On January 4, 2018 SpringWorks Therapeutics, a mission-driven medicines company dedicated to developing innovative potential new treatments for underserved patient communities, reported that it will present at the 36th Annual J.P. Morgan Healthcare Conference (Press release, SpringWorks Therapeutics, JAN 4, 2018, View Source [SID1234538854]). Lara S. Sullivan, M.D., President and Founder, and Saqib Islam, Chief Financial Officer and Chief Business Officer, are scheduled to present at 1:30 p.m. PT on Tuesday, January 9, 2017 at the Westin St. Francis in San Francisco, CA.

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"Having launched SpringWorks Therapeutics only four months ago, we are honored to have been selected to present at the J.P. Morgan Healthcare Conference," stated Mr. Islam. "Our collaborative, patient-centric business model is providing innovative ways to advance promising, investigational therapies that may be otherwise trapped on the sidelines in larger pharmaceutical and biotech companies. We look forward to sharing highlights of our novel approach and late-stage pipeline as we enter a catalyst-rich 2018."

On January 4, 2018 Cellectar Biosciences, Inc. (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, reported that company management will be participating in Biotech Showcase 2018 taking place January 8-10, 2018 at the Hilton San Francisco Union Square (Press release, Cellectar Biosciences, JAN 4, 2018, View Source [SID1234522906]). James Caruso, president and chief executive officer of Cellectar Biosciences, will present a company overview and update on January 10, 2018 at 10:00 a.m. Pacific time.

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Mr. Caruso’s presentation will be webcast live at View Source;tp_key=5c62464b98 and on the Events section of the company’s website where it will also be archived.

About Phospholipid Drug Conjugates (PDCs)

Cellectar’s product candidates are built upon its patented cancer cell-targeting delivery and retention platform of optimized phospholipid ether-drug conjugates (PDCs). The company deliberately designed its phospholipid ether (PLE) carrier platform to be coupled with a variety of payloads to facilitate both therapeutic and diagnostic applications. The basis for selective tumor targeting of our PDC compounds lies in the differences between the plasma membranes of cancer cells compared to those of normal cells. Cancer cell membranes are highly enriched in lipid rafts, which are glycolipoprotein microdomains of the plasma membrane of cells that contain high concentrations of cholesterol and sphingolipids, and serve to organize cell surface and intracellular signaling molecules. PDCs have been tested in more than 80 different xenograft models of cancer.

On January 4, 2018 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS), reported that it has entered into a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), part of the National Institutes of Health (NIH) (Press release, Onconova, JAN 4, 2018, View Source [SID1234522892]). Under the terms of the CRADA, the NCI will conduct research, including preclinical laboratory studies and a clinical trial, on rigosertib in pediatric cancer associated RASopathies.

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The RASopathies are a group of rare diseases which share a well-defined molecular basis in expression or defects involving Ras Effector Pathways. They are usually caused by germline mutations in genes that alter the RAS subfamily and mitogen-activated protein kinases that control signal transduction, and are among the most common genetic syndromes. Together, this group of diseases can impact more than 1 in 1000 individuals, according to RASopathiesNet.

Dr. Steve Fruchtman, Chief Medical Officer of Onconova, noted: "We are excited about the potential of our collaboration with the Pediatric Oncology Branch at NCI’s Center for Cancer Research in the study of rigosertib in children with both hematological and solid tumors that are driven by the Ras pathway. This collaboration could lead to important advances in the treatment of these refractory tumors. This novel approach directed at a specific mechanism driving the underlying neoplasm is the basis of personalized medicine for these indications".

As part of the CRADA, Onconova will provide rigosertib supplies and initial funding towards non-clinical studies. The NCI will fund the majority of the research, including the cost of the clinical trial, which is expected to start in 2018. A clinical trial protocol has been developed and will be reviewed by the Institutional Review Board.

Onconova is also collaborating with academic researchers and patient advocacy groups interested in developing novel therapeutics to address the needs of these patients. In October 2017, Onconova held a Key Opinion Leader Breakfast Symposium in New York City to bring attention to this unmet medical need and the potential for rigosertib in RASopathies. The meeting featured presentations by Bruce D. Gelb, M.D. (Mount Sinai, New York) and Elliot Stieglitz, M.D. (University of California, San Francisco), alongside Dr. Fruchtman. In July 2017, Onconova also presented a summary of its targeted approach at a symposium organized by RASopathiesNet.org.

While the NCI will conduct a trial for RASopathy related cancers in pediatric patients, Onconova will focus on Juvenile Myelomonocytic Leukemia (JMML), a well-described RASopathy affecting children which is incurable without an allogenic hematopoietic stem cell transplant.

