Associate degree: PTC’s hints of phase II HD efficacy studied
PTC Therapeutics Inc. CEO Matthew Klein said the firm “achieved all we set out to [achieve] in phase II” with PTC-518 in Huntington’s disease (HD), but it wasn’t enough to excite Wall Street, as some questioned whether the level of associations between trial findings and efficacy would appease the U.S. FDA. Shares (NASDAQ:PTCT) ended May 5 at $40.65, down $9.30, or 18.6%. Warren, N.J.-based PTC rolled out the most recent data from the Pivot-HD effort in stage 2 and stage 3 HD with PTC-518, an oral, centrally as well as peripherally distributed huntingtin (HTT) pre-mRNA splicing modifier.

Associate degree: PTC’s hints of phase II HD efficacy studied
PTC Therapeutics Inc. CEO Matthew Klein said the firm “achieved all we set out to [achieve] in phase II” with PTC-518 in Huntington’s disease (HD), but it wasn’t enough to excite Wall Street, as some questioned whether the level of associations between trial findings and efficacy would appease the U.S. FDA. Shares (NASDAQ:PTCT) ended May 5 at $40.65, down $9.30, or 18.6%. Warren, N.J.-based PTC rolled out the most recent data from the Pivot-HD effort in stage 2 and stage 3 HD with PTC-518, an oral, centrally as well as peripherally distributed huntingtin (HTT) pre-mRNA splicing modifier.
ICYMI: Week in review, April 28-May 2, 2025
A quick look back at top stories.
Biggest gainers and losers for April 28-May 2, 2025
The top 10 biopharma stock gainers and losers for the week.
Today's news in brief
BioWorld briefs for May 5, 2025.
Annual US 301 trade report has louder bark
Echoing President Donald Trump’s justification for reciprocal tariffs, the U.S. Trade Representative’s annual Special 301 Report is a bit more aggressive in tone this year as it calls out trading partners that don't adequately enforce intellectual property rights or that otherwise discriminate against products from foreign companies.
Australia follows Canadian lead; Labor win a sharp rebuke to Trump
Australian biopharma stakeholders welcomed the reelection of Prime Minister Anthony Albanese, who promised to expand Medicare and to invest more in Australian research.
Oral GLP-1 Wegovy chases approval in obesity
The U.S. FDA has accepted the NDA for an oral formulation of Novo Nordisk A/S’s Wegovy (semaglutide) in treating obesity. If approved, the daily pill will be the first oral GLP-1 for treating chronic weight management. So far, however, it has been injectables leading the way to approval.
Immutep’s efti shows strong survival data in head and neck cancer
Immutep Ltd. announced strong median overall survival of 17.6 months in cohort B of the TACTI-003 (Keynote-PNC-34) phase IIb trial that evaluated eftilagimod alfa in combination with Merck & Co.’s Keytruda (pembrolizumab) as first-line therapy in recurrent/metastatic head and neck squamous cell carcinoma in patients with PD-L1 expression below 1.

BioWorld Insider Podcast

Robert Williamson, CEO of Triumvira Immunologics, and Hernan Bazan, CEO of South Rampart Pharma, discuss bracing for tariffs, NIH grant cuts and gifting China a biopharma leadership position.

