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.
How Policy Pressures Are Driving DTP Adoption
Policy pressures such as Medicare price negotiations, Most Favored Nation rules, and new transparency mandates are reshaping how life‑science companies sell medicines. These forces are accelerating adoption of direct‑to‑patient (DTP) models that bypass traditional intermediaries to deliver drugs more affordably. Executives at AssistRX report that clients are restructuring distribution and pricing strategies to prioritize patient choice across cash‑pay, free‑drug, and commercial pathways. Operational challenges remain, especially in coordinating with providers and pharmacies to ensure seamless delivery.
HDX-MS Plus Computational Methods Provide Novel Approach to Study of Protein-Protein Interactions
Regeneron scientists have integrated hydrogen‑deuterium exchange mass spectrometry (HDX‑MS) with AI‑driven computational analysis to pinpoint binding sites, geometry, and stoichiometry in sandwich ELISA assays. The approach rapidly distinguishes protected versus exposed regions of antibodies, boosting assay specificity and sensitivity. By...
STAT+: Clearing Tumors in Mice, Azalea Therapeutics Advances Dream of in Vivo CAR-T Therapy
Azalea Therapeutics, a spinout from Jennifer Doudna’s lab, reported in Nature that its in vivo CAR‑T approach can generate functional CAR‑T cells directly within mice and eradicate both solid and hematologic tumors. The technique uses infused gene‑editing particles that precisely...
Valbenazine
Valbenazine (Ingrezza®), an oral selective VMAT2 inhibitor from Neurocrine Biosciences, received FDA approval for treating tardive dyskinesia and Huntington’s disease‑associated chorea. In the Phase 3 KINECT‑3 trial, a once‑daily 80 mg dose produced a statistically significant reduction in AIMS dyskinesia scores after...
STAT+: A Huntington’s Researcher on the UniQure-FDA Fray
UniQure’s experimental gene‑therapy for Huntington’s disease, which previously reported a 75% slowdown in disease progression, has received a third consecutive rejection from the FDA. The trial’s lead investigator, Ed Wild of University College London, praised the early data but warned...
AI Startup Basecamp Research Announces Trillion-Gene Project
Basecamp Research, an AI‑focused biotech startup backed by Microsoft and Nvidia, announced a trillion‑gene sequencing initiative. The company aims to collect genetic sequences for over a trillion proteins within the next two years. Leveraging high‑performance cloud computing and advanced generative‑AI...
China’s CCP Stifles Cure Innovation, FDA Lag Exposes Gap
My @8VC partner Francisco is giving key testimony today about CCP crushing our ability to develop cures. China has legit innovators - but also a culture of stealing, and working around patents. Our legacy FDA is so slow that they can...
Ionis Delays Prion Disease Readout; Arvinas' Parkinson's Biomarker Data
Ionis Therapeutics announced that the primary completion date for its Phase 1/2a prion disease trial has been pushed to February 2027, extending the study timeline by more than two years. In parallel, Arvinas reported new biomarker data from its Parkinson’s disease program,...
Kennedy’s Vaccine Agenda Stalled, Structure’s ‘Competitive’ Obesity Pill, Novo’s Warning Letter
Structure Therapeutics reported a 16.3% weight loss after 44 weeks in a Phase 2 trial of its oral GLP‑1 pill, positioning it as a competitive alternative to Eli Lilly’s and Novo Nordisk’s candidates. Rhythm Pharmaceuticals disclosed that its obesity drug Imcivree failed in...
Briggs Morrison's Crossbow Unveils $77M Series B for T Cell Engagers
Crossbow Therapeutics, founded by biotech veteran Briggs Morrison, announced a $77 million Series B round to accelerate its off‑the‑shelf T‑cell engager platform. The funding, led by a mix of venture capital and strategic investors, will support the advancement of three preclinical candidates...
Aspen's Personalized Parkinson's Therapy Shows Early Promise
Aspen Neuroscience reported encouraging early data from a small autologous cell therapy trial for Parkinson's disease. The treatment, derived from each patient's own skin cells and reprogrammed into dopaminergic neurons, was safely implanted in twelve participants, showing graft survival and...
First MHRA/NICE Aligned Guidance Due in June
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and the National Institute for Health and Care Excellence (NICE) will begin delivering aligned guidance in June, enabling simultaneous licensing and reimbursement decisions for new medicines. Twenty‑seven companies have signed up...
