Today's Pharma Pulse
Ona lands $86.6M Series B, one of Spain’s biggest biotech rounds
Spanish biotech firm Ona announced an $86.6 million Series B financing, ranking among the largest venture rounds in Spain’s life‑science sector this year. The round was led by Seventure Partners with participation from existing backers and new strategic investors. The funding will be used to scale Ona’s AI‑driven drug discovery platform and accelerate its pre‑clinical programs.
Verdiva Bio Completes Enrolment for Phase 2b Study of Oral GLP‑1 Candidate VRB‑101
Verdiva Bio announced that enrollment for its phase 2b EVOLVE‑2 trial of VRB‑101, a once‑weekly oral GLP‑1 peptide analog for weight loss, is complete with more than 200 participants across 22 U.S. sites. The double‑blind, placebo‑controlled study will assess safety, tolerability and efficacy over 20 weeks, with the primary endpoint measuring mean percentage body‑weight change. Topline results are slated for release by the end of 2026, and favorable outcomes would pave the way for phase 3 trials in 2027.
Male Infertility Care to Shift Treatment Burden From Women
Infertility has long been framed as a women's issue, yet about half of cases stem from male factors. New treatments such as Igyxos' IGX12 monoclonal antibody, which enhances follicle‑stimulating hormone activity, aim to improve sperm production and reduce the physical...
Division of Applied Regulatory Science
The Division of Applied Regulatory Science (DARS) operates within the FDA’s Office of Clinical Pharmacology and the Office of Translational Sciences. Its mandate is to translate emerging scientific advances into the agency’s regulatory framework, tackling complex questions that hinder drug...
Valo Health CEO: We Don’t Want Investors To Drive the Science
Valo Health is reshaping drug development by applying AI to human causal biology, aiming to raise clinical success rates from roughly 10% to 20%. The company leverages over 17 million de‑identified patient records and Mendelian randomization to pinpoint genetically validated targets....
Novo Nordisk Inks A $2.1 Billion Deal To Develop Next Generation Obesity Pills
Novo Nordisk has struck a deal with Boston biotech Vivtex worth up to $2.1 billion to develop next‑generation oral GLP‑1 obesity and diabetes pills. The partnership leverages Vivtex’s AI‑driven gastrointestinal‑on‑a‑chip platform that can boost drug absorption by orders of magnitude. Novo...
Generate Biomedicines (GENB) IPO Deck
Generate Biomedicines (GENB) filed its IPO prospectus, unveiling a programmable biology platform that engineers therapeutic proteins previously considered undruggable. The company highlighted a pipeline featuring oncology and immunology candidates that have progressed to Phase 2 trials, supported by early efficacy signals....
Patient Death Forces Partial Freeze on MacroGenics’ Gynecologic Cancer Study
A patient in MacroGenics' Phase 2 LINNET trial of the bispecific antibody lorigerlimab suffered grade 4 neutropenia and septic shock, leading to a fatality and prompting the FDA to place a partial clinical hold on the study. The company also reported three...
IDEAYA Biosciences Reports the First Patient Enrolment in P-I Trial of IDE034
IDEAYA Biosciences announced the enrollment of the first patient in a Phase‑I dose‑escalation and expansion study of IDE034, a bispecific B7H3/PTK7 TOP1 antibody‑drug conjugate for solid tumours. The trial will assess safety, tolerability and pharmacokinetics of IDE034 as a monotherapy...
Tooth Whitening without Enamel Damage? Vibration-Activated Powder Protects Pearly Whites for Better Oral Health
Researchers at Shanghai Xuhui District Stomatological Hospital and the Chinese Academy of Sciences have engineered a vibration‑activated teeth‑whitening powder (BSCT) that works with electric toothbrushes. The ceramic powder uses piezoelectric catalysis to generate reactive oxygen species for stain removal while...
IQVIA Signs Agreement to Acquire Drug Discovery Assets From Charles River Laboratories, Expanding End-to-End Drug Discovery Capabilities
IQVIA Holdings announced an agreement to acquire five drug discovery service sites from Charles River Laboratories, adding in‑vitro capabilities, New Approach Methodologies and a small‑molecule AI platform. The assets, built on more than 20 years of data, have helped launch...
Lilly Bests Novo Again, Rare Disease Week Goes Regulatory, More CDC Leadership Upheaval
Eli Lilly’s obesity drug Zepbound achieved a 25.5% weight loss in a head‑to‑head Phase 3 trial, outpacing Novo Nordisk’s CagriSema and sending Novo’s shares down 20%. The FDA launched Rare Disease Week, issuing draft guidance for a new Plausible Mechanism Pathway that...
