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U.S. FDA Lifts Partial Clinical Hold on Medivation’s Pidilizumab

On March 9, 2016 Medivation, Inc. (NASDAQ: MDVN) reported that the U.S. Food and Drug Administration (FDA) has lifted the partial clinical hold on the Investigational New Drug (IND) application for pidilizumab (MDV9300) in hematological malignancies and confirmed that the Phase II clinical trial in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), as well as other studies that cross reference the IND, may now proceed (Press release, Medivation, MAR 9, 2016, View Source [SID:1234509435]). The partial clinical hold was not related to any safety concerns.

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The investigator brochure, protocols, and informed consent documents related to the Phase II trial have satisfactorily been revised to reflect the Company’s understanding that PD-1 (Programmed Death-1) is not the target of pidilizumab. No patients had yet been enrolled in the trial, which commenced in late 2015. Patients who were receiving pidilizumab through investigator-sponsored trials have continued to receive treatment and those investigators have been informed to update their protocols and informed consent documents to state that pidilizumab is not an anti-PD-1 antibody.

"We are delighted that the FDA has lifted the partial clinical hold and that we may proceed with our potentially pivotal trial in this area of high unmet medical need," said David Hung, M.D., Founder, President & Chief Executive Officer of Medivation. "As we move forward, we also are working to determine the compound’s exact binding mechanism which, we believe, modulates the body’s innate immune response and differentiates it from the heavily crowded immuno-oncology space that targets the adaptive side of immunity."

The Company also intends to submit an amendment to the Chemistry, Manufacturing and Controls (CMC) section of the IND in the second quarter to provide for larger manufacturing lot sizes to better support the current and planned clinical activities for pidilizumab, which is intended for development in other hematologic malignancies, such as multiple myeloma. As such, the DLBCL trial is targeted to resume in the second half of this year.

About the Phase II Trial
The international, open-label, Phase II trial of pidilizumab is expected to enroll approximately 180 patients with an incomplete response following salvage therapy or autologous stem cell transplantation for relapsed or refractory CD20+ DLBCL, transformed indolent lymphoma, or primary mediastinal B-cell lymphoma. The patients will be assessed in two parallel cohorts of approximately 90 patients each: one cohort will enroll patients who have received an autologous stem cell transplant, and the second cohort will enroll patients who have received salvage chemotherapy, but who are ineligible for transplant. Pidilizumab will be administered at a dose of 200 mg by IV infusion. The primary endpoint of the trial is best overall response rate.

XBiotech Announces Submission of Marketing Authorization Application (MAA) for Candidate Colorectal Cancer Therapy to European Medicines Agency (EMA)

On March 08, 2016 XBiotech Inc. (NASDAQ:XBIT) reported that the Company has now submitted its Marketing Authorization Application (MAA) for its candidate therapy (Xilonix) for advanced colorectal cancer to the European Medicines Agency (EMA) (Press release, XBiotech, MAR 8, 2016, View Source [SID1234556367]). Submission of the MAA for Xilonix is necessary for the Company to gain approval to sell the drug in European Union member nations. The Company, which only recently was granted eligibility to submit its MAA through the Centralised Procedure, has made a remarkably quick turnaround for delivery of the extensive MAA documentation needed for the EMA review.

John Simard, CEO of XBiotech, commented, "With submission of the Marketing Authorization Application, we now look forward to the review process."

Few agents are reliably able to mediate durable responses in metastatic disease. And many agents have significant trade-offs in terms of side effects. In patients with advanced disease, often weakened from successive rounds of cytotoxic therapy, the risk-benefit associated with further therapy is often questionable. Based on novel objective response (OR) criteria, developed by XBiotech in conjunction with the EMA’s Scientific Advice Working Group, the candidate therapy subject of the MAA was recently evaluated in a Phase III clinical study for the ability to control disease-related symptoms that inversely correlate with survival. The Company believes using the symptom-based objective response criteria will allow better assessment of overall benefit from therapy in advanced cancer patients.

