News

XENLETA represents the first new antibiotic class approved for patients with CAP in Europe in nearly 20 years

XENLETA approval provides urgently needed short-course, empiric monotherapy treatment option for CAP aligned with core principles of antimicrobial stewardship DUBLIN, July 28, 2020 (GLOBE NEWSWIRE) -- Nabriva Therapeutics plc (NASDAQ: NBRV) announced today that the European Commission (EC) has issued a legally binding decision for approval of the marketing authorization application for XENLETA™ (lefamulin) for the treatment of community-acquired pneumonia (CAP) in adults following a review by the European Medicines Agency (EMA). The EMA approval of XENLETA in CAP patients when it is considered inappropriate to use antibacterial agents that are commonly recommended for initial treatment or when these agents have failed paves the way for the launch of XENLETA across Europe. The U.S. Food and Drug Administration (FDA) approved XENLETA for the treatment of adult patients with community-acquired bacterial pneumonia (CABP) in August 2019. The EMA approval is based on efficacy data from the Lefamulin Evaluation Against Pneumonia (LEAP) 1 and LEAP 2 studies and a safety database of 1,242 study participants. In the two Phase 3 clinical trials, the European Medicines Agency’s co-primary endpoint was the Investigator Assessment of Clinical Response (IACR) at Test of Cure (TOC) in both the clinically evaluable (CE) and modified intent-to-treat (mITT) populations. Both studies established XENLETA to be non-inferior to the standard-of-care moxifloxacin in the treatment of adults with CAP independently and when the pooled data were analyzed across PORT scores of II-V.  In the pooled analysis, the IACR success rate at TOC in the modified Intent-to-Treat (mITT) population was 85 percent in the XENLETA group and 87.1 percent in the moxifloxacin group (treatment difference ‑2.2 percent; 95 percent confidence interval (CI): ‑5.9, 1.6), and 88.5 percent in the lefamulin group and 91.8 percent in the moxifloxacin group (treatment difference ‑3.3 percent; 95 percent CI: ‑6.8, 0.1) in the clinically evaluable population. In these trials, lefamulin was generally well-tolerated.  The most frequently reported adverse reactions were administration site reactions, diarrhea, nausea, vomiting, hepatic enzyme elevation, headache, hypokalemia and insomnia.

- $32.5M Series A Financing Led by Aisling Capital, Vertex Ventures HC, Qiming Venture Partners USA, Driehaus Capital Management, and BVF Partners -

- Registration-Enabling Phase 2 CRESTONE Study Now Enrolling Patients with Solid Tumors of Any Origin that have an NRG1 Gene Fusion -

- CRESTONE Enrollment Enhanced Through Strategic Partnerships with Next Generation Sequencing Diagnostic Providers Including Ashion Analytics, Strata Oncology, and Tempus to Advance Patient Enrollment Practices for Genomically-driven, Tumor-agnostic Clinical Trials -

NEW YORK, July 21, 2020 /PRNewswire/ -- Elevation Oncology, a clinical stage biopharmaceutical company focused on the development of precision medicines for patients with genomically defined cancers, announced today the launch of the Company with a $32.5M Series A financing, initiation of the Phase 2 CRESTONE study, and new partnerships with Next Generation Sequencing diagnostic providers including Ashion Analytics, Strata Oncology, and Tempus to explore innovative models for real-time identification, patient referral, and enrollment of patients with tumors driven by rare genomic alterations. The Series A financing was led by Aisling Capital and a syndicate of investors including Vertex Ventures HC, Qiming Venture Partners USA, Driehaus Capital Management, and BVF Partners.

SAN DIEGO, July 9, 2020 /PRNewswire/ -- Poseida Therapeutics, Inc., a clinical-stage biopharmaceutical company dedicated to utilizing proprietary gene engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure, today announced the pricing of its initial public offering of 14,000,000 shares of its common stock at a price to the public of $16.00 per share. The gross proceeds to Poseida from the offering, before deducting underwriting discounts and commissions and estimated offering expenses, are expected to be $224.0 million. The shares are expected to begin trading on the Nasdaq Global Select Market on July 10, 2020 under the symbol "PSTX". The offering is expected to close on July 14, 2020, subject to satisfaction of customary closing conditions. In addition, Poseida has granted the underwriters a 30-day option to purchase up to an additional 2,100,000 shares of common stock at the initial public offering price less underwriting discounts and commissions. BofA Securities, Piper Sandler and William Blair are acting as joint book-running managers for the offering.

