On February 2, 2018 Acorda Therapeutics, Inc. (Nasdaq:ACOR) will host a conference call and webcast to report its fourth quarter 2017 financial results and pipeline updates on Thursday, February 15 at 8:30 a.m. ET (Press release, Acorda Therapeutics, FEB 2, 2018, View Source [SID1234523694]).

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To participate in the conference call, please dial (866) 393-4306 (domestic) or (734) 385-2616 (international) and reference the access code 8789908. The presentation will be available on the Investors section of www.acorda.com.

A replay of the call will be available from 11:30 a.m. ET on February 15, 2018 until 11:59 p.m. ET on March 15, 2018. To access the replay, please dial (855) 859-2056 (domestic) or (404) 537-3406 (international); reference code 8789908. The archived webcast will be available in the Investor Relations section of the Acorda website at www.acorda.com.

On February 2, 2018 Aclaris Therapeutics, Inc. (NASDAQ:ACRS), a dermatologist-led biopharmaceutical company focused on identifying, developing and commercializing innovative and differentiated therapies to address significant unmet needs in medical and aesthetic dermatology, reported that members of its management team will present and host investor meetings at the following upcoming investor events (Press release, Aclaris Therapeutics, FEB 2, 2018, View Source [SID1234523693]):

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Dr. Neal Walker, President and Chief Executive Officer, will present at the LEERINK Partners 7th Annual Global Healthcare Conference in New York, NY on Wednesday, February 14, 2018 at 3:30 PM EST. Management will also host investor meetings on Wednesday, February 14, 2018.
Dr. Neal Walker, President and Chief Executive Officer, will present at the Cowen and Company 38th Annual Health Care Conference in Boston, MA on Tuesday, March 13, 2018 at 10:00 AM EST. Management will also host investor meetings on Tuesday, March 13, 2018.
A live webcast of the LEERINK Partners 7th Annual Global Healthcare Conference and the Cowen and Company 38th Annual Health Care Conference presentation may be accessed through the Company’s web site, www.aclaristx.com, on the ‘Events and Presentations’ section. An archived version of the presentation will be available for 30 days.

On February 2, 2018 Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, reported upcoming oral and poster presentations at the 14th Annual WORLDSymposium Meeting being held in San Diego, CA, February 5 – 9, 2018 (Press release, Abeona Therapeutics, FEB 2, 2018, View Source;p=RssLanding&cat=news&id=2330088 [SID1234523692]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Platform Presentations:

A phase 1/2 clinical trial of systemic gene transfer of scAAV9.U1a.HSGSH for MPS IIIA: safety, tolerability, and preliminary evidence of biopotency
Presenter: Kevin Flanigan, M.D. – Principal Investigator and Director, Center for Gene Therapy and Neuromuscular Disorders, Nationwide Children’s Hospital, Columbus, OH
Date: Thursday, February 8, 2018
Time: 11:00 AM PST

Identification of novel AAV capsids for treatment of lysosomal disorders
Presenter: Daphne Chen, Ph.D., University of North Carolina at Chapel Hill, Chapel Hill, NC
Date: Tuesday, February 6, 2018
Time: 4:00 PM PST

Poster Presentations:

Poster 67: AAV vector comparability across mammalian and insect cell production platforms for treatment of lysosomal diseases
Presenter: Adam S. Davis, Ph.D. – Director of Manufacturing at Abeona Therapeutics
Tuesday, February 6, 2018
Time: 4:30 PM PST

Poster 185: Intravenous administration of CLN3 gene therapy for juvenile neuronal ceroid lipofuscinosis
Presenter: Scott Kerns – Product Development Scientist at Abeona Therapeutics
Tuesday, February 6, 2018
Time: 4:30 PM PST

Poster 329: Combination dosing of CLN1 gene therapy extends lifespan in a mouse model of infantile neuronal ceroid lipofuscinosis
Presenter: Alejandra Rozenberg, Ph.D. – Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC
Wednesday, February 7, 2018
Time: 4:30 PM PST

About WORLDSymposium: WORLDSymposium is an annual research conference dedicated to lysosomal diseases. W.O.R.L.D. is an acronym that stands for We’re Organizing Research on Lysosomal Diseases. Since its inception as a small group of passionate researchers in 2002, WORLDSymposium has grown to an international research conference that attracts over 1600 participants from more than 50 countries around the globe.

