Sangamo Therapeutics To Present At The 36th Annual J.P. Morgan Healthcare Conference

On January 4, 2018 Sangamo Therapeutics, Inc. (NASDAQ: SGMO) reported that Dr. Sandy Macrae, CEO of Sangamo, will present at the 36th Annual J.P. Morgan Healthcare Conference on Wednesday, January 10th at 4:00 p.m. PT. Dr. Macrae’s presentation will be followed by a separate Q&A session starting at 4:30 p.m. PT (Press release, Sangamo Therapeutics, JAN 4, 2018, View Source [SID1234522912]). The conference is being held in San Francisco, CA from January 8-11, 2018.

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The presentation and Q&A session will be webcast live and may be accessed via a link on the Sangamo Therapeutics website in the Investors and Media section under Events and Presentations. The presentation will be archived on the Sangamo website for two weeks after the event.

Illumina and KingMed Diagnostics Partner to Develop Next-Generation Sequencing Technology for Chinese FDA Approval

On January 4, 2018 Illumina, Inc. (NASDAQ: ILMN) and KingMed Diagnostics (SSE: 603882.SS) reported an agreement to jointly develop novel oncology and hereditary disease testing applications utilizing Illumina’s next-generation sequencing (NGS) technology (Press release, Illumina, JAN 4, 2018, View Source [SID1234522917]). The collaboration is a significant step toward China Food and Drug Administration (CFDA) review and approval, and serves as a starting point to deliver precision medicine to patients throughout China.

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Under the agreement, Illumina and KingMed Diagnostics will partner to co-develop an integrated NGS system that provides cost-effective and ready-to-use in-vitro diagnostic (IVD) assays for molecular oncology and hereditary cancer testing. The new system is based on Illumina’s MiniSeq System and related sequencing consumables, integrated with KingMed Diagnostics’ proprietary testing components, which include library preparation kits and analysis software.

The integrated system can reach cancer patients across China via KingMed Diagnostics’ extensive clinical network that serves more than 8,000 Class 2 and Class 3B hospitals.

"KingMed Diagnostics’ motivation is to improve diagnosis and treatment for the more than 4 million new cancer patients identified in China each year. Our mission is to bring state-of-the-art technology to Chinese patients by enhancing their standard of care and improving their outcomes," said Professor Yaoming Liang, Chairman and CEO of KingMed Diagnostics. "Illumina is the ideal collaborator because they have a proven track record of working with multiple domestic companies in China, in addition to being the first company with a U.S. FDA-cleared, next-generation sequencing instrument."

"KingMed Diagnostics is one of the leading independent clinical laboratory service providers in China, and as such, we are excited to partner with them to help customize the MiniSeq System under CFDA requirements for clinical oncology applications," said Garret Hampton, Ph.D., Executive Vice President of Clinical Genomics at Illumina.

"This agreement is a clear demonstration of our commitment to working with broader clinical testing service providers in China who want to develop and commercialize IVDs based on NGS," said Ruilin Zhao, Ph.D., Illumina’s Vice President and General Manager of Greater China. "As we continue to expand our clinical partner networks, we are focused on providing the best healthcare solutions to patients throughout China."

pellepharm appoints sanuj k. ravindran, m.d., as president and chief executive officer

On January 4, 2018 PellePharm, a clinical-stage biopharmaceutical company committed to targeting rare genetic dermatological conditions at the source, reported the appointment of Sanuj K. Ravindran, M.D., to the position of president and chief executive officer (Press release, PellePharm, JAN 4, 2018, View Source [SID1234576282]). In parallel, Dr. Ravindran will join BridgeBio Pharma, PellePharm’s lead investor, as CEO-in-Residence, to advance its broader orphan dermatology portfolio.

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"Dr. Ravindran’s biopharma industry experience and rare disease expertise will offer enormous strategic benefit to PellePharm as we move into our next stage of development," said Ervin Epstein, M.D., chief medical officer and co-founder of PellePharm. "Having been successful with the management and growth of multiple biopharma companies, Dr. Ravindran brings the right experience to PellePharm so that we may offer topical patidegib to patients as swiftly as possible."

