News

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Feb. 10, 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 new initial data from the first patient dosed in the investigational gene therapy program for cystinosis, showing improvements in early measures at three months compared to baseline. The company also unveiled new clinical data showcasing a sustained biomarker response in patients for up to 32 months after receiving the company’s investigational gene therapy for Fabry disease across metrics including vector copy number (VCN), substrate levels and enzyme activity. Additionally, the company reported on the clinical debut of its plato^TM gene therapy platform. These data showed improved enzyme activity, transduction efficiency and VCN in drug product manufactured using plato compared with drug product produced using the academic platform, as well as higher in vivo enzyme activity at one month in the first patient treated with plato, as compared to other patients treated using the academic platform. All these data will be presented today, during the 16^th Annual WORLDSymposium^TM in Orlando, Fla.

Early data trends from first patient dosed in the AVR-RD-04 investigational gene therapy program for cystinosis show improvements across multiple measures

Data from the Phase 1 and Phase 2 trials of AVR-RD-01 support potential long-term engraftment and durable, endogenous production of functional enzyme in patients with Fabry disease

First Phase 2 Fabry patient treated using plato gene therapy platform shows plasma enzyme activity at one month 4.0 times higher than mean activity of other Phase 2 patients treated using academic platform at same timepoint

BRISBANE, Calif.–(BUSINESS WIRE)–Feb. 5, 2020– Aimmune Therapeutics, Inc. (Nasdaq: AIMT), a biopharmaceutical company developing treatments for potentially life-threatening food allergies, today announced it has obtained an exclusive worldwide license to develop and commercialize the investigational humanized monoclonal antibody XmAb^®7195 from Xencor, Inc.

XmAb7195, which has been renamed AIMab7195, was originally developed by Xencor for the treatment of allergic asthma. It uses three distinct mechanisms of action to reduce blood serum IgE and suppress IgE-producing cells. Aimmune initially plans to develop AIMab7195 as an adjunctive treatment with select Characterized Oral Desensitized ImmunoTherapy (CODIT™) programs, including PALFORZIA^™, to explore treatment outcomes in patients with food allergies.

BRISBANE, Calif.–(BUSINESS WIRE)–Jan. 31, 2020– Aimmune Therapeutics, Inc. (Nasdaq: AIMT), a biopharmaceutical company developing and commercializing treatments for potentially life-threatening food allergies, today announced that the U.S. Food and Drug Administration (FDA) approved PALFORZIA™ [Peanut (Arachis hypogaea) Allergen Powder-dnfp]. PALFORZIA is the first approved treatment for patients with peanut allergy. It is an oral immunotherapy indicated for the mitigation of allergic reactions, including anaphylaxis, that may occur with accidental exposure to peanut. PALFORZIA is approved for use in patients with a confirmed diagnosis of peanut allergy. Initial dose escalation may be administered to patients aged 4 through 17 years. Up-dosing and maintenance may be continued in patients 4 years of age and older. PALFORZIA is to be used in conjunction with a peanut-avoidant diet. PALFORZIA is not indicated for the emergency treatment of allergic reactions, including anaphylaxis.

LONDON, Jan. 13, 2020 (GLOBE NEWSWIRE) — Verona Pharma plc (AIM: VRP) (Nasdaq: VRNA) (“Verona Pharma”), a biopharmaceutical company focused on respiratory diseases, announces positive top-line data from a 4 week, 416 patient, Phase 2b dose-ranging study evaluating nebulized ensifentrine (0.375 mg, 0.75 mg, 1.5 mg and 3.0 mg) or placebo as an add-on treatment to tiotropium (Spiriva^® Respimat^®), a long acting anti-muscarinic (“LAMA”) bronchodilator, in patients with moderate to severe chronic obstructive pulmonary disease (“COPD”).

The study met its primary endpoint of improved lung function, with ensifentrine added on to inhaled tiotropium, a LAMA commonly used to treat COPD. Ensifentrine produced a clinically and statistically significant, and dose-dependent improvement in peak forced expiratory volume in one second (“FEV₁”)¹ at week 4 compared to placebo added on to tiotropium.  

