CERo Therapeutics, Inc.

Johnson and Johnson kicked off first-quarter earnings season with a “modest” beat and an ambitious goal; Replimune failed again to gain approval for its advanced melanoma therapy, as analysts tout increased accountability brought by the FDA’s new policy of publicizing complete response letters; and Revolution Medicines’ pancreatic cancer candidate doubled survival in one of cancer’s most intractable foes. > Listen on Spotify > Listen on Apple Products > Listen on Amazon Music > Listen on iHeart Johnson & Johnson kicked off Q1 earnings season with a “modest” beat , raking in $24.1 billion in sales as it targets $100 billion in revenue in 2026. J&J, as always, is the first to report results from a first quarter that has seen a wave of deals across biopharma; this M&A rush is expected to headline investor calls as more companies, including Novo Nordisk and Eli Lilly, report their Q1 results in the coming days . Meanwhile, IPOs are also on an upswing, with Kailera Therapeutics eyeing a raise that could reach as high as $533 million. On the regulatory front, Replimune failed for a second time to secure approval for its advanced melanoma therapy RP1, as the FDA held its ground, requesting a Phase 3 trial that CEO Sushil Patel indicated in a statement last week “will not be viable.” This comes as analysts say the FDA’s new policy of making complete response letters public is increasing accountability. Along with transparency, another key theme for the FDA this year has been its promise to be flexible, particularly when it comes to therapies for rare diseases. While BioSpace has closely covered the myriad cases where the agency has appeared to reverse its guidance to sponsors, two companies—Rezolute & CERo Therapeutics— lauded recent interactions with the FDA, calling reviewers “collaborative” and “curious.” Finally, heading into the American Association for Cancer Research’s annual meeting this weekend, several companies—including Revolution Medicines , Allogene and IDEAYA Biosciences and Servier —got a jump start, reporting positive data in a number of indications. Revolution’s report was especially seminal. The company’s therapy, daraxonrasib, doubled survival in pancreatic cancer—a disease that has just a 13% survival rate five years after diagnosis.

iStock, Yutthana Gaetgeaw Replimune’s CEO Sushil Patel has already warned that the biotech will need to cut staff and substantially scale back its U.S. manufacturing operations. The second FDA rejection of the melanoma drug RP1 has plunged Replimune into uncertainty, with the company’s likely path to recovery paved with job cuts, site closures and strategic alternatives. “We have no choice but to eliminate jobs, including substantially scaling back our U.S. based manufacturing operations,” Replimune CEO Sushil Patel said in a prepared statement on Friday in response to RP1’s rejection. It remains unclear how many employees will be affected by Replimune’s savings push, or when the layoffs might occur. Replimune faces “a challenging path forward,” BMO Capital Markets wrote in a note to investors on Friday afternoon. “The company will need to secure funding,” the analysts said, pointing to the biotech’s $269.1 million cash position at the end of 2025. “Significant cost cuts are likely necessary.” The regulatory rebuff likely also signals the end of the road for RP1: “Without timely accelerated approval, the development of RP1 will not be viable,” Patel said on Friday, though he stopped short of definitively announcing that the drug’s development would be discontinued. Friday’s rejection triggered a 20% drop in Replimmune’s shares—and the selloff has only worsened since. The biotech is trading at $1.75 per share before the opening bell on Monday, a 63% crash from its previous closing price. Patel in his statement did not mince words. He called the FDA’s verdict “deeply disappointing,” claiming that the agency “has not exercised regulatory flexibility to meet patients’ needs.” The CEO also accused the agency of “inconsistent communication” and of employing a “fragmented and slow-moving regulatory process.” In particular, Replimune’s press announcement blasted the FDA’s use of a different review team for RP1’s resubmission, replacing the previous staffers “who had interacted with the company.” This new review team, the biotech claimed, “did not meet with the company during the review process despite the company offering.” Replimune also flagged what it characterized as contradictions between the FDA’s previous position on RP1’s data package and the reasons for rejection. Most notably, the biotech pointed out that the agency in 2021 agreed that “if the data was sufficiently compelling, a single arm trial could be acceptable for consideration under accelerated approval.” Regulatory FDA flexibility isn’t absent but evolving regulations come with growing pains The FDA has gained a reputation during the past year for being inconsistently flexible, particularly when it comes to rare diseases. Executives at Rezolute and CERo Therapeutics recently had positive interactions with the agency, in which they told BioSpace reviewers have been “collaborative” and “curious. April 13, 2026 · 5 min read · Heather McKenzie Read more But in the FDA’s complete response letter (CRL), which was made public under the agency’s new transparency initiative, the agency insisted that Replimune’s single-arm study “is not considered to be an adequate and well-controlled clinical investigation” and cannot support approval. Analysts at BMO took the regulator’s side. “While we are no fans of [Center for Biologics Evaluation and Research Director Vinay] Prasad, commentary from the CRL clarify the consistency in FDA’s decision to deny approval,” the analysts wrote. The CRL is clear, they continued, that Replimune and the agency had never truly aligned on the use of a single-arm study or the company’s methods to assess RP1’s efficacy. “While the FDA allowed the resubmission, they appeared to do so out of a desire to be open to the possibility that fundamental issues with the product’s clinical package could be addressed in a high unmet need indication,” the firm continued. “We can’t help but feel prior resubmission efforts were grounded more on hope than true alignment with FDA,” the analysts added. “Hope is never a winning strategy, unfortunately.”

