Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are resolvable
FDA clinical reviews acknowledged that the clinical data are robust and biomarker data are supportive NOVATO, Calif., July 11, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its Biologics License... Read more
Robust objective response rates were observed (31% overall, 44% in KRAS-mutant, and 17% in KRAS wild-type) in patients whose cancer had progressed after multiple prior lines of therapy
The majority of patients (82%) had some reduction in target lesions, regardless of KRAS mutation status
Median progression-free survival was 12.9 months overall, 31.0 months in KRAS-mutant, and 12.8 months in KRAS wild-type BOSTON / Jul 11, 2025 / Business Wire / Verastem Oncology (Nasdaq:... Read more
Absence of neurotoxicity of any grade in low-volume disease to-date
On track for first Biologics License Application (BLA) approved cell therapy in orphan indication relapsed/refractory AL Amyloidosis
Potential future indication expansion planned LOS ANGELES, July 11, 2025 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us” or ”IMMX”), a clinical-stage biopharmaceutical company developing cell therapies for AL Amyloidosis... Read more
MONTREAL and CHARLOTTE, N.C., July 11, 2025 (GLOBE NEWSWIRE) -- Milestone® Pharmaceuticals Inc. (“Milestone”) (Nasdaq: MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, today announced the pricing of its previously announced underwritten public offering (the “Offering”) of (i) 31,500,000 of its common shares (the “Shares”), accompanying Series A common warrants (the “Series A Common... Read more
Strong interest from Saudi investment funds in CEL-SCI, Multikine, and potential joint venture to address the wider Middle East and North Africa market
Patient access and reimbursement/sale in Saudi Arabia within approximately 60 days following filing for Breakthrough Medicine Designation VIENNA, Va. / Jul 11, 2025 / Business Wire / CEL-SCI Corporation (NYSE American: CVM) today announced it has reached an agreement with one of Saudi Arabia’s premier pharmaceutical... Read more
HOUSTON, July 11, 2025 (GLOBE NEWSWIRE) -- FibroBiologics, Inc. (Nasdaq: FBLG) (“FibroBiologics”), a clinical-stage biotechnology company with 275+ patents issued and pending with a focus on the development of therapeutics and potential cures for chronic diseases using fibroblasts and fibroblast-derived materials, today announced that Founder and Chief Executive Officer, Pete O’Heeron, will present at the Advanced Wound Care Summit USA in Boston, Massachusetts, on... Read more
Exploring Strategic Partnerships to Expand Platform Beyond Reproductive Health SAN DIEGO, July 11, 2025 (GLOBE NEWSWIRE) -- Daré Bioscience, Inc. (NASDAQ: DARE), a biopharmaceutical company driven by a mission to challenge the status quo, making women's health a priority, today announced the receipt of a $6 million non-dilutive funding installment under its multi-year grant agreement to support development of DARE-LARC1, the contraception-focused use case... Read more
BOSTON, July 11, 2025 (GLOBE NEWSWIRE) -- Ikena Oncology, Inc. (Nasdaq: IKNA, “Ikena,” the “Company”) today announced that Institutional Shareholder Services Inc. (“ISS”) and Glass, Lewis & Co. (“Glass Lewis”) recommend that stockholders vote FOR the issuance of shares in connection with the previously disclosed proposed merger with Inmagene Biopharmaceuticals (“Inmagene”) at Ikena’s upcoming Annual Meeting of Stockholders on July 15, 2025.
