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Deciphera Pharmaceuticals Announces Submission of New Drug Application to U.S. FDA for Ripretinib in Patients with Advanced Gastrointestinal Stromal Tumors

On December 16, 2019 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH) reported the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for ripretinib, the Company’s investigational broad-spectrum KIT and PDGFRα inhibitor, for the treatment of patients with advanced GIST who have received prior treatment with imatinib, sunitinib and regorafenib (Press release, Deciphera Pharmaceuticals, DEC 16, 2019, View Source [SID1234552382]).

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"The NDA submission for ripretinib marks an exciting milestone as we work towards delivering a potential new treatment option for people with advanced GIST," said Steve Hoerter, President and Chief Executive Officer of Deciphera. "We look forward to working with the FDA through their review of our application, and we remain focused on preparing for the potential launch of ripretinib in the United States, if approved."

The NDA is being reviewed by the FDA under the Oncology Center of Excellence Real-Time Oncology Review, or RTOR, pilot program. The pilot program aims to explore a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible, while maintaining and improving review quality. Additional information about RTOR can be found at: View Source

In October 2019, FDA granted Breakthrough Therapy Designation (BTD) for ripretinib for the treatment of patients with advanced GIST who have received prior treatment with imatinib, sunitinib and regorafenib. BTD is designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy.

The NDA submission is supported by positive data from the Company’s INVICTUS pivotal Phase 3 study of ripretinib in advanced GIST. INVICTUS is a randomized (2:1), double-blind, placebo-controlled, international, multicenter study designed to evaluate the efficacy and safety of ripretinib compared to placebo in 129 patients with advanced GIST whose previous therapies have included at least imatinib, sunitinib, and regorafenib. As previously reported, the study achieved the primary endpoint of improved progression free survival compared to placebo in patients with fourth-line and fourth-line plus GIST, as determined by blinded independent central radiologic review using modified Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1.

About Gastrointestinal Stromal Tumor

Gastrointestinal stromal tumor (GIST) is a cancer affecting the digestive tract or nearby structures within the abdomen, most often presenting in the stomach or small intestine. GIST is the most common sarcoma of the gastrointestinal tract, with approximately 4,000 to 6,000 new GIST cases each year in the United States and a similar incidence rate in European and other countries. Most cases of GIST are driven by a spectrum of mutations. The most common primary mutations are in KIT kinase, representing approximately 75% to 80% of cases, and PDGFRα kinase, representing approximately 5% to 10% of cases. Current therapies are unable to inhibit the full spectrum of primary and secondary mutations, which drives resistance and disease progression. Estimates for 5-year survival range from 48% to 90%, depending on the stage of the disease at diagnosis.

About Ripretinib

Ripretinib is an investigational tyrosine kinase switch control inhibitor that was engineered to broadly inhibit KIT and PDGFRα mutated kinases by using a unique dual mechanism of action that regulates the kinase switch pocket and activation loop. Ripretinib is currently in clinical development for the treatment of KIT and/or PDGFRα-driven cancers, including gastrointestinal stromal tumors, or GIST, systemic mastocytosis, or SM, and other cancers. Ripretinib inhibits initiating and secondary KIT mutations in exons 9, 11, 13, 14, 17, and 18, involved in GIST, as well as the primary exon 17 D816V mutation involved in SM. Ripretinib also inhibits primary PDGFRα mutations in exons 12, 14 and 18, including the exon 18 D842V mutation, involved in a subset of GIST. Ripretinib has been granted Fast Track Designation and Breakthrough Therapy Designation by the U.S. FDA for the treatment of patients with advanced GIST who have received prior treatment with imatinib, sunitinib and regorafenib. Ripretinib has also been granted Orphan Drug Designation for the treatment of GIST by the U.S. FDA and European Medicines Agency (EMA). For more information about the Company’s clinical trials with ripretinib, please visit www.clinicaltrials.gov.

Deciphera Pharmaceuticals has an exclusive license agreement with Zai Lab (Shanghai) Co., Ltd. for the development and commercialization of ripretinib in Greater China (MainlandChina, Hong Kong, Macau and Taiwan). Deciphera Pharmaceuticals retains development and commercial rights for ripretinib in the rest of the world.