20-F – Annual and transition report of foreign private issuers [Sections 13 or 15(d)]

(Filing, Annual, AstraZeneca, 2015, MAR 8, 2016, View Source [SID:1234509415])

Abeona Therapeutics Announces Fourth Quarter and Full Year 2015 Summary Financial Results and Recent Operational Highlights

On March 8, 2016 Abeona Therapeutics, Inc. (NASDAQ: ABEO), a biopharmaceutical company focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases, reported summary financial results for the fourth quarter and full fiscal year ended December 31, 2015 (Press release, Abeona Therapeutics, MAR 8, 2016, View Source;p=RssLanding&cat=news&id=2146834 [SID:1234510443]). The Company will provide a business update for investors and other stakeholders on a conference call, Wednesday, March 9th, at 4:45 pm (Eastern). Tim Miller, Ph.D., President and CEO and Jeffrey Davis, Chief Operating Officer, together with other executives, will conduct the call. Interested parties are invited to participate in the call by dialing 877-269-7756 (toll free domestic) or 201-689-7817 (international). The call will consist of an overview of the Company’s 4Q15 financials, and a discussion of business highlights.

"The past year has led to significant advancements in our goal of building a leadership position in the field of gene therapy and plasma protein therapies towards transforming the lives of patients with rare diseases," stated Steven H. Rouhandeh, Executive Chairman. "In 2015, we expanded our pipeline with two clinical stage AAV gene therapies for Sanfilippo syndrome types A and B, added a third AAV gene therapy product in Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) (also known as juvenile Batten disease), signed a license to an innovative CRISPR-Cas9 gene editing platform in rare blood disorders, with an initial focus in Fanconi anemia, strengthened our team, and added substantial financial resources to our balance sheet. In 2016, our priorities include driving our AAV gene therapy and alpha-1 protease inhibitor programs into the clinic, and advancing our gene editing programs including defining of regulatory pathways to bring our CRISPR product candidates to patients."

Tim Miller, Ph.D., President and CEO, stated, "2016 will be an exciting, transformative year for Abeona Therapeutics as we position ourselves to enter multiple human clinical trials with our pipeline of innovative product candidates. As recently announced, the FDA allowance of the IND for the Phase 1/2 clinical study of ABO-102 for patients with Sanfilippo syndrome type A (MPS IIIA) moves our programs into the clinic here in the US, and we look forward to working with our collaborators to expand this program into Europe and Australia later this year. We believe that our gene therapy programs in Sanfilippo syndrome type B (ABO-101) and Juvenile Neuronal Ceroid Lipofuscinosis (ABO-201) will follow shortly. Lastly, we would like to thank our dedicated researchers and clinical collaborators, as well as the many dedicated patient foundations, for their tireless efforts and commitment to advancing new treatment options for these devastating unmet medical needs."

Recent Abeona Highlights

Sanfilippo syndrome gene therapy programs: On February 29, 2016, Abeona announced the FDA allowance of an Investigational New Drug (IND) for systemic AAV Phase 1/2 clinical study with ABO-102 gene therapy for patients with Sanfilippo syndrome type A (MPS IIIA). On January 11, 2016, we announced that initial regulatory approvals from European bodies — the Genetically Modified Organism (GMO) Voluntary Release regulatory filings, and the ethical committee regulatory filings — for both the ABO-101 and ABO-102 programs in Spain. Abeona plans to commence both programs for the upcoming human clinical trials to be conducted at Cruces University Hospital in Bilbao, Spain. Both the ABO-101 and ABO-102 programs have received Orphan Drug and Pediatric Rare Disease designations from the FDA.
SDF-Alpha plasma protein program: Abeona has completed optimization of the downstream chromatography steps for our SDF-Alpha (alpha-1 protease inhibitor) for inherited COPD. Additional provisional patent applications have been filed to provide the Company with expanded intellectual property protection. The Company has expanded its CMO relationships to ensure it has the ability to manufacture clinical material for future trials. The Company confirms that its proprietary SDF platform provides significantly enhanced yields of alpha-1 protease inhibitor, at levels up to 10 times of that achievable with the industry standard Cohn processes, and with purity levels consistent with that achieved by other commercial processes.
Advanced pipeline programs: Together with its academic collaborators, the Company continued to progress its pre-clinical programs in juvenile Batten disease and its CRISPR-Cas9 program in Fanconi anemia (FA) and other rare blood disorders. Juvenile Batten disease is the most common form of a group of disorders known as neuronal ceriod lipofuscinosis (NCL), a lysosomal storage disease that affects the nervous system in children and for which there are no approved treatment options. Fanconi anemia is a rare pediatric blood disease characterized by multiple physical abnormalities, bone marrow failure and a higher than normal risk of cancer.
Fourth Quarter and Full 2015 Summary Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2015 were $40.1 million, compared to $11.6 million as of December 31, 2014. The increase was primarily driven by net proceeds from multiple equity financings partially offset by cash used to fund operations. Cash used in operations in 2015 was $10.4 million.
Revenues: Revenue was $215 thousand and $1.0 million for the fourth quarter of 2015 and the full year 2015, respectively, compared to $234 thousand and $925 thousand in comparable periods in 2014. Revenues consisted of a combination of royalties from marketed products, primarily MuGard, and recognition of deferred revenues related to upfront payments from early license agreements.
Loss per share: Loss per share was $0.07 and $0.53 for the fourth quarter of 2015 and the full year 2015, respectively, compared to a loss per share of $2.05 and $15.26 in comparable periods in 2014.
About ABO-101 (AAV NAGLU) and ABO-102 (AAV SGSH) are next generation adeno-associated viral (AAV)-based gene therapies for MPS IIIA and MPS IIIB, respectively. These gene therapies involve a one-time delivery of a genetically modified virus to deliver a normal copy of the defective gene to cells of the central nervous system (CNS) and peripheral organs with the aim of reversing the effects of the genetic errors that cause the disease. After a single dose in preclinical animal models of Sanfilippo syndrome, ABO-101 and ABO-102 induced cells in the CNS and peripheral organs to produce the missing enzymes and help repair damage caused to the cells. Preclinical in-vivo efficacy studies in animals with Sanfilippo syndrome have demonstrated functional benefits that remain for months after treatment. A single dose significantly restored normal cell and organ function, corrected cognitive defects that remained for months after drug administration, increased neuromuscular control and increased the lifespan of animals with MPS III over 100% one year after treatment compared to untreated control animals. These results are consistent with studies from several laboratories suggesting AAV treatment could potentially benefit patients with Sanfilippo syndrome Type A and B,. In addition, safety studies conducted in animal models of Sanfilippo syndromes have demonstrated that delivery of ABO-101 and ABO-102 are well tolerated with minimal side effects.

