BioMed Nexus Daily Updates
Your essential biotech, medtech, and pharma recap — no noise, just what matters.
📌TL;DR
Capricor says the FDA is willing to review an amended deramiocel BLA and to extend the August 22 PDUFA date once the amendment is received; the amendment adds 24 month open label extension data and new upper limb analyses. HOPE-3 was published in The Lancet with the ejection fraction result revised to p=0.09 from an initially reported p=0.04.
The Second Circuit revived UMB Bank's Celgene CVR lawsuit against Bristol Myers Squibb on August 13. The $9 per CVR payment died when Breyanzi was approved on February 5, 2021, past a December 31, 2020 deadline; Reuters puts the aggregate near $6.4 billion.
Silence Therapeutics closed an upsized offering for about $201.3 million gross, 14,907,407 ADSs at $13.50 including the fully exercised option, four days after divesiran's Phase 2 win.
Skye Bioscience and Redx Pharma will combine into Fibrx Therapeutics with about $125 million in financing led by Abingworth; existing Skye holders keep about 5.38 percent, and the deal closes in the fourth quarter.
MapLight raised $150 million at $11.38 a share to take its muscarinic candidate ML-007C-MA into a Phase 3 in schizophrenia and a Phase 2 in Alzheimer's disease psychosis.
Novo's Wegovy pill logged about 163,000 weekly US scripts against 38,928 for Lilly's Foundayo in the week of August 7, per IQVIA data cited by Citi and reported by Fierce Pharma; Novo's obesity share was 39.9 percent to Lilly's 60.1 percent.
⚡ Executive Takeaway
We said Friday that Monday we would start counting who holds the binaries. So here is the first line of the ledger, and it is not the entry we expected: a binary that did not resolve at all. The FDA has, on Capricor's account, agreed to read more data rather than rule on August 22, and the market paid roughly 70 percent overnight for that deferral, per market reports. Nothing about deramiocel's efficacy changed on Friday. If anything, peer review made the cardiac case weaker. What changed is that a near certain no became an open question with a later date, and an open question is worth a lot more than a closed one to the person holding it.
That is the week's through line: the binary ledger. Every entry is someone deferring, transferring, litigating, or cashing a coin flip, and the price they pay or receive is the sector's real risk appetite, printed in public. Entry two arrived the same day, from 2019: Celgene's CVR holders got their $9 claim back in front of a judge, seven years after a five week miss killed it. Structured consideration does not remove the binary. It relocates it. Keep both entries in mind as the milestone heavy deals from last week season. 👉 Read Full Analysis
🏛️ FDA and Regulatory
Capricor's August 22 verdict is set to move, and the market treated a delay like a win. CAPR
On its second quarter call, chief executive Linda Marbán said Capricor will amend the deramiocel BLA with 24 month open label extension results from HOPE-3 and new analyses focused on the upper limb primary endpoint, and that the FDA "has indicated it is willing to review this amendment and upon receipt to extend the PDUFA action date accordingly." The FDA has not confirmed that publicly, and the length of any extension was not given. The same release disclosed that peer review at The Lancet left the upper limb primary endpoint at p=0.029 while the ejection fraction secondary slipped to p=0.09 from an initially reported p=0.04, which is a problem for the cardiomyopathy question the July 30 panel voted 3 to 9 against and an argument for moving the file onto the endpoint the panel liked better. Shares roughly doubled at Friday's open per market reports, from $4.21 to around $7, still far below the pre panel level near $20. Cash was $237.9 million at June 30. This is our FDA thread again: last week the same agency issued a manufacturing only rejection to ITM; here it is offering a sponsor with a shaky efficacy package more time. Biology risk, yes; factory risk, no. The Pro brief prices what a deferral is actually worth.
⚖️ Litigation and Deal Structure
The Celgene CVR is back from the dead, and every milestone written last week should notice. BMY
A unanimous Second Circuit panel revived UMB Bank's suit over the Celgene contingent value rights on August 13, ruling that a defect in the trustee's appointment did not bar the case because everyone, Bristol Myers included, had treated UMB as trustee. The CVR paid $9 in cash only if Zeposia, Abecma, and Breyanzi were each approved by their deadlines; Breyanzi's was December 31, 2020, and the FDA approved it on February 5, 2021, so the whole payment lapsed. Reuters puts the aggregate near $6.4 billion. The trustee alleges Bristol Myers left mandatory information out of the initial filing and did not have its plants inspection ready; the company did not comment on the ruling. Judge Furman had dismissed twice on the appointment issue; the case now goes back to him on the merits. Read this next to Sangamo's auction, where PTC pushed $100 million of the Fabry price into FDA contingent milestones: the buyer of a binary gets to hold it, and the seller keeps a claim on how carefully it was held.
💰 Capital Markets
Silence won its coin flip on Tuesday and sold $201 million of the winnings by Friday. SLN
Silence Therapeutics closed an upsized public offering of 14,907,407 ADSs at $13.50, about $201.3 million gross, with the underwriters' option on 1,944,444 ADSs fully exercised. The deal was launched at $150 million and priced at $175 million on August 12, two days after divesiran met its SANRECO Phase 2 endpoints in polycythemia vera. Book runners were Jefferies, Morgan Stanley, Cantor, and William Blair. For the ledger, this is the right tail entry: the holder of a binary that paid out immediately converted part of the win into permanent capital, funding a Phase 3 that starts in the first half of 2027 against rusfertide, whose FDA decision lands this quarter. Zealand sold its rusfertide royalty before that verdict; Silence sold equity after its own. Both cashed certainty; only one had to wait for it.