ISS... Read more
Median Overall Survival (OS) of 13.9 months in triple negative breast cancer (TNBC) exceeds Trodelvy (11.8 months) and doubles control (6.9 months)
OS of 17.3 months in HR+ metastatic breast cancer surpasses Trodelvy (14.4 months) and control (11.2 months)
No treatment related discontinuations reported PHILADELPHIA and VANCOUVER, British Columbia, July 11, 2025 (GLOBE NEWSWIRE) -- BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW, BCTXZ) (TSX: BCT) (“BriaCell”... Read more
CELEBRATION, Fla., July 11, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced an oral presentation and two poster presentations on MIPLYFFA® (MY-PLY-FAH) (arimoclomol) are being featured at the National Niemann Pick Disease Foundation (NNPDF) Conference, taking place July 10-13, 2025, in Concord, North Carolina. The p... Read more
New report adds to growing evidence of iNKT cell therapy’s potential in solid tumors NEW YORK, July 11, 2025 (GLOBE NEWSWIRE) -- MiNK Therapeutics, Inc. (NASDAQ: INKT), a clinical-stage biopharmaceutical company pioneering allogeneic, off-the-shelf invariant natural killer T (iNKT) cell therapies, today announced the publication of another landmark case in Nature’s Oncogene describing a complete and durable remission in a patient with metastatic, treatment-refractory... Read more
FDA issued Complete Response Letter
Capricor plans to resubmit its BLA to include data from the ongoing Phase 3 HOPE-3 trial in Q3 2025 to continue pursuing the indication for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy
FDA advised Capricor to request a meeting to determine next steps toward potential approval
Conference call and webcast scheduled for today at 8:30 a.m. ET SAN DIEGO, July 11, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics... Read more
MONTREAL, Quebec and CHARLOTTE, N.C., July 11, 2025 (GLOBE NEWSWIRE) -- Milestone® Pharmaceuticals Inc. (“Milestone”) (Nasdaq: MIST) today announced that it has commenced an underwritten public offering (the “Offering”) of its common shares, accompanying Series A warrants to purchase common shares (the “Series A Warrants”) and accompanying Series B warrants to purchase common shares (the “Series B Warrants”), and, in lieu of common shares to certain... Read more
New PDUFA Action Date of December 13, 2025
$75 Million Royalty Purchase Agreement Payment from RTW Extended Through 2025 MONTREAL and CHARLOTTE, N.C., July 11, 2025 (GLOBE NEWSWIRE) -- Milestone® Pharmaceuticals Inc. (Nasdaq: MIST) (Milestone) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review Milestone’s response to issues raised in the Complete Response Letter (CRL) for CARDAMYST™ (etripamil) nasal spray, an... Read more
SANTA ANA, Calif., July 10, 2025 (GLOBE NEWSWIRE) -- NKGen Biotech, Inc. (OTC: NKGN) (“NKGen” or the “Company”), a clinical-stage biotechnology company focused on the development and commercialization of innovative autologous and allogeneic natural killer (“NK”) cell therapeutics, today announced the administration of the first dose of troculeucel, an expanded autologous NK cell therapy, under a single compassionate use, Investigational New Drug (“IND”)... Read more
WALTHAM, Mass. and BOULDER, Colo., July 10, 2025 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced the closing of its previously announced upsized underwritten public offering of 25,555,556 shares of its common stock, which includes 3,333,333 shares issued pursuant to the exercise in full by the underwriters of their option to purchase additional... Read more
Multimodal foundation model enables personalized cognitive forecasts and expands access to Alzheimer's diagnostics POTOMAC, MD / ACCESS Newswire / July 10, 2025 / IGC Pharma, Inc. (NYSE American:IGC) a clinical-stage biopharmaceutical company focused on Alzheimer's disease, today announced the development of MINT-AD, Multimodal Interpretable Transformer for Alzheimer's, the Company's proprietary AI-powered diagnostic platform designed to identify individuals at high... Read more
Construct including CT domain demonstrated higher levels of microdystrophin protein, increased muscle force, and improved resistance to damage in mice lacking dystrophin
REGENXBIO's next-generation investigational gene therapy, RGX-202, is the only microdystrophin construct that includes the CT domain
