2025, Disease and Licensing - Drug Discovery Digest

Collaboration aims to reduce animal use in drug safety testing

Broad Institute

JULY 29, 2025

Collaboration aims to reduce animal use in drug safety testing By Leah Eisenstadt July 29, 2025 Breadcrumb Home Collaboration aims to reduce animal use in drug safety testing OASIS consortium is evaluating omics tools in cells, including Cell Painting and transcriptomics, to predict liver toxicity of potential new medicines and agrochemicals.

Animal Testing

Animal Testing Drugs Science Pharmaceuticals

Arrowhead’s Subsidiary Visirna Sells China Rights for Plozasiran to Sanofi

The Pharma Data

AUGUST 1, 2025

“Greater China has long been seen as a critical growth market in biopharma, and when we founded Visirna in 2022, we recognized that our RNAi platform had the potential to make a meaningful impact on cardiometabolic disease there,” said Christopher Anzalone, Ph.D., The application was formally accepted in January 2025.

Licensing

Licensing Licensing Therapies RNA

From siloed data to breakthroughs: multimodal AI in drug discovery

Drug Target Review

JUNE 11, 2025

NGS has revolutionised genomic analysis, enabling the identification of disease-related genetic variants. This ability to correlate seemingly disparate data is crucial for tackling complex targets and discovering new treatments for diseases considered difficult to treat with conventional approaches.

Drugs

Drugs Clinical Trials Drug Development Pharmaceuticals

Protein degraders: chasing undruggable targets

BioPharma Drive: Drug Pricing

JULY 8, 2025

Published July 8, 2025 Gwendolyn Wu Senior reporter post share post print email license Proteasomes are cellular machines for breaking down proteins. Love Employee via Getty Images For decades now, scientists have been experimenting with a new way to target and destroy proteins linked to disease.

Targeted Protein Degradation

Targeted Protein Degradation Small Molecule Disease Therapies

Fitusiran

New Drug Approvals

APRIL 11, 2025

2] Fitusiran was approved for medical use in the United States in March 2025. [2] 1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] 26 March 2025.

Clinical Trials

Clinical Trials FDA FDA Approval Trials

Otsuka’s Sibeprenlimab Shows Significant Proteinuria Reduction in Phase 3 IgAN Trial

The Pharma Data

JUNE 6, 2025

Proteinuria reduction is a well-established surrogate marker for the progression of kidney disease, and this level of decline is considered both statistically significant and clinically meaningful in slowing disease progression toward end-stage kidney disease (ESKD). in the group treated with sibeprenlimab relative to placebo.

Trials

Trials Treatment Therapies FDA

2025 Merkin Prize in Biomedical Technology awarded to pioneers of CAR T-cell therapy

Broad Institute

MAY 21, 2025

The $400,000 prize, to be shared among the four winners, recognizes their role in creating and advancing a technology that has reshaped how physicians treat leukemia, lymphoma, and multiple myeloma, and is now showing promise in treating autoimmune and infectious diseases. Their work puts us on the doorstep of finding cures to help millions.

Therapies

Therapies FDA Approval Engineering Immune Response

Intellia Reports Positive 3-Year Phase 1 Data for Lonvo-z in Hereditary Angioedema

The Pharma Data

JUNE 15, 2025

The data, which encompass up to 3 years of follow-up after a single infusion of the treatment, were shared in an oral presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025, held from June 13–16 in Glasgow, United Kingdom. President and Chief Executive Officer of Intellia Therapeutics.

Treatment

Treatment Therapies Licensing Licensing

Tofersen

New Drug Approvals

MAY 17, 2025

Retrieved 17 April 2025. ^ “Qalsody- tofersen injection” DailyMed. “FDA grants accelerated approval for Biogen ALS drug that treats rare form of the disease” CNBC. “FDA advisors vote against effectiveness of Biogen’s ALS drug for rare and aggressive form of the disease” CNBC.