Listen now

Analysis and data insight

Three Korea biotechs prep for May IPOs
Financings
Among three Korean biotech companies slated to sell shares on the Korea Exchange this month, Immuneoncia Therapeutics Inc. is the latest to price a ₩33.9 billion (US$24 million) Kosdaq IPO for May 19. Regenerative cell therapy maker Organoid Sciences Ltd. and antibody-drug conjugate specialist...
Biopharma funding drops 71% from 2024, but in line with prior years
Financings
Total biopharma financings for the first four months of 2025 reached $15.48 billion, a 71% decline from $53.49 billion in 2024. Biopharma companies raised $2.4 billion in April 2025, reflecting a 37% drop from March's $3.82 billion.
Navigating the Trump tariffs: Biopharma execs weigh in
Europe
First quarter 2025 results presentations were the first chance analysts have had to quiz pharma companies in public about their exposure to the threat of U.S. import tariffs and what action they are taking to mitigate the risks. BioWorld reporters have listened in to the analyst meetings and...
Filling the NIH funding gap to protect biopharma research
U.S.
The Trump administration’s broad slash to university research budgets raises pertinent questions over impacts to the biopharma ecosystem, specifically how a changed trajectory of early research programs will affect tomorrow’s treatments and cures. Who is going to bridge research from idea to...

Financings

‘Everything is possible’: Pharmas navigate US market uncertainties
Europe
“We are prepared for every scenario, even though we don’t know what some of those are.” That sums up the message from executives of Astrazeneca plc, GSK plc and Sanofi SA, when quizzed during presentations of their first-quarter 2025 results this week, about the fallout if pharmaceuticals they...

Science

AACR 2025: Induced proximity strategy, beyond degraders
Conferences
Targeted protein degradation has yet to notch its first approval. But with more than two dozen agents now in clinical trials, the strategy’s ultimate clinical validation appears to be a matter of time.

AACR 2025: Nouscom reports early but promising response in Lynch syndrome
Conferences
Off-the-shelf cancer vaccine specialist Nouscom AG announced positive final results for its lead product, Nous-209, showing there was a “highly potent” and durable immune response in carriers of Lynch syndrome, a common hereditary condition that can increase the lifetime risk of cancer by as much...
Vaccine Integrity Project launched to tackle ‘unfortunate reality’
Regulatory
Nervousness about the Trump administration’s attitude to vaccines has spurred the formation of the Vaccine Integrity Project, which has the aim of safeguarding the use of vaccines and ensuring vaccine policy “remains grounded in the best available science,” and is “free from external influence.”

Newco news

Brink’s recombinase enzymes offer new gene editing approach
Financings
Newco Brink Therapeutics SAS is poised to work on the next chapter in genome editing after raising €3.5 million (US$4 million) in seed funding to discover and develop programmable recombinase enzymes.
Aiming for autoimmunity, Merida Bio launches with $121M series A
Financings
When an autoimmune disease disrupted the life of someone close to scientist Dario Gutierrez, an idea emerged for a new company called Merida Biosciences focused on removing misdirected antibodies and their negative effects using Fc biotherapeutics.
Antag aiming to fill therapeutic gaps in obesity space
Clinical
The latest obesity contender, Antag Therapeutics A/S, has dosed the first participants in a phase I trial of a glucose-dependent insulinotrophic polypeptide (GIP) agonist it said will address key gaps in the current treatment options, not least the gastrointestinal side effects seen with GLP-1...
Hillstar launches with $67M series A to target TRBV9+ T cells
Financings
Hillstar Bio came out of stealth mode, announcing a $67 million series A financing round with investors including Droia Ventures, Frazier Life Sciences, Novo Holdings A/S and Lifearc Ventures.
Augustine Therapeutics closes $85M series A for HDAC6 inhibitors
Science
Eight months after announcing the $18.5 million first tranche of its series A, Augustine Therapeutics has closed the oversubscribed round at $85 million and is now ready to begin clinical development of its novel histone deacetylase-6 (HDAC6) inhibitors.

U.S.

AUA hoorays in bladder cancer for J&J, Urogen, more
Clinical
Clinical results offered at the recent meeting of the American Urological Association in Las Vegas signal that better treatments may lie ahead for non-muscle invasive bladder cancer.

Europe

Argenx’s Vyvgart, Camurus’ Oczyesa among 16 recommended by CHMP
Regulatory
Argenx NV is now well on the way to establishing a Vyvgart (efgartigimod alfa) franchise in severe autoimmune diseases, after getting the nod from the EMA in the treatment of progressive or relapsing chronic inflammatory demyelinating polyneuropathy.

