REDWOOD CITY, Calif., Dec. 10, 2018 (GLOBE NEWSWIRE) — Menlo Therapeutics Inc. (NASDAQ: MNLO), a late-stage biopharmaceutical company focused on the development of serlopitant for the treatment of pruritus (itch), today announced positive top-line results from MTI-109 (PSORIXA), a 204-patient Phase 2 clinical trial of serlopitant for the treatment of pruritus associated with psoriasis.  The trial successfully met its primary endpoint, showing a statistically significant reduction in pruritus based upon a 4-point improvement responder analysis.  In the trial, 33% of patients treated with serlopitant 5 mg daily achieved a 4-point or greater improvement on the worst-itch numeric rating scale, or WI-NRS, at week 8 compared to baseline (primary efficacy endpoint) vs. 21% of patients treated with placebo (p= 0.028).

The trial also prospectively defined three key secondary endpoints for sequential step-down analyses at week 4 and days 7 and 3. The trial successfully met the secondary endpoint of WI-NRS responder rate at week 4.  At week 4, 21% of patients treated with serlopitant achieved a 4-point or greater improvement on the WI-NRS vs. 11% of patients treated with placebo (p=0.039).  Assessment of the secondary endpoints of the absolute change in WI-NRS from baseline to day 7 and day 3 for serlopitant compared to placebo showed a greater numerical, but not statistically significant, improvement for the serlopitant group.  At every assessed time point in the trial (daily in week 1 and average weekly scores through week 8), the serlopitant treated group demonstrated greater numerical improvement than the placebo group in both the WI-NRS 4-point responder analysis and in the mean change in WI-NRS from baseline.

Serlopitant was well-tolerated in this clinical trial.  No serious adverse events were reported for serlopitant treated patients.  Treatment-emergent adverse events assessed as likely related to treatment were observed with similar frequency in both groups (4.0% for placebo and 4.9% for serlopitant).  The consolidated safety summary for serlopitant now includes more than 1,600 evaluable patients, including patients who have received treatment for up to one year.

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• Phase 1b study includes Parkinson’s disease patients with and without a genetic LRRK2 mutation

SOUTH SAN FRANCISCO, Calif., Dec. 10, 2018 (GLOBE NEWSWIRE) — Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates for neurodegenerative diseases, today announced initiation of dosing in a Phase 1b clinical study of DNL201 in patients with Parkinson’s disease.

“Based on the positive outcome of our Phase 1 study in 122 healthy volunteer subjects, we are excited to evaluate DNL201 in Parkinson’s disease patients,” said Carole Ho, M.D., Chief Medical Officer of Denali. “This study will provide additional important safety and biomarker data in patients to support rational dose selection. The results from this study will inform decisions on further clinical testing of DNL201, including potential registrational trials.”

DNL201, Denali’s lead LRRK2 therapeutic candidate, is a small molecule inhibitor of leucine-rich repeat kinase 2 (LRRK2). LRRK2 is a regulator of lysosomal function, which is impaired in Parkinson’s disease and may be restored by LRRK2 inhibition. Inhibition of LRRK2 activity may potentially slow the progression of disease in patients with a genetic LRRK2 mutation as well as in patients with sporadic Parkinson’s disease.

Mutations in the LRRK2 gene are among the most frequent genetic causes of Parkinson’s disease and a major driver of lysosomal dysfunction, which contributes to the formation of Lewy body protein aggregates and neurodegeneration.

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SpaceX Falcon 9 Delivering ARCUS Gene Editing to the International Space Station

CAPE CANAVERAL, Fla.–(BUSINESS WIRE)–Precision BioSciences today announced that the world’s first genome editing experiment in space has been launched aboard a SpaceX Dragon cargo spacecraft on a Falcon 9 rocket. The mission was launched yesterday from Cape Canaveral Air Force Station and is anticipated to deliver its cargo to the International Space Station (ISS). Astronauts aboard the ISS are expected to perform the first genome editing experiment in space using Precision’s proprietary, next-generation genome editing technology, ARCUS.

Precisioneers JoAnn Hux and Ginger Tomberlin have been working with Karen Kingera, STEM Director of Immaculata Middle School in Durham, North Carolina, and her dedicated group of 15 students for over a year on this project. The program was made possible by Space Center University at Space Center Houston and the DreamUp program. DreamUp places select student projects on SpaceX ISS payloads, and students whose projects are chosen are also invited to attend the launch and participate in presentations and poster sessions at Cape Canaveral.

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Unum Therapeutics Inc. (NASDAQ: UMRX), announced today that the United States Patent and Trademark Office has issued US patent 10,144,770, entitled “Chimeric Receptors and Uses Thereof in Immune Therapy.” The ‘770 patent covers design and use of the chimeric Antibody-Coupled T-cell Receptor (ACTR) platform technology that enables an engineered immune cell to be targeted via an antibody to attack certain cell types, including cancer cells. An ACTR-expressing T cell offers a number of potential advantages over alternative cell therapy approaches and clinical proof-of-concept for the ACTR technology has been demonstrated in two independent clinical trials sponsored by Unum Therapeutics. ACTR therapeutic programs targeting non-Hodgkin lymphoma, multiple myeloma, and HER2+ advanced cancers are currently in early stages of clinical testing.