FDA Enforcement on Copycat GLP-1's Could Reshape Retail Pharmacy Economics: Q&A with David Graziano
The FDA’s crackdown on non‑approved or “copycat” GLP‑1 drugs is expected to push patients toward FDA‑approved therapies, elevating the role of retail pharmacies as primary access points. GoodRx’s head of retail network, David Graziano, says the company’s cost‑plus pricing model...
SteinCares Forges Licensing Agreement with Shilpa Biologicals for Biosimilars Across Latin America
SteinCares has signed a strategic licensing agreement with Shilpa Biologicals to commercialize a biosimilar across Latin America. Under the deal, SteinCares receives exclusive rights to register, market and distribute the product throughout the region, while Shilpa Biologicals will complete development...
Vico Therapeutics Initiates Patient Dosing for VO659 Phase I/IIa Trial
Vico Therapeutics has initiated patient dosing in an expanded European cohort for its Phase I/IIa VO659 trial, targeting Huntington's disease, spinocerebellar ataxia type 3 and type 1. The study employs a twice‑annual intrathecal regimen and will monitor safety, tolerability, pharmacodynamics and pharmacokinetics...
NorthX Biologics and Demeetra Collaborate on GMP Manufacturing and CLD
NorthX Biologics and Demeatriz have formed a strategic alliance to deliver an end‑to‑end platform that couples Demeatriz’s CleanCut CHO cell line development with NorthX’s GMP manufacturing capabilities. The partnership leverages a high‑titre GS‑knockout CHO cell line that carries no royalty...
Evolution Summit By Marcus Evans | May 7-8, 2026 | The Ritz-Carlton, Fort Lauderdale, Florida
The Evolution Summit, organized by Marcus Evans, will take place May 7‑8, 2026 at the Ritz‑Carlton in Fort Lauderdale. The invitation‑only event convenes senior clinical‑trial executives with innovative suppliers to discuss pressing challenges in clinical operations and drug development. Featured speakers include...
STAT+: GSK Picks up Pulmonary Hypertension Drug with 35Pharma Acquisition
GlaxoSmithKline announced a $950 million cash acquisition of 35Pharma, a privately held biotech focused on lung diseases. The deal brings the experimental compound HS235, slated to enter pulmonary arterial hypertension (PAH) trials, into GSK’s pipeline. PAH has become a lucrative target,...
Five-Year Data Show Sustained Benefits with Briumvi in Relapsing MS
Long‑term data from the Phase 3 ULTIMATE I and II trials show that continuous treatment with Briumvi (ublituximab) provides sustained clinical benefits for relapsing multiple sclerosis over five years. More than 80 % of patients who started Briumvi remained free of relapses and...
J&J Reports the FDA’s sBLA Submission for Imaavy to Treat Warm Autoimmune Hemolytic Anemia
Johnson & Johnson has filed a supplemental Biologics License Application (sBLA) with the U.S. FDA for Imaavy (nipocalimab‑aahu), a monoclonal antibody intended to treat warm autoimmune hemolytic anemia (wAIHA). The filing is supported by the Phase II/III ENERGY trial, which demonstrated...
In Brief SOTU Remarks, Trump Asks Congress to "Codify" MFN
President Donald Trump used a brief four‑minute segment of his State of the Union address to push Congress to codify his Most‑Favoured Nations (MFN) drug‑pricing program. He claimed the voluntary agreements would give Americans the lowest prescription prices worldwide, though...
Opinion: Balancing Safety Access in Rare Disease—Lessons From Sarepta
In July 2025 the FDA ordered a complete halt of Sarepta’s gene‑therapy Elevidys after two non‑ambulatory Duchenne muscular dystrophy (DMD) patients died, sparking a market plunge and patient access concerns. Sarepta argues that ambulatory patients, who had been receiving Elevidys...
Cashing In on ‘Creative Fundamentals’ To Fund Rare Disease R&D
The FDA reauthorized its rare pediatric disease priority review voucher program, bolstering a wave of regulatory incentives that include new guidance on plausible mechanisms and a Rare Disease Evidence Principles framework. Yet ultra‑rare indications still struggle to attract traditional venture...
TrumpRx Cuts Cash Prices, but Offers Little New Savings
President Trump negotiated lower prices for some drugs on TrumpRx, but discounts only apply to cash paying patients not using their insurance. Many drugs already had big discounts through manufacturers or coupons, and some have generic equivalents that are already...
