Last updated: August 25, 2026
The FDA moved Capricor’s deramiocel decision to November 22, 2026 after classifying new HOPE-3 extension data as a major amendment, while Genentech licensed Hanmi’s muscle preserving obesity candidate HM17321 for $190 million upfront and up to $2.3 billion total.
This page covers the Capricor extension and its refined indication, the Genentech and Hanmi obesity license, the FDA clinical hold on REGENXBIO’s Hunter syndrome gene therapy, Robert F. Kennedy Jr.’s vaccine classification inquiry, and the FDA’s expanded TEMPO pilot, with sources, for August 25, 2026.
Why did the FDA extend Capricor’s deramiocel review to November 22?
Capricor Therapeutics said on August 24 that the FDA extended the deramiocel PDUFA target action date to November 22, 2026, after the agency’s Center for Biologics Evaluation and Research classified a BLA amendment containing 24 month HOPE-3 open label extension data as a major amendment.
The announcement ends two days of silence. The original target action date was Saturday, August 22, and it passed with no approval, no complete response letter, and no company statement, a vacuum we documented in detail in Monday’s analysis of the empty PDUFA date. According to the company’s August 24 release, Capricor submitted an amendment to its biologics license application that includes 24 months of open label extension data from the Phase 3 HOPE-3 study plus additional robustness analyses, and asked the agency to review existing and new data supporting a refined proposed indication focused on upper limb function in Duchenne muscular dystrophy. A major amendment classification permits the FDA to extend its review clock by three months, which is exactly what happened.
Chief executive officer Linda Marbán said the additional year of follow up gives Capricor one of the most extensive clinical datasets evaluating upper limb outcomes in the disease. CAPR closed at $6.80 on August 24, up 8.1 percent from Friday’s $6.29 close, per market data.
The deramiocel timeline
| Date | Event |
|---|---|
| March 10, 2026 | Capricor announces the FDA set a PDUFA target action date of August 22, 2026 for the resubmitted deramiocel BLA |
| July 30, 2026 | FDA advisory committee votes 3 yes and 9 no on effectiveness in DMD cardiomyopathy, with no benefit and risk vote taken |
| August 13, 2026 | Second quarter report; CEO Linda Marbán describes an expected extension on the earnings call, with no length stated and no FDA confirmation |
| August 21, 2026 | Kaos Capital, a self described significant shareholder, publishes an open letter demanding board changes, an M&A committee, and a cash preservation plan |
| August 22, 2026 | PDUFA target action date passes with no public FDA action and no company statement |
| August 24, 2026 | Capricor announces the new November 22, 2026 target action date, a major amendment classification, and a refined proposed indication focused on upper limb function |
What is a major amendment to a BLA?
A major amendment is a substantial new data submission made while an application is under review. When the FDA accepts one, it may extend the review goal date by three months to evaluate the new information, which resets the PDUFA clock without any judgment on approvability.
The mechanism matters here because it resolves the two competing readings of Saturday’s silence. An extension driven by a sponsor’s own data submission is procedural, and the FDA does not announce these extensions; disclosure is the sponsor’s choice. Capricor’s August 13 characterization of an expected extension is now backed by a written release with a date attached, which is what the market was missing while CAPR fell 21 percent into the weekend.
What does the refined indication focused on upper limb function mean?
Capricor asked the FDA to evaluate deramiocel for a proposed indication centered on upper limb function rather than the broader cardiomyopathy claim its advisory committee rejected in July. The primary endpoint HOPE-3 met was upper limb function; the cardiac secondary endpoint did not reach significance.
The numbers explain the pivot. HOPE-3 met its prespecified primary endpoint on the Performance of the Upper Limb 2.0 scale with p equal to 0.029, while the revised left ventricular ejection fraction secondary analysis came in at p equal to 0.09, and the July 30 advisory committee voted 3 to 9 against effectiveness in DMD cardiomyopathy specifically. Anchoring the label ask to the endpoint that won, supported by 24 months of open label extension data, is the strongest remaining path through the review. It is also a narrower commercial claim than the one the company originally filed, and the review that concludes November 22 will decide whether even that narrower claim clears an agency whose advisers were unconvinced.
How much is Genentech paying Hanmi for HM17321?
Hanmi Pharmaceutical granted Genentech, a member of the Roche Group, an exclusive worldwide license outside South Korea to HM17321, a Phase 1 obesity candidate, for $190 million upfront and up to $2.3 billion in total value including milestones, plus tiered royalties on net sales.
