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.

On January 4, 2018 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) reported that Raul Rodriguez, the company’s president and chief executive officer, will present an update on its product pipeline and a financial overview at the upcoming 36th Annual J.P. Morgan Healthcare Conference in San Francisco, California on January 10, 2018, at 1:30pm PST (see webcast details below) (Press release, Rigel, JAN 4, 2018, View Source;p=RssLanding&cat=news&id=2324845 [SID1234522884]).

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Rigel’s presentation will include a review of the company’s New Drug Application (NDA) for fostamatinib, an oral spleen tyrosine kinase (SYK) inhibitor, in adult patients with immune thrombocytopenia (ITP). The FDA has confirmed that it does not plan to convene an Oncology Drugs Advisory Committee (ODAC) meeting to discuss the NDA. The action date for the FDA to complete its review is April 17, 2018 under the Prescription Drug User Fee Act (PDUFA).

"In 2017, we achieved the critical milestones necessary to transition Rigel into a fully integrated research, development and commercial company prepared to launch its first product into the US market," said Mr. Rodriguez. "2018 will be an exciting year. Fostamatinib, if approved by the FDA, could become an important alternative treatment option for patients with chronic ITP. Additionally, we have now shown in preliminary results that 47% of the patients treated with fostamatinib in our autoimmune hemolytic anemia study had a clinical response as measured by a defined increase in hemoglobin. There are currently no approved therapies for this indication."

Portfolio Update
Fostamatinib in ITP
In June, Rigel announced that the FDA had accepted for filing its NDA for fostamatinib for the treatment of adult patients with ITP. The NDA is supported by data from the FIT clinical program, which included three Phase 3 studies, two randomized placebo-controlled studies (Studies 047 and 048) and an open-label extension study (Study 049). Since the NDA filing, Rigel has worked closely with the FDA to address any questions and will continue to do so in 2018. Rigel has undergone two routine FDA inspections at its headquarters (BIMO and PAI) which did not result in any FDA Form 483 observations. In addition, the FDA has inspected the two highest enrolling FIT clinical sites, both without 483 observations.

The organization is ramping up for a potential product launch, anticipated in the second quarter of 2018. In addition to hiring key personnel, Rigel is developing relationships with external partners to establish distribution channels and the systems needed to provide medication access.

Fostamatinib in Autoimmune Hemolytic Anemia (AIHA)
The Phase 2, open-label, multi-center, Simon two-stage study of fostamatinib for the treatment of warm AIHA completed enrollment of Stage 1. The study is evaluating the safety and efficacy of fostamatinib, at 150mg BID (twice daily), in patients with warm AIHA who have previously received at least one treatment for this disease, but did not have a meaningful benefit and are still anemic.

Stage 1 of the study enrolled 17 evaluable patients. 47% of these patients (8 patients out of 17) have responded to fostamatinib treatment as of December 2017. Six patients, including the last two patients enrolled, responded during the 12-week evaluation period and an additional two patients met the response criteria in the extension study after 12 weeks of dosing. A response was defined as achieving a hemoglobin level of greater than 10 g/dl and at least a 2 g/dl increase from baseline. The safety profile was consistent with the existing fostamatinib safety database. Stage 2 enrollment commenced in late 2017. Stage 2 follows the same protocol as Stage 1 and will include 20 patients.

These data are planned to be presented at a future medical meeting. Rigel plans to meet with the FDA in the first half of 2018 to determine the regulatory path for approval of fostamatinib in AIHA.

Additional Product Development

Rigel has completed enrollment of the second cohort in its blinded Phase 2 study of fostamatinib in IgA Nephropathy (IgAN). The study is evaluating the efficacy, safety, and tolerability of fostamatinib as measured by change in proteinuria, renal function, and histology (comparing the pre- and post-study renal biopsies). The second cohort evaluates a higher dose of fostamatinib, 150mg BID, while the first cohort evaluated 100mg BID. The primary efficacy endpoint is the mean change of proteinuria from baseline at 24 weeks. Rigel expects the study to be complete at the end of the first quarter of 2018.
During 2017, Rigel selected a molecule from its IRAK program for preclinical development. The molecule is differentiated in that it inhibits both the IRAK 1 and IRAK 4 signaling pathways, with potential to treat autoimmune and inflammatory diseases such as psoriasis, lupus, gout, psoriatic arthritis and multiple sclerosis. The company expects to initiate clinical trials in 2018.
Financial Update
Based upon preliminary estimates, Rigel expects to end 2017 with approximately $115.6 million in cash, cash equivalents, and short-term investments, which it believes will be sufficient to fund its operations into 2019. These operations include the potential commercial launch of fostamatinib in the U.S. in 2018 including hiring the sales force associated with the launch, the fostamatinib clinical trials described above for AIHA and IgAN, and continuing to advance the research pipeline.

About ITP
In patients with ITP, the immune system attacks and destroys the body’s own blood platelets, which play an active role in blood clotting and healing. Common symptoms of ITP are excessive bruising and bleeding. People suffering with chronic ITP may live with increased risk of severe bleeding events that can result in serious medical complication, or even death. Current therapies for ITP include steroids, blood platelet production boosters (TPOs) and splenectomy. However, not all patients are adequately treated with existing therapies. As a result, there remains a significant medical need for additional treatment options for patients with ITP.

About AIHA
Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder where the immune system produces antibodies that result in the destruction of the body’s own red blood cells. AIHA affects approximately 40,000 adult patients in the US and can be a severe, debilitating anemia. To date, there are no approved therapies disease-targeted therapies for AIHA, despite the tremendous medical need that exists for these patients.

