Bone Disease Readout Sinks Mereo, Ultragenyx Shares: Clinical Roundup
Mereo BioPharma and Ultragenyx reported disappointing readouts from their bone disease programs, triggering sharp declines in both stocks. Mereo’s trial failed to meet its primary endpoint, while Ultragenyx showed only modest efficacy signals. The market reaction erased roughly 15% of Mereo’s market value and about 12% of Ultragenyx’s. The setbacks raise questions about the companies’ near‑term growth trajectories in the rare‑bone‑disorder space.
Brinsupri Setback Slices Insmed Market Cap — Clinical Report
Insmed’s experimental therapy Brinsupri failed to meet its primary efficacy endpoint in a Phase 2 trial involving 150 patients with rare lung disease, triggering a sharp market reaction. The company announced the setback on Dec. 19, 2025, and its market...
A Non-Profit Brought an Abandoned Rare Disease Gene Therapy to Market. Can the Model Scale?
A non‑profit, Fondazione Telethon, partnered with a U.S. charity to bring a lentiviral stem‑cell gene therapy for Wiskott‑Aldrich syndrome to market. The FDA approved the product, Waskyra etuvetidigene autotemcel, marking the first time a non‑profit acted as the regulatory applicant. The therapy...
Raising the Sun: Japan Biotech Looks to Level Up
Japan’s biotech industry is entering a growth phase as the government unveils a ¥200 billion fund and regulatory sandbox to speed drug development. Venture capital activity surged 45% year‑over‑year, fueling a wave of startups focused on gene therapy and rare‑disease platforms....
Seeking ‘Continuous’ Run of Deals, BioMarin Adds Fabry, Pompe Drugs via $4.8B Amicus Takeout
BioMarin Pharmaceutical announced a $4.8 billion acquisition of Amicus Therapeutics, a move designed to fuel a continuous stream of strategic deals. The transaction will bring two marketed orphan drugs—one for Fabry disease and another for Pompe disease—into BioMarin’s portfolio, together projected...
Cai Succeeding Zhang as Head of CSPC
Cai has been appointed to replace Zhang as head of China Starch & Pharmaceutical Co. (CSPC), the country’s largest generic drug manufacturer. The transition was announced in December 2025 and is effective immediately. Cai brings a background in AI-driven drug...
Stelios Papadopoulos Brings the Long View on Biotech on The BioCentury Show
Stelios Papadopoulos, former Biogen chair and current Exelixis leader, warned that biotech faces heightened pricing pressure and rising competition from China despite a surge of over $3 billion in capital in a single day. He argued that the sector can no...
How Alphamab Is Differentiating in Crowded Cancer Targets
Alphamab is launching a next‑generation bispecific antibody‑drug conjugate (ADC) platform that simultaneously engages two tumor antigens while employing a lower drug‑to‑antibody ratio (DAR). The company argues that this design improves the therapeutic index by delivering potent payloads more selectively and...
Third Rock Backs Steve Paul’s Latest Schizophrenia Spinout in $165M Round: Venture Report
Third Rock Ventures led a $165 million financing round for Steve Paul’s newest schizophrenia‑focused biotech, marking a significant venture capital commitment to neuropsychiatric innovation. Paul, a serial biotech founder, will use the capital to move novel mechanisms from discovery into early...
Early Signals Stack Up: Two Small Molecules Activate GCase in Parkinson’s
Gain Therapeutics reported Phase Ib data showing that its small‑molecule program reduces glucosylceramide substrate in the central nervous system, confirming target engagement of glucocerebrosidase (GCase). In parallel, Vanqua Bio presented early evidence that its distinct compound activates peripheral GCase while...
New Role for Tam Man Hong at Xtalpi
Xtalpi announced that veteran biotech executive Tam Man Hong will assume the role of President, Business Development, effective immediately. The appointment comes as the company finalizes a $150 million Series B financing round to expand its AI‑driven protein therapeutics platform. Hong, who...
Science Spotlight: Dialing in Pathway Balance in Friedreich Ataxia
Two independent research groups have pinpointed reduced expression of the mitochondrial protein FDX2 as a novel therapeutic axis for Friedreich ataxia, publishing their findings in Nature. The studies argue that frataxin (FXN) and FDX2 function as a stoichiometric pair, and...
Addition Therapeutics: One Genomic Safe Site, Many DNA Insertions
Addition Therapeutics, a newly launched biotech, is deploying engineered retrotransposons to insert therapeutic payloads into a single ribosomal DNA (rDNA) locus, a recognized genomic safe harbor. By concentrating multiple genes at this highly transcribed, repetitive site, the platform promises stable...
RTW Leans Into NewCo Model with GenSci Deal for Ocular Autoimmune Therapy: Deals Report
RTW Therapeutics announced a strategic partnership with GenSci to form a new joint‑venture focused on developing an ocular autoimmune therapy. The collaboration adopts a NewCo model, with both parties contributing capital and expertise to accelerate drug discovery for non‑infectious uveitis....