Dr. Ravindran brings more than 15 years of strategic and operational biopharma experience to PellePharm. Most recently, he was chief business officer at aTyr Pharma ("LIFE"), a clinical stage rare disease-focused biotechnology company, where he led corporate and financial strategy, business development, and investor relations. Prior to that, Dr. Ravindran was senior vice president of corporate development for The Medicines Company ("MDCO"), where he worked to execute multiple transactions totaling more than $2 billion in potential aggregate value. Previously a practicing physician, Dr. Ravindran began his industry career as a venture capitalist for 10 years with Burrill & Company, Radius Ventures, and Asian Healthcare Fund. Dr. Ravindran is trained in Internal Medicine and completed his residency training at Thomas Jefferson University Hospital. Dr. Ravindran received his B.A. from Northwestern University, his M.D. from Jefferson Medical College and his MBA from the Kellogg School of Management.

"I am thrilled to join PellePharm at such an important juncture, as the Company prepares to advance topical patidegib one step closer to patients with Gorlin Syndrome. With PellePharm’s scientific premise, clinical progress, and recently strengthened leadership team, the company is well on its way to meeting its mission of delivering therapies for rare genetic dermatological conditions," said Dr. Ravindran.

PellePharm today also announced that it has expanded its executive team to enhance regulatory and operational capabilities. Alix Alderman is now vice president of regulatory affairs at PellePharm, and Gerd Kochendoerfer, Ph.D., is vice president of technical operations and program management. Both Ms. Alderman and Dr. Kochendoerfer bring many years of experience in drug development, quality management and global regulatory affairs.

"We are pleased to welcome Dr. Ravindran, Dr. Kochendoerfer and Ms. Alderman," said Neil Kumar, CEO and co-founder of BridgeBio Pharma. "At this inflection point, having the right team in place, with the collective experience this group brings, enables PellePharm to more ably achieve its goal of helping patients with serious unmet dermatologic conditions."

About Patidegib

Topical patidegib gel has shown early promise in a Phase 2 clinical study in Gorlin Syndrome by blocking the disease at its source within the hedgehog signaling pathway. Topical patidegib was developed to provide the efficacy previously demonstrated by oral patidegib in Phase 1 trials, but without the adverse systemic side effects of oral hedgehog inhibitors. Patidegib’s gel formulation is stable at room temperature for at least two years, making it a viable potential therapy for ongoing, at-home management of Gorlin Syndrome. Topical patidegib currently is being studied in a United States-based Phase 2 clinical trial for the treatment of sporadic basal cell carcinomas (BCCs).

About Gorlin Syndrome

Gorlin Syndrome is a rare, genetic disease characterized by mutations in the tumor suppressor gene encoding Patched1 (PTCH1), which acts as the primary inhibitor of the hedgehog signaling pathway. This leads to hundreds of basal cell carcinomas, especially on the face and sun-exposed areas.

With no FDA-approved drugs available for Gorlin Syndrome, also known as Basal Cell Carcinoma Nevus Syndrome (BCCNS), the standard of care is surgery. People with severe Gorlin Syndrome may have as many as 30 surgeries per year, which can be repetitive and scarring. Approximately 10,000 people in the United States, or one in 31,000, are believed to be affected by Gorlin Syndrome. Gorlin Syndrome is known by several names, including BCCNS, Gorlin-Goltz Syndrome, Basal Cell Nevus Syndrome, or Nevoid Basal Cell Carcinoma Syndrome.

Teva to Present at the 36th Annual J.P. Morgan Healthcare Conference

On January 4, 2018 Teva Pharmaceutical Industries Ltd. (NYSE: TEVA) reported that it will host a live audio webcast at the 36th Annual J.P. Morgan Healthcare Conference (Press release, Teva, JAN 4, 2018, View Source;p=RssLanding&cat=news&id=2324950 [SID1234522913]). Kåre Schultz, President & CEO will present on Monday, January 8, 2018 at 9:30 AM PST.

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What:

Teva Presentation at the 36th Annual J.P. Morgan Healthcare Conference

Who:

Kåre Schultz, President & CEO
Teva Pharmaceutical Industries Ltd.

When:

Monday, January 8, 2018 at 9:30 AM PST

Where:

www.ir.tevapharm.com

How:

Live over the Internet – log on to the Web at the address above and register for the event (approximately 10 minutes before). An archive of the webcast will be available on Teva’s website.