FDA Approval

FDA Approval FDA Treatment Clinical Trials

Taletrectinib

New Drug Approvals

JUNE 29, 2025

Taletrectinib CAS 1505514-27-1 as salt: 1505515-69-4 , Taletrectinib adipate FDA 6/11/2025, Ibtrozi, To treat locally advanced or metastatic ROS1-positive non-small cell lung cancer AB-106, DS-6051a 405.5 1] Taletrectinib was approved for medical use in the United States in June 2025. [3] April 2025. Retrieved 14 June 2025.

International

International FDA Clinical Trials Treatment

Inavolisib

New Drug Approvals

APRIL 25, 2025

Retrieved 17 April 2025. January 2025. Archived from the original on 21 January 2025. Retrieved 21 January 2025. 3] Structure, reactivity, and synthesis Inavolisib is a synthetic, organic, small compound (the full structure can be seen here ). [8] 3] Names Inavolisib is the international nonproprietary name. [22]

FDA

FDA FDA Approval Therapies Trials

Jazz Pharma Gets EU Nod for Ziihera in Advanced HER2+ Biliary Cancer

The Pharma Data

JULY 2, 2025

“Ziihera offers a targeted monotherapy with a favorable safety profile and compelling efficacy, representing a crucial advance in the fight against this challenging disease.” May 2025 : China’s National Medical Products Administration (NMPA) issued conditional approval for the same indication. formerly BeiGene, Ltd.)

Therapies

Therapies Trials Medical Schools Pharmaceuticals

Ropotrectinib

New Drug Approvals

AUGUST 7, 2025

5] Repotrectinib was approved for medical use in the United States in November 2023, [5] [6] and in the European Union in January 2025. [3] Retrieved 23 May 2025. 14 January 2025. Retrieved 16 January 2025. 2] [5] It is taken by mouth. [2] g, 0.000611 mol, 1.0 in DMF (4.0 mL, 0.00488 mol, 8.0 in DCM (1.8 3 November 2006.

FDA

FDA FDA Approval Production Treatment

FDA clears Nuvation lung cancer drug, setting up battle with Bristol Myers and Roche

BioPharma Drive: Drug Pricing

JUNE 11, 2025

Published June 11, 2025 Ben Fidler Senior Editor post share post print email license The FDA on June 11, 2025 approved Nuvation's Ibtrozi for ROS1-positive non-small cell lung cancer.

FDA

FDA Drugs Therapies Science

Vor, with new CEO, changes course to target autoimmune disease

BioPharma Drive: Drug Pricing

JUNE 26, 2025

Published June 26, 2025 Gwendolyn Wu Senior reporter post share post print email license B cells, like those pictured above, malfunction in autoimmune disease. Vor Biopharma licensed a drug in June 2025 that targets proteins essential to B cell survival. and European drugmakers. His research, writing offer clues.

Disease

Disease Licensing Licensing Therapies

Targeting HER3 – a little wave of drug development that’s about to get a lot bigger

SugarCone Biotech

JULY 6, 2025

A post-ASCO update on tumor targets DLL3, B7H3 and HER3 July 2025 Paul D Rennert, SugarCone Biotech LLC Introduction: DLL3, B7H3 and HER3 are compelling tumor antigens to target with cancer therapeutics. However, HER3-DXd missed achieving a statistically significant improvement in overall survival, leading to the withdrawal of its U.S.

Drug Development

Drug Development Drugs Clinical Trials Therapies

FDA Grants Priority Review for WINREVAIR Label Update Based on ZENITH Trial

The Pharma Data

JULY 2, 2025

Food and Drug Administration (FDA) has accepted for review a new supplemental Biologics License Application (sBLA) and granted it priority review status, seeking to update the U.S. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of October 25, 2025. If approved, the updated U.S. Source link

Trials

Trials FDA Licensing Licensing

Leniolisib

New Drug Approvals

MAY 15, 2025

2017, 8, 9, 975980 [link] The predominant expression of phosphoinositide 3-kinase (PI3K) in leukocytes and its critical role in B and T cell functions led to the hypothesis that selective inhibitors of this isoform would have potential as therapeutics for the treatment of allergic and inflammatory disease. 16 April 2025.

