Capricor Is Amending Its Deramiocel BLA Ahead of the August 22 PDUFA: What the HOPE-3 Lancet Data and the Revived Celgene CVR Lawsuit Mean for Biotech in 2026

Last updated: August 17, 2026

Capricor Therapeutics says the FDA is willing to review an amended deramiocel BLA and extend the August 22, 2026 action date once the amendment arrives. The amendment adds 24 month open label extension data and new analyses of the upper limb primary endpoint from HOPE-3, which The Lancet has now published.

This page covers the deramiocel regulatory situation as of August 17, 2026, the published HOPE-3 data, how PDUFA amendments work, the revived Celgene CVR lawsuit against Bristol Myers Squibb, and the weekend’s other capital markets moves: Silence Therapeutics’ $201.3 million raise, the Skye and Redx combination into Fibrx, MapLight’s $150 million placement, and the latest oral GLP-1 prescription data.

What did Capricor announce about deramiocel on August 13 and 14, 2026?

Capricor said on its second quarter call that it will submit an amendment to the deramiocel Biologics License Application, and that the FDA has indicated it is willing to review the amendment and to extend the August 22 PDUFA action date on receipt. The FDA has not publicly confirmed the discussion.

Capricor Therapeutics (Nasdaq: CAPR) reported second quarter 2026 results on August 13. The written release said only that the BLA for deramiocel, an allogeneic cardiosphere derived cell therapy for Duchenne muscular dystrophy, remains under active FDA review, that the company continues to believe there is a path to approval, and that HOPE-3 has now been published in The Lancet after peer review. The amendment plan came on the call and in subsequent coverage by BioSpace and Fierce Biotech. Chief executive Linda Marbán said the amendment will contain 24 month results from the HOPE-3 open label extension plus additional analyses of the existing data, with the focus moved to the trial’s primary endpoint, upper limb function, rather than the cardiomyopathy question the July 30 advisory committee voted on. Her words, as reported: the FDA “has indicated it is willing to review this amendment and upon receipt to extend the PDUFA action date accordingly.” The length of any extension was not disclosed.

Shares roughly doubled at Friday’s open before settling; BioSpace put the move at 68 percent to $6.95 in early trading and Fierce Biotech at 71 percent to $7.19, against a Thursday close of $4.21 and a level near $20 before the advisory committee. Those are market reports, not company figures.

What does the HOPE-3 Lancet publication show for deramiocel?

Peer review left the upper limb primary endpoint significant and weakened the cardiac secondary endpoint. Per Capricor’s August 13 release, the Performance of the Upper Limb 2.0 result held at p=0.029, while the left ventricular ejection fraction result moved to p=0.09 from an initially reported p=0.04. The prespecified cardiomyopathy subgroup stayed at p=0.02.

That revision matters because the July 30 meeting of the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee was framed around cardiomyopathy. Per Capricor’s July 30 release, the panel voted 3 in favor and 9 against, with no abstentions, on effectiveness in DMD cardiomyopathy, a narrower question than the indication Capricor proposed, and did not vote on overall benefit and risk. Capricor said at the time that the committee’s feedback on the upper limb data was directionally supportive. Fierce Biotech quoted panel member Christopher Coffey saying he felt “stronger that there might be something there for upper limb than I do for the [left ventricular ejection fraction].” The amendment is, in effect, an attempt to move the review onto the endpoint the panel found more persuasive.

What happens to a PDUFA date when a sponsor submits a major amendment?

Under the FDA’s PDUFA review goals, a major amendment submitted late in review can extend the goal date, typically by three months, so the agency has time to evaluate the new material. Capricor has not said how long its extension will be, and the FDA has not commented, so treat August 22 as a date that is set to move rather than a date with a new value.

