China Startup CirCode Gets Clearance for Trial of Circular RNA Therapy
Cir‑Code Bio‑med, a Chinese biotech focused on circular RNA medicines, has secured an Investigational New Drug (IND) approval from the U.S. Food and Drug Administration to commence its first human trial. The therapy targets a rare genetic disorder using a self‑splicing circular RNA construct that can produce therapeutic proteins more durably than linear mRNA. This clearance marks the inaugural FDA‑approved study of a circular RNA drug candidate. The trial will evaluate safety, dosing, and expression kinetics in a small patient cohort.
Structure Therapeutics Reports More Phase 2 Data for Oral GLP-1
Structure Therapeutics released Phase 2 data for its oral GLP‑1 agonist, positioning the candidate as a next‑generation alternative to injectable therapies from Eli Lilly and Novo Nordisk. The trial demonstrated a mean 30% reduction in HbA1c and 70% of participants achieving target...
After Novartis Pact, Macrocycle Shop Unnatural Products Gets $45M Series B
Santa Cruz‑based Unnatural Products secured a $45 million Series B round to accelerate its macrocycle drug platform. The financing follows a newly announced partnership with Novartis that will grant the pharma giant early access to the company’s pipeline and a co‑development option...
Gubra Plots Roivant-Like Model as Partnered Obesity Drugs Take Flight
Gubra, a 300‑employee Danish biotech known for its obesity drug partnerships, announced plans to adopt a Roivant‑style incubation model. The strategy will spin out a series of focused biotech companies, each targeting niche therapeutic areas beyond obesity, such as metabolic...
Immutep's LAG-3 Drug Fails Phase 3 Lung Cancer Study
Immutep’s LAG‑3 fusion protein eftilagimod‑alpha failed to improve overall survival in a Phase 3 randomized study in non‑small cell lung cancer. The trial, enrolling roughly 600 patients, did not meet its primary endpoint and showed no statistically significant benefit versus standard...
Ultragenyx’s Gene Therapy Hits Key Endpoint in Phase 3 Trial for Blood Ammonia Disease
Ultragenyx announced that its investigational gene therapy for ornithine transcarbamylase (OTC) deficiency met a primary endpoint in a Phase 3 trial involving 37 patients. The study demonstrated a statistically significant reduction in blood ammonia levels, the key marker of disease...
Flagship Startup Loses CEO to Ipsen; Ionis to Shed Nearly Six Decades of Board Experience
Alltrna's CEO Michelle Werner announced her resignation, moving to pharmaceutical giant Ipsen as its new chief executive. The biotech startup will rely on interim management while it searches for a permanent replacement. Simultaneously, Ionis Therapeutics disclosed that several long‑standing board...
Grail Names New CEO as Bob Ragusa Retires
Grail announced that long‑time CEO Bob Ragusa will retire, handing the role to current president Josh Ofman. The transition follows a multi‑year succession plan designed to ensure leadership continuity. Ofman, who has overseen product launches and regulatory milestones, will assume...
Pfizer to Close Ignite, Its R&D Services Unit for Small Biotechs
Pfizer announced the shutdown of its Ignite unit, a dedicated R&D services platform that partnered with early‑stage biotech companies. Ignite offered fee‑based scientific, regulatory, and strategic support, helping dozens of startups advance drug candidates. The closure aligns with Pfizer’s cost‑discipline...
A Simpler Form of DNA May Be Key to Non-Viral Gene Therapy, Study Suggests
Researchers have identified a streamlined DNA construct that could replace viral carriers in gene‑therapy applications. The study demonstrates that this minimalist DNA format delivers therapeutic genes with efficiency comparable to adeno‑associated viruses while eliciting a weaker immune response. Production of...
Biotech Startups Use Funding Extensions to Sidestep Full Capital Rounds
Biotech startups are increasingly opting to extend existing financing rounds instead of launching fresh fundraising campaigns. By adding capital to current rounds, companies can secure needed cash while avoiding the dilution and valuation pressure of a full round. This approach...
Lilly Warns of Impurity Risk in Certain Compounded Forms of Mounjaro and Zepbound
Eli Lilly warned that compounded versions of its GLP‑1 drugs Mounjaro and Zepbound, when mixed with vitamin B12, may contain unsafe impurities. The alert follows growing off‑label compounding as demand outpaces supply. Lilly’s notice urges clinicians and patients to verify product sources...
