Allogene: FDA Clears Application for BCMA Allogeneic CAR T Therapy

• ALLO-715 Utilizes Gene-Editing of TRAC and CD52 Loci to Enable Allogeneic CAR T Therapy
• ALLO-715 will be Evaluated in Combination with ALLO-647, Allogene’s Proprietary anti-CD52 Antibody as Part of the Lymphodepletion Regimen 
• Allogene Plans to Initiate the UNIVERSAL Study for ALLO-715 in Relapsed/Refractory Multiple Myeloma in the Second Half of 2019

Allogene Therapeutics, Inc. (ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T™) therapies for cancer, today announced that the U.S. Food & Drug Administration (FDA) has cleared Allogene’s Investigational New Drug (IND) application for ALLO-715 in patients with relapsed/refractory multiple myeloma. The Phase 1 portion of the UNIVERSAL study, which will include ALLO-647 as part of the lymphodepletion regimen, is expected to be initiated in the second half of 2019.

https://finance.yahoo.com/news/allogene-therapeutics-announces-fda-clearance-114500176.html

Celgene, Acceleron: FDA Accepts Luspatercept License Application

U.S. Food and Drug Administration grants priority review for beta-thalassemia indication and sets target action date of December 4, 2019

U.S. Food and Drug Administration sets target action date of April 4, 2020 for myelodysplastic syndromes indication

Luspatercept EU Marketing Authorization Application also validated

Celgene Corporation (CELG) and Acceleron Pharma Inc. (XLRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted Celgene’s Biologics License Application (BLA) for luspatercept, an investigational erythroid maturation agent, for the treatment of adult patients with very low to intermediate-risk myelodysplastic syndromes (MDS)-associated anemia who have ring sideroblasts and require red blood cell (RBC) transfusions, and for the treatment of adult patients with beta-thalassemia-associated anemia who require RBC transfusions. The FDA has granted Priority Review to this BLA for the evaluation of the beta-thalassemia indication and set a Prescription Drug User Fee Act (PDUFA), or target action, date of December 4, 2019. The FDA has also set a PDUFA date of April 4, 2020 for the evaluation of the MDS indication.

https://finance.yahoo.com/news/celgene-corporation-acceleron-pharma-announce-113000756.html

VBI Vaccines Expands on Data from Phase 1/2a Glioblastoma Study

• Well tolerated at all doses with no safety signals
• Three patients in high dose (10 µg) cohort showed evidence of stable disease by MRI
• 10 µg dose selected for Part B of the study, which is expected to initiate enrollment mid-year 2019

VBI Vaccines Inc. (NASDAQ: VBIV) (“VBI”), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, presented a poster on Sunday, June 2, 2019, at the American Society of Clinical Oncology (ASCO) Annual Meeting that exhibited expanded clinical data from Part A of the Phase 1/2a study of VBI-1901 in recurrent Glioblastoma (GBM) patients.

The poster presented data on a total of 18 patients enrolled in Part A of the study, which was a multi-center, open-label, dose-escalation study across three dose cohorts of VBI’s vaccine immunotherapeutic, VBI-1901 – 0.4 µg, 2.0 µg, and 10.0 µg.  Part A was designed to evaluate the safety and tolerability of VBI-1901, and to define the optimal immunogenic dose level to test in the Part B extension phase of the study, which is expected to initiate enrollment mid-year 2019.  Part B of the trial will further assess immunologic responses and potential correlations with tumor and clinical responses.

Andrew B. Lassman, M.D., Chief of Neuro-oncology at Columbia University Irving Medical Center and Associate Director for Clinical Infrastructure at Herbert Irving Comprehensive Cancer Center, and principal investigator of the study commented, “Though early, the data we’ve seen to-date in this Phase 1/2a study of VBI-1901 are intriguing, yet of course require confirmation in later phase and additional trials.  The patients in this study, and more generally in the recurrent GBM setting, are immunocompromised and have very few effective treatment options available to them. Any treatment that could demonstrate even some benefit would be incredibly meaningful for these patients and their families.  I look forward to seeing additional data from Part B of the study.”

https://www.globenewswire.com/news-release/2019/06/04/1863878/0/en/VBI-Vaccines-Reports-Data-from-Part-A-of-the-Ongoing-Phase-1-2a-Study-of-VBI-1901-in-Recurrent-Glioblastoma-GBM-Patients-at-ASCO-2019.html

Veru Adds Androgen Deprivation Therapy to Prostate Cancer Pipeline

— VERU-100 Is a Proprietary Peptide Formulation Designed with Multiple Beneficial Clinical Attributes Addressing the Shortfalls of the Current Multi-Billion-Dollar Androgen Deprivation Therapy Market —

— Reached Agreement with FDA on an Expedited Regulatory Pathway and Clinical Development Strategy —

Veru Inc. (NASDAQ: VERU), an oncology and urology biopharmaceutical company developing novel medicines for prostate cancer treatment and prostate cancer supportive care, today announced that it has added to its robust prostate cancer drug development pipeline, a proprietary biologic drug candidate, VERU-100, for the treatment of hormone sensitive advanced prostate cancer, an established multi-billion-dollar global market.  VERU-100 was internally developed in collaboration with Drug Delivery Experts, LLC of San Diego, California (DDE Labs).