FDA Approval

FDA Approval FDA Treatment International

Crinecerfont

New Drug Approvals

APRIL 18, 2025

4] A medication used to reduce the amount of steroid replacement required in patients with a genetic disease that causes, amongst other symptoms, a steroid deficiency. Retrieved 25 January 2025. January 2025. Archived from the original on 21 January 2025. . : 321839-75-2 Molecular Weight 519.50 1] It is taken by mouth. [1]

FDA

FDA FDA Approval Drugs Trials

Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks

BioPharma Drive: Drug Pricing

JULY 16, 2025

Published July 16, 2025 • Updated 2 hours ago Ben Fidler Senior Editor post share post print email license Sarepta Therapeutics announced a workforce reduction on July 16, 2025. Some Wall Street analysts predicted sales might top $2 billion in 2025. Shares in the company rose in after-hours trading on the news.

Therapies

Therapies FDA Clinical Trials Licensing

Moderna COVID vaccine gets full approval for children

BioPharma Drive: Drug Pricing

JULY 10, 2025

Published July 10, 2025 Delilah Alvarado Staff Reporter post share post print email license The Food and Drug Administration on July 10, 2025, issued Moderna a full approval in children for its COVID-19 vaccine Spikevax. Kennedy Jr., who has pushed for policy changes around mRNA vaccines in particular. Kennedy Jr. Kennedy Jr.

Vaccine

Vaccine FDA Therapies Licensing

Panel urges FDA to remove warnings on hormonal menopause therapy

BioPharma Drive: Drug Pricing

JULY 18, 2025

Published July 18, 2025 Delilah Alvarado Staff Reporter post share post print email license Food and Drug Administration Commissioner Martin Makary speaks on stage during The Semafor 2025 World Economy Summit on April 24, 2025 in Washington, DC. The panelists were generally dismissive of the WHI study and its findings.

Therapies

Therapies FDA Clinical Trials Licensing

Soleno sales of new Prader-Willi drug rise faster than expected

BioPharma Drive: Drug Pricing

JULY 10, 2025

Published July 10, 2025 Ben Fidler Senior Editor post share post print email license Soleno Therapeutics on July 10, 2025 announced preliminary sales for its new Prader-Willi Syndrome drug Vykat. The disease, which affects an estimated 10,000 to 20,000 people in the U.S.,

Drugs

Drugs Licensing Licensing Therapies

FDA to ask Sarepta to stop shipping Duchenne gene therapy

BioPharma Drive: Drug Pricing

JULY 18, 2025

Published July 18, 2025 • Updated 3 hours ago Ned Pagliarulo Lead Editor post share post print email license The U.S. The FDA expanded Elevidys’ approval the next year, allowing a substantially larger group of patients to receive it, including those whose disease had eroded their ability to walk.

Therapies

Therapies FDA Clinical Trials Licensing

Lexeo to help launch spinout around RNA drugs for the heart

BioPharma Drive: Drug Pricing

JUNE 24, 2025

Published June 24, 2025 Gwendolyn Wu Senior reporter post share post print email license Lexeo Therapeutics and two life sciences investors are working together to launch a startup to develop cardiac RNA therapies. Earlier this year, it laid off 15% of its workforce.

RNA

RNA Drugs Therapies Licensing

Madrigal buys into GLP-1 from CSPC; Apellis wins expanded drug approval

BioPharma Drive: Drug Pricing

JULY 30, 2025

Published July 30, 2025 Ned Pagliarulo Lead Editor post share post print email license Daniel Tadevosyan via Getty Images Today, a brief rundown of news involving Madrigal Pharmaceuticals and Viridian Therapeutics, as well as updates from Apellis Pharmaceuticals, PTC Therapeutics and Arrowhead Pharmaceuticals that you may have missed.