Lilly-Backed China Startup Debuts With $68.7M Seed to Advance Next-Gen T Cell Engagers
Excalipoint Therapeutics, a Shanghai‑based biotech, closed a $68.7 million seed round, including a $41 million founding raise and a $27.7 million extension led by MPCi, Centurium Capital, Lilly Asia Ventures, and Eisai Innovation. The capital will fund six tri‑specific T‑cell engager candidates, notably...
STAT+: J&J Wins Approval for First-of-Its-Kind Psoriasis Pill
Johnson & Johnson received FDA clearance for Icotyde, the first oral daily pill for moderate‑to‑severe plaque psoriasis. The drug, originally called icotrokinra, is approved for patients aged 12 and older and is designed to replicate the efficacy of injectable biologics...
Myriad Genetics Receives the US FDA Approval for MyChoice CDx Test as a Companion Diagnostic for GSK’s Zejula
Myriad Genetics announced FDA approval of its MyChoice CDx test as a companion diagnostic for GSK’s Zejula (niraparib) in advanced ovarian cancer. The clearance follows the PRIMA trial, which showed that patients identified as HRD‑positive derived significant benefit from Zejula...
Navigating the Valley of Death: A How-To From an Investor’s Perspective
Biotech startups face the notorious "valley of death" as they move from proof‑of‑concept to commercialization, but investors remain active, with a 70.9% jump in venture‑financing deal value between Q2 and Q3 2025. A GlobalData survey shows 39% of respondents are...
Hope Rises for Vaccine Against Hookworm Parasite
A phase 2 trial of the Na‑GST1/Al–CpG vaccine demonstrated near‑complete protection against hookworm infection in healthy adults, with vaccinated participants shedding a median of zero eggs per gram versus 67 in the placebo group. The study, conducted in Washington, DC,...
Seaweed Shield: Marine Molecules May Block Norovirus Infection
Researchers from Griffith University and biotech firm Marinova evaluated brown and green seaweed polysaccharides for their ability to block norovirus attachment. Fucoidan, a sulfated fiber from brown seaweed, demonstrated the strongest and most consistent inhibition of virus‑like particles binding to...
Samsung Bioepis Partners with Sandoz for Up to Five Biosimilar Candidates
Samsung Bioepis and Sandoz have signed a global license, development and commercialization agreement covering up to five biosimilar candidates, including the preclinical SB36 biosimilar to Entyvio (vedolizumab). Samsung Bioepis will manage development, manufacturing and regulatory submissions, while Sandoz will handle...
US Court Says Pharma Giants Must Face False Claims Suit
The U.S. Ninth Circuit Court of Appeals denied dismissal motions by AbbVie, AstraZeneca, Novartis and Sanofi, allowing a whistleblower False Claims Act suit over alleged 340B program overcharges to proceed. The lawsuit, filed by Adventist Health System/West, claims the manufacturers...
Xaira Unveils AI Model X-Cell for Predicting Virtual Cells
Xaira, the biotech AI startup with the deepest funding round, launched its first model, X‑Cell, on Tuesday. The platform uses deep learning to generate virtual cell representations from multi‑omics data, enabling researchers to simulate cellular responses without physical experiments. Early...
How Is eClinical Positioning Its Platform Capabilities to Help Sponsors?
eClinical Solutions is leveraging its elluminate platform to help sponsors adapt to the FDA’s new requirement for a single pivotal trial, a move aimed at strengthening efficacy evidence and patient safety. The platform offers highly customizable centralized monitoring, AI‑driven risk...
R1 Therapeutics Raises $77.5m to Progress AP306 for Hyperphosphatemia
R1 Therapeutics announced a $77.5 million oversubscribed Series A round to advance AP306, a first‑in‑class pan phosphate transporter inhibitor for hyperphosphatemia in chronic kidney disease patients. The funding, led by Abingworth, F‑Prime and DaVita Venture Group, also secured an exclusive global licence...
Tick-Borne Encephalitis Study Identifies ABCG1 as Potential Target for Future Treatments
Researchers at the Medical University of Graz conducted one of the largest genome‑wide association studies on tick‑borne encephalitis (TBE), analyzing about 1,600 patients from multiple European nations. The study pinpointed variants in the cholesterol‑transport gene ABCG1 as a genetic risk...