Findings support the positive functional data seen in Phase I/II AFFINITY DUCHENNE® trial of RGX-202 ROCKVILLE, Md., July 10, 2025 /PRNewswire/ -- REGENXBIO Inc.... Read more
ROCKVILLE, Md. and SUZHOU, China, July 10, 2025 (GLOBE NEWSWIRE) -- Ascentage Pharma (NASDAQ: AAPG; HKEX: 6855), a global biopharmaceutical company dedicated to addressing unmet medical needs in cancers, announced that its proprietary novel Bcl-2 selective inhibitor lisaftoclax (APG-2575) has been approved by China’s National Medical Products Administration (NMPA) for the treatment of adult patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL)... Read more
PRISM-1, the ongoing registrational, Phase 3 study evaluating quemliclustat plus chemotherapy as a first-line treatment for metastatic pancreatic cancer, is expected to be fully enrolled by the end of this year. HAYWARD, Calif. / Jul 10, 2025 / Business Wire / Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for patients with cancer, today announced that q... Read more
KOL webinar to take place on July 22, 2025, at 1:00 p.m. ET SAN DIEGO and CALGARY, AB, July 10, 2025 /CNW/ -- Oncolytics Biotech® Inc. (NASDAQ: ONCY) (TSX: ONC), a leading clinical-stage company specializing in immunotherapy for oncology, announced that it will host a key opinion leader (KOL) webinar to discuss pelareorep in metastatic pancreatic ductal adenocarcinoma (mPDAC) and other gastrointestinal cancers. The webinar will take place on Tuesday, July 22,... Read more
Company strategy: In discussions with multiple potential animal health company partners to collaborate to bring Canalevia®, conditionally approved for CID, to regulatory approval and commercialization for general diarrhea globally
Diarrhea is one of the most common reasons owners bring their dog to the veterinarian and the second most common reason for visits to the veterinary emergency hospital, yet there are currently no FDA-approved drugs to treat general diarrhea... Read more
Metastatic Colorectal Cancer Cohort in NCI-Led Multi-Cohort Study Demonstrates Promising Response Rate, Triggering Enrollment Expansion Under Simon Two-Stage Design PRINCETON, N.J., July 10, 2025 (GLOBE NEWSWIRE) -- PDS Biotechnology Corporation (Nasdaq: PDSB) (“PDS Biotech” or the “Company”), a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers, today announced patient recruitment has been completed in... Read more
BOTHELL, Wash., July 10, 2025 (GLOBE NEWSWIRE) -- Cocrystal Pharma, Inc. (Nasdaq: COCP) announces that its first-in-class pan-viral protease inhibitor CDI-988 has been selected for an oral presentation at the 9th International Calicivirus Conference being held September 7-11, 2025 in Banff, Alberta, Canada. Sam Lee, PhD, the Company’s President and co-CEO, will present “Oral Direct-Acting Antiviral CDI-988 for Norovirus Infection Prevention and Treatment: Mechanism... Read more
ZELSUVMI is now commercially available via prescription through retail pharmacies, ASPN pharmacy services and for at-home delivery through mail-order pharmacies
Once-daily topical prescription medication can be applied by patients, parents and caregivers outside of a physician's office, at home or on the go
Molluscum contagiosum is a highly contagious viral skin condition that afflicts an estimated 16.7 million people with up to 6 million new incidents per year in the... Read more
NEWTON, Mass., July 10, 2025 (GLOBE NEWSWIRE) -- Acumen Pharmaceuticals, Inc. (NASDAQ: ABOS), a clinical-stage biopharmaceutical company developing a novel therapeutic that targets soluble amyloid beta oligomers (AβOs) for the treatment of Alzheimer’s disease (AD), will present new findings at the upcoming Alzheimer's Association International Conference (AAIC®) 2025 in Toronto. The presentations include a cost savings analysis of the use of pTau217 as a screening... Read more
JUPITER, Fla., July 10, 2025 (GLOBE NEWSWIRE) -- Ligand Pharmaceuticals Incorporated (Nasdaq: LGND) today announced that its partner Pelthos Therapeutics Inc. (NYSE American: PTHS) has commercially launched ZELSUVMI™ (berdazimer) topical gel 10.3%, the first and only FDA-approved at-home treatment for molluscum contagiosum. The Company has earned a $5 million milestone payment from Pelthos following the commercial launch of ZELSUVMI.