Drugs

Drugs Clinical Trials FDA Therapies

Organon drug for endometriosis falls short in mid-stage study

BioPharma Drive: Drug Pricing

JULY 2, 2025

Published July 2, 2025 By Kristin Jensen post share post print email license Endometriosis involves the outgrowth of womb lining tissue in the pelvis or abdomen. Organon acquired the drug in its purchase of Forendo Pharma in 2021. Company Announcements View all | Post a press release Nobel Laureate Morten Meldal Joins Receptor.AI

Clinical Trials

Clinical Trials Drugs Therapies Licensing

Merck to buy Verona and its lung drug in $10B deal

BioPharma Drive: Drug Pricing

JULY 9, 2025

Published July 9, 2025 By Kristin Jensen post share post print email license Merck & Co. has agreed to pay $10 billion to buy London-based Verona Pharma, scooping up a potential multibillion-dollar new drug to treat chronic obstructive pulmonary disease in a deal announced Wednesday. Alamy Merck & Co.

Clinical Trials

Clinical Trials Drugs Therapies Licensing

Incyte replaces CEO Hoppenot with dealmaker Meury

BioPharma Drive: Drug Pricing

JUNE 26, 2025

Published June 26, 2025 Ned Pagliarulo Lead Editor post share post print email license An Incyte researcher works in a laboratory. On June 26, 2025, the company announced the replacement of longtime CEO Hervé Hoppenot with Bill Meury. Last year, Jakafi brought in nearly $2.8 billion in sales. Track funding rounds here.

Therapies

Therapies Licensing Licensing Vaccine

Biocryst sells Europe business; Peter Marks criticizes new FDA vaccine rules

BioPharma Drive: Drug Pricing

JUNE 27, 2025

Published June 27, 2025 By BioPharma Dive staff post share post print email license Daniel Tadevosyan via Getty Images Today, a brief rundown of news involving Biocryst and Peter Marks, as well as updates from UCB and Altimmune that you may have missed. By Delilah Alvarado • May 13, 2025 Keep up with the story.

Vaccine

Vaccine FDA Therapies Licensing

Genmab Unveils Executive Committee Restructuring

The Pharma Data

JULY 2, 2025

Genmab Announces Leadership Transition as Chief Legal Officer Birgitte Stephensen Retires, Greg Mueller Appointed Successor Genmab A/S (Nasdaq: GMAB), a leading international biotechnology company focused on antibody-based therapeutics for cancer and other serious diseases, today announced a major leadership transition within its executive team.

Compliance

Compliance Pharmaceutical Companies Government International

Regulatory uncertainty and access challenges changing go-to-market models, DTC, and DTP

BioPharma Drive: Drug Pricing

AUGUST 11, 2025

11, 2025 post share post print email shutterstock.com/ PeopleImages.com - Yuri A Sponsored content By GoodRx SPONSORED CONTENT BY While direct-to-consumer (DTC) advertising is effective at top-of-funnel awareness and driving action early in the patient journey, it cannot pull through conversions at the pharmacy. © 2025 TechTarget, Inc.

Marketing

Marketing Pharmacy Therapies Clinical Trials

Replimune drug rejected by FDA; Omega raises $647M biotech fund

BioPharma Drive: Drug Pricing

JULY 22, 2025

Published July 22, 2025 By BioPharma Dive staff post share post print email license Daniel Tadevosyan via Getty Images Today, a brief rundown of news involving Replimune Group and Johnson & Johnson, as well as updates from Omega Funds, iTeos Therapeutics and Roche that you may have missed. and European life sciences companies.