Under the August 24 agreement, Hanmi completes the ongoing Phase 1 program, which began after FDA clearance of the investigational new drug application in November 2025, and Genentech assumes development from Phase 2 onward. Some coverage has put the total at $2.5 billion; the companies’ own release says up to $2.3 billion, and that is the figure of record here.
| Term | Detail |
|---|---|
| Asset | HM17321, a UCN2 (urocortin 2) analog |
| Mechanism class | Non incretin; designed to promote weight loss while preserving lean body mass |
| Indications | Obesity and associated conditions including type 2 diabetes and cardiovascular disease |
| Upfront | $190 million |
| Total potential value | Up to $2.3 billion including development, regulatory, and commercial milestones |
| Royalties | Tiered, on future net sales |
| Territory | Worldwide excluding South Korea |
| Stage and handoff | Phase 1 ongoing; Hanmi completes Phase 1, Genentech leads from Phase 2 |
What makes a UCN2 analog different from GLP-1 drugs?
Incretin drugs such as semaglutide and tirzepatide reduce weight mainly by suppressing appetite, and part of the weight lost is muscle. A UCN2 analog works outside the incretin axis and is designed to shift body composition, preserving lean mass while fat is lost.
Hanmi’s chief development officer In-Young Choi framed the license around exactly that distinction, saying the paradigm of obesity treatment is evolving beyond simple weight reduction toward improving body composition. That places HM17321 in the next generation mechanism bucket we have tracked all year: amylin analogs, RNA interference approaches such as Arrowhead’s ARO-INHBE, and now muscle preservation, all aimed at the qualities the current GLP-1 franchises cannot easily add. For a deeper map of the incumbent race this challenger class is aiming at, see our breakdown of retatrutide, CagriSema, and the triple agonist generation.
Why did the FDA place REGENXBIO’s Hunter syndrome gene therapy on clinical hold?
The FDA halted clinical work on RGX-121, REGENXBIO’s gene therapy for MPS II, after asymptomatic spine MRI findings in five CAMPSIITE study participants dosed three to six years earlier. REGENXBIO said it no longer expects to resubmit the BLA in the near term.
According to the company’s August 24 release, the findings are small nodules or small cystic masses seen on spinal imaging in participants who received RGX-121, known generically as clemidsogene lanparvovec, by intracisternal or intraventricular administration. Investigators deemed the findings nonserious, radiologists consider them likely benign, no brain findings were identified, and all five participants remain clinically well with stable to improved neurocognitive and neurobehavioral assessments. REGENXBIO and partner NS Pharma are evaluating additional imaging and long term follow up data and are awaiting the FDA’s full clinical hold letter. Chief executive Curran Simpson said the company believes the findings are unique and limited to the Hunter syndrome program. BioSpace reported the hold upends the company’s plan to resubmit an application the agency previously declined. RGNX closed at $8.055 on August 24, down 24.9 percent from Friday, per market data.
What does the RGX-121 hold mean for gene therapy timelines?
The hold removes any near term resubmission and adds a new question for every program that delivers AAV vectors into cerebrospinal fluid: what long term imaging surveillance will the FDA now expect, and how many years of follow up will it want reviewed before approval.
It also completes a pairing we have been tracking. This FDA has shown it will take biology risk at approval, clearing Ultragenyx’s GENGLYCOS on 44 evaluable patients and Regeneron’s Pasatru on 63, while giving no ground on manufacturing compliance, as covered in our August analysis of the ITM and Scholar Rock actions. RGX-121 shows the third surface: emergent safety signals in long term follow up. Ultragenyx’s UX111, with its September 19 action date, remains the factory test; RGX-121 is now the biology surveillance test.
What did Kennedy’s vaccine classification inquiry ask?
Per BioSpace, Health Secretary Robert F. Kennedy Jr. filed a Request for Information asking whether the CDC’s vaccine recommendation categories inadvertently imply that parental permission, individual consent, or meaningful clinical discussion applies only to shared clinical decision making vaccines, and soliciting comment on evidence standards for immunization guidelines.
The filing, reported August 24, follows the August 10 executive order directing the health department to separate the measles, mumps, and rubella combination vaccine into three individual shots given at different visits. BioSpace reports that healthcare providers have pushed back on the order and that manufacturers including Merck, Pfizer, GSK, and Sanofi have indicated such changes would take years, with some experts estimating a decade. The CDC currently sorts recommendations into routine, risk based, and shared clinical decision making categories. The RFI cites reduced public trust following pandemic era mandates as part of its rationale. No regulatory change has been made; an RFI is a solicitation of comment, not a rule.
Which companies are in the FDA’s TEMPO pilot now?
The FDA added Limbic and SonderMind on August 24, per MedTech Dive, bringing the TEMPO digital health pilot to four participants alongside Dexcom and Cadence. TEMPO pairs a premarket authorization exemption and real world data collection with a Medicare payment model that rewards patient outcomes.
| Participant | Product | Focus |
|---|---|---|
| Dexcom | Continuous glucose monitoring | Diabetes |
| Cadence | Hypertension management tool | Cardiovascular |
| Limbic | Unpacked, an AI voice agent delivering cognitive behavioral therapy by telephone under clinician oversight | Anxiety and depression |
| SonderMind | Adjunctive Care Application, a smartphone app used alongside therapy or medication | Anxiety and depression in adults |
The move into behavioral health is notable because both products carry explicit contraindications, including suicidality, mania, and psychosis for SonderMind’s app, and both will reach Medicare patients without conventional premarket authorization while the agency separately runs a public comment period on regulating generative AI in medical devices.
