Key approvals, positive study results and a partnership deal to fast track new medications. 11.09.2026 The US Food and Drug Administration and the European Medicines Agency have granted two biotechs with Fast Track designation and Orphan Drug designation, respectively for therapies targeting ventilator-associated bacterial pneumonia and thymic epithelial tumours. Windward Bio and Pharvaris announced postive results from late-stage clinical trials. A new strategic collaboration for Avrios Therapeutics will see the company advance its AVR-001, an advanced preclinical gene therapy candidate for certain familial forms of amyotrophic lateral sclerosis. BioVersys, a multi-asset biotech company focused on the development of novel antibacterial products for serious life-threatening infections caused by multi-drug resistant (MDR) bacteria, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for its lead candidate BV100 under the Qualified Infectious Disease Product (QIDP) designation. BV100 is currently being advanced for the treatment of ventilator-associated bacterial pneumonia (VABP) and hospital-acquired bacterial pneumonia (HABP) caused by carbapenem-resistant Acinetobacter baumannii-calcoaceticus complex (CRABC). BV100 possesses a novel mode of action via uptake via the siderophore pathway in Acinetobacter, providing a much-needed novel non-β-lactam therapy for this infection. BV100 is currently studied in two clinical trials: the pivotal Phase 3 global registrational trial, RIV-TARGET and the open-label Phase 2b differentiation trial, RIV-CARE in collaboration with ADVANCE-ID. These studies follow the successful Phase 2 study that showed BV100 had an overall 50% reduction in mortality compared with the best available therapy. In 2019, BV100 received the Qualified Infectious Disease Product (QIDP) Designation by the U.S. FDA. With the Fast Track designation, BioVersys can benefit from more frequent interactions with the FDA, increased communication on clinical trial design and data requirements, and eligibility for rolling review. EU Orphan Drug Designation for Torqur's thymic epithelial Torqur AG, a Swiss Rockets company advancing innovative clinical-stage treatments for oncology and dermatology, announced that the European Commission has granted orphan drug designation (ODD) for bimiralisib for the treatment of thymic epithelial tumours - severe and life-threatening cancers that lacks effective treatment options, are chronically debilitating due to their association with severe autoimmune comorbidities, such as myasthenia gravis, and they have the potential to progress into refractory disease. Receiving the EU ODD follows a positive opinion from the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP), marking bimiralisib's third orphan drug designation, and its first in a solid tumour indication, building on the company's broader oral bimiralisib oncology pipeline. An EU ODD reflects the intention to diagnose, prevent or treat a life-threatening or chronically debilitating condition affecting not more than five in 10 thousand people in the Community when the application is made. The ODD recognizes the potential of bimiralisib to address the needs of patients affected by this devastating disease. Orphan designation in the EU provides access to protocol assistance and reduced regulatory fees during development and, upon marketing authorisation, up to ten years of market exclusivity in the designated indication. Windward Bio: positive Phase 2 results of WIN378 in asthma Windward Bio, a clinical-stage biotechnology company developing ultra-long-acting antibodies for patients with serious respiratory and dermatologic diseases, announced positive interim results from the Phase 2 portion of the POLARIS-1 clinical trial evaluating twice-yearly dosing of WIN378 in asthma. The Phase 2 portion of the study is designed to evaluate the pharmacokinetics, safety, immunogenicity and pharmacodynamic activity of WIN378 in patients with uncontrolled, moderate-to-severe asthma. The study also evaluates the effects of WIN378 on lung function and biomarkers of airway inflammation and informed dose selection for Phase 3 development. The Phase 2 portion of the study enrolled a total of 147 patients, exceeding the initial enrollment target of 120 patients, with the interim analysis conducted on the first 98 patients. The interim analysis demonstrated favorable safety and clinically meaningful effects across key measures of lung function and inflammation in asthma. Two twice-yearly doses have been selected for the Phase 3 portion of POLARIS-1 which is designed to evaluate the effect of WIN378 on annualized asthma exacerbation rate and other key efficacy measures in patients with severe asthma. The Phase 3 portion of the study has been initiated, and a second Phase 3 study, POLARIS-2, is planned to begin in the first half of 2027. Pharvaris: positive Phase 3 topline data in all three types of HAE Pharvaris, a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to help address unmet needs of those living with bradykinin-mediated angioedema (AE-BK), such as hereditary angioedema (HAE) and acquired angioedema due to C1 inhibitor deficiency (AAE-C1INH), today announced statistically significant and clinically meaningful topline results of the CHAPTER-3 pivotal Phase 3 study evaluating deucrictibant extended-release (XR) tablet for the prevention of HAE attacks. The primary endpoint and all secondary efficacy endpoints were met with statistical significance. Data from the CHAPTER-3 study will serve as the basis for marketing authorization applications, which are planned to be submitted starting in the first half of 2027. HAE is a complex, rare, life-long genetic condition with significant burden from recurrent, unpredictable, disabling, and potentially life-threatening attacks of swelling affecting various body parts. Common attack locations include the extremities, face and neck, abdomen, and larynx. If untreated, HAE attacks can last multiple days. HAE affects all genders and ethnicities. Different forms of HAE are currently recognized and genetically identifiable due to their pathogenesis. Avrion Therapeutics enters strategic collaboration to advance AV-001 Lausanne-based Avrion Therapeutics focused on the developing transformative therapies for ALS and other neurodegenerative diseases, together with Institut Charcot, an initiative of ARSLA and ReMedys Foundation have signed a memorandum of understanding setting out a collaboration to advance AVR-001, an advanced preclinical gene therapy candidate for certain familial forms of amyotrophic lateral sclerosis (ALS) associated with mutations in SOD1. AVR-001 arises from research led by Dr. Bernard Schneider at EPFL, is jointly developed by ReMedys and Avrion, and will be included in Institut Charcot's integrated translational pipeline. The collaboration brings together ARSLA's patient-community perspective, Institut Charcot's scientific network, ReMedys' translational development expertise, and Avrion's gene therapy capabilities. Together, the partners aim to move the AVR-001 program towards clinical evaluation. 0Comments.

Pharvaris, a late-stage biopharmaceutical company developing oral treatments for bradykinin-mediated angioedema, reported second quarter 2026 financial results. The company held €318 million in cash and cash equivalents as of 30 June 2026. The FDA is reviewing Pharvaris' New Drug Application for deucrictibant IR, with a PDUFA date of 23 April 2027. The EMA validated the marketing authorisation application in July 2026. Pharvaris expects topline data from CHAPTER-3, a pivotal Phase 3 study of deucrictibant XR for hereditary angioedema prophylaxis, in the third quarter of 2026. Enrolment continues in CREAATE, a pivotal study for acquired angioedema treatment. The company closed a $132 million underwritten offering in May, extending its cash runway into 2028. Second quarter loss was €47.8 million, with R&D expenses of €35.0 million and G&A expenses of €15.8 million.