iStock, wildpixel The FDA has gained a reputation during the past year for being inconsistently flexible, particularly when it comes to rare diseases. Executives at Rezolute and CERo Therapeutics recently had positive interactions with the agency, in which they told BioSpace reviewers have been “collaborative” and “curious. In March 2025, CERo Therapeutics kicked off a Phase 1 trial for its lead candidate, an autologous T cell therapy called CER-1236, focused on acute myeloid leukemia. In October, however, the company detected an unexpectedly positive response in a patient whose AML had progressed to myelodysplastic syndrome. Prior to treatment with CERo’s drug, this patient was burdened with frequent platelet transfusions. After four rounds of CER-1236, however, they started making their own platelets, said Robert Sikorski, chief medical officer at the Bay Area biotech. “We hadn’t expected that,” Sikorski told BioSpace . “Obviously, when you see that . . . we made the decision to add more myelodysplastic syndrome patients to the trial.” So, CERo went to the FDA with a proposal to amend the trial to include both indications. The request was approved within 30 days, enabling the company to “really quickly move into a new set of patient populations in an active trial without having any downtime,” Sikorski said. This was critical, he added, given the serious nature of the diseases, and the FDA seemed to understand that. The agency, he said, was “very responsive and collaborative.” This feedback stands out from recent headlines . The FDA has been repeatedly criticized by politicians, patient advocates and biotech executives alike for a lack of flexibility—not to mention clarity —in its regulation, particularly of rare disease therapies. Earlier this month, the Rare Disease Advocacy, Biotechnology, and Investor Coalition penned a letter to the Trump administration in which it called on FDA and Department of Health and Human Services (HHS) leaders “to restore regulatory clarity” as they consider candidates to replace outgoing Center for Biologics Evaluation and Research (CBER) chief Vinay Prasad. Certainly, myriad companies—including Capricor Therapeutics , Replimune and uniQure —have been thrown for a loop this past year as guidance given by previous FDA leaders was reversed and their rare disease candidates were either rejected or punted to further trials. Regulatory experts acknowledge that these reversals are frustrating but say they are not necessarily inappropriate. FDA leadership is not obligated to follow previous guidance, Holly Fernandez Lynch, associate professor of Medical Ethics and Health Policy at the University of Pennsylvania School of Medicine, told BioSpace last month. “Just because the FDA said something doesn’t mean that future FDAs should not be able to say, ‘That was wrong. We have an obligation to not approve a drug that doesn’t work.’” Rare diseases Rare Disease Leaders Call for Regulatory Clarity as FDA Balances Urgency With Rigor With CBER director Vinay Prasad set to depart the agency at the end of the month, a coalition of patient groups and biotech executives penned a letter imploring the Trump administration to “restore regulatory clarity” for rare disease therapies. Experts on a BioSpace panel last week also acknowledged the challenges faced by a more stringent FDA. April 2, 2026 · 6 min read · Heather McKenzie Read more As for why some companies find themselves derailed at the final checkpoint, and others like CERo report positive feedback, Sikorski noted that some of the apparently reversed guidance was provided under the previous administration. That was the case for Capricor and uniQure, as guidance on the design of their registrational studies was given by agency leadership while Joe Biden was president and Robert Califf ran the FDA. Moreover, Sikorski noted that the agency’s approach could differ between the FDA’s actions in early- and late-stage clinical trials. “I have not observed a lack of flexibility in recent early-stage trials and have found the agency’s technical staff to be constructive overall,” he said. The most publicized reversals of guidance primarily occurred either in late-stage meetings with the FDA or during the product’s review. Sikorski additionally noted that his experience has been limited to that