On August 7, 2019 Biologics by McKesson, an independent specialty pharmacy for oncology and other complex therapeutic areas, reported that it was selected by Daiichi Sankyo, Inc. as the exclusive specialty pharmacy provider for TURALIO (pexidartinib) for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery (Press release, McKesson, AUG 7, 2019, View Source [SID1234538337]).

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TURALIO, a first-in-class oral tyrosine kinase inhibitor for TGCT, was approved by the FDA on August 2, 2019. Surgery is the primary mode of treatment for TGCT associated with severe morbidity or functional limitations; however, some patients have disease that is not amenable to resection. For these patients, treatment options are limited because there are no approved systemic therapies for the disease. In February 2019, the FDA accepted, with Priority Review, a New Drug Application (NDA) for TURALIO based on results of the phase 3 ENLIVEN study, the first placebo-controlled study of a systemic investigational therapy in patients with TGCT.

TURALIO is approved with a Boxed Warning for the risk of serious and potentially fatal liver injury. Hepatotoxicity with ductopenia and cholestasis has occurred in patients treated with TURALIO. Across 768 patients who received TURALIO in clinical trials, there were two irreversible cases of cholestatic liver injury. One patient died with advanced cancer and ongoing liver toxicity, and one patient required a liver transplant. The mechanism of cholestatic hepatotoxicity is unknown, and its occurrence cannot be predicted. It is unknown whether liver injury occurs in the absence of increased transaminases.

"We are pleased to be able to dispense this first systemic therapy for TGCT to patients," said Ann Steagall, director of Clinical Policy, Biologics by McKesson. "It offers patients a treatment where surgery may not be an option. We’re excited to support TGCT patients as well as distribute another therapy in our growing portfolio of complex care. Due to the risk of serious and potentially fatal hepatotoxicity, TURALIO is only available through a Risk Evaluation and Mitigation Strategy (REMS) program. As a leading provider of risk assessment and care planning, Biologics is proud to help ensure that patients taking TURALIO get the level of care they need and deserve."

Biologics is committed to and recognized for its high level of customer service as well as its innovative, high-touch and multidisciplinary patient-centric approach. Each team includes a pharmacist with in-depth knowledge of therapies, an experienced nurse and a financial counselor who is familiar with various financial assistance programs and organizations that help patients. Each patient speaks to a PharmD prior to dispense and has access to them 24 hours a day, seven days a week. This highly-skilled care team works together to develop individualized care plans that address each patient’s unique clinical, financial and emotional needs and streamlines communication back to the treating provider, enabling high-quality care, differentiated outcomes and improved adherence. In addition, the Biologics team works closely with payers to ensure patients can access the specialty medications they need.

Clinicians may submit prescriptions to Biologics via phone (800.850.4306), fax (800.823.4506) or eScribe. For electronic prescribing systems, clinicians may search for Biologics within their EMR system

On August 7, 2019 Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of hematological cancers, reported the appointment of Robert Ang, MBBS, MBA as its President and Chief Executive Officer (Press release, Vor BioPharma, AUG 7, 2019, View Source [SID1234538336]). Vor also announced a move into new integrated headquarters in Cambridge, Mass. This expansion follows a recent $42 million Series A financing directed at advancing the Company’s lead eHSC-based candidate for the treatment of acute myeloid leukemia towards the clinic and further building the pipeline.

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Dr. Ang is the former Chief Business Officer of Neon Therapeutics, part of the early team establishing the company prior to its Series A investment and through its initial public offering. Prior to Neon, he served as Senior Vice President of Business Development at Bavarian Nordic, where he was primarily responsible for conducting a $975 million transaction between the company and Bristol-Myers Squibb for PROSTVAC, a Phase 3 immuno-oncology asset. Before joining Bavarian Nordic, Dr. Ang served as Head of both Business Development and Medical Affairs for Cadence Pharmaceuticals (now Mallinckrodt) and worked at Frazier Healthcare Ventures, a leading life sciences venture capital firm. At Frazier, he was involved in several pharmaceutical and biotechnology investments including Cadence Pharmaceuticals, Incline Therapeutics (now The Medicines Company), Alnara Pharmaceuticals (now Eli Lilly) and Collegium Pharmaceuticals. Dr. Ang also has experience in strategy consulting at the Boston Consulting Group and has general surgical training. He holds an MBBS (Doctor of Medicine) from the University of Western Australia and an MBA with honors from Columbia Business School.

"I am privileged to join Vor Biopharma, a company already equipped with leading science from Dr. Siddhartha Mukherjee’s lab at Columbia University and strong financial backing from eminent life science investors. Vor’s unique and scientifically elegant eHSC approach places it among the most exciting opportunities in oncology, with the potential to change the treatment paradigm of liquid tumors," said Dr. Ang. "An excellent team has already been established in Cambridge, Mass., and new headquarters in Kendall Square will catalyze our research and development in gene editing, hematopoietic stem cell biology, process development and translational medicine."