Frequently asked questions
What is deramiocel?
Deramiocel is Capricor Therapeutics’ allogeneic cell therapy for Duchenne muscular dystrophy, derived from cardiosphere derived cells. Its BLA is under FDA review with a target action date of November 22, 2026.
What is the new PDUFA date for Capricor’s deramiocel?
November 22, 2026. The FDA extended the original August 22 date by three months after classifying Capricor’s submission of 24 month open label extension data as a major amendment.
Did the FDA approve or reject deramiocel?
Neither. The August 22 date passed without action, and on August 24 Capricor disclosed a three month extension. The application remains under review at the Center for Biologics Evaluation and Research.
Why did Capricor’s stock rise on the extension?
CAPR closed up 8.1 percent on August 24, per market data. The formal extension replaced an unexplained silence that the market had been pricing as potentially worse news, and it confirmed the review is proceeding.
What indication is Capricor now seeking for deramiocel?
Capricor asked the FDA to review data supporting a refined proposed indication focused on upper limb function in Duchenne muscular dystrophy, narrower than the cardiomyopathy centered claim its July advisory committee voted against.
What is HM17321?
HM17321 is Hanmi Pharmaceutical’s UCN2 analog, a non incretin Phase 1 obesity candidate designed to preserve lean body mass during weight loss. Genentech licensed it worldwide outside South Korea on August 24, 2026.
How much did Roche’s Genentech pay for HM17321?
$190 million upfront, with total potential value up to $2.3 billion including development, regulatory, and commercial milestones, plus tiered royalties on net sales, per the companies’ announcement.
What is urocortin 2?
Urocortin 2, or UCN2, is a peptide in the corticotropin releasing factor family that acts on receptors expressed in skeletal muscle and other tissues. An analog of it aims to improve body composition rather than suppress appetite the way incretin drugs do.
What is RGX-121?
RGX-121, generic name clemidsogene lanparvovec, is REGENXBIO’s AAV gene therapy for mucopolysaccharidosis type II, also called Hunter syndrome, delivered into the central nervous system. It is now on FDA clinical hold.
Why is RGX-121 on clinical hold?
The FDA halted the program after asymptomatic spine MRI findings, small nodules or small cystic masses, in five CAMPSIITE participants dosed three to six years earlier. All five remain clinically well, and REGENXBIO awaits the agency’s full hold letter.
Does the RGX-121 hold affect REGENXBIO’s Duchenne program?
The company said it believes the findings are unique and limited to the Hunter syndrome program. The Duchenne gene therapy is a separate program with a separate filing plan.
What is the FDA TEMPO pilot?
TEMPO is a joint FDA and CMS pilot that lets selected digital health products gather real world data under a premarket authorization exemption while participating in a Medicare payment model that rewards improved outcomes. It now has four participants and can take up to ten.
What did Trump’s MMR executive order do?
The August 10 order directed the health department to move toward separating the combined measles, mumps, and rubella vaccine into three individual shots at different visits. Manufacturers have indicated implementing separate shots would take years, per BioSpace.
Is the Amylyx offering closed?
As of the evening of August 24, Amylyx had not announced the closing of its $500.2 million offering, which was expected on or about August 21, nor any exercise of the underwriters option.
Sources
Primary sources
- Capricor Therapeutics press release, August 24, 2026: extension of the deramiocel PDUFA target action date to November 22, 2026 (capricor.com)
- Hanmi Pharmaceutical announcement, August 24, 2026: exclusive license of HM17321 to Genentech (PR Newswire)
- REGENXBIO press release, August 24, 2026: regulatory update on RGX-121 for MPS II (PR Newswire)
- MiniMed press release, August 17, 2026: MiniMed Flex shipping with Abbott’s Instinct sensor (news.minimed.com)
- Capricor Therapeutics press release, March 10, 2026: establishment of the August 22 PDUFA date
Reporting and market data
- BioSpace, August 24, 2026: Kennedy vaccine classification RFI; REGENXBIO hold coverage; Roche and Hanmi deal coverage
- MedTech Dive, August 24, 2026: FDA adds Limbic and SonderMind to the TEMPO pilot
- Endpoints News, August 24, 2026: Roche and Hanmi deal coverage
- Exchange close data for CAPR, RGNX, and AMLX, August 21 and August 24, 2026 (market data)
Related coverage
- Capricor’s August 22 PDUFA date passed with no public FDA action as an activist pushed for a board overhaul
- The binary ledger tally: how the market priced a week of binary events
- Retatrutide vs CagriSema: why 2026 is the year of the triple agonist
- Why the FDA rejected ITM’s radiopharma therapy and stalled Scholar Rock’s SMA filing
- PTC and Lilly just bought Sangamo for parts: the $163.55M bankruptcy auction
