Skyhawk Therapeutics, Inc.

The vibe at BIO 2026 in San Diego last week was overwhelmingly positive, with attendees observing noticeable changes at the FDA and an uptick in dealmaking and IPOs. Plus, a top medical journal this week retracted a pivotal study for Amgen’s rare disease drug Tavneos, which has been in the FDA’s crosshairs since January. > Listen on Spotify > Listen on Apple Products > Listen on Amazon Music > Listen on iHeart Amgen’s Tavneos continues to face serious challenges, with the FDA still pushing for its removal from the market. Early this year, the agency identified issues with an analysis from the drug’s original developer, ChemoCentryx, which Amgen acquired for $3.7 billion in August 2022 after the drug’s 2021 approval. The regulator in January asked Amgen to voluntarily pull Tavneos from the market, but the pharma refused . Since then, eight deaths have been tied to drug, and now, a top-tier medical journal has retracted its pivotal study . Elsewhere, optimism prevailed at the BIO International Convention in San Diego last week. Celebrating the 50 th anniversary of Genentech —and of biotech itself—the conference highlighted an uptick in deals and IPOs , while noting that these transactions have gotten more nuanced. Attendees also drew attention to continued threats from China and Trump’s Most Favored Nation drug pricing policy. Meanwhile, the FDA seems to be turning a corner after a year and a half of turnover and controversial policymaking under former Commissioner Marty Makary. Acting leadership is now making an effort to support the staff they have left while also hiring 2,200 more , and the FDA’s new IND initiative was highlighted as a sign that normal procedures are returning to the agency. Still, the FDA lacks permanent leadership, and the industry is left to speculate who might serve as the next chief . For now, though, the agency is taking another look at some controversial decisions made under Makary’s leadership. After the FDA’s reversal of course on uniQure’s gene therapy for Huntington’s disease, Replimune announced that the FDA had accepted a resubmission for its twice-rejected melanoma asset. REGENXBIO , meanwhile, announced that it will resubmit its Hunter syndrome gene therapy thanks to an FDA change of heart and seek accelerated approval for its Duchenne muscular dystrophy gene therapy. And Skyhawk Therapeutics pointed to uniQure’s situation as a positive sign for its own Huntington’s candidate, for which the company reported 12-month functional data from a midstage trial this week. Capricor Therapeutics, another company that was blindsided by FDA decisions under Makary, was back on track with an accepted resubmission of its once-rejected Duchenne muscular dystrophy cardiomyopathy cell therapy, but last week, the company got another surprise when the FDA decided this decision required an advisory committee meeting. Finally, with more than 50 deals in the first half of 2026—nine of them from Eli Lilly—the business environment also seems to be headed in the right direction.

iStock, bpawesome Sarepta Therapeutics is seeking to convert the accelerated approval of its therapeutic exon-skippers for Duchenne muscular dystrophy to full despite the drugs’ failure to improve motor function in a confirmatory trial. The FDA has formally accepted Sarepta Therapeutics’ application for the full approval of its two Duchenne muscular dystrophy medicines, with a verdict expected on or before Feb. 28, 2027. Sarepta is looking to convert the accelerated approval for Amondys 45 and Vyondys 53 —exon-skipping therapies given the greenlight in 2021 and 2019, respectively—into full approval. In November last year, results from the confirmatory Phase 3 ESSENCE trial showed that neither agent elicited significant motor function improvements versus placebo. Still, analysts at Oppenheimer view the FDA’s acceptance of Sarepta’s applications as a “positive signal,” especially because Sarepta on Tuesday said the submission also includes supplemental “substantial published real-world evidence and the favorable and consistent safety profiles of both exon-skipping therapies.” “We think inclusion of positive real-world safety data improves likelihood of conversion to full approval,” the analysts told investors in a note on Tuesday, noting that “safety will be key” for the FDA’s verdict. Jefferies shared this sentiment, saying in its own Tuesday note that real-world data suggest the exon-skippers can “help patients remain ambulatory, off ventilators, and out of ERs/hospitals.” Given that side effects are mostly mild or moderate, “the overall benefit/risk pro favorable” for Amondys and Vyondys, the analysts added. FDA Sarepta Plans FDA Run for Duchenne Exon Skippers Despite Confirmatory Trial Failure Sarepta Therapeutics says the FDA has agreed to review a regulatory package for Amondys 45 and Vyondys 53 after they failed a confirmatory trial, but whether the agency will agree to approve them is still unknown. March 19, 2026 · 2 min read · Annalee Armstrong Read more Beyond the beefed-up submission, Jefferies also pointed to other broader changes that could boost Sarepta’s chances of an approval. For one, the firm noted the exit of former Commissioner Marty Makary—as well as that of Vinay Prasad, ex-director of the Center for Biologics Evaluation and Research—which in turn could give way to a regulator that is more lenient toward rare diseases. “We are closely monitoring the new FDA’s stance toward rare diseases,” Jefferies wrote. The firm also noted some “softening” on the regulator’s part regarding recent rejections, “alongside cases where the agency has revised its view on supportive data.” Replimune, which owns the twice-rejected melanoma therapy RP1, said last week that the FDA has accepted a resubmission after some “collaborative dialogue” with the company. A decision is expected by Aug. 2, with an advisory committee meeting planned. Similarly, uniQure now plans a Q3 submission for its Huntington’s disease gene therapy after a 180-degree-turn from the FDA, which had previously wanted a sham surgery–controled trial to establish the benefits of the investigational treatment—but has now instead agreed to the use of uniQure’s externally controlled Phase 1/2 trial. FDA FDA’s uniQure, REGENXBIO reversals could bolster other near-term rare disease applications The FDA’s recently altered outlook on the evidence required for approval of rare disease drugs could have immediate benefits for companies including Skyhawk Therapeutics, Capricor Therapeutics and Biohaven. June 25, 2026 · 6 min read · Heather McKenzie Read more Full approvals for Amondys and Vyondys would hand Sarepta a much-needed win after the biotech last year was rocked by patient deaths associated with its gene therapies. Its FDA-approved Duchenne gene therapy Elevidys was linked to two deaths—one in March 2025 and another in June —followed by a third mortality in a patient treated with an investigational gene therapy for limb-girdle muscular dystrophy. Following this episode, the biotech pivoted away from gene therapies to instead focus on RNA interference modalities .

Twelve-month findings showed favorable and consistent trends for participants receiving SKY-0515 across all four cUHDRS subcomponents, including Total Functional Capacity (TFC), Total Motor Score (TMS), Symbol Digit Modalities Test (SDMT), and Stroop Word Reading Test (SWRT)Skyhawk also releases Clinician and Patient Global Impression (CGI and PGI) improvement of disease survey data, which shows that at twelve months no clinician or patient assessed disease progression – all saw either disease stabilization, or improvement BOSTON, June 30, 2026 /PRNewswire/ -- Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule therapies designed to modulate RNA targets, announces cUHDRS subcomponent data from the twelve-month interim analysis of data from its Phase 1/2 clinical trial evaluating SKY-0515, an oral investigational treatment for Huntington's disease (HD). Source: Skyhawk Therapeutics Source: Skyhawk Therapeutics Source: Skyhawk Therapeutics Source: Skyhawk Therapeutics Source: Skyhawk Therapeutics