Trump Pushes Secret Drug Deals Into Law Despite Opacity
President Trump has called for his deals with drug companies to be enacted into legislation. It's hard to know what that means since the deals are not public.
Ironwood Pharmaceuticals Inc (IRWD) Q4 2025 Earnings Call Transcript
IQVIA reported fourth‑quarter 2025 revenue of $4.34 billion, a 10.3% year‑over‑year increase, driven by strong organic growth and roughly two percentage points from recent acquisitions. Adjusted EBITDA rose 5% to $1.046 billion and adjusted diluted EPS climbed 9.6% to $3.42. The company...
GSK's New CEO Targets Proven Science, Eyes Obesity Drugs
The FT's report on GSK's $950M deal to buy 35Pharma and its pulmonary arterial hypertension drug includes an interesting unnamed insider's quote that new CEO Luke Miels is going after assets where "the science is relatively settled." Maybe leaning into...
Circulating piRNAs May Predict Elderly Survival and Longevity
Strong claim that needs independent replication: "Our findings provide compelling evidence that circulating smRNAs—especially piRNAs—are powerful predictors of survival in older adults and potential biomarkers of longevity" https://t.co/oi4220zxGY https://t.co/yk3MX4VdQj
United Therapeutics Corp (UTHR) Q4 2025 Earnings Call Transcript
United Therapeutics reported record Q4 2025 revenue of $800 million, a 7% year‑over‑year increase driven by double‑digit growth of its Tyvaso DPI inhaler and Orenitram. The company unveiled new 80‑112 µg Tyvaso DPI cartridges, expanding dosing flexibility, and highlighted unprecedented results from...
Novo's Vivtex Deal Could Unlock Superior Oral Obesity Meds
With Vivtex deal, Novo gains a chance at better oral obesity drugs https://t.co/DhIelbC9jI by @gwendolynawu $NVO #obesity
China's Asset Prices Rise, Ending Bargain-Basement Era
Assets from China are ubiquitous but China is no longer the 'bargain basement' in terms of deals. Will be interesting to see how rising prices impact deal interest https://t.co/yM3P28QCDc $XBI $IBB $BBC https://t.co/W61XACPX7O
Geron Corp (GERN) Q4 2025 Earnings Call Transcript
Geron Corp reported full‑year 2025 Rytelo net revenue of $184 million, with Q4 sales of $48 million, and projected 2026 revenue between $220 million and $240 million. Operating expenses are expected to dip to $230‑$240 million, a $20 million reduction year‑over‑year, while cash balances sit at...
Sarepta Therapeutics Inc (SRPT) Q4 2025 Earnings Call Transcript
Sarepta Therapeutics reported a blockbuster fourth quarter, with ELEVIDYS gene‑therapy sales of $384 million, surpassing guidance by $60 million, and total net product revenue of $1.79 billion for 2024, up 56% year‑over‑year. The company achieved GAAP profitability and cash‑flow positivity, confirming the financial...
Schrodinger Inc (SDGR) Q4 2025 Earnings Call Transcript
Schrödinger reported Q2 FY2025 revenue of $54.8 million, up 16% year‑over‑year, driven by 15% software growth to $40.5 million and 19% drug‑discovery growth to $14.2 million. Gross margin slipped to 68% as the mix shifted toward lower‑margin services and new predictive‑toxicology investments. Net...
ACADIA Pharmaceuticals Inc (ACAD) Q4 2025 Earnings Call Transcript
ACADIA Pharmaceuticals reported record adjusted 2025 revenue of $1.08 billion, up 14% year‑over‑year, driven by strong performance of NUPLAZID and DAYBUE. The company forecast 2026 total revenue between $1.22 billion and $1.28 billion, with NUPLAZID sales expected to reach $760‑$790 million and DAYBUE $460‑$490 million....
Revolution Medicines Inc (RVMD) Q4 2025 Earnings Call Transcript
Revolution Medicines reported that its lead KRAS inhibitor doraxonrasib secured breakthrough therapy, orphan drug, and a FDA National Priority Voucher, underscoring its potential in pancreatic cancer. Phase I data revealed median overall survival of 13.1‑15.6 months and a 47% objective...
Soleno Therapeutics Inc (SLNO) Q4 2025 Earnings Call Transcript
Soleno Therapeutics reported $190.4 million in net revenue for fiscal 2025, representing less than nine months of commercial sales of its VICAT XR therapy. The company turned profitable, posting $20.9 million net income and generating $48.7 million of operating cash in Q4, ending the...