Salarius Pharmaceuticals Receives FDA Fast Track Designation for Lead Drug Candidate, Seclidemstat, in Relapsed or Refractory Ewing Sarcoma

On December 16, 2019 Salarius Pharmaceuticals, Inc. (Nasdaq: SLRX), a clinical-stage biotechnology company targeting cancers caused by mis-regulated gene expression, reported that its lead investigational drug candidate, Seclidemstat, has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with Ewing sarcoma who have relapsed or are refractory to standard-of-care therapy (Press release, Flex Pharma, DEC 16, 2019, View Source [SID1234552381]). Seclidemstat, a potent reversible LSD1 inhibitor, is the subject of an ongoing Phase 1/2 clinical study in Ewing sarcoma.

David Arthur, President and Chief Executive Officer of Salarius, stated, "Securing FDA Fast Track Designation for Seclidemstat in Ewing sarcoma is an achievement for Salarius in the ongoing development of the drug and recognition that there is an unmet need to bring much needed hope to patients and their families suffering through this devastating disease. Coupled with Seclidemstat’s previously granted Orphan Drug Designation and Rare Pediatric Disease Designation by the U.S. Food and Drug Administration, we feel well positioned to take advantage of the FDA’s expedited programs for drug development and review."

"Ewing sarcoma is a rare and deadly bone cancer that most often strikes children and young adults and for which there are no targeted therapies approved. Seclidemstat has demonstrated a potential to address this considerable unmet need, and we look forward to rapidly advancing its development so that it soon may be available to those patients most in need," stated Damon Reed, M.D., Director of the Adolescent and Young Adult Program at the Moffitt Cancer Center and Principle Investigator of the Salarius Ewing sarcoma clinical trial.

Fast Track is a process designed by the FDA to expedite the development and review of new drugs with the potential to treat serious or life-threatening conditions and fill unmet medical needs. The aim is to streamline regulatory submissions and enable more frequent communications with the agency to assure that questions and issues are resolved quickly, which often leads to earlier drug approval and access by patients.

About Seclidemstat
Seclidemstat is a first-in-class, oral, small molecule designed for the reversible and noncompetitive inhibition of the LSD1 enzyme. Seclidemstat is based on the research of Dr. Sunil Sharma, Salarius’ co-founder, into LDS1 inhibition during his tenure at the University of Utah’s Huntsman Cancer Institute. As a reversible inhibitor, Seclidemstat could offer more efficacy, more flexible dosing and less toxicity. Salarius expects to release early cohort data early next year from its Ewing sarcoma study and a second Phase 1 clinical study in advanced solid tumors, including prostate, breast and ovarian cancers. A preclinical program in glioblastoma is underway at the Barrow Neurological Institute ‘s Ivy Brain Tumor Center.

More information about Salarius’ ongoing Ewing sarcoma trial is available at ClinicalTrials.gov and on the company website, salariuspharma.com. Active clinical trial sites include, Memorial Sloan Kettering Cancer Center in New York City, Nationwide Children’s Hospital in Columbus, OH, Johns Hopkins All Children’s Hospital in St. Petersburg, FL; Children’s Hospital of Los Angeles in Los Angeles, CA; Moffitt Cancer Center in Tampa, FL; Dana-Farber Cancer Institute in Boston, MA; MD Anderson Cancer Center in Houston, TX; and the Sarcoma Oncology Center in Santa Monica, CA.

Astellas Commences Tender Offer to Acquire All Outstanding Shares of Audentes

On December 16, 2019 Astellas Pharma Inc. (TSE:4503, Headquarters: Tokyo, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") reported that it has commenced, through its indirect, wholly-owned subsidiary Asilomar Acquisition Corp. ("Asilomar"), a tender offer ("Tender Offer") for all of the issued and outstanding shares of common stock of Audentes Therapeutics, Inc. (NASDAQ: BOLD, Headquarters: San Francisco, Chairman and CEO: Matthew R. Patterson, "Audentes") for a price of US$60.00 per share, net to the seller in cash, on December 16, 2019, New York City time (Press release, Astellas, DEC 16, 2019, View Source [SID1234552380]). The Tender Offer is scheduled to expire at 12:00 midnight, New York City time, at the end of the day on January 14, 2020, unless extended or earlier terminated.

The Tender Offer was commenced pursuant to the definitive agreement dated December 2, 2019, by and among Astellas, Asilomar and Audentes, and promptly upon successful completion of the Tender Offer, Asilomar will be merged into Audentes, with Audentes surviving the merger as a subsidiary of Astellas.