“Molluscum impacts millions of... Read more
End of Phase 2 Meeting with FDA scheduled for August 27, 2025 to review OST-HER2 recurrent, pulmonary metastatic osteosarcoma program
Scientific Advice Meetings confirmed with global regulators in major markets - including the United Kingdom and European Union
All patients enrolled in its Phase 1 clinical study of OST-504 in second line prostate cancer have completed treatment, with data expected to be announced later in 2025 New York, New York--(Newsfile Corp. -... Read more
Positive Discussion on Path Forward for Registrational Program PURCHASE, N.Y., July 10, 2025 (GLOBE NEWSWIRE) -- Cognition Therapeutics, Inc., (the Company or Cognition) (NASDAQ: CGTX), a clinical stage company developing drugs that treat neurodegenerative disorders, conducted an end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) on July 9, 2025. The objective of this meeting was to review results from the Phase 2 study of zervimesine (CT1812) and... Read more
The Joint Society for Neuro-Oncology (SNO) and the American Society of Clinical Oncology (ASCO) focus on the rapidly evolving area of central nervous system metastases
Presentations will highlight the clinical utility of the company’s CNSide Cerebrospinal Fluid (CSF) Assay Platform for patients at risk for central nervous system metastases HOUSTON, July 10, 2025 (GLOBE NEWSWIRE) -- Plus Therapeutics, Inc. (Nasdaq: PSTV) (the “Company”), a clinical-stage... Read more
[IMAGES BELOW] Sustained complete resolution of temporal lobe brain metastasis and continued orbital tumor reduction after >18 months of treatment
“Eye bulging” metastatic breast cancer patient had failed 8 prior regimens, including antibody-drug conjugate (ADC) therapy
Patient remains on BriaCell’s Phase 2 study with 29 treatment cycles completed PHILADELPHIA and VANCOUVER, British Columbia, July 10, 2025 (GLOBE NEWSWIRE) -- BriaCell Therapeutics Corp.... Read more
Hot Line Presentation of MAPLE-HCM to Elaborate on Positive Topline Results in Patients with Obstructive Hypertrophic Cardiomyopathy
Late Breaking Clinical Science Session to Present Incidence and Impact of Atrial Fibrillation Across Three Clinical Trials of Aficamten in Obstructive Hypertrophic Cardiomyopathy SOUTH SAN FRANCISCO, Calif., July 10, 2025 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced five presentations related to aficamten... Read more
Data from an analysis designed to evaluate the potential effects of the Phase 3 zorevunersen dosing regimen showed improvements in cognition and behavior at Week 68
These findings support the inclusion of key secondary endpoints assessing cognition and behavior in the Phase 3 EMPEROR study and contrast with outcomes observed in natural history data BEDFORD, Mass., & CAMBRIDGE, Mass. / Jul 10, 2025 / Business Wire / Stoke Therapeutics, Inc. (Nasdaq: STOK),... Read more
Received FDA approval of EKTERLY® (sebetralstat)—the first and only oral on-demand treatment for hereditary angioedema; U.S. launch underway
Six additional global regulatory submissions under review
Entered licensing agreements for sebetralstat commercialization in Japan and Canada
$220.6M in cash, providing runway into 2027 CAMBRIDGE, Mass. & SALISBURY, England / Jul 10, 2025 / Business Wire / KalVista Pharmaceuticals, Inc. (Nasdaq:... Read more
TNX-801 is up to 100,000-fold less virulent than live smallpox vaccine strains and a single dose provides robust immunogenicity and protection against mpox and rabbitpox (more than one year) in animals
Subcutaneous administration of TNX-801 yielded equivalent protection to the traditional percutaneous administration CHATHAM, N.J., July 10, 2025 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a fully integrated biopharmaceutical company... Read more
REDWOOD CITY, Calif., July 10, 2025 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (Soleno) (Nasdaq: SLNO), a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, announced today certain preliminary unaudited financial and operational results for the three months ended June 30, 2025: Soleno expects its net revenue from the sales of VYKATTM XR, for the three months ended June 30, 2025 to be between $31.0 million and $33.0 million.... Read more
REDWOOD CITY, Calif., July 10, 2025 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (Soleno) (Nasdaq:SLNO), a biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, announced today that it intends to offer and sell $200 million of shares of its common stock in an underwritten public offering. In addition, Soleno intends to grant the underwriters a 30-day option to purchase up to an additional $30 million of shares of common stock.... Read more
Selection Follows Robust Succession Process Led by the Board
Bruce Cozadd to Retire as CEO and Remain Chairperson of the Board of Directors DUBLIN, July 10, 2025 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the Jazz Board of Directors has unanimously selected Renee Gala, Jazz's President and Chief Operating Officer, as the President and Chief Executive Officer, effective August 11, 2025, when she will also... Read more
SAN CARLOS, Calif., July 10, 2025 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a commercial biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, today announced the appointment of Marc. R Theoret, M.D. to assume the newly created position of Senior Vice President, Regulatory Strategy, where he will focus on clinical regulatory strategy for Iovance’s... Read more
Kelowna, British Columbia – July 10, 2025 – TheNewswire - Rize Oncology Inc. (the “Company”), a clinical-stage pharmaceutical company focused on developing innovative oncology therapeutics, today announced that it has appointed Executive Chairperson Daren Graham as the Company’s interim Chief Executive Officer.