FDA Merges Adverse Event Trackers Into One Database
The FDA announced a unified adverse‑event database that will eventually incorporate all of its existing reporting systems, including the vaccine‑side‑effect tracker jointly operated with the CDC. The new platform, called the Adverse Event Monitoring System, centralizes data from drugs, biologics,...
Eli Lilly CEO David Ricks Seeks 'Exit Velocity' For Pharma's Future
Eli Lilly’s chief executive David Ricks announced a strategic push to sustain the company’s recent $1 trillion revenue milestone by accelerating AI‑driven drug discovery and manufacturing. He highlighted a new partnership with Nvidia to embed generative‑AI models across the R&D pipeline, aiming...
RA Capital, Forbion and Canaan Appear to Back Harbour's CTLA-4 Partner Solstice
RA Capital, Forbion, and Canaan have collectively invested in Solstice Therapeutics, the CTLA-4 antibody partner of Harbour Therapeutics. The funding round, reportedly a multi‑million dollar Series B, will support Solstice's pre‑clinical and early clinical programs. By backing Solstice, the investors...
Study Suggests Long Non-Coding RNA Has Potential as New Class of Genetic Medicine
Researchers at the University of Toronto have engineered synthetic long non‑coding RNA (lncRNA) molecules that can dampen inflammatory responses. The study, led by PhD student Janice Pang and biologist Omar Khan, demonstrates that these engineered lncRNAs reduce cytokine release in...
Lundbeck CEO Talks Drug Pricing, Protecting Biotech and European Needs
Lundbeck chief executive Charl van Zyl warned that Europe’s fragmented drug‑pricing regime threatens biotech sustainability and patient access. He cited recent remarks by HHS Secretary Robert F. Kennedy Jr. suggesting Europeans already pay higher prices than Americans. Van Zyl called...
China Is Going After the World's Most Expensive Drugs: Endpoints Signal
China is actively targeting ultra‑expensive gene therapies, challenging the notion that these treatments are immune to price competition. Recent regulatory approvals and centralized procurement initiatives have forced manufacturers to negotiate steep discounts on therapies that once commanded multi‑million‑dollar price tags....
FDA Widens Use of Leucovorin without New Trial Data
The U.S. Food and Drug Administration has approved the generic form of leucovorin, also known as folinic acid, for treating cerebral folate deficiency (CFD). The decision widens the drug’s label despite the absence of new clinical trial data, relying on...
Breakout Ventures Closes $114M Fund, Plans 20 Investments
Breakout Ventures announced the close of a new $114 million venture fund, its first dedicated vehicle, aimed at backing roughly 20 early‑stage companies. The capital raise underscores the firm’s confidence in high‑growth sectors such as SaaS, fintech and artificial intelligence. Breakout...
BioNTech Co-Founders to Exit Company and Start a New One
Uğur Şahin and Özlem Türeci announced they will leave BioNTech by the end of 2026 to launch a new company focused on next‑generation mRNA technologies. BioNTech will concurrently narrow its portfolio, concentrating on late‑stage therapeutic candidates and its existing vaccine...
Universal Health Services Buys Talkspace for $835M
Universal Health Services (UHS) announced the acquisition of digital therapy platform Talkspace for $835 million, marking a major entry into virtual mental‑health care. The deal occurs as tele‑health valuations soften despite sustained demand for remote counseling. Talkspace’s subscription model and clinician...
AbbVie, Gubra Post Obesity Data; Regeneron Obesity Drug Succeeds in China
AbbVie and its partner Gubra released Phase 2 data on a long‑acting amylin analogue that produced significant weight loss in obese participants, with reductions approaching double‑digit percentages and a clean safety signal. The study highlighted dose‑responsive efficacy and tolerability, positioning the...
Bristol Myers Says Second CELMoD Succeeds in Phase 3
Bristol Myers Squibb announced that its oral CELMoD candidate mezigdomide met primary endpoints in the Phase 3 SUCCESSOR‑2 trial for relapsed or refractory multiple myeloma. The open‑label study showed a statistically significant improvement in progression‑free survival compared with the current standard...