Webcast Details
To access the live audio webcast or the subsequent archived recording, log on to www.rigel.com. Please connect to Rigel’s website several minutes prior to the start of the live webcast to ensure adequate time for any software download that may be necessary.

On January 4, 2018 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, and Riken Genesis, a subsidiary of Sysmex Corporation (TOKYO, 6869), reported that they have entered into a partnership to introduce nCounter-based diagnostic assays in Japan (Press release, NanoString Technologies, JAN 4, 2018, View Source [SID1234522864]).

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Under this agreement, NanoString and Riken Genesis will collaborate to register, obtain reimbursement and commercialize companion diagnostic assays in Japan, including NanoString’s Lymphoma Subtyping Test which will be marketed as the nCounter Dx LymphMarkTM assay.

LymphMark is a 20-gene signature that classifies cell-of-origin subtypes of Diffuse Large B-cell Lymphoma (DLBCL) tumors. The initial indication for the LymphMark assay is expected to be a potential companion diagnostic to aid in identifying DLBCL patients for treatment. The LymphMark assay is based on the Lymph2Cx gene signature that was originally developed by the Lymphoma/Leukemia Molecular Profiling Project (LLMPP) and has demonstrated analytical robustness and potential clinical utility.

The LymphMark assay was used to select patients in a Phase III clinical trial. The LymphMark assay is being evaluated in more than 40 research studies that are being conducted with 23 companies.

"Through this collaboration, we will work closely with Riken Genesis to launch novel diagnostic products on the nCounter platform that have the potential to guide decision making in the Japanese oncology community," said Brad Gray, chief executive officer of NanoString.

"We are pleased to partner with NanoString to bring new highly multiplexed molecular assays to clinicians and patients in Japan," said Dr. Naoto Kondo, president and chief executive officer of Riken Genesis. "These innovative assays will be an important addition to our portfolio of diagnostic tests."

On January 4, 2018 Deciphera Pharmaceuticals, Inc. (NASDAQ:DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, reported that it has initiated a Phase 3 clinical study to evaluate the safety and efficacy of DCC-2618, a pan-KIT and PDGFRα inhibitor, in patients with advanced gastrointestinal stromal tumors (GIST) (Press release, Deciphera Pharmaceuticals, JAN 4, 2018, View Source [SID1234522871]).

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"We are extremely pleased to initiate the INVICTUS study with DCC-2618 in heavily pretreated GIST patients, specifically fourth-line and fourth-line plus patients," said Michael D. Taylor, Ph.D., President and Chief Executive Officer of Deciphera. "We expect to report top-line results in 2019 and, if successful, this pivotal Phase 3 study could serve as the basis for a New Drug Application (NDA), providing a much-needed therapeutic option for these patients for whom there are no approved treatments. We also plan to initiate a second Phase 3 study later this year evaluating DCC-2618 in second-line GIST patients who have progressed or are intolerant to front-line therapy with imitanib."

"While effective treatments are available for patients with early-stage GIST, in 9 out 10 patients the disease will eventually progress due to the development of secondary drug resistance mutations," said Professor Jean-Yves Blay, Medical Oncologist, General Director Centre Léon Bérard, Comprehensive cancer Centre of Lyon, France. "A therapy with the potential to provide broad coverage across the full spectrum of KIT and PDGFRα mutations would represent a much-needed improvement over currently approved treatment options for patients with later-stage GIST."

Initiation of the INVICTUS study follows results from the ongoing Phase 1 clinical trial presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in September 2017, in which durable disease control by DCC-2618 was observed in heavily pretreated patients with GIST.

INVICTUSPhase 3 Study
The INVICTUS Phase 3 clinical study is a randomized, double-blind, placebo-controlled, international, multicenter trial to evaluate the safety, tolerability, and efficacy of DCC-2618 compared to placebo in patients with advanced GIST patients whose previous therapies have included imatinib, sunitinib, and regorafenib. The trial is expected to enroll approximately 120 patients randomized 2:1 to either 150 mg once daily of DCC-2618 or placebo. The primary efficacy endpoint is median progression free survival (PFS) as determined by independent radiologic review using modified Response Evaluation Criteria in Solid Tumors (RECIST). Secondary endpoints as determined by independent radiologic review using modified RECIST include Objective Response Rate (ORR); Time to Tumor Progression (TTP); and Overall Survival (OS). See www.clinicaltrials.gov for further information (NCT03353753).

About DCC-2618
DCC-2618 is a pan-KIT and PDGFRα kinase switch control inhibitor in clinical development for the treatment of KIT and/or PDGFRα-driven cancers, including gastrointestinal stromal tumors, glioblastoma multiforme and systemic mastocytosis. DCC-2618 was specifically designed to improve the treatment of GIST patients by inhibiting the full spectrum of mutations in KIT and PDGFRα. DCC-2618 is a pan-KIT and pan-PDGFRα inhibitor that blocks initiating KIT mutations in exons 9, 11, 13, 14, 17, and 18, known to be present in GIST patients and the D816V exon 17 mutation known to be present in ASM patients. DCC-2618 inhibits PDGFRα mutations in exon 18, including the D842V mutation that drives a subset of GIST.

On January 4, 2018 Intrexon Corporation (NYSE: XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, reported that Randal J. Kirk, Chairman and Chief Executive Officer will present at the 36th Annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 10th at 9:30 a.m. Pacific Time (Press release, Intrexon, JAN 4, 2018, View Source [SID1234522882]).

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A live webcast of the presentation will be available on the Investors section of Intrexon’s website at View Source Replay of the webcast will be available for 30 days following the event.