Additional information highlighting Onconova’s approach to studying rigosertib in RAS mediated diseases can be found in the presentation, "Strategies to RASopathies and JMML," located in the "Scientific Presentations" section of Onconova’s website.

For Patients

Patients interested in enrolling please contact NCI’s toll-free number 1-800-4-Cancer (1-800-422-6237) (TTY: 1-800-332-8615).

On January 4, 2018 Galera Therapeutics, Inc., a clinical-stage biotechnology company focused on the development of drugs targeting oxygen metabolic pathways with the potential to transform cancer radiotherapy, reported data from its Phase 2b clinical trial of lead product candidate GC4419 for the treatment of severe oral mucositis (SOM) in patients with head and neck cancer will be presented during an oral presentation at the 2018 Multidisciplinary Head and Neck Cancers Symposium being held February 15-17, 2018, at The Westin Kierland Resort & Spa in Scottsdale, Ariz (Press release, Galera Therapeutics, JAN 4, 2018, View Source [SID1234522908]).

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Details of the presentation are as follows:

Abstract ID: 20496
Title: GC4419, a small molecule superoxide dismutase (SOD) mimetic: Randomized, placebo (PBO)-controlled, double blind trial to reduce oral mucositis (OM) from chemoradiotherapy (CRT) in patients (pts) with oral cavity (OC) or oropharyngeal (OP) carcinoma (OCC)
Presentation #: LBA2
Session: Breakout Session II: Survivorship and Acute and Late Effects
Date/Time: Friday, February 16, 2018, 3-4:30 p.m. MT
Presenter: Carryn M. Anderson, M.D., Department of Radiation Oncology, University of Iowa

"We are very pleased with the robust results of this clinical trial, one of the largest ever conducted for this indication, and look forward to presenting the data at this important meeting for patients with head and neck cancer," said Mel Sorensen, M.D., President and CEO of Galera. "We believe GC4419, which leverages our dismutase mimetic platform, has the potential to represent an important new treatment approach for patients with chemoradiotherapy-related severe oral mucositis."

Co-sponsored by the American Society for Radiation Oncology (ASTRO), the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the American Head & Neck Society (AHNS), the Multidisciplinary Head and Neck Cancers Symposium brings researchers and clinicians together to discuss advances in research and treatments for head and neck cancers. For more information about the meeting, visit: View Source

About GC4419

GC4419 is a highly selective and potent small molecule dismutase mimetic that closely mimics the activity of human superoxide dismutase enzymes. GC4419 works to reduce elevated levels of superoxide free radical levels caused by radiation therapy by rapidly converting superoxide free radical molecules to hydrogen peroxide and oxygen. Left untreated, elevated superoxide free radicals can damage noncancerous tissues and lead to debilitating side effects, including oral mucositis (OM), which can limit the antitumor efficacy of radiation therapy.

GC4419 is initially being studied for its ability to reduce the incidence, duration and severity of radiation and chemotherapy-induced OM in patients with head and neck cancer. As reported in December 2017, top-line results from Galera’s 223-patient, double blind, randomized, placebo-controlled Phase 2b clinical trial demonstrate GC4419’s ability to dramatically reduce the duration of severe OM from 19 days to 1.5 days (92 percent), the incidence of severe OM through completion of radiation by 34 percent and the severity of patients’ OM by 47 percent, while preserving healthy tissue. In addition, in preclinical study GC4419 demonstrated an increased tumor response to radiation therapy while preventing toxicity in normal tissue.

The U.S. Food and Drug Administration granted Fast Track designation to GC4419 for the reduction and incidence of radiotherapy induced OM in patients with head and neck cancer. GC4419 also has potential in other indications in which mucosa is damaged by radiation.

About Oral Mucositis

Oral mucositis (OM) is a painful and problematic complication during cancer treatment, especially radiation therapy, caused by excessive superoxide generated during treatment that breaks down epithelial cells that line the mouth. Patients suffering from OM experience severe pain, inflammation, ulceration and bleeding of the mouth.

In the United States, more than 50 percent of patients with cancer receive radiotherapy at some time in their treatment. In patients with head and neck cancer, radiotherapy is a mainstay of treatment and approximately 70 percent of patients receiving chemoradiotherapy develop severe oral mucositis (SOM) as defined by the World Health Organization as Grade 3 or 4, which is the most debilitating side effect of the radiotherapy.

SOM can adversely affect cancer treatment outcomes by causing interruptions in radiotherapy, which may compromise the otherwise good prognosis for tumor control in many of these patients. SOM may also inhibit patients’ ability to eat solid food or even drink liquids, and can cause serious infections. Further, the costs of managing these side effects are substantial, particularly when hospitalization and/or surgical placement of PEG tubes to maintain nutrition and hydration are required. There is currently no drug approved to prevent or treat SOM in patients with head and neck cancer.

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.