START and Trialing Collaborate on Oncology Clinical Trials
START Center for Cancer Research has partnered with Trialing to broaden access to early‑phase oncology clinical trials throughout Europe. The exclusive agreement lets START distribute curated trial information and real‑time enrollment updates via Trialing’s platform, directly notifying oncologists when slots...
Promising Biotechs in Maryland Right Now
Maryland’s life‑science cluster, home to over 2,700 institutions, is projected to generate a $16.7 billion R&D market by 2026. Six home‑grown biotech firms—SciNeuro, Gain Therapeutics, Theriva Biologics, Supernus Pharmaceuticals, Elixirgen Therapeutics and Altimmune—have secured sizable financing and strategic partnerships in the...
Co PSMA Prostate Imaging Data Presented at EAU Congress
Clarity Pharmaceuticals presented Phase II Co‑PSMA data at the EAU Congress, showing its 64Cu‑SAR‑bisPSMA PET tracer outperforms the standard 68Ga‑PSMA‑11 in detecting prostate cancer recurrence at low PSA levels. In 50 men post‑radical prostatectomy, 64Cu‑SAR‑bisPSMA identified 63 lesions versus 24...
Cogent Biosciences Reports US FDA’s NDA Acceptance of Bezuclastinib for NonAdvanced Systemic Mastocytosis (NonAdvSM)
Cogent Biosciences announced that the U.S. FDA has accepted its new‑drug application for bezuclastinib in non‑advanced systemic mastocytosis (NonAdvSM), with a PDUFA target date of December 30 2026 and no advisory committee required. The filing is supported by the SUMMIT trial, which...
Ascendis Pharma Reports Topline P-II (New InsiGHTS) Trial Data on TransCon hGH in Turner Syndrome
Ascendis Pharma released topline results from its Phase‑II New InsiGHTS trial evaluating TransCon hGH (lonapegsomatropin) administered once‑weekly versus daily somatropin in 49 prepubertal children with Turner syndrome. At week 52, the weekly formulation achieved an LS mean annualized height velocity...
CTMX Shows Higher ORR Than Temab‑A, Needs Validation
Late line CRC therapies in development following $CTMX's data this week (from Guggenheim). A lot is riding on $CTMX's 10 mg/kg cohort which had just 19 evaluable patients (3 patients were excluded so ITT response rate is lower) so this needs...
MRK Unveils PD-1/VEGF Bispecific at AACR26
$MRK's PD-1 x VEGF bispecific reveal, & other key #AACR26 presentations, via @APEXONCO -> https://t.co/ob1eswxCWF $RVMD $AMGN $GILD $BCYC $SDGR
Outsmarting Resistance with Rhythm
In this episode, Immuneering CEO Ben Zeskin explains the company’s novel “deep cyclic inhibition” dosing strategy, which delivers intense, short‑duration MEK inhibition pulses instead of continuous suppression. By restoring the natural intermittent signaling rhythm in healthy cells while repeatedly ambushing...
RBC Flags Revolution, Xenon, Arrowhead as Top Biotech Takeover Targets
RBC: Revolution, Xenon, Arrowhead among top #biotech takeover targets https://t.co/h1toH9szrU by @realJacobBell $ARWR $XENE $RVMD $DYNE
5‑MeO‑DMT Shows Promise for Treatment‑Resistant Depression
Emerging evidence supports 5-MeO-DMT as a promising, ultra-short-acting psychedelic for treatment-resistant depression and other psychiatric conditions, warranting larger randomized controlled trials. https://t.co/6CcEOSat1F
Voro Therapeutics Collaborates with Daiichi Sankyo to Develop Tumor-Activated ADCs
Voro Therapeutics has signed a research collaboration with Daiichi Sankyo’s San Diego research institute to create tumor‑activated antibody‑drug conjugates (ADCs) using Voro’s PrimeBody platform. The partnership will focus on masked ADCs that employ proprietary masking domains and protease‑cleavable linkers to achieve...
Mitochondrial Transfer Shows Promise Against Parkinson’s in Animals
Potential of mitochondrial transfer to prevent or treat Parkinson' s disease, in mouse and monkey models @CellCellPress https://t.co/c0oqfagddX https://t.co/KyBc1zQttc
Comparing Psychedelic Trials to Antidepressants Skews Results
Does it stack the deck in favor of the standard antidepressants to pull data from placebo-controlled trials for one condition (the psychedelic) and compare it with single condition data for the standard antidepressants? Also...
