On August 7, 2019 Transgene (Paris:TNG), a biotech company designing and developing virus-based immunotherapies for the treatment of solid tumors, reported an update on the interim futility analysis of the PHOCUS study of Pexa-Vec in liver cancer (Press release, Transgene, AUG 7, 2019, View Source [SID1234538332]). The independent Data Monitoring Committee (IDMC) of the PHOCUS trial has recommended to stop the study (see press release distributed on August 2, 2019). Transgene is currently analyzing the data of the trial it received from its partner SillaJen, notably in the context of the ongoing Phase 2 clinical trial evaluating the combination regimen of Pexa-Vec and the immunotherapy nivolumab in the same indication. The recommendation to stop the PHOCUS trial is not caused by safety issues of Pexa-Vec.

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Transgene is convinced of the great potential of oncolytic viruses (OV) as this therapeutic class displays numerous advantages that are acknowledged by the scientific and medical community. These include the ability of the viruses to infect and selectively replicate within the tumor, inducing cancer cell destruction, and to elicit a strong immune response against the tumor.

Transgene novel proprietary OV platform Invir.IO allows the arming of these viruses to trigger the expression of anticancer weapons directly in the tumor, thus increasing the efficacy of these molecules while reducing their possible side effects.

The oncolytic viruses derived from the Invir.IO platform have been designed using an optimized strain of Vaccinia Copenhagen. These carry a double deletion TK-RR-, which makes their replication more selective than viruses carrying a simple TK deletion such as Pexa-Vec. In addition, the Invir.IO platform is designed to incorporate several transgenes encoding for a range of specific anticancer weapons which involve well-established immunotherapy mechanisms such as anti-CTLA-4 antibodies.

The collaboration with AstraZeneca was formed on the merits of this platform. As described in the press release distributed on May 2, 2019, Transgene is currently working to design five new oncolytic viruses for AstraZeneca.

Philippe Archinard, PhD, Chairman and CEO of Transgene, commented: "We are obviously disappointed with the outcome of the PHOCUS study; however we remain convinced in the potential of our oncolytic virus pipeline. Our recent collaborative agreement with AstraZeneca highlights the industry interest in the multi-armed OVs that we can generate using our unique Invir.IO platform. We also expect to announce the first clinical data with TG6002 in patients with colorectal cancer later this year. In addition, we expect to announce important clinical results from our most advanced therapeutic vaccines; TG4010 in lung cancer and TG4001 in HPV-Positive head and neck cancer. With funding through to 2022, a clear strategy and novel technology platforms such as Invir.IO and myvac, Transgene is well placed to demonstrate the potential of its novel medicines designed to improve the treatment of solid tumors."

A conference call in English is scheduled on August 7, 2019, at 6:30 p.m. CET (12:30 pm EST).

Webcast link to English language conference call:
https://channel.royalcast.com/webcast/transgene/20190805_1/

Participant telephone numbers:

France: +33 (0) 1 7037 7166

United Kingdom: +44 20 3003 2666

United States: +1 202 204 1514

Confirmation code: Transgene

A replay of the call will be available on the Transgene website (www.transgene.fr) following the live event.

-Ends-

About the PHOCUS trial
The PHOCUS trial was a Phase 3 clinical trial evaluating the oncolytic immunotherapy Pexa-Vec for advanced liver cancer patients who have not received prior systemic treatment for their cancer. The study was conducted by Transgene’s partner, SillaJen.
In the PHOCUS study, patients were randomized to one of two treatment groups: one receiving Pexa-Vec followed by sorafenib and one receiving sorafenib alone. The primary objective of the study was to determine the overall survival of patients treated with Pexa-Vec, followed by sorafenib versus sorafenib alone. Secondary objectives included safety as well as assessments for tumor responses between the two groups as measured by the following endpoints: time to progression, progression-free survival, overall response rate and disease control rate.

About Pexa-Vec
Pexa-Vec (formerly JX-594/TG6006 – pexastimogene devacirepvec) is an oncolytic immunotherapeutic based on an oncolytic vaccinia virus armed with a GM-CSF gene that promotes an anti-tumor immune response. Pexa-Vec is designed to selectively target and destroy cancer cells through three different mechanisms of action: selectively destroy cancer cells through the direct lysis (breakdown) of cancer cells through viral replication, reduce the blood supply to tumors through vascular disruption, and stimulate the body’s immune response against cancer cells.
In a Phase 2 study, results of patients with advanced liver cancer showed that patients receiving the high dose had a statistically significant clinical improvement in terms of overall survival compared to the group receiving the low dose. Median overall survival was respectively 14.1 months in the high-dose group and 6.7 months in the low-dose group, which compares favorably with current approved treatments. (Heo J. et al., Nature Medicine, March 2013, doi: 10.1038/nm.3089)
Transgene has exclusive rights to develop and commercialize Pexa-Vec for the treatment of solid tumors in Europe. Its partner SillaJen, Inc. is focused on developing Pexa-Vec for the North American market and has also granted exclusive development and commercial rights to Pexa-Vec in Hong Kong and The People’s Republic of China to Lee’s Pharmaceutical.