Mirum Pharmaceuticals Inc (MIRM) Q4 2025 Earnings Call Transcript
Mirum Pharmaceuticals reported $521 million in 2025 net product sales, a 55% year‑over‑year increase driven by Livmarli and bile‑acid medicines, surpassing guidance. Operating expenses rose to $543 million, yet the company generated positive cash flow and ended the year with $391 million in...
Biomarker for Craving and Acamprosate Treatment Response in Patients with Alcohol Use Disorder: Insights From Multi-Omics
A multi‑omics investigation identified molecular signatures that link alcohol craving to acamprosate treatment response in patients with alcohol use disorder (AUD). The study highlighted IL17RB genetic variants and elevated plasma TNFSF10 as robust predictors of therapeutic success, while proteomic and...
AI-Powered Platform Accelerates Discovery of New mRNA Delivery Materials
University of Toronto researchers unveiled LUMI‑lab, an AI‑driven self‑driving lab that combined a 28‑million‑molecule foundation model with active‑learning robotics to synthesize and test over 1,700 lipid nanoparticles. The system independently identified brominated lipid tails as a potent new class for...
New Protein-Like Polymers Target, Degrade “Undruggable” Proteins Driving Cancer
Researchers at Northwestern University have created HYDRACs, hybrid degrading copolymers that bind and eliminate traditionally "undruggable" oncogenic proteins such as MYC and KRAS. The polymers display target‑recognizing peptides on one side and degron motifs on the other, directing the proteins...
FDA Approves Drug for Adult and Pediatric Patients Aged 6 and Older with Allergic Fungal Rhinosinusitis
The FDA has granted approval for Dupixent (dupilumab) to treat allergic fungal rhinosinusitis (AFRS) in adults and children six years and older, marking the first indication for this rare sinus disease. The approval follows a 52‑week trial that demonstrated significant...
Filkri Becomes Fifth FDA-Approved Neupogen Biosimilar
Accord BioPharma’s Filkri (filgrastim‑laha) received FDA approval as the fifth biosimilar to Amgen’s Neupogen. The indication set mirrors Neupogen’s, covering chemotherapy‑induced neutropenia, AML, bone‑marrow transplant, severe chronic neutropenia, and radiation‑induced syndrome. Approval was based on two randomized trials in healthy...
Sustained HIV Viral Suppression Restores Immune Potential: Victor Appay, PhD
New data presented at CROI 2026 show that people living with HIV who have maintained viral suppression for 25‑30 years on antiretroviral therapy regain robust HIV‑specific CD8⁺ T‑cell function. Using flow cytometry and single‑cell RNA sequencing, researchers found these cells display...
Verified Clinical Benefit | Cancer Accelerated Approvals
The FDA has granted traditional approvals to ten oncology drugs that originally received accelerated approval after post‑marketing studies verified clinical benefit. The list includes agents such as Braftovi, Epkinly, and Padcev, with conversion dates ranging from 2022 to 2026. These...
AI Must Model Dynamic Biology, Not Static Proteins
There is one major barrier blocking AI from truly reshaping biology and no one is talking about it. Biology is not static. Most AI breakthroughs so far have treated proteins, cells, and systems as frozen objects. That abstraction worked to get...
Bull Slams Wedbush's ABVX Report as Terrible
Lot's of requests to discuss the Wedbush report on $ABVX today. It's a doozy. Obviously I'm a biased bull so sure, take my thoughts with a grain of salt, but this is truly, awful work from my perspective....
Engineered Bacteria Can Consume Tumors From the Inside Out
University of Waterloo researchers have engineered the anaerobic bacterium Clostridium sporogenes to eat solid tumors from the inside out. By inserting an oxygen‑tolerant gene and coupling it to a quorum‑sensing circuit, the bacteria survive at the tumor periphery only after...
FDA Says Data, Not Numbers, Prove IBRX Benefit
Th FDA is absolutely correct, and confirms what I’ve said all along. This $IBRX @DrPatrick “lymphopenia” push means nothing until or unless they come up with real data showing patient benefit. The “number” doesn’t matter.
Former Pfizer CMO Joins $HELP, Credibility Boosted
$HELP adds former Pfizer CMO Dr. Freda Lewis-Hall to its board. Big pharma credibility bump. Now the pipeline has to earn it. https://t.co/CK7PsBE9B8
FDA Needs Clear Standards for Rare Disease Trials
If FDA is serious about moving with urgency in rare disease, the key question is evidentiary standards. Speed matters. But clarity on what constitutes “adequate and well-controlled” in ultra-small populations matters more. Predictability > rhetoric https://t.co/XVXg71NmGq