Endpoints News

FDA widens use of leucovorin based on external controls, no new trial data The FDA approved the now-generic drug leucovorin to treat patients with a form of cerebral folate deficiency, satisfying a monthslong effort to widen the drug’s use for a condition whose symptoms can ...

The FDA has approved leucovorin to treat patients with a form of cerebral folate deficiency, a condition with symptoms that can resemble a form of autism. The agency did not base its decision on additional clinical data.
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China is going after the world's most expensive drugs: Endpoints Signal Chinese companies dominate global gene therapy competition with 48 programs vs 20 in US, targeting expensive Western treatments like Zynteglo, Elevidys, and Roctavian at fraction of cost. More evidenc...

China is going after gene therapies at a fraction of the cost in the US, an Endpoints News analysis found.
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Breakout Ventures closes $114M fund, with plans for about 20 investments The latest fund announced Tuesday is the firm’s first raise in five years and third overall, and includes both new and existing investors.

Breakout Ventures reels in a new $114M fund and is planning to invest it in about 20 companies.
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Who won the Hims vs Novo feud? YouTube video by Endpoints News

@shelbylivingston.bsky.social and @lydiapflanzer.bsky.social, the pens behind Health Tech, chat with senior biopharma journalist @lizcairns.bsky.social later this morning, March 10, at 11:30 a.m. ET to dive deep into the saga. Join here: www.youtube.com/live/W2-lDqy...

Hims agrees to limit compounding, ending feud with Novo Nordisk Hims & Hers agrees to offer Novo Nordisk's GLP-1 drugs and limit compounded versions, ending lawsuit. CEO Dudum cites shift in market toward FDA-approved treatments.

⚔️ The Hims-Novo feud has come to an end (maybe?): Hims agrees to limit its compounding offer and list Novo's GLP-1s on its website.
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BioNTech co-founders to exit company and start a new one BioNTech founders Sahin and Türeci to depart by year-end to start new mRNA company; BioNTech to focus on late-stage pipeline and take minority stake in new venture.

BioNTech co-founders Uğur Şahin and Özlem Türeci will leave the company to start a new next-generation mRNA enterprise.
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Exclusive: RNA editing startup Tacit Therapeutics launches with $19M for brain diseases Tacit Therapeutics raises $19M to develop RNA editing therapies, targeting Huntington's disease and Dravet syndrome. CEO David Nelles leads effort using RNA splicing to fix genetic mutations.

RNA editing startup Tacit Therapeutics launches with $19M to make "trans-splicing" therapies that replace large swathes of broken genetic code in brain diseases including Dravet syndrome and Huntington's disease. My exclusive for @endpts.com has the details: endpoints.news/rna-editing-...

What Prasad's exit means for the FDA moving forward Vinay Prasad exits FDA leadership roles amid controversy over vaccine reviews and staff issues. CBER director and chief medical officer positions now vacant, with Tracy Beth Høeg possible interim repl...

Vinay Prasad’s exit leaves a leadership gap at the FDA’s biologics center — and raises new questions about how the agency will handle vaccines and rare disease drugs.

Exclusive: RNA editing startup Tacit Therapeutics launches with $19M for brain diseases Tacit Therapeutics raises $19M to develop RNA editing therapies, targeting Huntington's disease and Dravet syndrome. CEO David Nelles leads effort using RNA splicing to fix genetic mutations.

Exclusive: RNA editing startup Tacit Therapeutics has launched with $19M to develop therapies for brain diseases, starting with Huntington’s.

Updated: Roche’s big-hope breast cancer drug fails in crucial first-line trial Roche's breast cancer drug giredestrant fails key Phase 3 trial when tested with Pfizer's Ibrance, falling short of progression-free survival goals vs placebo.

Roche’s breast cancer pill failed a key Phase 3 first-line trial, undermining hopes it could become the company’s biggest-selling drug.

Xenon gets Phase 3 win with epilepsy drug, fueling blockbuster hopes for its first launch Azetukalner greatly reduced the frequency of seizures in a Phase 3 epilepsy study and would be Xenon's first commercial product since its founding in 1996.

Xenon scored a Phase 3 win with its epilepsy drug azetukalner, cutting seizure frequency and setting up an FDA filing for what could become the company’s first blockbuster.

Universal Health Services buys Talkspace for $835M Universal Health Services is acquiring Talkspace in an area closely watched for consolidation, as virtual mental health companies grapple with falling valuations despite high demand after a pandemic-e...

Universal Health Services is buying virtual mental health company Talkspace for $835M, marking a major consolidation move in the teletherapy sector.

Lonza sells capsule and health ingredients unit to UK's Lone Star for $2.2B The separation has been a long time coming for Lonza, which first divulged plans to divest the unit in December 2024.

Lonza is selling its capsules and health ingredients unit to UK investment firm Lone Star for $2.2B upfront, completing its shift to a pure-play CDMO.

Servier to buy Day One for $2.5B, gaining rare oncology medicines The French pharma said on Friday it will pay $21.50 a share in cash, marking a 68% premium over Day One's Thursday closing price.

Servier is buying Day One Biopharmaceuticals for $2.5B, gaining its rare cancer portfolio including the marketed brain tumor drug Ojemda.

Lilly plucks Flagship, bluebird vet for regenerative medicine unit; Novo's new China president → Anne-Virginie Eggimann announced on LinkedIn that she has jumped to Eli Lilly as chief development officer of its regenerative medicine business. Eggimann spent the last ...

In this week’s Peer Review: Eli Lilly hires Anne-Virginie Eggimann to lead development for its regenerative medicine unit, Novo Nordisk names Yan Cai as China president, and Takeda taps Alan Walshe to run its Canada operations.