On February 1, 2018 Boston-based cancer company Partner Therapeutics, Inc. (PTx) reported that it has acquired the global rights to develop, manufacture, and commercialize Leukine (sargramostim) from Sanofi (Press release, Partner Therapeutics, FEB 1, 2018, View Source [SID1234610372]). Leukine is an immuno-stimulant that promotes the growth and activation of a broad range of white blood cells important in activating the body’s immune response to fight infections. Leukine is used to treat or prevent severe and life-threatening infections and is the only immune modulator approved by the FDA for the treatment of acute myelogenous leukemia (AML) in older patients and for use in both allogeneic and autologous bone marrow transplantation.

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In connection with the acquisition of Leukine, PTx will also acquire a dedicated manufacturing facility in Lynnwood, Washington. The facility is a state of the art biologics manufacturing plant that was certified for commercial production in 2012. The Lynnwood facility will serve as the core manufacturing and supply chain center for PTx’s operations.

Leukine is the only FDA-approved recombinant human granulocyte-macrophage colony stimulating factor (GM-CSF). It has been demonstrated to promote growth and activation of monocytes, macrophages, neutrophils and dendritic cells. It is currently indicated for the treatment of AML in older adults to reduce the incidence of severe and life-threatening infections resulting in death; use in the treatment of allogeneic bone marrow transplants to reduce the incidence of bacteremia and other culture positive infections and shorten the median duration of hospitalization; and to prolong the survival of patients who are experiencing bone marrow transplant failure or delay.

PTx will support the development of Leukine for new indications. The product is being tested in a diverse set of clinical trials for its potential to improve survival and reduce adverse events in combination with leading immuno-oncology therapies. A 250 patient, randomized Phase II study in refractory melanoma in combination with ipilimumab demonstrated an improvement in survival (hazard ratio of 0.64) over ipilimumab alone1. Leukine is currently being tested in a Phase III trial in front-line melanoma in combination with ipilimumab and nivolumab, being conducted by the ECOG-ACRIN Cancer Research Group (Principal Investigator: F Stephen Hodi, MD, Director of the Center for Immuno-Oncology at Dana-Farber Cancer Institute) and sponsored by the National Cancer Institute (ClinicalTrials.gov Identifier: NCT02339571).

Leukine is also in development for the treatment of Hematopoietic Syndrome of Acute Radiation Syndrome (H-ARS). Data presented at the 2016 annual meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), demonstrated Leukine’s ability to increase survival in non-human primates exposed to myelosuppressive doses of radiation without supportive whole blood transfusions or individualized antibiotics2. A supplemental biologics licensing application (sBLA) was filed in September of 2017 with the FDA requesting approval of Leukine for the treatment of H-ARS. In December, the application was granted Priority Review with a PDUFA date of March 29, 2018.

"We are delighted to have the opportunity to build a new future for Leukine and welcome the talented and dedicated team in Lynnwood to the PTx family", said Robert Mulroy, CEO of PTx. "The acquisition of Leukine provides us with an established commercial business, a product that has demonstrated a clear and substantial impact on outcomes, and a program with the potential to become a core component of immuno-oncology, the treatment of acute radiation syndrome and the treatment of infections."

"In contrast to other approved growth factors that stimulate one cell type, Leukine’s ability to stimulate a broader variety of cells, endows it with unique clinical potential to address serious medical needs across hematologic diseases and cancer as well as infectious, neurological and pulmonary disorders," said Dr. Debasish Roychowdhury, Chief Medical Officer. "We are excited to have the opportunity to work with investigators and healthcare professionals to explore new indications that can take advantage of Leukine’s unique biological and immuno-stimulatory attributes and clinical properties."

PTx plans to provide commercial and medical support of Leukine in the United States and explore commercialization opportunities outside the U.S.

On February 1, 2018 SciTech Development reported that the U.S. Food & Drug Administration (FDA) has recently granted its lead drug product ST-001 Orphan Drug Status for the treatment of T-cell lymphoma (Press release, SciTech Development, FEB 1, 2018, View Source [SID1234523786]).

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SciTech’s lead drug product (ST-001) is comprised of the active pharmaceutical ingredient (API) fenretinide, a proven anti-cancer drug, and a specifically selected mixture of bioavailability enhancing phospholipids combined in a proprietary nano formulation. Fenretinide has previously demonstrated human efficacy in the treatment of neuroblastoma and leukemia as well as for lymphoma. ST-001 may be utilized as a standalone drug or in combination with other drugs and immunotherapy agents.

"The granting of orphan drug status by the FDA is a significant milestone in the development of our ST-001 drug program" said Earle Holsapple, President of SciTech Development. "We intend to make use of other FDA expedited programs including fast track designation, priority review and expedited new drug application (NDA) approval in bringing ST001 to market."

The FDA confers orphan status to drugs and biologics that treat rare diseases and disorders that affect fewer than 200,000 people in the United States. Benefits to companies receiving orphan drug status include tax credits and seven years of additional market exclusivity.