The practical sequence is: Capricor files the amendment; the FDA classifies it; if the agency treats it as a major amendment it resets the goal date and typically notifies the sponsor in writing. Until that happens, the August 22 date remains formally on the books. Investors should also note the difference between a deferral and a decision. An extension is not an approval signal. It says the agency would rather read new data than issue a complete response on the existing package, which is a meaningfully different posture from the one the same agency took with ITM’s ITM-11 in August, where a complete response letter cited only manufacturing items. Our running FDA thread has held that this agency takes biology risk more readily than manufacturing risk; a willingness to reopen the file on efficacy analyses is consistent with that.

Deramiocel regulatory timeline, 2026

Why did an appeals court revive the Celgene CVR lawsuit against Bristol Myers Squibb?

On August 13, a unanimous three judge panel of the US Court of Appeals for the Second Circuit reversed the dismissal of UMB Bank’s suit over the Celgene contingent value rights, holding that a defect in the trustee’s appointment did not bar the case because all parties, including Bristol Myers Squibb, had accepted UMB’s role. The suit goes back to district court.

The contingent value right was issued in Bristol Myers Squibb’s 2019 acquisition of Celgene. Each CVR paid $9 in cash only if three Celgene pipeline drugs won FDA approval by set deadlines. Two did: Zeposia (ozanimod) and Abecma (idecabtagene vicleucel). The third, Breyanzi (lisocabtagene maraleucel), had to be approved by December 31, 2020 and was approved on February 5, 2021, about five weeks late, which extinguished the entire payment. Reuters and Fierce Pharma put the aggregate at roughly $6.4 billion; the company has not restated that figure in connection with the ruling. UMB Bank, as trustee for CVR holders, alleges that Bristol Myers left critical and mandatory information out of its initial Breyanzi filing and failed to ready manufacturing sites for FDA inspection, delaying approval past the deadline. Judge Jesse Furman of the Southern District of New York had dismissed the case twice on the appointment issue while allowing a properly appointed trustee to refile. Bristol Myers did not comment on the ruling, per Reuters.

The relevance for 2026 dealmaking is direct. Last week’s Sangamo auction priced PTC Therapeutics’ Fabry asset with $100 million of milestones tied to FDA outcomes, and we have argued that structured consideration is now the price of exit for clinical stage assets. The Celgene case is the reminder that a milestone or CVR does not eliminate the binary; it moves the argument about who caused a miss into a courtroom, sometimes years later. Anyone writing a CVR today should be reading the diligence obligation and effort covenants with this docket in mind.

What did Silence Therapeutics raise after the divesiran data?

Silence Therapeutics closed an upsized underwritten public offering of 14,907,407 American Depositary Shares at $13.50 each on August 14, including full exercise of the underwriters’ option on 1,944,444 ADSs, for gross proceeds of about $201.3 million.

The offering was launched at $150 million and priced at $175 million on August 12, two days after Silence reported that divesiran met its endpoints in the SANRECO Phase 2 trial in polycythemia vera. Jefferies, Morgan Stanley, Cantor Fitzgerald, and William Blair were joint book runners. Silence plans a Phase 3 start in polycythemia vera in the first half of 2027, where it will chase Takeda and Protagonist’s rusfertide, which has an FDA decision due this quarter.

What is Fibrx Therapeutics and what did Skye and Redx agree to?

Skye Bioscience and Redx Pharma agreed on August 14 to combine into Fibrx Therapeutics, a Nasdaq listed fibrosis company, backed by about $125 million in aggregate financing led by Abingworth, with Redmile, British Business Bank, NEXTBio Capital, and 5AM Ventures participating. Closing is expected in the fourth quarter of 2026.

Per Skye’s release, the financing stacks a roughly $68 million PIPE, a $36 million Series A led by Abingworth, and an equity line of up to $22 million from a Redmile affiliated fund. On a pro forma basis, existing Skye holders would own about 5.38 percent, Redx holders about 46.17 percent, and the financing investors about 48.45 percent. The lead asset is RXC008, a gastrointestinal restricted pan ROCK inhibitor for fibrostenotic Crohn’s disease with an open US IND and Fast Track designation; a Phase 2 study is due to start in the second half of 2026 with topline data expected in the second half of 2028. RXC007, zelasudil, is a selective ROCK2 inhibitor that has completed a Phase 2 program in idiopathic pulmonary fibrosis. A preclinical discoidin domain receptor inhibitor is slated for an IND in 2027. Lisa Anson, formerly of AstraZeneca, will be chief executive; the company will be headquartered at Alderley Park in the United Kingdom, and says its runway extends through 2029.

