Interim data from two early stage Friedreich’s ataxia (FA) cardiomyopathy studies from Lexeo Therapeutics Inc. hit the mark by reducing heart muscle thickness, a key cause of death among patients. The results came from the Sunrise-FA phase I/II study and an investigator-initiated phase Ia study of LX-2006, an adeno-associated, virus-mediated gene therapy encoding the human frataxin gene. The drug is designed to improve frataxin protein expression to improve mitochondrial cell function. FA is a progressive, autosomal recessive and neurodegenerative disease characterized by the body slowly losing its ability to control itself. FA cardiomyopathy, the heart’s inability to pump blood to where it’s needed, is the leading cause of death for patients. FA has no approved treatments. LX-2006 received fast track designation from the U.S. FDA in April. The data weren’t enough to enthuse RBC Capital Markets analysts, who said the study had multiple limitations. Investors agreed as the company’s stock (NASDAQ:LXEO) had fallen 29% at midday to $12.42 per share.

US Senate passes effort to prune patent thickets for biologics

A bipartisan bill aimed at limiting patent thickets on biologics moved a step closer to law last week when the Senate passed it with unanimous consent in an unexpected vote that came more than one-and-a-half years after the legislation was favorably sent to the Senate floor. Offered as an amendment to the Federal Trade Commission Act, the Affordable Prescriptions for Patients Act, S. 150, would limit the type and number of patents that can be litigated under the Biologics Price Competition and Innovation Act. “By stopping abuses of our patent system, this legislation will pave the way for biosimilars to compete with branded drugs and aggressively lower drug prices for consumers in the process,” according to the bill’s sponsors, Sens. John Cornyn (R-Texas) and Richard Blumenthal (D-Conn).

Opyl, L39 Capital pick biotech winner with $100M fund and AI

AI-driven clinical trial design company Opyl Ltd. formed a partnership with L39 Capital Pty Ltd. to establish a $100 million biotech fund that will showcase the predictive power of Opyl’s Trialkey software in selecting successful biotech and pharmaceutical stocks. Melbourne, Australia-based Opyl leverages AI to elevate clinical trial design and forecast outcomes, empowering clinical researchers, biopharma companies and investors to enhance trial design, drug development and market delivery. “If you're running clinical trials, we can tell you with 90% certainty whether that trial is going to succeed or not,” Opyl Executive Chairman Saurabh Jain told BioWorld.

US House, Senate eye different appropriations for FDA’s 2025 monies

A committee of the U.S. House of Representatives proposed an FDA discretionary spending bill of less than $26 billion for fiscal year 2025, but that amount is significantly less than the $27 billion offered by a Senate committee, a difference that will have to be ironed out if the agency is to avoid the need to deal with yet another continuing budget resolution.

ISSCR 2024: Expanding niche definition gives insights into stem cells

The word “niche” implies a specialized environment. But to Fiona Doetsch, the stem cell niche is anything but. For brain stem cells, “the whole organism is the niche,” Doetsch told the audience at the third plenary session of the International Society for Stem Cell Research (ISSCR) annual meeting in Hamburg. It’s a surprising idea at first, given the brain’s protection from many circulating substances via a series of barriers, including the blood-brain barrier and the blood-cerebrospinal fluid barrier. But over the past few years, Doetsch, who is an associate professor at EPFL Basel, and her team have identified a series of signals that influence the major brain stem cell niche in ways that are time-, location- and cell type-specific.

US Medicare program offers pay boost for radiopharmaceuticals

The U.S. Medicare outpatient draft for 2025 is rich with applications for pass-through payment, but the draft also would boost payment for radiopharmaceuticals, a proposal that drew the applause of industry and physicians alike. The outpatient draft for CY 2025 tackles the implications of some new technologies for the pass-through payment program, but nestled in the draft rule is a proposal to pay separately for diagnostic radiopharmaceuticals.

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