News

- Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR. Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A and to demonstrate benefit on cardiovascular outcomes

- Attruby demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date:

- In as few as 3 months, the time to first event (all-cause mortality (ACM) or cardiovascular-related hospitalizations (CVH)) durably separated relative to placebo
- A 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30
- A 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30

- With this approval, BridgeBio will receive a $500 million payment under our royalty funding agreement

 

Palo Alto, CA -- November 22, 2024 -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio or the Company), a new type of biopharmaceutical company focused on genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) approved Attruby™ (acoramidis), an orally-administered near-complete (≥90%) stabilizer of Transthyretin (TTR) for the treatment of adults with ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalization. The FDA approval is based on positive results seen in the ATTRibute-CM Phase 3 study, where Attruby significantly reduced death and cardiovascular-related hospitalization, and improved quality of life.

“We are excited to be part of the celebration for the FDA approval of Attruby. The need for more treatment options for patients living with ATTR-CM is crucial to achieving the goal of better outcomes and improved quality of life. Access to this new therapy means more hope and more opportunity to improve the lives of patients with amyloidosis,” said Muriel Finkel, President of Amyloidosis Support Groups, a non-profit organization dedicated to the support of amyloidosis patients and caregivers.

Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR. Attruby was designed to mimic a naturally occurring "rescue mutation” of the TTR gene (T119M) that targets the root cause of ATTR-CM, destabilization of the native TTR tetramer. Through near-complete TTR stabilization, Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A and to demonstrate benefit on cardiovascular outcomes.

The ATTRibute-CM Phase 3 study enrolled 632 participants with symptomatic ATTR-CM, associated with either wild-type or variant TTR. Participants were randomized 2:1 to receive Attruby or placebo for 30 months. As published in The New England Journal of Medicine, the trial successfully met its primary endpoint of a 4-component composite endpoint of ACM, CVH, N-terminal prohormone of brain natriuretic peptide (NT-proBNP), and 6-minute walk distance with a Win Ratio of 1.8 (p<0.0001). Attruby demonstrated a statistically significant treatment effect at 30 months on the Kansas City Cardiomyopathy Questionnaire and 6-minute walk test. Additionally, the increase in NT-proBNP on treatment was about half that of placebo.

• First and only therapy FDA approved in both R/R acute myeloid leukemia (AML) with an NPM1 mutation and R/R acute leukemia with a KMT2A translocation –
• Second approved indication for Revuforj in less than one year further solidifies Syndax’s leadership in menin inhibition –
• Included in NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for R/R NPM1 mutated AML –
• Syndax to host conference call today at 2:30 p.m. ET

New York, NY -- October 24, 2025 -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today announced that the U.S. Food and Drug Administration (FDA) has approved Revuforj® (revumenib) for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options. Revuforj previously received FDA approval in 2024 for the treatment of R/R acute leukemia with a KMT2A translocation in adult and pediatric patients one year and older. Revuforj is the first and only FDA-approved therapy for both R/R AML with an NPM1 mutation and R/R acute leukemia with a KMT2A translocation.

Rahway, NJ -- October 07, 2025 -- Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced the completion of the Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma”) acquisition. Verona Pharma is now a wholly-owned subsidiary of Merck and the American Depositary Shares (ADS) of Verona Pharma will no longer be listed or traded on the Nasdaq Global Market.

• Novartis receives an exclusive license to an undisclosed discovery target
• Novartis also receives options to license two programs from Monte Rosa’s growing preclinical immunology portfolio
• Monte Rosa to receive an upfront payment of $120 million, plus option maintenance payments, and is eligible for option exercise payments and development, regulatory, and sales milestones, as well as tiered royalties on global net sales
• Extended cash runway enables Monte Rosa to accelerate preclinical and clinical-stage immunology & inflammation (I&I) pipeline

Boston, MA -- September 15, 2025 -- Monte Rosa Therapeutics, Inc. (Nasdaq: GLUE), a clinical-stage biotechnology company developing novel molecular glue degrader (MGD)-based medicines, today announced an agreement to collaborate with Novartis to develop novel degraders for immune-mediated diseases. The agreement is the Company’s second with Novartis, in addition to the global exclusive license agreement for Monte Rosa’s VAV1 degraders including MRT-6160, announced in October 2024.

The agreement announced today was uniquely structured by the companies to collaborate on accelerating development of degraders for important immune-mediated diseases driven by highly credentialed and difficult-to-drug targets. Under the agreement, Monte Rosa’s scientists will apply their proprietary AI/ML-enabled QuEEN™ product engine for the discovery and development of degraders to be further developed and commercialized by Novartis.

Watertown, MA -- September 03, 2025 -- Treeline Biosciences (or "Treeline") today announced the initiation of Phase 1 trials for two internally discovered programs, TLN-121 and TLN-372, and a third in-licensed program, TLN-254. TLN-121, a BCL6 degrader, and TLN-254, an EZH2 inhibitor, are both being studied in patients with lymphoma. TLN-372, a pan-KRAS inhibitor, is being studied in patients with cancers expressing certain KRAS mutations. The company also announced the close of a $200 million Series A extension, bringing total funding to $1.1 billion.