What did MapLight Therapeutics raise and what will it fund?

MapLight Therapeutics announced a $150 million private placement on August 13 at $11.38 per share, to fund ML-007C-MA through the ZEPHYR-2 Phase 3 study in schizophrenia and the VISTA Phase 2 study in Alzheimer’s disease psychosis, with runway through 2028.

ML-007C-MA is a muscarinic agonist combination in the same mechanistic family as Bristol Myers Squibb’s Cobenfy. MapLight plans an end of Phase 2 meeting with the FDA to settle the Phase 3 design. Fierce Biotech reported the company is winding down preclinical work to concentrate resources on the schizophrenia program. For readers tracking the Cobenfy story, this is the best funded direct challenger and it is now aimed squarely at both indications where Bristol Myers has struggled for traction or delayed data.

How are the oral GLP-1 pills tracking in August 2026?

Per IQVIA data cited by Citi analysts and reported by Fierce Pharma on August 14, Novo Nordisk’s Wegovy pill logged about 163,000 US prescriptions in the week of August 7 versus 38,928 for Eli Lilly’s Foundayo. Novo’s share of the US obesity market was 39.9 percent to Lilly’s 60.1 percent. These are analyst reported figures, not company confirmed.

Two readings coexist. Novo’s pill is far ahead on volume, and the company has called it one of the strongest US launches by volume on record. But its weekly count has flattened while Foundayo’s has roughly doubled since late June, and oral semaglutide has stayed at 30 to 35 percent of total Wegovy scripts, which is what Fierce means by a market share ceiling. Lilly’s Zepbound has held the majority of the obesity market throughout. Lilly reported $98 million of Foundayo revenue in its first quarter on the market, so the pill remains a small line in a $23.0 billion quarter.

What else happened in life sciences from August 14 to 16, 2026?

The weekend was quiet. The remaining Friday items: ten pharmacy benefit managers committed, per a PCMA release, to display TrumpRx prices in their real time benefit tools; Abcuro raised a $66 million Series C to run a new Phase 3 of ulviprubart in inclusion body myositis after its Phase 2/3 study missed in the broad population, per Endpoints News; BioCryst said it is ending internal discovery, per Fierce Biotech; BioSpace reported that newly released EU documents cite serious breaches in the pivotal Tavneos study; and shareholders of AMS, a tissue healing company, approved its sale to H.B. Fuller, per MedTech Dive.

Frequently asked questions

Is deramiocel approved?

No. Deramiocel is under FDA review with a current PDUFA action date of August 22, 2026, which Capricor expects to be extended once it submits an amendment to the BLA.

Did the FDA confirm it will extend the deramiocel PDUFA date?

Not publicly. The statement that the FDA is willing to review an amendment and extend the date came from Capricor’s chief executive on the company’s second quarter call. The FDA has not commented.

What is in the deramiocel BLA amendment?

Per the company, 24 month results from the HOPE-3 open label extension and additional analyses of the existing data focused on the upper limb primary endpoint.

What did the FDA advisory committee vote on deramiocel?

On July 30, 2026, the Cellular, Tissue and Gene Therapies Advisory Committee voted 3 in favor and 9 against on effectiveness for DMD cardiomyopathy. The vote is non binding and did not address overall benefit and risk.

What did The Lancet publication of HOPE-3 change?

The upper limb primary endpoint remained significant at p=0.029. The left ventricular ejection fraction result was revised to p=0.09 from an initially reported p=0.04. The prespecified cardiomyopathy subgroup stayed at p=0.02.

