The Friday Biotech Perspective #40
After the Whiplash
Since the April 2 Liberation Day, several positive signals surfaced in both the biotech market and the overall global markets. None are definitive but three indicate the changes at the FDA will not extend to abandoning support for vital drug development. Who will decide the standard for vital treatment, however, is going to change.
New FDA commissioner Martin Makary said this month he would push for “radical transparency” to mitigate industry influence in FDA decisions. Specifically, he set out a new policy to scale back participation by drug company scientists and executives as official members on FDA advisory committees (AdComs). The rationale for Makary’s transparency initiative was for the agency to avoid any appearance of a “conflict of interest” in its decisions. Though the intent appears obvious, pharmaceutical company staffers often have as much or more expertise on a new science approach than FDA staffers or independent researchers. Hence, participation by a drug company staffer genuinely can strengthen the depth of an AdCom review. Makary, however, seeks to minimize the conflict-of-interest agenda, which may or may not improve the depth and speed of AdCom reviews.
A second signal occurred on April 24 when China-based Akeso Pharmaceuticals, Inc. (OTCPK:AKESF) received FDA approval to market its PD-1 monoclonal antibody penpulimab in combination with the chemotherapy cisplatin, or carboplatin and gemcitabine, as a first-line treatment of adult recurrent or metastatic non-keratinizing nasopharyngeal carcinoma. Even though the nasopharyngeal carcinoma indication is small, the approval is striking as Akeso owns the drug outright and now can market in the US, in spite of all the saber rattling around tariffs to be imposed on China.
A third flag was waved at uniQure N.V.’s (NASDAQ:QURE) gene therapy treatment for Huntington’s disease, AMT-130, when the FDA granted Breakthrough Therapy designation (BTD) to the drug on April 17. Even after 20 years of study, many regulators and scientists remain skeptical of the efficacy of gene therapy treatments. The BTD signifies the FDA wants to make an extra effort to move AMT-130 into the market, as there are no effective, long-term treatments for Huntington’s disease. Shares of uniQure rose 38% on the news while most biotechs declined during the day.
In November 2024, the FDA informed uniQure that data from the current phase I/II studies of AMT-130, compared to a natural history external control, may serve as the primary basis for a BLA submission under the FDA’s accelerated approval pathway. uniQure plans to provide clinical and regulatory updates on AMT-130 by the end of June 2025. A biological license application (BLA) could be sent to the FDA in the second half of the year, setting the stage for a potential launch in 2026.
While the positive news for Akeso and uniQure only may reflect the spring bloom of daffodils, it’s significant the FDA was not completely stagnant during April. As mentioned earlier this month, another key test will be on April 29 when the FDA is set to decide the approval of Abeona Therapeutics Inc.’s (NASDAQ:ABEO) prademagene zamikeracel (pz-cel), an autologous gene therapy, to treat the rare disease dystrophic epidermolysis bullosa (DEB).
Catalyst results from companies like Akeso, uniQure and Abeona are primary drivers of stock selections by Biotech Currents. While our approach and fundamental analysis remains unchanged, for the rest of 2025 we may tilt more toward revenue-producing companies with catalysts that provide a tailwind, such as Ascendis Pharma (NASDAQ:ASND). Ascendis’ skytrofa gained approval in 2021 as a once weekly injection to treat children ages 1 and older with growth hormone deficiency (gHD). Revenue growth of skytrofa has been impressive since 2022 and Ascendis wants to expand its technology with a new drug to treat achondroplasia, or dwarfism, in children between the ages of 2 and 11. Shares of Ascendis are up ~35% since initiation by Biotech Currents in November 2024.
Another rising revenue-producing company appears to be Australia-based Telix Pharmaceuticals Limited (NASDAQ:TLX). Telix, which markets radiopharmaceutical diagnostics, listed in the US in early January 2025. Revenue at the company increased 62% in the first quarter of 2025. A full report on Telix will follow shortly.
Unfortunately, manufacturers of current vaccines are likely to be big losers in 2025 unless Robert F. Kennedy Jr. does a U-turn on his vaccine stance. The stocks of Moderna, Inc. (NASDAQ:MRNA), BioNTech SE (NASDAQ:BNTX) and Vaxcyte, Inc., (NASDAQ:PCVX) have not recovered much from their highs of early 2025, even as the XBI crossed the $80 threshold this past week. Introducing new vaccines may be one bridge the FDA chooses not to cross this year, unless the “vaccine” also is intended as a therapeutic, as cancer vaccines are.