WAYNE, Pa., Jan. 09, 2020 (GLOBE NEWSWIRE) — Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a physician-led biopharmaceutical company focused on immuno-inflammatory diseases, today announced positive results from ATI-450-PKPD-101, a Single Ascending Dose and Multiple Ascending Dose (SAD/MAD) Phase 1 clinical trial of the investigational compound ATI-450. Preliminary data demonstrated that ATI-450:

• resulted in marked inhibition of TNFα, IL1β, IL8, and IL6;
• was generally well-tolerated at all doses tested in the trial;
• had dose proportional pharmacokinetics (PK) with a terminal half-life of 9-12 hours; and
• had no meaningful food effect or drug-drug interaction (DDI) with methotrexate.

BOSTON, December 18, 2019 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) subsidiary Origin Biosciences, a company focused on developing and commercializing a treatment for Molybdenum Cofactor Deficiency (MoCD) Type A, and Medison Pharma Ltd., a leading commercial biotech partner that operates in Israel, Canada and Central and Eastern European countries, have entered into an exclusive license agreement under which Medison received rights from Origin to distribute, market, sell and otherwise commercialize Origin’s drug product known as fosdenopterin (BBP-870/ORGN001) in Israel.

Collaboration leverages Genentech’s MORPHEUS studies to accelerate development of AB928, a potential best-in-class therapy targeting the adenosine axis to stimulate immune responses against cancer

HAYWARD, Calif.–(BUSINESS WIRE)– Arcus Biosciences, Inc. (NYSE:RCUS), an oncology-focused biopharmaceutical company discovering and developing highly-differentiated therapies, today announced a clinical collaboration with Genentech, a member of the Roche group, for the evaluation of novel combinations with AB928, Arcus’s dual antagonist of adenosine receptors A_2aR and A_2bR, for colorectal (CRC) and pancreatic (PDAC) cancers. The collaboration will utilize the MORPHEUS Phase 1b/2 platform for rapid and efficient combination development, with upfront randomization versus control groups, in two studies:

• Third-line metastatic CRC: two combination arms, (1) AB928 plus atezolizumab (TECENTRIQ^®) and regorafenib, and (2) atezolizumab plus regorafenib, will be randomized versus regorafenib monotherapy.
• First-line metastatic PDAC: the combination of AB928 plus atezolizumab and gemcitabine/nab-paclitaxel will be randomized versus gemcitabine/nab-paclitaxel.

• Phase 3 trial (PRIMROSE 2) met primary and secondary efficacy endpoints
• 93.9% responder rate for 200 mg with ABT (p < 0.001)
• 56.7% responder rate for 100 mg without ABT (p < 0.001)
• Both doses achieved important clinically relevant secondary endpoints including amenorrhea (p < 0.001), reduction in pain (p < 0.001), improvement in quality of life (p < 0.001) and improvement in hemoglobin levels (p < 0.002)

GENEVA, Switzerland and BOSTON, MA (December 9, 2019) – ObsEva SA (NASDAQ: OBSV; SIX: OBSN), a biopharmaceutical company developing and commercializing novel therapies to improve women’s reproductive health, today reported positive Phase 3 trial results from the PRIMROSE 2 trial of linzagolix for the treatment of heavy menstrual bleeding (HMB) due to uterine fibroids.

SOUTH SAN FRANCISCO, Calif., Dec. 05, 2019 (GLOBE NEWSWIRE) — Sunesis Pharmaceuticals, Inc. (Nasdaq: SNSS) today announced data from the Company’s Phase 1b/2 clinical trial of its non-covalent BTK inhibitor vecabrutinib in adults with relapsed/refractory chronic lymphocytic leukemia (CLL) and other B-cell malignancies.

REDWOOD CITY, Calif., Dec. 03, 2019 (GLOBE NEWSWIRE) — Atreca, Inc. (Atreca) (NASDAQ: BCEL), a biotechnology company focused on the development of novel cancer therapeutics generated through a unique discovery platform based on interrogation of the active human immune response, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application. Atreca expects to initiate a first-in-human Phase 1b clinical trial of ATRC-101 in patients with solid tumors in early 2020.