• Business combination transaction with Helix Acquisition Corp. II, a special purpose acquisition company sponsored by affiliates of Cormorant Asset Management, completed on August 11, 2025
• Closed concurrent $261 million private investment in public equity (“PIPE”), led by Cormorant
• Combining approximately $120 million from the former Helix II trust account (reflecting a final redemption rate of approximately 39% which is the second lowest redemption rate for a biotech de-SPAC transaction since 2022) with the PIPE provides approximately $382 million in gross proceeds
• Net proceeds will be used to accelerate the development of BBOT's pipeline of clinical-stage RAS-targeted oncology drug candidates
• BBOT to commence trading on the Nasdaq Global Market under the ticker symbol “BBOT” on August 12, 2025

South San Francisco, CA and Boston, MA -- August 11, 2025 -- BridgeBio Oncology Therapeutics, Inc. (“BBOT”) (Nasdaq: BBOT), a clinical-stage biopharmaceutical company focused on RAS-pathway malignancies, today announced the closing of its previously announced business combination with Helix Acquisition Corp. II (formerly Nasdaq: HLXB) (“Helix”), a special purpose acquisition company (SPAC) sponsored by affiliates of Cormorant Asset Management. The business combination was approved by Helix’s shareholders on August 4, 2025, and closed on August 11, 2025.

In connection with the closing, Helix changed its name to “BridgeBio Oncology Therapeutics, Inc.”, and beginning on August 12, 2025, the shares of common stock of the combined company are expected to begin trading under the new ticker symbol “BBOT” on the Nasdaq Global Market.

 

RAHWAY, NJ & RALEIGH, NC -- July 9, 2025 -- Merck (NYSE: MRK), known as MSD outside of the United States and Canada, and Verona Pharma plc (Nasdaq: VRNA) (“Verona Pharma”), a biopharmaceutical company focused on respiratory diseases, today announced that the companies have entered into a definitive agreement under which Merck, through a subsidiary, will acquire Verona Pharma for $107 per American Depository Share (ADS), each of which represents eight Verona Pharma ordinary shares, for a total transaction value of approximately $10 billion.

Through this acquisition Merck will add Ohtuvayre® (ensifentrine), a first-in-class selective dual inhibitor of phosphodiesterase 3 and 4 (PDE3 and PDE4), to its growing cardio-pulmonary pipeline and portfolio. The U.S. Food and Drug Administration approved Ohtuvayre in June 2024 for the maintenance treatment of chronic obstructive pulmonary disease (COPD) in adult patients. Ohtuvayre is the first novel inhaled mechanism for the treatment of COPD in more than 20 years and combines bronchodilator and non-steroidal anti-inflammatory effects. Ohtuvayre is also being evaluated in clinical trials for the treatment of non-cystic fibrosis bronchiectasis.

• Dren Bio deep B-cell depleter program has the potential to reset the immune system
• Acquisition underpins path for Sanofi becoming leading immunology company
• Sanofi to pay $600 million up front  

Paris -- March 20, 2025 -- Sanofi and Dren Bio, Inc., a private clinical-stage biopharmaceutical company, have entered into a definitive agreement under which Sanofi has agreed to acquire DR-0201, a targeted bispecific myeloid cell engager (MCE) that has shown robust B-cell depletion in pre-clinical and early clinical studies. DR-0201 is a potential first-in-class CD20-directed bispecific antibody that targets and engages specific tissue-resident and trafficking myeloid cells to induce deep B-cell depletion via targeted phagocytosis. Recent early clinical study data in autoimmune diseases suggest that deep B-cell depletion might have the potential to reset the adaptive immune system, leading to sustained treatment-free remission in patients with refractory B-cell mediated autoimmune diseases such as lupus, where significant unmet medical needs remain. 

Wayne, PA -- November 18, 2024 --  Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, today announced that it has entered into an exclusive license agreement with Biosion, Inc. (Biosion) for worldwide rights (excluding Greater China) to BSI-045B, a potential best-in-class, clinical-stage, novel anti-TSLP monoclonal antibody, and BSI-502, a potential best-in-class, pre-clinical stage, novel bispecific antibody that is directed against both TSLP and IL4R. “This transformative transaction, which marks the completion of our strategic review process, accelerates our evolution into becoming a leading immunology company with both small and large molecule drugs," said Dr. Neal Walker, Interim CEO and Chair of the Board of Directors of Aclaris. “Despite recent advances with anti-TSLP and anti-IL4R therapies, there remains substantial unmet need for more effective and convenient treatment options. BSI-045B’s compelling Phase 2a proof-of-concept data in atopic dermatitis, together with BSI-502's dual-targeting approach, complement our existing ITK inhibitor portfolio, resulting in a pipeline of differentiated assets that targets multiple high-value indications. In addition, the proceeds from the private placement financing announced this morning bolsters our balance sheet and provides us with enhanced financial flexibility to support our strategic growth while maintaining our cash runway into 2028.”

Wayne, PA -- November 18, 2024 -- Aclaris Therapeutics, Inc. (NASDAQ: ACRS) (the “Company” or “Aclaris”), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, today announced that it has entered into a securities purchase agreement with a group of accredited investors for the private placement of 35,555,555 shares of common stock at a purchase price of $2.25 per share, for gross proceeds of approximately $80.0 million. The private placement is expected to close on or about November 19, 2024, subject to the satisfaction of customary closing conditions.

The private placement was led by Vivo Capital, with participation by new and existing investors including Forge Life Science Partners, Rock Springs Capital, RA Capital Management, Adage Capital Partners LP, Decheng Capital, Logos Capital and Samsara BioCapital.