On August 7, 2019 Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (Prometic), a biopharmaceutical company focused on developing novel therapeutics to treat unmet needs in patients with liver, respiratory and kidney disease, reported that it will report its financial results for the second quarter ended June 30th, 2019 on Monday August 12th, 2019 after market close (Press release, ProMetic Life Sciences, AUG 7, 2019, View Source [SID1234538331]).

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Prometic will host a conference call at 11:00 a.m. (EDT) on Tuesday August 13th, 2019. The
telephone numbers to access the conference call are 1-888-231-8191 and 647-427-7450.
An audio replay of the call will be available as of Tuesday August 13th 2019 at 2:00 p.m. (EDT).
The numbers to access the audio replay are 416-849-0833 and 1-855-859-2056 using
the following password: 3058704.

A live audio webcast of the conference call will be available via:

View Source

On August 7, 2019 TRACON Pharmaceuticals (Nasdaq:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer, wet age-related macular degeneration through our license to Santen Pharmaceutical Co. Ltd., and utilizing our product development platform to partner with ex-U.S. companies to develop and commercialize innovative products in the U.S., reported that Charles Theuer, M.D., Ph.D., President and CEO, will participate in the 2019 BTIG Biotechnology Conference on Monday, August 12, 2019 in New York (Press release, Tracon Pharmaceuticals, AUG 7, 2019, View Source [SID1234538330]).

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On August 7, 2019 Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, reported that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for avapritinib for the treatment of adult patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST (Press release, Blueprint Medicines, AUG 7, 2019, View Source [SID1234538329]). The FDA granted Priority Review and set an action date of February 14, 2020 under the Prescription Drug User Fee Act (PDUFA). At this time, the FDA is not planning to hold an advisory committee meeting to discuss this application. Avapritinib, an investigational therapy, is a potent and highly selective KIT and PDGFRA inhibitor for patients with advanced GIST.

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"Patients with PDGFRA Exon 18 mutant GIST and fourth-line GIST are in need of new treatment options that address the underlying drivers of the disease," said Andy Boral, M.D., Ph.D., Chief Medical Officer at Blueprint Medicines. "The FDA’s acceptance of our application for Priority Review brings us closer to our goal of delivering avapritinib to patients with GIST, and we look forward to working closely with the FDA during the review process."

The FDA’s acceptance of the NDA indicates the application is sufficiently complete to permit a substantive review. A Priority Review designation accelerates the FDA’s review time from 12 months to a goal of eight months from the NDA submission date, and is granted to drugs that may offer a significant improvement in the safety or effectiveness of the treatment, prevention or diagnosis of a serious condition. Previously, the FDA granted avapritinib Breakthrough Therapy Designation for the treatment of patients with unresectable or metastatic GIST harboring the PDGFRα D842V mutation.

In July 2019, the European Medicines Agency validated Blueprint Medicines’ Marketing Authorization Application for avapritinib in adult patients with PDGFRα D842V mutant GIST, regardless of prior therapy, and fourth-line GIST.

About GIST
GIST is a sarcoma, or tumor of bone or connective tissue, of the gastrointestinal (GI) tract. Tumors arise from cells in the wall of the GI tract and occur most often in the stomach or small intestine. Most patients are diagnosed between the ages of 50 to 80, and diagnosis is typically triggered by GI bleeding, incidental findings during surgery or imaging and, in rare cases, tumor rupture or GI obstruction.

Most GIST cases are caused by a spectrum of clinically relevant mutations that force the KIT or PDGFRA protein kinases into an increasingly active state. Because currently available therapies primarily bind to the inactive protein conformations, certain primary and secondary mutations typically lead to treatment resistance and disease progression.

In unresectable or metastatic GIST, clinical benefits from existing treatments can vary by mutation type. Mutational testing is critical to tailor therapy to the underlying disease driver and is recommended in expert guidelines. Currently, there are no approved therapies for patients with KIT-driven GIST whose disease progresses beyond imatinib, sunitinib and regorafenib. In patients with metastatic PDGFRα D842V-driven GIST, progression occurs in a median of approximately three to four months with available therapy.

About Avapritinib
Avapritinib is an investigational, oral precision therapy that selectively and potently inhibits KIT and PDGFRA mutant kinases. It is a type 1 inhibitor designed to target the active kinase conformation; all oncogenic kinases signal via this conformation. Avapritinib has demonstrated broad inhibition of KIT and PDGFRA mutations associated with GIST, including potent activity against activation loop mutations that are associated with resistance to currently approved therapies.

Blueprint Medicines is initially developing avapritinib for the treatment of advanced GIST, advanced systemic mastocytosis (SM), and indolent and smoldering SM. The FDA has granted Breakthrough Therapy Designation to avapritinib for two indications: one for the treatment of unresectable or metastatic GIST harboring the PDGFRα D842V mutation and one for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia.

Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of avapritinib and certain other drug candidates in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for avapritinib in the rest of the world.

On August 7, 2019 Pacira BioSciences, Inc. (NASDAQ: PCRX) reported that it will present at the 2019 Wedbush Pacgrow Healthcare Conference at 12:45 PM ET on Tuesday, August 13, 2019 (Press release, Pacira Pharmaceuticals, AUG 7, 2019, View Source [SID1234538328]). Live audio of the event can be accessed by visiting the "Events" page of the company’s website at investor.pacira.com. A replay of the webcast will also be available for two weeks following the event.

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