technical staff, which includes reviewers, medical officers and safety officers. In contrast, Prasad reportedly played a role in the review of Capricor’s deramiocel for Duchenne muscular dystrophy cardiomyopathy, and both Prasad and FDA Commissioner Marty Makary made comments to the press about uniQure’s Huntington’s disease gene therapy. Positive resolutions for Rezolute CERo is not alone in its praise for the FDA under Makary. Another company with a positive experience in the past year is Rezolute, which in August 2025 received the agency’s blessing to pivot from a randomized, placebo-controlled Phase 3 trial to a truncated single-arm, open-label study comprising as few as 16 patients. Rezolute’s lead candidate, ersodetug, had shown success in tumor hyperinsulinism (HI) in several patients treated through an expanded access program and onlookers questioned the rationale for conducting a randomized study in this indication, CEO Nevan Charles Elam told BioSpace . The FDA appears to be on board with innovative trial designs —at least in theory. Last year, the agency issued draft guidance encouraging cell and gene therapy developers to use alternative trial designs including externally controlled trials and single-arm studies to streamline the development of these products. The FDA at the time cited the “urgent need for safe and effective” medicines to address severe conditions affecting “small populations.” Recent decisions regarding products tested through these innovative trials, however, have led to confusion . FDA FDA Reversals in Rare Disease Space Highlight Confusion Around External Controls While recent FDA guidance speaks to the agency’s support of innovative trial designs—including the use of external controls—the application of this flexibility appears to be inconsistent. One former regulator says the situation is more nuanced. April 6, 2026 · 6 min read · Heather McKenzie Read more But according to Harpreet Singh, chief medical officer at Precision for Medicine and a former oncology regulator at the FDA, final acceptability of an external control “really can’t be determined until the review process is complete, or throughout the course of the review process.” Nuance aside, these stories do indicate a nod toward flexibility at the FDA. Last month, the agency again extended flexibility to Rezolute when it acknowledged that a missed primary endpoint in another trial of ersodetug—this time for congenital hyperinsulinism—could have been caused partly by behavioral factors. The FDA requested more data from the trial. “For a division to say ‘We’re going to do our own analysis, if you give us the data set, because we’re curious’… I haven’t seen that in years,” CEO Nevan Charles Elam told BioSpace last month. Notably, the FDA offers a number of pathways through which increased communication is possible, including Breakthrough Therapy and Fast Track designations. Most recently, in June 2025, the agency introduced the Commissioner’s National Priority Voucher program , which aims to expedite the timeline for decisions for products that align with certain national priorities from 10–12 months down to 1–2 months. Being in receipt of one of these vouchers also offers enhanced communication with the FDA. And just this month, the FDA proposed a new “clinical trial notification pathway” that would offer an alternative to the current Investigational New Drug application system, which the agency in its 2027 budget document called “burdensome.” While details remain sparse, the program “would create an accelerated path to initiate U.S.-based Phase 1 clinical programs,” according to the proposal. Given the evolving regulations, Singh recommends early and continuous interaction with the agency. “From the leadership, we are seeing more enthusiasm, or perhaps more openness, to engage directly with industry,” Singh observed. Her advice to sponsors: “[It’s] that old adage; engage early and often.” Government FDA’s 2027 Budget Proposes Permanent Rare Disease Vouchers, Easier Entry to Clinic If the Trump administration’s proposal passes, the FDA’s budget will be more than $200 million bigger in 2027, with plans to launch new programs that expedite drug development, boost national security and promote “radical transparency.” April 7, 2026 · 3 min read · Tristan Manalac Read more