Mr. Graham’s appointment will be effective July 10, 2025 and coincides with the resignation of Roland Boivin as the Company’s chief executive officer... Read more
BOSTON, July 09, 2025 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM) (“Rhythm”), a global, commercial-stage biopharmaceutical company dedicated to transforming the lives of patients living with rare neuroendocrine diseases, today announced a proposed public offering of $150 million of its common stock. All securities in the offering will be offered by Rhythm. In addition, Rhythm intends to grant the underwriters a 30-day option to purchase up to an... Read more
Data from Orbit and Cosmic studies expected around the end of the year NOVATO, Calif., July 09, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and Mereo BioPharma Group plc (NASDAQ: MREO), today announced that the randomized, placebo-controlled Phase 3 portion of the Orbit study evaluating UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (OI) is progressing toward a final analysis consistent with the original... Read more
Collaboration extension through 2029 accelerates access to MRD technology across four major cancer types FREMONT, Calif. / Jul 09, 2025 / Business Wire / Personalis, Inc. (Nasdaq: PSNL) today announced an expansion and extension of its strategic collaboration with Tempus AI, Inc. (Nasdaq: TEM), adding a new indication, colorectal cancer (CRC), to the existing, exclusive commercialization agreement. The update means Personalis and Tempus will now work to bring to market... Read more
The previously disclosed and now published RESILIENT data show that once-nightly TNX-102 SL achieved statistically significant improvement in the primary endpoint of reducing fibromyalgia pain versus placebo, and was generally well tolerated
These results confirm findings from the previously published RELIEF phase 3 trial, which also demonstrated a statistically significant reduction in fibromyalgia pain
FDA target PDUFA date for TNX-102 SL is August 15, 2025 and, if... Read more
Presented updated data for bexobrutideg (NX-5948) at EHA2025 and ICML-18, demonstrating a favorable safety profile and deepening responses in patients with r/r chronic lymphocytic leukemia (CLL) and Waldenström macroglobulinemia (WM)
Secured $15M license fee as Sanofi extends STAT6 collaboration to target type 2 inflammatory diseases
Announced FDA clearance of IND application for novel IRAK4 degrader GS-6791/NX-0479, enabling collaboration partner Gilead to initiate... Read more
SAN DIEGO, July 09, 2025 (GLOBE NEWSWIRE) -- Calidi Biotherapeutics, Inc. (“Calidi” or the “Company”) (NYSE American: CLDI), a clinical-stage biotechnology company pioneering the development of targeted therapies with the potential to deliver genetic medicines to distal sites of disease, today announced the entry into a definitive agreement for the immediate exercise of certain outstanding Series A, Series B-1, Series C-1, Series D, Series E and Series F Warrants... Read more
CHICAGO / Jul 09, 2025 / Business Wire / MAIA Biotechnology, Inc. (NYSE American: MAIA), a clinical-stage biopharmaceutical company focused on developing targeted immunotherapies for cancer, today announced dosing of the first patient in Taiwan in the expansion phase of its THIO-101 Phase 2 trial for advanced non-small cell lung cancer (NSCLC). The trial’s entry into another continent marks a key milestone for MAIA, opening a significantly larger patient pool for its evaluations... Read more
SAN DIEGO, July 09, 2025 (GLOBE NEWSWIRE) -- Connect Biopharma Holdings Limited (Nasdaq: CNTB) (“Connect Biopharma”, “Connect”, or the “Company”), a clinical-stage biopharmaceutical company focused on transforming care for the treatment of inflammatory diseases, today announced that the Company’s collaborator and exclusive licensee in China, Simcere Pharmaceutical Co., Ltd. (“Simcere”), has submitted its New Drug Application for rademikibart to the National... Read more
Secured approval from the Regulation Agency for Medical and Pharmaceutical Activities (RAMPA) in Georgia for Phase 2B/3 MIRACLE trial
Recent EU approval from the European Medicines Agency (EMA) boosts trial profile
Enrollment in Part A of MIRACLE reaches seven subjects treated and one additional subject in screening
16 additional clinical sites in Europe and the US expected to begin recruitment by the end of August; building to an expected 30 plus sites for Part... Read more
Company’s New Groundbreaking Preclinical Data in NSCLC Models Provides Further Validation for SIL204's Innovative Delivery System; Company is Currently Conducting Additional Studies into New and Previously Untested KRAS Mutation with Results expected in the Near Future Grand Cayman, Cayman Islands, July 09, 2025 (GLOBE NEWSWIRE) -- Silexion Therapeutics Corp. (NASDAQ: SLXN) ("Silexion" or the "Company"), a clinical-stage biotechnology company pioneering RNA interference... Read more