The Evolving BTK Story in Multiple Sclerosis: Clinical Report
Sanofi’s Bruton’s tyrosine kinase (BTK) inhibitor failed to meet its primary endpoint in a pivotal trial for primary progressive multiple sclerosis, while the company awaits an FDA decision on a secondary progressive indication. Roche, meanwhile, reported encouraging efficacy and safety...
$12B-Plus in New Life Sciences Funds This Year
Venture capital activity in life sciences has surged in 2025, with at least 27 firms announcing new funds. Collectively, these vehicles have attracted more than $12 billion in capital, marking a record inflow for the sector. Notably, four of the funds...
Elkedonia: Antidepressants that Act Downstream of Neurotransmitters
Elkedonia, a seed‑funded French biotech, is developing a novel class of antidepressants that target the transcription factor ELK1 to reboot neuroplasticity downstream of classic neurotransmitter pathways. The company announced a €5 million seed round led by European venture partners and presented...
Obesity Data, Kymera and FDA Sentiment Survey Results — a BioCentury Podcast
Obesity therapeutics dominated the latest BioCentury podcast, with Eli Lilly unveiling new top‑line data for its triple‑agonist retatrutide, positioning it as a best‑in‑class weight‑loss candidate. Structure Therapeutics reported strong Phase IIb results for its oral GLP‑1, reigniting interest in non‑injectable options, while...
Pursuing a Rare Autoimmune Disease Accelerated Kyverna’s Path to BLA
Kyverna Therapeutics announced that its lead autoimmune CAR‑T, mivocabtagene autoleucel (miv‑cel), achieved both primary and secondary endpoints in a registrational Phase II trial for stiff person syndrome. The data positions the therapy as the first autoimmune CAR‑T to pursue a Biologics...
MHRA Deepens Global Strategy with Singapore Regulatory Pathway
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has launched a regulatory innovation corridor with Singapore’s Health Sciences Authority, offering companies coordinated scientific advice from both regulators. Flagship Pioneering is the first industry partner, giving its biotech portfolio a...
The Busiest Biotech Seed and Series A Investors of 2025
In 2025 more than a dozen venture capital firms have each disclosed at least five seed or Series A biotech investments, making them the busiest early‑stage investors of the year. BioCentury’s data shows that capital is flowing into a wide range...
CEO Moves at Prolynx, Centessa, Kyowa Kirin, Enliven, Expression
BioCentury reports a wave of CEO appointments across five life‑science firms. Prolynx named a former biotech COO to steer its next growth phase, while Centessa tapped a veteran pharma executive to accelerate its pipeline expansion. Kyowa Kirin promoted its internal R&D...
Makary’s Embrace of Ideology, Conspiracies Puts FDA at Risk: A Perspective
FDA Commissioner Marty Makary is openly endorsing debunked conspiracy theories while linking drug approvals to political pricing deals and onshoring incentives. His recent podcast remarks claimed Lyme disease and AIDS originated from government labs, blurring the line between science and...
EMA’s CHMP Backs New Medicines From Hansoh, Cytokinetics
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval for five new drugs and vaccines in its December meeting. Two products originate from Chinese biotech Hansoh, while three are from U.S. firm Cytokinetics. The...
BeOne Strengthens Its BTK Position at ASH
BeOne Medicines presented six‑year follow‑up data for its BTK inhibitor Brukinsa (zanubrutinib) at the ASH meeting, showing roughly 75% of treatment‑naïve chronic lymphocytic leukemia (CLL) patients remain progression‑free. The company also shared early results from a novel BTK degrader that...
Lilly’s Triple Agonist Contender for Best-in-Class Weight Loss
Eli Lilly announced Phase III TRIUMPH-4 data for its triple‑agonist retatrutide, showing a 26.6% placebo‑adjusted weight loss after 68 weeks at the 12 mg weekly dose. The study enrolled 445 obese adults with knee osteoarthritis, and the highest dose translated to...
BioCentury’s FDA Survey Reflects an Industry in Distress
BioCentury’s 2025 FDA survey paints a picture of deep distress across biopharma, flagging “inconsistency” as the sector’s most pressing worry. Respondents praised FDA staff effort but argued senior leadership decisions appear driven by political agendas rather than science. The perceived...
Front Row with BioCentury: Inside the Turbulent Ride of Cell and Gene Therapy
Cell and gene therapy continues to attract attention despite a recent wave of setbacks that have slowed commercial momentum. Manufacturing complexities, heightened regulatory scrutiny, and pricing debates are creating a turbulent environment for developers. Nevertheless, breakthrough scientific data and expanding...
Science Spotlight: Chinese Researchers Advance PROTAC Design
Chinese scientists have unveiled a next‑generation PROTAC scaffold that delivers oral bioavailability and heightened target selectivity. The platform successfully degrades previously undruggable proteins, including KRAS(G12C), and demonstrates tumor‑growth inhibition in mouse models. By integrating AI‑guided linker optimization, the researchers mitigated...