Start of phase II clinical trial in four types of terminal cancers

On December 15, 2019 N. N. The Alexandrov National Cancer Centre reported that begins phase II case-control clinical trial of its immuno-oncology agent, Elenagen (Press release, N N The Alexandrov National Cancer Centre, DEC 15, 2019, View Source [SID1234553262]). Inoperable late-stage cancer patients with triple-negative breast cancer, ovarian cancer, stomach cancer or prostate cancer will be randomized and then subsequently receive either standard chemotherapy or chemotherapy combined with Elenagen. For each disease, the experimental and control groups will be compared for time to disease progression, overall survival, as well as tumor regression and the dynamics of clinical conditions. One can also expect a reversal of a tumor grade to an operable stage, which would make the tumor surgically removable.

"We have conducted a comprehensive worldwide search for the most promising adjuvants to standards of care and selected Elenagen", said prof. Oleg Sukonko MD, Ph.D., D.Sc., Head of the Alexandrov National Cancer Centre, "We assessed hundreds of potential products according to the following criteria: high safety as demonstrated in phase I/IIa clinical trial, encouraging first signs of synergistic effects with chemo-, radio- and immunotherapies, the economic feasibility of the future product and its accessibility for the healthcare system, and scientific novelty. Elenagen satisfied all of these criteria".

Elenagen was developed by, CureLab Oncology, Inc., a Boston-based biotech startup which provided the product to Belarussian colleagues. Aldevron, Inc. (Fargo, ND) was the contract manufacturer for the product. "Although in the coming year we are planning to start phase II clinical trials of Elenagen in breast and ovarian cancers in the US and multiple other countries, it was very important for us to begin with Belarus, the country which was the most hit by Chernobyl", said Dr. Alex Shneider, CEO of CureLab Oncology.

"We are very thankful to our colleagues from NYU and MD Andersen Cancer Centers who donated their ideas and expertise, went with us through multiple iterations of the clinical protocol, and helped to create the final version which we will now implement in our studies. Without their generous assistance and continuous expert support, we would not be where we are now", said prof. Sergey Krasny MD, Ph.D., D.Sc., deputy director of the Alexandrov National Cancer Centre and corresponding member of the Belarus National Academy of Science. The study is unanimously approved by the Belarus Ministry of Health. The team of clinical scientists conducting the trial in Belarus will maintain constant contact and consistently exchange information with their colleagues in the US, EU, and Israel serving as observers and scientific co-authors to the study.

Ally Bridge Group Leads More Than US$140 million in Financings for Three New Medtech Investments

On December 15, 2019 Ally Bridge Group ("ABG"), a leading global life science investment group, reported that it has announced that it recently led investments in three new medical technology companies (Press release, Ally Bridge Group, DEC 15, 2019, View Source [SID1234552379]). Each of the three companies is a commercial-stage, leading innovator re-setting the standard of care in its respective category. They include:

Imperative Care (Campbell, CA) — A company that is singularly dedicated to improving the way stroke is treated by advancing medical technology, equipping healthcare providers to evolve best practices and elevate the standard of care for stroke patients. Imperative Care has received FDA approval for its first families of access and aspiration catheters. Website: View Source
Venclose Medical (Santa Clara, CA) — A commercial-stage company developing next-generation solutions for the treatment of venous reflux disease, also known as chronic venous insufficiency. Venclose has been commercializing in the US and Europe since 2018. Website: View Source
Mavrik Dental Systems (Tel Aviv, Israel) — A company with proprietary technologies and products for in-office teeth whitening with unparalleled efficiency and outcomes, and gum care. Mavrik represents ABG’s first investment in an Israel-based company. Website: View Source
"Medtech, as with biotech, remains a must-invest sector for ABG, where we focus on best-in-class technologies to disrupt current standards of care," said Frank Yu, CEO and CIO of Ally Bridge Group. "The closing of these investments brings the aggregate amount of ABG-led private life science investments to above $500 mm over the last 18 months, which include ABG’s lead role in investments in Grail (early cancer detection), Pulmonx (interventional pulmonology), Quantum Surgical (interventional oncology). All of these companies, with the exceptions of one in Israel and one in France, are based in Silicon Valley, California, USA."

"Our active investments that leverage powerful synergies across private and public markets, coupled with our M&A knowhow and cross-border value-add, position ABG uniquely as a leading investor in late-stage best-in-class life science technologies", adds Yu.