About Abeona: Abeona Therapeutics, Inc.

Soricimed Granted Orphan-Drug Designation from the U.S. FDA for Treatment of Ovarian Cancer

On March 8, 2016 – Soricimed Biopharma Inc. ("Soricimed"), a clinical-stage pharmaceutical company discovering and developing peptide-based cancer therapeutics, reported that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to peptide SOR-C13 for the treatment of ovarian cancer (Press release, Soricimed Biopharma, MAR 8, 2016, View Source [SID:1234509843]).

Orphan drug status is granted following review of the rarity and severity of the medical condition, as well as the potential benefit of the product treating this condition. Orphan drug status qualifies Soricimed for various development incentives, including tax credits and reduced filing fees for clinical trials undertaken in the U.S. If approved for commercialization by the FDA, SOR-C13 may qualify for seven years of marketing exclusivity in the United States.

According to the American Cancer Society, ovarian cancer ranks fifth in cancer deaths among women, accounting for more deaths than any other cancer of the female reproductive system, with an incidence rate of 11.9 per 100,000 and a death rate of 7.7 per 100,000. In some cases, orphan drugs can be made available to patients before marketing approval on a compassionate use basis.

"Receiving orphan drug status is significant", stated Paul Gunn, President and CEO at Soricimed. "It is an important regulatory milestone, offering special incentives to Soricimed through the development stage of SOR-C13." Soricimed announced that it had reached its target enrollment and treatment duration in its Phase 1 trial of SOR-C13 in patients with solid tumor cancers who had failed other treatments. Topline results were released last month.

About SOR-C13: SOR-C13 is a first-in-class peptide in development for the treatment of cancer. SOR-C13 binds with high selectivity and affinity to TRPV6, a calcium channel that is highly elevated in prostate, breast, lung and ovarian cancer and is correlated with poor outcomes. TRPV6-mediated Ca2+ entry is responsible for maintaining a high tumour proliferation rate, as well as increasing tumour cell survival and fortifying mechanisms that withstand cell destruction. By binding to this channel, SOR-C13 starves cancer cells of calcium that is needed for cell growth and division. As demonstrated in animal models, the result is an inhibition of tumor growth. Due to the high specificity of SOR-C13 for its target and its unique mechanism of action this drug candidate may result in fewer and less severe side effects compared to standard cancer chemotherapy. SOR-C13 is the first drug candidate targeting TRPV6 to have entered clinical development anywhere in the world.