Rasna Therapeutics, Inc. Announces Follow Up Phase II Clinical Data Confirming Efficacy of Actinomycin D in Patients with NPM1-mutated Acute Myeloid Leukemia

On January 3, 2018 Rasna Therapeutics, Inc. (OTCQX: RASP), a clinical stage biotechnology company focused on the development of disease-modifying drugs for hematological malignancies, reported follow up Phase II clinical data showing that 4 out of 9 (44%) evaluable AML patients carrying the NPM1 gene mutation treated with actinomycin D ("Act D"), achieved complete remission (CR) (Press release, Rasna Therapeutics, JAN 3, 2018, View Source [SID1234522873]). Treatment with Act D was well tolerated, except that patients experienced oral mucositis as the major toxicity. Rasna Therapeutics has developed a proprietary nanoparticle based formulation of Act D (RASP-101), which is anticipated to maximize efficacy while minimizing oral mucositis. RASP-101 could potentially be a first-in-class modality for treatment of NPM1-mutated acute myeloid leukemia (AML).

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NPM1-mutated AML is a specific genetic leukemia entity that accounts for approximately one-third cases of AML in adults. As we reported previously, intravenous treatment of refractory or relapsed NPM1-mutated AML patients with Act D (12.5 µg or 15 µg/day) for 5 consecutive days produced hematological complete response in some of them (Falini et al., N Eng J Med. 373: 12, 2015).

In a follow-up phase II clinical study (ActD-AML-PG01, EudraCT 2014-000693-18) in refractory/relapse (R/R) AML patients carrying NPM1 gene mutation, treatment with Act D at 15 µg/kg/day for 5 days every 28 days induced CR in 4 out of the 9 evaluable patients (44.4%) with only 1 or 2 cycles of therapy. Three out of the 4 (75%) patients who obtained CR relapsed after 3, 5 and 7 months, respectively. One patient underwent haploidentical allogeneic PBSC transplantation at 3 months after CR achievement and is alive in molecular CR (MRD-negative) after 24 months.

"The precise mechanism of action of Act D in NPM1-mutated AML is still not clearly understood. Follow up data confirms our earlier findings and further support the use of Act D to induce complete hematological remissions with possible long-term molecular responses in NPM1-mutated AML patients. To note, our first previously reported NPM1-mutated AML patient (resistant to hypomethylating therapy) treated with Act D remains in molecular remission at over 3 years since CR achievement," said Dr. Brunangelo Falini, a member of the scientific advisory board of Rasna Therapeutics, Inc.

"We have developed a proprietary formulated Act D (RASP-101), which we anticipate will produce a superior clinical outcome with improved efficacy and safety profile. Emerging data from the multicenter clinical studies will allow us to develop a biomarker strategy to select responsive patients and improve clinical outcome for this unmet clinical need" commented Alessandro Padova, Chairman of Rasna.

About Actinomycin D (Dactinomycin)

Actinomycin D, also known as dactinomycin, a cytotoxic antibiotic produced by Streptomyces parvullus, was approved for medical use in the United States in 1964. It is on the World Health Organization’s List of Essential Medicines, the most effective and safe medicines needed in a health system. It is believed to work by blocking RNA synthesis. The drug has been used to treat a number of types of cancers, including Wilms tumor, rhabdomyosarcoma, Ewing’s sarcoma, trophoblastic neoplasm, testicular cancer, and certain types of ovarian cancer.

About Dr. Brunangelo Falini, M.D.

Brunangelo Falini is the head of the Institute of Hematology and Hemopoietic Stem Cell Transplantation at the University of Perugia, Perugia, Italy. His research activity has mainly focused on the genetic characterization of lymphomas and leukemias using monoclonal antibodies and, more recently, NGS technologies. He led the research group who discovered NPM1 mutations in AML in 2005 and the BRAF-V600E mutation in hairy cell leukemia in 2011. Both these seminal discoveries have already translated into a better diagnosis and therapy of patients affected by these hematological malignancies. Dr. Falini is the recipient of numerous prestigious prizes, including the "Josè Carreras Award" from EHA (Free EHA Whitepaper) (Barcelona, 2010), the "Leopold Griffuel Prize" from ARC (Paris, 2015) and the "Prize for Excellence in Medicine" from the American-Italian Cancer Foundation (New York, 2017).