| | | |   | Hello, all. Damian here with a reminder that you can still get tickets to STAT's Thursday event in Washington, D.C., featuring patients, policymakers, and executives discussing the challenges of bringing forward new treatments for rare diseases. STAT+ subscribers get in free, and if you can't make it in person, a video recording will be available. Find out more here. | | | The brains behind CAR-T and mRNA have a new idea Carl June and Bruce Levine, two pioneers of CAR-T cancer therapy, have joined forces with the pathbreaking mRNA researcher Drew Weissman on a promising marriage of their respective disciplines. And Capstan Therapeutics, a company launched today, has $165 million to push the idea forward.
As STAT’s Allison DeAngelis reports, the plan is to subvert the normal CAR-T process — which requires genetically engineering T cells outside the body — by using mRNA to do the job in vivo. Using a revamped version of the lipid nanoparticles made famous by Covid-19 vaccines, Capstan would ferry mRNA to a patient’s T cells and coax them into becoming tumor-hunting CAR-Ts, all without the costly and time-consuming process of doing it the old-fashioned way.
Capstan has a long road ahead, with only preclinical data to support its ambitions. And it’s not alone. Vector BioPharma, Umoja Biopharma, and Ensoma are all working on in vivo CAR-T projects of their own.
Read more. | An IPO to test biotech’s mini-recovery Just six biotech companies have executed IPOs in 2022, a dramatic decline from the prior year in which more than 100 firms went public and raised nearly $20 billion in the process. Third Harmonic Bio, lined up to be the seventh, is expected to price its offering this week in the latest test of whether biotech’s summer recovery can endure.
Third Harmonic, at work on a treatment for multiple inflammatory diseases, is seeking to raise about $150 million and aiming to price its shares at between $16 and $18 a piece. Whether it can price within that range — and potentially even trade upward on its debut — will be closely watched among the many private biotech companies forced to delay their own plans as 2021’s boom collapsed into a downturn.
The XBI index of biotech stocks, once down about 50% in 2022, has recovered in fits and starts since June, making Third Harmonic a potential bellwether for the rest of the year. | Using longitudinal qualitative research to capture the patient voice One of the best ways to ensure that a sponsor’s clinical and commercial programs reflect how patients experience a disease or a treatment is to interview patients directly over time. This practice – longitudinal qualitative research – is an emerging science that can guide development efforts and inform communication strategies for treatments in any therapy area. Download this whitepaper to discover the basic principles and best practices of sound longitudinal qualitative research to ensure a patient centric approach in clinical development. | Akero’s NASH drug succeeds in Phase 2 After the drug industry’s years of failure to develop effective treatments for the prevalent liver disorder NASH, a small company may have come up with a treatment that can safely reduce the disease’s hallmark scarring.
As STAT’s Adam Feuerstein reports, a drug from Akero Therapeutics improved liver scarring at twice the rate of a placebo without worsening other NASH symptoms, meeting the goals of a Phase 2 study. In the study, about 40% of patients treated with Akero’s drug showed a clinically meaningful reduction in liver fibrosis. That number was 20% for patients on placebo.
If Akero can replicate those results in a Phase 3 study, its drug could become an effective treatment for a chronic disease that has grown into one of the leading causes of liver transplantation and cancer.
Read more. | What is a platform biotech that abandons its platform? Rubius Therapeutics first rose to attention with a 2018 press release claiming its red blood cell platform would lead to “life-changing” treatments, fueling a $2 billion IPO, then the largest in biotech’s history. Now Rubius is worth just $80 million, and, as of yesterday, the entire impetus for its foundation is no more.
Rubius said in a statement that it had discontinued its only drugs, cut 75% of its staff, and turned its attention to a new approach to making medicines with “the potential for substantive improvements over our existing platform,” in the words of CEO Pablo Cagnoni. In order to save enough cash to remain in business through next year, the company is also looking to sell its Rhode Island manufacturing facility.
That means a company founded on a scientific platform that would create a clinical pipeline now has neither. | More reads - ‘This is pharma’s dream’: How drugmakers are turning telehealth into a marketing goldmine, STAT
- China biotech rout shows growing pain from U.S. decoupling drive, Bloomberg
- Complexity of Covid vaccine program leads to concerns about potential for error, STAT
- AstraZeneca beats U.S. shareholder lawsuit over COVID vaccine disclosures, Reuters
| Thanks for reading! Until tomorrow,  | | | |
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