First patient dosed in AVROBIO’s global Phase 1/2 clinical trial of AVR-RD-02 for Gaucher disease type 1

Second patient dosed in investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 6, 2020-- AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the first patient has been dosed in the company’s GuardOne clinical trial, a Phase 1/2 investigational study evaluating AVR-RD-02 for Gaucher disease type 1. The company also announced that the second patient has been dosed in the ongoing investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis. “The first patient dosed is an important milestone for the Gaucher disease community and our AVR-RD-02 program. Gaucher disease type 1 leads to an array of serious symptoms and the current standard of care does not halt disease progression,” said Geoff MacKay, AVROBIO’s president and CEO. “With a single dose of our investigational lentiviral gene therapy, we aim to prevent the buildup of a fatty substrate in specialist immune cells called macrophages, as well as debilitating symptoms throughout the body, including the brain.” The company’s Phase 1/2 trial of AVR-RD-02 for Gaucher disease type 1 is currently recruiting patients in Australia and Canada, with new clinical sites expected to open in the U.S. and Israel by year-end.

SAN DIEGO, June 25, 2020 — Poseida Therapeutics, Inc., a clinical-stage biopharmaceutical company dedicated to utilizing proprietary gene engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure, today announced the closing of a Series D financing round, raising $110 million. The financing was led by funds advised by Fidelity Management Research Company, LLC, with participation  by Adage Capital Management and Schonfeld Strategic Advisors.  A number of current investors also participated in the financing. BofA Securities is acting as sole placement agent for the financing. “This financing supports the approach we are taking to leverage our broad proprietary gene engineering platform technologies, including the piggyBac DNA Modification System and Cas-CLOVER site-specific gene editing system, for the creation of numerous differentiated cell and gene therapy product candidates,” said Eric Ostertag, M.D., Ph.D., Chief Executive Officer of Poseida. Poseida’s portfolio includes allogeneic and autologous CAR-T product candidates in both hematological and solid tumor oncology indications, as well as liver-directed gene therapy programs in orphan genetic diseases.

— After Two Years of Daily Treatment, More Than 80% of Patients Were Successfully Desensitized to 2000 mg Peanut Protein or Equivalent of About 14 Peanut Kernels—

— Majority of Patients Treated Daily Also Reported Lower Rates of Adverse Events and Showed Higher Rates of Desensitization Compared to Non-Daily Dosing Groups —

BRISBANE, Calif.--(BUSINESS WIRE)--Jun. 8, 2020-- Aimmune Therapeutics, Inc. (Nasdaq: AIMT), a biopharmaceutical company developing and commercializing treatments for potentially life-threatening food allergies, today announced new two-year data that suggest long-term efficacy of daily treatment with PALFORZIA™ [Peanut (Arachis hypogaea) Allergen Powder-dnfp] in patients with peanut allergy. Patients dosed daily in the study also showed ongoing immunomodulation and higher rates of desensitization that increased over time compared to those patients who were administered non-daily dosing. Furthermore, the majority of patients in the daily dosing groups reported lower adverse event (AE) rates compared to those in the non-daily dosing groups, with an overall safety profile of long-term daily dosing with PALFORZIA that was better than non-daily dosing.

SAN DIEGO, May 20, 2020 — Poseida Therapeutics, Inc., a clinical-stage biopharmaceutical company dedicated to utilizing proprietary non-viral gene engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure, today announced the first patient has been dosed in its Phase 1 clinical trial evaluating P-PSMA-101, its autologous CAR-T therapeutic candidate, in metastatic castration-resistant prostate cancer. The study represents a promising advancement in evolving cell therapies to treat solid tumors. As part of its research, Poseida’s Phase 1 open label, multi-center, dose-escalating trial will include cohorts receiving single and multiple doses of P-PSMA-101, with the goal of determining the best dose with the fewest side effects.

Houston, TX—April 29, 2020—Marker Therapeutics, Inc. (Nasdaq:MRKR), a clinical-stage immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, today announced that the United States Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug designation to MT-401, a multi-tumor-associated antigen (MultiTAA)-specific T cell product for the treatment of patients with acute myeloid leukemia (AML), following allogeneic stem cell transplant.

- Preliminary Phase 1 results represent first clinical evidence that inhibition of the menin-MLL1 interaction can induce response in patients with MLL-r acute leukemias -- Data featured in New Drugs on the Horizon oral session at the 2020 AACR Virtual Annual Meeting I -- U.S. FDA grants orphan drug designation to SNDX-5613 for the treatment of adult and pediatric acute myeloid leukemia -

AUSTIN, Texas, April 08, 2020 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing next-generation human enzyme therapeutics as solutions for diseases with high unmet medical need, today announced the approval of its Clinical Trial Application (CTA) by the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) for ACN00177, a novel engineered human enzyme therapy designed to treat Homocystinuria, a serious metabolic disorder characterized by elevated plasma homocysteine levels, leading to a wide range of life-altering complications and reduced life expectancy.