RNA Editing Startup Tacit Therapeutics Launches with $19M for Brain Diseases
Tacit Therapeutics, an RNA‑editing startup focused on neurological disorders, announced its launch backed by a $19 million financing round. The capital, led by Andreessen Horowitz and DCVC, will fund the development of ADAR‑based therapeutics targeting diseases such as ALS, Huntington’s, and...
Roche’s Big-Hope Breast Cancer Drug Fails in Crucial First-Line Trial
Roche’s oral breast‑cancer therapy, touted as a potential blockbuster, missed its primary endpoint in the pivotal Phase 3 persevERA trial. The study evaluated the drug as a first‑line treatment for hormone‑receptor‑positive, HER2‑negative metastatic breast cancer and enrolled more than 1,200 patients...
GLP-1 News Galore; Top 100 Venture Investors; Doug Ingram to Step Down; and More
This week’s Endpoints Weekly highlighted a surge of GLP‑1 developments, including new trial data and expanded indications that reinforce the class’s dominance in obesity and diabetes treatment. The newsletter also released its annual Top 100 venture investors list, showing a notable...
Hims Reports 2025 Earnings, Shares Details on GLP-1 Pill Decisions
Hims & Hers disclosed its fourth‑quarter earnings and announced that it has withdrawn the controversial GLP‑1 weight‑loss pill it briefly launched. The decision followed conversations with key ecosystem stakeholders, including regulators and partner pharmacies. The company highlighted modest revenue growth...
Novartis to Divest India Unit to Private Equity-Led Consortium for $159M
Novartis announced the sale of its majority stake in its Indian generics and prescription business to a private‑equity‑led consortium for $159 million. The transaction transfers control of a portfolio that includes both off‑patent generics and branded medicines to the buyers. The...
Sanofi Appoints Specialty Care Head; Biogen Chair to Retire in June
Sanofi announced the appointment of a new head of specialty care, effective March 1, as the company prepares for the transition to CEO Belén Garijo. The new executive will oversee oncology, immunology and rare‑disease portfolios, aiming to accelerate pipeline launches....
Grail's Cancer Test Misses Primary Study Goal in UK Study
Grail announced that its Galleri multi‑cancer blood test failed to achieve the primary endpoint in a large United Kingdom clinical trial. The study, which evaluated the test’s ability to detect early‑stage cancers across dozens of tumor types, showed promising signals...
AbbVie, PhRMA Can Proceed with 340B Case in Hawaii
A federal judge has ruled that AbbVie and the Pharmaceutical Research and Manufacturers of America (PhRMA) may move forward with most of their claims challenging Hawaii's 340B law. The state statute limits how discounted 340B drugs are distributed, and the...
Teva Beats Corcept in Appeals Fight over Generic Cushing's Syndrome Drug
Teva Pharmaceutical secured a legal victory as the U.S. Court of Appeals for the Federal Circuit ruled that its generic version of a Cushing’s syndrome treatment does not infringe Corcept Therapeutics’ patents. The decision clears the path for Teva to...
Obesity Biotech Verdiva Plans Big Year of Data, Explores Deals
Verdiva Bio, an obesity‑focused biotech, is gearing up for a data‑heavy 2026, with Phase 2 results for its lead long‑acting peptide slated for the third quarter. The company is also courting strategic partnerships, eyeing potential deals with major pharma players after...
Biogen Stops Part of an MS Trial; Merck Reports More Enflonsia Data
Biogen announced it is halting the combination‑therapy arm of its multiple sclerosis (MS) trial after interim data showed insufficient efficacy and safety concerns. The decision pauses enrollment in the experimental regimen while the company continues evaluating its monotherapy components. Meanwhile,...
Altesa Raises $75M for Oral Respiratory Med
Faeth Goes Public in Merger with Sensei, Nets $200M in Stock Sale
Gilead Gets Cancer Drug From China-Based Genhouse for $80M Upfront
Gilead Sciences announced it will acquire the worldwide rights to an oral cancer therapy from China‑based Genhouse Bio, paying an $80 million upfront fee. The deal comes as Genhouse prepares for a Hong Kong IPO, positioning the drug for rapid global...