“The target product profile for VERU-100 is most compelling having a number of advantages over currently available androgen deprivation therapies. VERU-100 is a long-acting gonadotropin-releasing hormone (GnRH) antagonist designed to be administered as a small volume subcutaneous 3-month depot injection without a loading dose. As a GnRH antagonist, it will immediately suppress testosterone with no testosterone surge upon initial or repeated administration and no testosterone micro-increases which may adversely affect patient outcomes — a problem which potentially occurs with approved LHRH agonist drugs like LUPRON®, ZOLADEX® and ELIGARD®,” said Peter N. Schlegel, M.D., James J. Colt Professor and Chairman of the Department of Urology at Weill Cornell Medicine and Urologist-in-Chief at New York-Presbyterian / Weill Cornell Medical Center.  “Currently, there are no GnRH antagonists commercially approved beyond 1 month, making VERU-100, if approved, the only commercially available GnRH antagonist 3-month depot — an attractive choice for androgen deprivation therapy.”

The Company recently met with the FDA and received agreement that VERU-100 qualifies for an expedited regulatory pathway. Based on FDA input, the Company plans to commence a single open label, multicenter dose-finding Phase 2 clinical trial in approximately 50 men, followed by a single open label, multicenter Phase 3 clinical trial in approximately 100 men. Veru is in the process of scaling up GMP manufacturing of drug product to prepare for the clinical trials of VERU-100. The Company plans to submit an Investigational New Drug application by no later than calendar Q1 2020.

Based on current cash on hand and expected cash from current sales forecasts, along with existing sources of capital, the Company does not anticipate the need for a new equity financing until at least fiscal 2021, even with additional costs related to the VERU-100 clinical development.

https://www.globenewswire.com/news-release/2019/06/04/1863861/0/en/Veru-Adds-Breakthrough-Novel-Androgen-Deprivation-Therapy-Formulation-to-its-Advancing-Prostate-Cancer-Drug-Pipeline-Following-Successful-Meeting-with-FDA.html

Foamix Announces Publication of Phase 3 FMX101 Acne Study

Foamix Pharmaceuticals Ltd. (FOMX), a clinical-stage pharmaceutical company that specializes in developing and commercializing proprietary topical therapies for dermatological conditions, announced today the peer review publication of the Phase 3 study FX2017-22 (“Study 22”) in the Journal of the American Academy of Dermatology(JAAD).  Study 22 was conducted by Foamix to support the New Drug Application (NDA) submission of FMX101, which is currently under review by the FDA for the treatment of inflammatory lesions of non-nodular moderate-to-severe acne vulgaris in patients nine years of age and older.

• The study showed high statistically significant superiority of FMX101 compared with vehicle in both primary endpoints of absolute inflammatory lesion reduction and IGA treatment success at week 12.
• FMX101 also met all secondary endpoints, showing statistically significant improvement in absolute reductions in both inflammatory lesions (papules, pustules and nodules) and non-inflammatory lesions (open and closed comedones).
• There was a statistically significant reduction in inflammatory lesions versus vehicle as early as week 3 of treatment, and all subsequently assessed timepoints throughout the entire treatment course of the study.
• Statistically, participants were no more likely to experience treatment-emergent adverse events (TEAEs) from FMX101 than from vehicle treatment. The majority of TEAEs were mild to moderate.
• More than 95% of participants using FMX101 had no or mild skin tolerability scores at the treatment application site at week 12.

https://finance.yahoo.com/news/foamix-announces-publication-phase-3-120000773.html

Soliton studies acoustic cellulite treatment

RAP technology combines selective disruption of fibrotic structures with production of new collagen in single non-invasive procedure

Soliton, Inc., (SOLY) (“Soliton” or the “Company”), a medical device company with a novel and proprietary platform technology licensed from The University of Texas on behalf of the MD Anderson Cancer Center (“MD Anderson”), today announced results of preclinical studies of the use of a new version of its acoustic pulse technology which reveal its RAP device appears to be capable of selective disruption of the fibrotic septa that contribute to the appearance of cellulite.  The new discovery, referred to as “acoustic subcision,” helps explain the recent proof-of-concept trial results showing an improvement in the appearance of cellulite following use of the Company’s RAP device.  Together with the device’s demonstrated ability to stimulate new collagen production in animal models, this represents what the Company believes is a potentially important new way to treat cellulite and improve the appearance of the skin.  The RAP device for the treatment of cellulite is investigational and not available for sale in the United States.

“While the proof-of-concept clinical trial results we have recently announced regarding the improvement in the appearance of cellulite have been very encouraging,” commented Dr. Chris Capelli, President and CEO of Soliton, “Understanding the basic science and biology behind these results is very important.  We believe the discovery we are outlining here helps explain the promising results we saw from this trial, as well as setting the foundation for a platform technology that may have potential in a number of aesthetic indications.”

https://finance.yahoo.com/news/soliton-announces-significant-discovery-acoustic-120000462.html

AstraZeneca searches for successor to Chairman Johansson

British drugmaker AstraZeneca Plc is preparing to search for a successor to Chairman Leif Johansson, Sky News reported on Monday.

AstraZeneca’s board is in the early stages of planning for an eventual replacement for Johansson, who has chaired the company for seven years, according to the report.

There was no scheduled timetable in place yet for Johansson’s departure, Sky News reported, citing sources.

https://www.streetinsider.com/General+News/AstraZeneca+searches+for+successor+to+Chairman+Johansson%3A+Sky+News/15571636.html