Roche follows other pharmas, committing $50B in US investment
Financings
Roche AG has become the latest pharmaceutical company to respond to the Trump administration’s threat to impose tariffs, saying it will invest $50 billion in drug and diagnostics manufacturing in the U.S. over the next five years. That figure matches a similar commitment by its Basel,...
Cyprumed, Merck ink $493M peptide pill licensing deal
Deals and M&A
Oral peptide delivery specialist Cyprumed GmbH is about to find out if the high bioavailability of its tablet formulations seen in animal models will translate across to humans, after signing a $493 million license and option agreement with Merck & Co. Inc.

Clinical

AACR 2025: D3 Bio, Immvira present solid tumor, brain cancer data
Conferences
Shanghai-based D3 Bio (Wuxi) Co. Ltd. showed positive results for its lead candidate, next-generation KRAS G12C inhibitor, D3S-001, also known as elisrasib, in patients with KRAS G12C mutation cancers, including patients previously treated with...
AACR 2025: Revolution has early stage KRAS success in NSCLC
Conferences
Early phase I data for Revolution Medicines Inc.’s RAS(ON) G12D-selective inhibitor zoldonrasib encouraged both the company and the analysts. Initial study data produced a 61% objective response rate in 11 solid tumor patients receiving the...
Akeso hot with FDA cancer win, nods in China, new data
Regulatory
The intriguing VEGFxPD-1 bispecific antibody space gained oomph with phase III results disclosed April 23 by Akeso Pharmaceuticals Inc. related to Harmoni-6, testing ivonescimab in combination with platinum-based chemotherapy compared with...
‘Complement’-ary approaches? FcRn holds ground in myasthenia gravis
Analysis and data insight
Watchers of the percolating myasthenia gravis space are waiting eagerly for data from Dianthus Therapeutics Inc.’s phase II Magic study testing DNTH-103, an active C1s inhibitor, compared to placebo in patients with anti-AChR-positive generalized...
Telix’s brain cancer radiotherapy shows phase II survival benefit
Cancer
Telix Pharmaceuticals Ltd.’s glioblastoma theranostic, TLX-101, is showing promising overall survival in a phase II trial in recurrent high-grade glioma. The targeted radiation therapy, combined with external beam radiation therapy, demonstrated a...
Post-Pfizer dropout, Lilly Achieves GLP-1 win in phase III
Diabetes
Days after Pfizer Inc. pulled the plug on its oral GLP-1 candidate danuglipron, Eli Lilly and Co. aired positive top-line data from the phase III trial called Achieve-1 testing orforglipron vs. placebo in adults with type 2 diabetes and inadequate...

podcast microphone, sound waves on purple background Breakthrough medicines, billion-dollar deals, spectacular clinical successes and crushing failures all play a part in biopharma’s dynamic story. Developers make scientific advancements with the potential to change everything, only to face regulatory conundrums and ever-fluctuating markets. BioWorld tracks key events in the fast-moving sector every business day. Now, the BioWorld Insider podcast lets you hear directly from the movers and shakers whose collective work is changing how we all live. Join us each week for a new conversation.

Recent episodes:

View all

BioWorld

The news source of record covering the development of innovative human therapies for 25+ years

BioWorld MedTech

Actionable and timely intelligence on advances in medical devices and technologies for 20+ years

BioWorld Asia

A weekly monitor of biopharmaceutical news from the industry’s fastest-growing region

BioWorld Science

Essential discovery and preclinical research news to support crucial drug R&D decisions at the earliest stages

BioWorld Premium

Enjoy extended coverage for the most complete market view with BioWorld, BioWorld MedTech, and BioWorld Asia in a single, easy to access subscription

Today's news in brief

BioWorld

BioWorld MedTech

BioWorld Asia

BioWorld Science

BioWorld Premium