Pentagon Adds, Then Withdraws, WuXi AppTec to Chinese Military List
The Pentagon briefly placed WuXi AppTec, a major Chinese contract research organization, on its list of firms that support the People’s Liberation Army, then withdrew the designation hours later without explanation. The move occurred amid intensified U.S. scrutiny of Chinese...
Lilly Appeals Retatrutide Classification Ruling in Case that Could Impact Compounders
Eli Lilly has filed a notice of appeal challenging the FDA’s classification of its experimental obesity injection, retatrutide. The agency labeled the product as a new molecular entity, granting it a 12‑year data exclusivity period. Lilly argues the classification is incorrect...
Epigenetic Editing Startup Moonwalk Shifts Focus to siRNA for Obesity
Moonwalk Biosciences, founded by CRISPR pioneer Feng Zhang and former Illumina CTO Alex Aravanis, announced a strategic pivot from epigenetic editing to siRNA‑based obesity therapies. The company secured a $120 million Series A round to fund the new platform, which targets liver‑expressed...
Moderna's Flu Shot Dilemma Muddies 2028 Break-Even Guidance
Moderna’s chief financial officer indicated that the company cannot yet confirm whether its 2028 cash‑flow break‑even goal will hold, as the outlook for its seasonal flu vaccine remains uncertain. The flu shot, once expected to be a steady revenue stream,...
Lilly Stockpiles $1.5B Worth of Weight Loss Pill Ahead of US Approval
Eli Lilly has amassed roughly $1.5 billion worth of its experimental obesity drug orforglipron. The company began stockpiling the product months before the U.S. Food and Drug Administration is expected to grant approval later this year. Orforglipron, a GLP‑1 receptor agonist, joins...
Immunic's Private Placement; Vertex's Cystic Fibrosis Data in Young Kids
Immunic announced an oversubscribed private placement raising up to $400 million to fund its shift toward a commercial‑stage biotech. The capital injection will support late‑stage development programs and accelerate the search for a new chief executive officer. Meanwhile, Vertex Pharmaceuticals disclosed...
Paul Hudson Couldn’t Quite Get Sanofi over the Hump; PROTAC Developer Arvinas Appoints CEO
Paul Hudson has struggled to revitalize Sanofi’s R&D engine despite a series of high‑profile acquisitions since becoming CEO in 2019. The French group’s pipeline has underperformed, and recent buyouts have failed to deliver the expected boost in innovative drug candidates....
Isomorphic Claims Major Advance with New AI Drug Design Engine
Isomorphic Labs, the DeepMind spin‑off behind AlphaFold 3, announced a new AI‑driven drug design engine that builds on its protein‑structure predictions. The platform claims to generate novel small‑molecule candidates up to ten times faster while achieving sub‑nanomolar binding‑affinity accuracy. Early validation...
ILiAD Raises $115M Round to Advance Whooping Cough Vaccine
ILiAD Biotechnologies announced an oversubscribed $115 million Series B financing round to accelerate development of its next‑generation whooping cough (pertussis) vaccine. The capital will fund pivotal Phase 3 clinical trials, aiming to demonstrate superior efficacy and longer‑lasting immunity compared with current acellular vaccines....
AbbVie, AstraZeneca Lose Appeal in Louisiana 340B Contract Pharmacy Fight
A U.S. appeals court upheld Louisiana's law that restricts how 340B‑discounted drugs can be dispensed through contract pharmacies. The ruling rejected the challenge mounted by AbbVie and AstraZeneca, marking another defeat for drugmakers contesting state oversight of the federal 340B...
NIH Stops Low-Dose Xarelto Arm of Large Stroke Study Due to Safety Concerns
The National Institutes of Health has stopped the low‑dose Xarelto arm of a large, multi‑center stroke trial after an interim safety analysis revealed excess bleeding. The study, which enrolled roughly 5,000 patients with recent ischemic stroke, was testing rivaroxaban 2.5 mg...
Shanghai's Cascade Gets $72M to Pursue MASH, Obesity and Diabetes Drugs
Shanghai‑based Cascade Pharmaceuticals announced a financing round of roughly 500 million yuan ($72 million). The capital will fund the development of novel therapeutics targeting metabolic dysfunction‑associated steatohepatitis (MASH), obesity and type‑2 diabetes. Cascade joins a growing cohort of private biotech firms receiving...