Chinese Scientists Use E Coli to Fight Breast Tumours From Within in Mice Study
Chinese researchers at Shandong University have engineered the probiotic strain Escherichia coli Nissle 1917 to synthesize and deliver the anticancer drug Romidepsin directly within breast‑tumor tissue in mice. The bacteria colonized the tumors, releasing the drug locally and achieving tumor‑inhibiting...
New FDA Draft Bayesian Guidance Discussed with Dr. Gibson
Thrilled to be interviewed by imminent cardiologist and clinical trialist Mike Gibson yesterday about the new draft Bayesian guidance at FDA: https://t.co/I3uNISITKQ #Statistics #bayes #pharma #clinicaltrials #rct @CMichaelGibson
Europe's Biotech Funding Gap Spurs Urgent Investment Push
Europe's Biotech Investing Crisis: @OtelloVC Otello Stampacchia of Omega Funds discusses the drive to increase EU biotech investment on The Long Run. Sponsored by @AlphaSenseInc & Dash Bio https://t.co/ORIeN8IDEq
How TrumpRx Drug Prices Compare With Those in Other Countries
President Trump’s TrumpRx website claims to offer the world’s lowest prescription‑drug prices, but a New York Times analysis finds the opposite. The site lists only a few dozen negotiated U.S. prices, omitting widely used drugs such as statins and many high‑cost cancer...
Key Strategies for Building Biotech: Science, Tech, Finance
It was a fun privilege to share a few thoughts on building biotechs with Nature Biotechnology for their 30th Anniversary issue - covering a broad range of topics including translating science into medicines, technology cycles, and financing biotech companies... https://t.co/LbnWMSJI9f
Excalipoint Secures $69M for Next‑Gen T‑Cell Engagers
Chinese biotech startup Excalipoint banks $69M for next-gen T cell engagers https://t.co/809RqRWJNL by @gwendolynawu #biotech #startups
Middle East War Forces Pharma Reroute Critical Medicines to Safeguard Australian Supply
On March 17‑18, 2026, major pharmaceutical companies, represented by Medicines Australia, began diverting critical medicines from the Strait of Hormuz sea lane to air freight and alternative routes to ensure delivery to Australia. CEOs Liz de Somer and Leyla Hannbeck...
TerraPower Isotopes Launches cGMP Plant to Boost Actinium‑225 Supply Twenty‑Fold
TerraPower Isotopes announced on March 18, 2026 that it will build a new cGMP‑compliant manufacturing facility designed to raise actinium‑225 output by twenty times. The plant, slated for construction later this year, aims to address the growing demand for the...
EU Strikes Compromise on Pharma Reform: New Launch Rules and Antibiotic Voucher
On 6 March 2024, EU ambassadors in the Committee of Permanent Representatives (COREPER) reached a political compromise on a continent‑wide pharma reform. The deal tightens launch obligations for centrally authorised medicines, restricts the transferable exclusivity voucher, and creates a new antibiotic...
FDA Clears AstraZeneca’s All‑Oral Calquence‑Venetoclax Regimen for First‑Line CLL and SLL
On March 18, 2024 the U.S. Food and Drug Administration approved AstraZeneca’s all‑oral, fixed‑duration combination of Calquence (acalabrutinib) and venetoclax for first‑line treatment of adult chronic lymphocytic leukaemia (CLL) and small lymphocytic lymphoma (SLL). The decision follows the Phase III Amplify...
Prodrug-Tethered Lipid Nanoparticles for Synergistic Messenger RNA Cancer Immunotherapy
Researchers have engineered lipid nanoparticles tethered with a prodrug that releases an IDO‑1 inhibitor while simultaneously delivering mRNA encoding interleukin‑12. The dual‑action formulation, administered intratumorally, generated robust CD8⁺ T‑cell responses, mitigated T‑cell exhaustion, and produced complete tumor regressions in several...
Largest Review Finds No Evidence Cannabis Relieves Anxiety, Depression, PTSD
No evidence that medicinal cannabis effectively treats anxiety, depression, or PTSD - according to the largest review of cannabinoids ever conducted.
FTC Monitoring How Drug Companies React to Patent Cliff, Official Says
The U.S. Federal Trade Commission announced it is closely monitoring how pharmaceutical companies respond to the upcoming patent cliff affecting numerous blockbuster drugs. FTC antitrust director Dan Guarnera said the agency’s “laser focus” on healthcare will target any anticompetitive conduct...