme therapy restores boy's mobility

NYU Langone scientists used a chemical precursor to CoQ10, a commonly available enzyme, to reverse debilitating symptoms in an 8-year-old boy with HPDL deficiency — a rare, fatal mitochondrial disoder. The team administered 4-HB, a compound they discovered could cross the blood-brain barrier and jumpstart energy production in brain cells, offering hope where standard CoQ10 supplements fail.

The child, once unable to walk, is now hiking and thriving a year into treatment, STAT’s Marissa Russo writes. The success, details of which were published in Nature, underscores both the life-saving potential of basic science and the role of continued NIH funding, as researchers now plan broader trials to validate the treatment in more patients.

Read more.

More around STAT
Check out more exclusive coverage with a STAT+ subscription
Read premium in-depth biotech, pharma, policy, and life science coverage and analysis with all of our STAT+ articles.

More reads

  • Veteran venture capitalists launch new firm to Vie for autoimmune success, Fierce Biotech

  • Use of antibiotic to reduce STIs appears to be fueling some antibiotic resistance, research shows, STAT

  • HHS backtracks on pledge to disclose new vaccine advisers’ conflicts of interest, STAT

  • Novartis inks $175 million option deal for Sironax’s blood-brain barrier tech, FierceBiotech

Thanks for reading! Until tomorrow,