What will the US government do about China biotech? Live at 12:30 p.m. ET National Security Commission on Emerging Biotechnology's Caitlin Frazer to discuss US-China biotech competition and policy recommendations on Post-Hoc Live at 12:30 ET

🎙️Live today at 12:30 ET: Endpoints reporters @maxbayer.bsky.social and @zacharybrennan.bsky.social speak with NSCEB Executive Director Caitlin Frazer about the rise of China’s biotech industry, the commission’s policy proposals, and what steps the US could take to protect its biotech sector.

As FDA decisions get harder to predict, top official blasts outside advisory panels FDA senior official criticizes advisory committees as costly and ineffective, questions their value during press briefing, following uniQure gene therapy feedback and amid concerns about agency transp...

A top FDA official blasted advisory committees as “costly” and “slow,” questioning their value as companies complain that agency decisions are becoming harder to predict.

Multimillion-dollar gene therapies weren’t supposed to face competition. China has other plans Chinese firm Belief BioMed launches $350K hemophilia B gene therapy, challenging $3.5M Hemgenix by CSL Behring/uniQure, as China leads global race for cheaper gene therapies.

Multimillion-dollar gene therapies weren’t supposed to face competition. China has other plans

Latest feature + data analysis

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Merck KGaA drops two cancer trials on assets from SpringWorks deal The German pharma has removed studies of two drugs it picked up from the SpringWorks Therapeutics buyout, and cut a PARP1 inhibitor that it licensed from Hengrui.

Merck KGaA is dropping two cancer trials tied to its SpringWorks acquisition, trimming its pipeline after a strategic review.

Tenaya wins support from Alnylam in cardio disease pact Alnylam partners with Tenaya Therapeutics, paying $10M upfront for access to cardiovascular genetic targets, with potential milestone payments of $1.13B.

Tenaya landed a partnership with Alnylam, securing $10M upfront in a cardiovascular genetics deal that could be worth up to $1.13B in milestones.

Multimillion-dollar gene therapies weren’t supposed to face competition. China has other plans Chinese firm Belief BioMed launches $350K hemophilia B gene therapy, challenging $3.5M Hemgenix by CSL Behring/uniQure, as China leads global race for cheaper gene therapies.

Multimillion-dollar gene therapies weren’t supposed to face real price competition. China’s pipeline suggests otherwise. @jaredwhitlock.bsky.social explores the shift in the latest Endpoints In Focus.

Cell and Gene Day 2026 Cell and gene therapy has some wind beneath its sails. New regulatory frameworks from the Food and Drug Administration could help streamline development and manufacturing. Large pharma companies are i...

Today at 11 AM ET: The FDA is streamlining CGT R&D and big pharma is back at the table, but can the new science, regulatory and manufacturing leaps address the fundamental issues that still haunt the field? Find out what’s actually different this time — join us.

FDA puts PepGen’s Phase 2 muscle disease trial on partial hold The agency raised questions about the previously submitted preclinical pharmacology and toxicology studies with PepGen's drug for myotonic dystrophy type 1.

The FDA has put PepGen’s Phase 2 muscle disease trial on partial hold over safety concerns tied to earlier animal data, sending shares down 18% in premarket trading.
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FDA's top operating official Butler to retire, deputy to take over in April FDA's Barclay Butler to retire, with Melanie Keller taking over as deputy commissioner for operations and COO on April 6, per Commissioner Makary's email to staff.

FDA operations chief Barclay Butler is set to retire, with deputy Melanie Keller stepping into the role in April amid ongoing turbulence at the agency.

Yale’s Craig Crews builds model to guide biotech startups out of academia’s ‘valley of death’ Craig Crews' Quarry is a 'venture studio for serial academic entrepreneurs' that has already spawned four companies.

Yale’s Craig Crews is launching a new venture studio model to help spin biotech startups out of academia and bridge the scientific “valley of death.”

BIO chief maintains support for Makary, but calls some FDA decisions 'head-scratchers' John Crowley says he still supports FDA Commissioner Marty Makary even after a series of regulatory decisions on his watch have caused frustration and consternation among the biotech industry.

BIO CEO John Crowley says he still supports FDA Commissioner Marty Makary, but calls some recent agency decisions on rare disease and gene therapy “head-scratchers.”

Sanofi licenses Sino Biopharm's 'breakout star' transplant drug for $135M upfront Sanofi pays $135M upfront to Sino Biopharmaceutical for rovadicitinib, a transplant rejection therapy, with potential milestone payments of $1.4B plus royalties.

Sanofi is paying $135M upfront to license Sino Biopharm’s transplant drug, securing global rights in a deal worth up to $1.4B in milestones.

Moderna will pay $950M to settle Covid vaccine patent claims with Arbutus, Roivant Moderna to pay $950M to Arbutus & Genevant in Covid vaccine patent dispute; may owe $1.3B more pending court decision. CEO Bancel says it removes uncertainty.

Moderna will pay $950M to settle Covid-19 vaccine patent claims with Arbutus and Genevant.

UCB boards T cell engager train in $60M upfront deal with Antengene UCB partners with Antengene on CD19xCD3 T cell engager ATG-201 in $60M upfront deal, joining competitive space with Amgen, AstraZeneca, Merck and others.

UCB is paying $60M upfront to partner with China’s Antengene on a T cell engager, joining the crowded race in next-gen immunotherapy.
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In reversal, Prime Medicine will seek FDA approval of previously shelved gene therapy While other drugmakers have criticized the FDA’s recent judgment on rare disease treatments, Prime Medicine says the agency’s rhetoric pushed it to move forward with a rare immune disease therapy it h...

In a reversal, Prime Medicine will seek FDA accelerated approval for a previously shelved gene editing therapy in rare immune disease, citing the agency’s evolving stance on rare conditions.