News
Mar 9
2020
AVROBIO Receives Orphan Drug Designation from U.S. FDA for AVR‑RD‑04 for Cystinosis
Mar 9
2020
AVR-RD-04 is the third AVROBIO investigational gene therapy to receive orphan status
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- 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 U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s investigational gene therapy, AVR-RD-04, for the treatment of cystinosis. The gene therapy consists of the patient’s own hematopoietic stem cells, which are genetically modified to express cystinosin, the protein that is functionally deficient in people with cystinosis. A Phase 1/2 clinical trial to evaluate the safety and efficacy of the investigational gene therapy in patients with cystinosis is ongoing, sponsored by AVROBIO’s academic collaborator at the University of California, San Diego (UCSD).
Mar 4
2020
Zosano Pharma Announces FDA Acceptance of 505(b)(2) New Drug Application for Qtrypta™ for the Acute Treatment of Migraine
Mar 4
2020
FREMONT, Calif., March 04, 2020 (GLOBE NEWSWIRE) -- Zosano Pharma Corporation (Nasdaq: ZSAN), a biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for Qtrypta™ for filing and substantive review.
The Prescription Drug User Fee Act goal date for the completion of the FDA’s review of Qtrypta™ is set for October 20, 2020. This date reflects a standard 10-month review period and is consistent with the review timeline for a 505(b)(2) NDA submission.
Feb 26
2020
Arcus Biosciences and Taiho Pharmaceutical Co., Ltd. Jointly Announce Taiho’s Exercise of Its Option for an Exclusive License to Zimberelimab (AB122) for Its Territories in Japan and Other Asian Countries
Feb 26
2020
Taiho’s in-licensing facilitates global development and commercialization of zimberelimab as a monotherapy and as a combination backbone for Arcus’s and Taiho’s oncology portfolios
Zimberelimab exhibits clinical activity and a safety profile consistent with those of currently approved anti-PD-1 therapies
Broad development program delivering randomized data in 2020 for zimberelimab in multiple novel combinations, including with AB154, Arcus’s anti-TIGIT antibody, and with AB928, the first and only dual A2a/A2b adenosine receptor antagonist in the clinic
HAYWARD, Calif.--(BUSINESS WIRE)-- Arcus Biosciences, Inc. (NYSE:RCUS), an oncology-focused biopharmaceutical company working to create best-in-class cancer therapies, and Taiho Pharmaceutical Co. Ltd., (“Taiho”), today announced Taiho’s exercise of its option to exclusively license zimberelimab, an anti-PD-1 monoclonal antibody, from Arcus Biosciences for commercialization in Japan and certain other territories in Asia (excluding China). This is Taiho’s second exercise of an Arcus program and follows their 2018 decision to license AB928, the first and only dual A2a/A2b adenosine receptor antagonist in the clinic.
Feb 19
2020
Spruce Biosciences Raises $88 Million in Series B Financing, co-led by Omega Funds and Abingworth
Feb 19
2020
– Proceeds to advance development of Tildacerfont in Congenital Adrenal Hyperplasia and other indications
– Existing Investors Novo Holdings and RiverVest Venture Partners support the deal with new Investors HealthCap, Rock Springs Capital, Aisling Capital, Surveyor Capital (a Citadel company) and Sands Capital joining the syndicate
San Francisco, California — February 19, 2020 – Spruce Biosciences today announced it has raised $88 million in Series B financing co-led by Omega Funds and Abingworth and supported by existing investors Novo Holdings and RiverVest Venture Partners, as well as new investors HealthCap, Rock Springs Capital, Surveyor Capital (a Citadel company), Aisling Capital and Sands Capital. Spruce is a clinical stage biopharmaceutical company developing therapeutics for rare endocrine diseases. The proceeds will be used to further fund the clinical development of the company’s lead product, tildacerfont, in classic congenital adrenal hyperplasia (CAH) and other conditions.
Tildacerfont, a second generation CRF-1 receptor antagonist, is the first non-steroidal molecule to demonstrate an ability to normalize elevated androgens in patients with CAH in clinical studies. With the closing of the financing, Spruce will evaluate the ability of tildacerfont to reduce glucocorticoid (GC) steroid usage and improve clinical outcomes in a late-stage clinical program in adults with CAH. In addition, the company plans to complete proof-of-concept trials in the pediatric population.
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Feb 11
2020
AVROBIO Presents Positive Initial Data for its Investigational Cystinosis Program and Plato TM Platform, as well as Positive Data Out to 32 Months for its Ongoing Investigational Fabry Program
Feb 11
2020
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 platoTM 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 16th Annual WORLDSymposiumTM in Orlando, Fla.
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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
Feb 6
2020
Aimmune Licenses Exclusive Worldwide Rights to Xencor’s XmAb®7195 for the Development of Next-Generation Food Allergy Treatments
Feb 6
2020
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.
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Feb 1
2020
FDA Approves Aimmune’s PALFORZIA™ as First Treatment for Peanut Allergy
Feb 1
2020
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.
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Jan 31
2020
Syndax Announces $35.0 Million Offering of Common Stock and Enters Term Sheet for Loan with Hercules Capital for up to $30 Million
Jan 31
2020
WALTHAM, Mass., Jan. 31, 2020 /PRNewswire/ -- Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq:SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that it has entered into an agreement with five leading life sciences investors, including Biotechnology Value Fund, L.P., Boxer Capital and AI Life Sciences Investments LLC, an affiliate of Access Industries Inc., for the purchase of common stock at $8.00 per share, representing a premium of 20% to the share price as of market close on Thursday, January 30, 2020. Syndax anticipates aggregate gross proceeds from the offering will be approximately $35.0 million. Closing of the transaction is expected to occur on or about February 4, 2020.
In addition, Syndax Pharmaceuticals announced that it has entered into a term sheet with Hercules Capital, Inc. (NYSE: HTGC) for a term loan of up to $30.0 million, consisting of an initial tranche of $20.0 million to be funded at the closing with the potential for a second tranche of $10.0 million subject to satisfaction of certain terms and conditions. The debt facility is expected to be used to support the Company's pipeline programs. The term loan is subject to negotiation and execution of definitive agreements.
Jan 30
2020
Science Magazine Publishes Results from Preclinical Study on the Activity of Menin-MLL Inhibition for the Treatment of NPM1 Acute Myeloid Leukemia
Jan 30
2020
WALTHAM, Mass., Jan. 30, 2020 /PRNewswire/ -- Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that Science magazine has published a preclinical report supporting the potential role of MLL1-Menin inhibition in the management of nucleophosmin (NPM1) mutant acute myeloid leukemia (AML). The article, "Therapeutic targeting of preleukemia cells in a mouse model of NPM1 mutant acute myeloid leukemia," will be published in the journal's January 31, 2020 issue and is currently available online.
This study examined the activity of VTP-50469, an orally-available inhibitor of MLL1-Menin interaction and close analog of the Company's lead Menin inhibitor, SNDX-5613, for the treatment of established NPM1 AML and the possible prevention of the disease in high-risk populations. Using preclinical models of NPM1 AML, the authors established that the presence of an NPM1 mutation is a clear indicator of pre-leukemic activity and represents a critical step in the development of AML. VTP-50469 was shown to eradicate NPM1 mutant cells at various stages of disease development, suggesting that Menin-MLL inhibition could potentially serve either as a targeted preventive therapy or as a treatment of established disease.
Jan 14
2020
Verona Pharma Reports Positive Top-line Data in 4 Week Phase 2b COPD Study with Nebulized Ensifentrine on Top of Tiotropium Therapy
Jan 14
2020
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.
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