🧬 Fibrosis and Reverse Mergers
Abingworth and Redmile just built a fibrosis company out of two subscale biotechs and $125 million. SKYE
Skye Bioscience and Redx Pharma will combine into Fibrx Therapeutics, a Nasdaq listed company backed by about $125 million in aggregate financing: 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 affiliate. Pro forma, existing Skye holders keep about 5.38 percent, Redx holders about 46.17 percent, and the new money about 48.45 percent. The lead asset is RXC008, a gut restricted pan ROCK inhibitor for fibrostenotic Crohn's disease with Fast Track, into Phase 2 this half with topline data in the second half of 2028; RXC007, zelasudil, has finished a Phase 2 in IPF. Lisa Anson, ex AstraZeneca, runs it from Alderley Park; close is expected in the fourth quarter with runway through 2029. Column two of the barbell count, specialist creation vehicles, just added an entry, and the 5.38 percent left to Skye's public holders is the price of arriving at the table with no cash.
💊 Obesity
The Wegovy pill has a volume lead and a ceiling; Foundayo has a slope. NVO | LLY
Fierce Pharma's oral GLP-1 tracker, built on IQVIA data cited by Citi, put Novo's Wegovy pill at about 163,000 weekly US prescriptions in the week of August 7 versus 38,928 for Lilly's Foundayo. Those are analyst reported figures, not company confirmed. The pill's weekly count has bounced between roughly 147,000 and 172,000 since mid July, and oral semaglutide has held at 30 to 35 percent of all Wegovy scripts, while Foundayo has roughly doubled from 19,830 in late June. Overall obesity share, per the same data, sits at 39.9 percent Novo to 60.1 percent Lilly. This does not change our standing call. Lilly's lead on the current generation is intact, Foundayo is a $98 million line inside a $23.0 billion quarter, and the pill fight is a share of a share. The line worth watching is the ceiling, because if oral semaglutide is cannibalizing the injectable rather than expanding the class, Novo's best launch by volume is also its most expensive.
📋 Quick Hits
MapLight raised $150 million at $11.38 a share to fund ML-007C-MA through the ZEPHYR-2 Phase 3 in schizophrenia and the VISTA Phase 2 in Alzheimer's psychosis, per its release, and is winding down preclinical work to pay for it, per Fierce Biotech. That is the best funded muscarinic challenger to Cobenfy, aimed at both indications where Bristol Myers has stumbled or slipped.
Ten PBMs, including CVS, Express Scripts, and OptumRx, committed to display TrumpRx prices in their real time benefit tools, per a PCMA release.
Abcuro raised a $66 million Series C to run a new Phase 3 of ulviprubart in inclusion body myositis after its Phase 2/3 missed in the broad population, per Endpoints News.
BioSpace reports newly released EU documents cite serious breaches in the pivotal Tavneos study; our call that the evidence base was unraveling stands.
BioCryst is ending internal discovery, per Fierce Biotech, and shareholders of tissue healing company AMS approved its sale to H.B. Fuller, per MedTech Dive.
📖 In Today's Full Analysis
Be sure to read the full article for all the details, including:
The full deramiocel timeline and the HOPE-3 Lancet endpoint table, including what moved in peer review
How a major amendment resets a PDUFA goal date, and what to watch for from the FDA
The Celgene CVR term sheet: the $9 trigger, the three drugs, the deadlines, and the five week miss
Deal tables for Silence's $201.3 million offering and the Fibrx combination, plus the weekly oral GLP-1 script table
📋 The Week in Review
Last Monday: Replimune's Tudriqev won on the third try; Curium bid up to $8.0 billion for Lantheus' radiodiagnostics; the through line was set: infrastructure over pipeline.
Tuesday: Sionna's SION-719 missed, Tenax's LEVEL missed, Silence's divesiran won. The left tail and the right tail on one morning.
Wednesday: the FDA rejected the factory, not the drug: ITM's manufacturing only CRL and Scholar Rock's fill finish surgery to protect September 30.
Thursday: Definium's DT120 went two for two in Phase 3; Prader-Willi absorbed the Vykat XR safety cluster and Aardvark's termination.
Friday: the Sangamo auction priced platform and drug in one courtroom; Zealand sold its rusfertide royalty; the thesis paid off.
Weekend: quiet. Capricor's deferral, the CVR revival, Silence's close, and Fibrx were the Friday carryovers, and the ledger opens today.
🔒 BioMed Nexus Pro
In Today's Pro Brief...
🧠 What a PDUFA deferral is actually worth: why the market paid roughly 70 percent for a delay, and the one number in the Lancet paper that decides whether it was right
📊 The Celgene CVR as the ledger's oldest entry: the clause every milestone written last week is now missing, and what to demand in the next one
💰 The right tail cash out and the creation vehicle: how Silence and Fibrx fit the barbell count, and what the 5.38 percent left to Skye's holders tells you about the price of arriving without cash
🎯 Plus the full catalyst calendar, with August 22 restated and three new entries
The ledger opened with a coin flip the FDA agreed not to call and a coin flip a court agreed to re examine seven years late. Both tell you the same thing: a binary is never really gone, it just changes hands. What are you watching? Reply to this email.
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