How much cash does Capricor have?

$237.9 million in cash, cash equivalents, and marketable securities at June 30, 2026, per its second quarter release, with runway of at least twelve months.

How long does a major amendment extend a PDUFA goal date?

Under PDUFA review goals, a major amendment submitted late in the review cycle typically extends the goal date by three months. Capricor has not disclosed the length of any extension.

What was the Celgene CVR?

A contingent value right issued in Bristol Myers Squibb’s 2019 purchase of Celgene, paying $9 in cash per CVR only if Zeposia, Abecma, and Breyanzi were each approved by set deadlines. Breyanzi missed its December 31, 2020 deadline and the CVR expired worthless.

What did the Second Circuit decide in the Celgene CVR case?

On August 13, 2026, a unanimous panel reversed the district court’s dismissal, ruling the suit could proceed despite a defect in the trustee’s appointment because all parties had accepted UMB Bank’s role as trustee.

How much is at stake in the Celgene CVR lawsuit?

Reuters and Fierce Pharma put the aggregate value of the CVRs at roughly $6.4 billion. Some outlets have reported a higher figure; the company has not restated the number.

How much did Silence Therapeutics raise?

About $201.3 million gross, from 14,907,407 ADSs at $13.50 including the fully exercised underwriters’ option, closed August 14, 2026.

What is Fibrx Therapeutics?

The name of the combined company formed by Skye Bioscience and Redx Pharma, a Nasdaq listed fibrosis company with about $125 million in committed financing, expected to close in the fourth quarter of 2026.

What is RXC008?

A gastrointestinal restricted pan ROCK inhibitor for fibrostenotic Crohn’s disease with FDA Fast Track designation. A Phase 2 study is expected to begin in the second half of 2026 with topline data in the second half of 2028.

How many prescriptions is the Wegovy pill writing per week?

About 163,000 in the week of August 7, 2026, per IQVIA data cited by Citi and reported by Fierce Pharma, versus 38,928 for Foundayo.

What did MapLight raise?

$150 million in a private placement at $11.38 per share, announced August 13, 2026, to fund ML-007C-MA through a Phase 3 in schizophrenia and a Phase 2 in Alzheimer’s disease psychosis.

Sources

Primary sources
Capricor Therapeutics, second quarter 2026 results and corporate update, August 13, 2026 (company release).
Capricor Therapeutics, update on FDA advisory committee meeting for deramiocel, July 30, 2026 (company release).
Skye Bioscience, transaction agreement with Redx Pharma and $125 million in financings, August 14, 2026 (company release).
Silence Therapeutics, closing of upsized public offering, August 14, 2026, and pricing of upsized $175 million offering, August 12, 2026 (company releases).
MapLight Therapeutics, $150 million private placement, August 13, 2026 (company release).
Pharmaceutical Care Management Association, PBM commitment on TrumpRx prices, August 13, 2026 (release).
US Food and Drug Administration, Breyanzi approval, February 5, 2021.

Trade press and wire coverage
BioSpace, “Capricor soars on potential FDA review extension, filing amendments,” August 14, 2026.
Fierce Biotech, “Capricor soars on plan to amend DMD filing after adcomm setback,” August 14, 2026.
Reuters (via Yahoo Finance), “Lawsuit against Bristol Myers over delayed cancer drug is revived,” August 13, 2026.
Fierce Pharma, “It’s BMS vs. Celgene investors once more after US appeals court revives lawsuit,” August 14, 2026.
Fierce Pharma, “The Oral GLP-1 Tracker,” August 14, 2026 (IQVIA data via Citi and Jefferies).
Fierce Biotech, “MapLight turns off preclinical tap to fund schizophrenia drug,” August 14, 2026.
Endpoints News, “Abcuro gets $66M for Phase 3 in muscle-weakening condition,” August 14, 2026.
MedTech Dive, medical devices coverage, August 13 to 14, 2026.

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