FDA

FDA Specialty Pharmacies Drugs Therapies

Sanofi to acquire vaccine biotech in billion-dollar deal

BioPharma Drive: Drug Pricing

JULY 22, 2025

Published July 22, 2025 • Updated 2 hours ago Delilah Alvarado Staff Reporter post share post print email license A sign bearing Sanofi's logo sits outside the company's U.S. An antibody drug, Beyfortus , that Sanofi developed with partner AstraZeneca has become a blockbuster product for preventing RSV disease in infants.

Vaccine

Vaccine Clinical Trials FDA Therapies

The rise of GLP-1 drugs: Transforming weight loss treatment

BioPharma Drive: Drug Pricing

JUNE 23, 2025

Expanding potential uses Emerging research suggests GLP-1 drugs might effectively treat chronic kidney disease, liver conditions, and certain cardiac diseases. By Jacob Bell • May 6, 2025 Keep up with the story. © 2025 TechTarget, Inc. or its subsidiaries. All rights reserved.

Treatment

Treatment Drugs Therapies FDA

Drug candidates with blockbuster potential for CNS diseases

Drug Target Review

SEPTEMBER 19, 2024

Based on Nobel Prize-winning research, IRLAB has grown rapidly to become recognised and respected as a world-leader in understanding the complex neuropharmacology of CNS disorders, especially Parkinson’s disease (PD). Top-line data from the Phase IIb study is expected at the beginning of 2025.

Disease

Disease Drugs Treatment Drug Development

Acoltremon

New Drug Approvals

JUNE 26, 2025

Acoltremon AR-15512 CAS 68489-09-8 WeightAverage: 289.419 Monoisotopic: 289.204179113 Chemical FormulaC 18 H 27 NO 2 FDA 2025, 5/28/2025, To treat the signs and symptoms of dry eye disease Tryptyr WS-12 WS 12 (1R,2S,5R)-N-(4-methoxyphenyl)-5-methyl-2-(propan-2-yl)cyclohexane-1-carboxamide Fema No. 29 May 2025.

FDA

FDA Clinical Trials Small Molecule FDA Approval

Sentiment & Themes Emerging From JPM 2024

LifeSciVC

JANUARY 16, 2024

recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Rise of the best-in-class biologics plays: Are you looking to in-license or invest in “de-risked” biology that is already in the clinic? Join the club. Additional trials (e.g.,

Small Molecule

Small Molecule Trials Therapies Disease

Levacetylleucine

New Drug Approvals

APRIL 30, 2025

1] [2] [3] Levacetylleucine is the second medication approved by the US Food and Drug Administration (FDA) for the treatment of Niemann-Pick disease type C. [2] 2] The US Food and Drug Administration (FDA) granted the application for levacetylleucine priority review , fast track , orphan drug , and rare pediatric disease designations. [2]

FDA

FDA FDA Approval Disease Trials

ELRIG Announces USA Forum on Neuroimmunology in Drug Discovery

Elrig

APRIL 29, 2025

Rahway NJ USA) research facility in the Longwood medical area of Boston, Massachusetts, on 24 June 2025. Understanding the role of neuroimmune interactions has become increasingly important in tackling complex neurological diseases. The event is held in partnership with Drug Discovery News. Matt received his B.Sc.

Drugs

Drugs Science Clinical Trials Laboratories

A new roadmap for cannabis and cannabis policy research

National Institute on Drug Abuse: Nora's Blog

JANUARY 3, 2025

A new roadmap for cannabis and cannabis policy research mfleming Fri, 01/03/2025 - 11:38 Nora's Blog January 14, 2025 Image Getty Images/ Fiordaliso The greatly increased availability of cannabis over the last two decades has outpaced our understanding of the public-health impacts of the drug.

Research

Research Production Licensing Licensing

AstraZeneca to Acquire Alexion, Accelerating the Company’s Strategic and Financial Development

The Pharma Data

DECEMBER 12, 2020

Dedicated rare disease unit to be headquartered in Boston. Double-digit revenue growth through 2025; acquisition strengthens AstraZeneca’s broad-based revenue and the company will further globalise Alexion’s portfolio . Geographical presence to be enhanced with broad coverage across primary, speciality and highly specialised care.