The initial ACTR construct was developed by Dr. Dario Campana, a well-renowned cancer researcher recognized as an inventor of the chimeric antigen receptor that forms the basis for tisagenlecleucel, the world’s first approved CAR-T therapy. Working initially at St. Jude Children’s Research Hospital and later at the National University of Singapore, Dr. Campana and his team were able to demonstrate in mouse studies that ACTR-expressing T cells exhibit potent anti-tumor activity. Researchers at Unum Therapeutics extended upon this work, demonstrating that a wide range of functional ACTR receptors could be constructed using different building blocks derived from various human immune cell receptors.

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First demonstration of potential gene therapy treatment in this population leading to sustained levels of functioning neutrophils after 12 months

BOSTON and LONDON, Dec. 05, 2018 (GLOBE NEWSWIRE) — Orchard Therapeutics (NASDAQ:ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced that Scientific Advisory Board Member, Donald B. Kohn, MD, presented clinical proof-of-concept data from an ongoing academic clinical trial evaluating OTL-102 for the treatment of X-Linked Chronic Granulomatous Disease (X-CGD) during the Presidential Symposium at the 2018 American Society of Hematology (ASH) Annual Meeting & Exposition on December 4, 2018. X-CGD is a life-threatening inherited immunodeficiency disorder which is caused by a genetic mutation that results in the inability of neutrophils to effectively kill bacterial and fungal pathogens. This immune deficiency leads to repeated chronic and severe infections often requiring hospitalization and resulting in chronic sequelae leading to early mortality. Preclinical and clinical development of OTL-102 had originally been initiated by Genethon (Evry, France) before licensing to Orchard.

“These clinical proof-of-concept data demonstrate, for the first time, 12 months or more of restoration of immunity in X-CGD patients treated with ex-vivo autologous gene therapy,” said Adrian Thrasher, professor of pediatric immunology and Wellcome Trust Principal Research Fellow at UCL Great Ormond Street Institute of Child Health in London. “In six of the seven evaluated patients, we are encouraged to see sustained levels of functioning neutrophils at greater than 10%, which prior publicly available data suggests is the level sufficient to see potential clinical benefit. This important milestone, together with improved clinical outcomes and an emerging safety profile with no signs of genotoxicity, suggest OTL-102 may provide a transformative treatment for X-CGD patients.”

SUZHOU, China, Dec. 4, 2018 /PRNewswire/ — Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class China-based biopharmaceutical company that develops and commercializes high quality drugs, announced today that its IND application for IBI101, a recombinant fully human anti-OX40 monoclonal antibody (mAb) drug candidate, has been approved by the US Food and Drug Administration (FDA), and plans to initiate the US phase I clinical trial based on results from the China phase I study in patients with advanced solid tumors.

IBI101 is the third molecule from Innovent approved for clinical trials by FDA. The company also has received FDA IND approvals for IBI308 (Sintilimab, an anti-PD-1 antibody) in January, 2018 and IBI188 (an anti-CD47 antibody) in September, 2018 respectively. Innovent is the first Chinese biopharmaceutical company to receive clinical trial approval from FDA for an anti-OX40 monoclonal antibody.

“Innovent was built according to international R&D and production standards and has been exploring the latest cutting-edge research in the area of biopharmaceutical development. The FDA IND approval of anti-OX40 monoclonal antibody once again demonstrates Innovent’s research and development capability. We hope that through our efforts, we can make more breakthroughs in the field of innovative biopharmaceuticals and benefit patients around the world,” said Michael Yu, Founder, Chief Executive Officer and Chairman.

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NEW YORK & OTTAWA, Ontario–(BUSINESS WIRE)–Turnstone Biologics, a clinical-stage immuno-oncology company leading the next generation of oncolytic viral therapies, presented pre-clinical data today supporting the development of a new Vaccinia therapeutic platform at the American Association for Cancer Research Tumor Immunology and Immunotherapy Meeting in Miami. This platform was developed by Dr. John Bell and his colleagues at The Ottawa Hospital Research Institute and the University of Ottawa, and engineered to be potent, highly selective and immune-stimulatory. Furthermore, the versatility of the platform enables additional therapeutic agents to be encoded in the virus and produced at tumor sites. This single platform virus has the potential to create a portfolio of diverse products. Turnstone has exclusively licensed this technology.

This proprietary Vaccinia virus can be delivered systemically and is designed to target and kill cancer cells throughout the body, modulate the tumor microenvironment and stimulate both innate and adaptive immune responses. The virus also contains a very large transgene capacity, allowing for the insertion of multiple therapeutic agents. These agents will be produced selectively at tumor sites as the virus replicates only in the tumor cells, effectively enabling a local combination therapy from a single therapeutic promoting cancer cell killing. Turnstone is conducting IND-enabling studies on TBio-6517, its lead product candidate from this platform, consisting of the Vaccinia virus expressing three potent immune modulators, and anticipates initiating clinical studies in 2019.

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