Regulations

Regulations International Disease Small Molecule

Ascendis Pharma A/S Provides Vision 3×3 Update at 39th Annual J.P. Morgan Healthcare Conference

The Pharma Data

JANUARY 10, 2021

In 2019, we introduced Vision 3×3, the company’s strategic roadmap through 2025, to build a leading biopharma company by achieving sustainable growth through multiple approaches. The company submitted its Biologic License Application to the FDA which has set a Prescription Drug User Fee Act (PDUFA) date for June 25, 2021.

Clinical Trials

Clinical Trials Clinical Development Small Molecule Trials

AbbVie Reports Full-Year and Fourth-Quarter 2020 Financial Results

The Pharma Data

FEBRUARY 4, 2021

AbbVie announced positive top-line results from the Phase 3 ADVANCE and MOTIVATE studies, which evaluated the efficacy and safety of Skyrizi (risankizumab) for induction therapy in adult patients with moderate to severe Crohn’s disease (CD). AbbVie and Frontier Medicines, Corp.,

Production

Production Clinical Trials International Small Molecule

Collaboration aims to reduce animal use in drug safety testing

Arrowhead’s Subsidiary Visirna Sells China Rights for Plozasiran to Sanofi

Trending Sources

From siloed data to breakthroughs: multimodal AI in drug discovery

Protein degraders: chasing undruggable targets

Fitusiran

Otsuka’s Sibeprenlimab Shows Significant Proteinuria Reduction in Phase 3 IgAN Trial

2025 Merkin Prize in Biomedical Technology awarded to pioneers of CAR T-cell therapy

Intellia Reports Positive 3-Year Phase 1 Data for Lonvo-z in Hereditary Angioedema

Tofersen

Taletrectinib

Inavolisib

Jazz Pharma Gets EU Nod for Ziihera in Advanced HER2+ Biliary Cancer

Ropotrectinib

FDA clears Nuvation lung cancer drug, setting up battle with Bristol Myers and Roche

Vor, with new CEO, changes course to target autoimmune disease

Targeting HER3 – a little wave of drug development that’s about to get a lot bigger

FDA Grants Priority Review for WINREVAIR Label Update Based on ZENITH Trial

Leniolisib

Crinecerfont

Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks

Moderna COVID vaccine gets full approval for children

Panel urges FDA to remove warnings on hormonal menopause therapy

Soleno sales of new Prader-Willi drug rise faster than expected

FDA to ask Sarepta to stop shipping Duchenne gene therapy

Lexeo to help launch spinout around RNA drugs for the heart

Madrigal buys into GLP-1 from CSPC; Apellis wins expanded drug approval

Organon drug for endometriosis falls short in mid-stage study

Merck to buy Verona and its lung drug in $10B deal

Incyte replaces CEO Hoppenot with dealmaker Meury

Biocryst sells Europe business; Peter Marks criticizes new FDA vaccine rules

Genmab Unveils Executive Committee Restructuring

Regulatory uncertainty and access challenges changing go-to-market models, DTC, and DTP

Replimune drug rejected by FDA; Omega raises $647M biotech fund

Sanofi to acquire vaccine biotech in billion-dollar deal

The rise of GLP-1 drugs: Transforming weight loss treatment

Drug candidates with blockbuster potential for CNS diseases

Acoltremon

Sentiment & Themes Emerging From JPM 2024

Levacetylleucine

ELRIG Announces USA Forum on Neuroimmunology in Drug Discovery

A new roadmap for cannabis and cannabis policy research

AstraZeneca to Acquire Alexion, Accelerating the Company’s Strategic and Financial Development

Ascendis Pharma A/S Provides Vision 3×3 Update at 39th Annual J.P. Morgan Healthcare Conference

AbbVie Reports Full-Year and Fourth-Quarter 2020 Financial Results

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