"Robert has a deep understanding of the oncology landscape and a proven track record in strategic planning and business development," said Kush Parmar, MD, PhD, Managing Partner at 5AM Ventures and Chairman of Vor’s Board of Directors. "We are thrilled to welcome Robert to Vor at this pivotal moment as we bring the company to a new stage of growth and development while advancing our lead candidate, VOR33, toward the clinic."

About VOR33
Vor’s lead engineered hematopoietic stem cell (eHSC) product candidate, VOR33, is in development for acute myeloid leukemia (AML). VOR33 is designed to produce healthy blood cells that lack the receptor CD33, thus enabling the targeting of AML cells through the CD33 antigen while avoiding toxicity to the blood and bone marrow. Currently, treating AML with therapy that targets CD33 can be effective but may be limited in dose and duration due to toxicity to the blood and bone marrow. By rendering healthy blood and bone marrow cells ‘invisible’ to CD33-targeted therapies, VOR33 aims to significantly improve the therapeutic window and effectiveness of these AML therapies with potential to broaden clinical benefit to different patient populations.

On August 7, 2019 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for cancer, reported that The University of New Mexico (UNM) Comprehensive Cancer Center and the New Mexico Alliance for Cancer Care will utilize Personalis’ cancer immunogenomics platform, ImmunoID NeXT, for the investigation of biomarkers of response to a novel treatment paradigm in women with ovarian cancer (Press release, Personalis, AUG 7, 2019, View Source [SID1234538335]). The basis of this trial, which evaluates the combination of Olaparib and Tremelimumab in women with recurrent BRCA-deficient ovarian cancers, is based on the work of Sarah Adams, MD, that indicated that immune priming with targeted cytotoxic therapy using a PARP-inhibitor can sensitize ovarian tumors to immune therapy and optimize patient survival. The clinical trial is being conducted at several ORIEN centers across the United States. For more information please visit clinicaltrials.gov identifier NCT02571725.

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"We are delighted to collaborate with Personalis on this study," said the principal investigator of the study, Dr. Adams, Associate Professor, Division of Gynecologic Oncology at the UNM Comprehensive Cancer Center. "Comprehensive immuno-profiling will inform on biomarkers of response for this experimental treatment in women with BRCA1 or BRCA2 germline mutated ovarian cancers, for which there aren’t standard curative measures."

Personalis ImmunoID NeXT Platform provides a comprehensive interrogation and analysis of ~20,000 genes in both DNA and RNA. The platform is an end-to-end solution for immuno- and precision oncology biomarker discovery applications, simultaneously enabling the analysis of: tumor escape mechanisms (including HLA typing and somatic mutation detection), immune repertoire profiles, neoantigen load, tumor mutational burden (TMB), microsatellite instability (MSI), oncoviruses, and immune checkpoint gene expression.

On August 7, 2019 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform clinical-stage gene therapy company, reported financial results for the quarter ended June 30, 2019, and provides an update on the Company’s recent pipeline developments, as well as upcoming milestones (Press release, Rocket Pharmaceuticals, AUG 7, 2019, View Source [SID1234538334]).

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2019 is a transformative year for Rocket as we transition to a registrational-stage development company focused on the growth of our multi-platform gene therapy pipeline," said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. "This quarter, we made a lot of progress, and we now have three Rocket-sponsored gene therapy programs in the clinic, including our first AAV gene therapy program. The initiation of our Phase 1 clinical trial for Danon disease marked an important milestone for Rocket as we advanced the first AAV gene therapy for a monogenic heart failure syndrome into the clinic. With each clinical milestone, we are one step closer to rapidly providing potentially curative treatments to patients severely in need."

Recent Pipeline Developments

Two patients treated with RP-L102 "Process B" in the Phase 1 trial for Fanconi Anemia (FA) at Stanford University with initial data anticipated in six to nine months. Two patients were treated earlier this year at the Center for Definitive and Curative Medicine at Stanford University School of Medicine, the lead U.S. clinical site. "Process B" incorporates a modified cell enrichment process, transduction enhancers, and commercial-grade vector manufacturing and cell processing. The global Phase 2 study is anticipated to commence in the fourth quarter with alignment from the U.S. Food and Drug Administration (FDA). Ongoing follow-up data on patients from "Process A" and preliminary data on patients from "Process B" will be presented in the fourth quarter.
Patient dosing continues in first cohort in the Phase 1 clinical trial of RP-A501 for the treatment of Danon disease. Rocket continues to enroll patients in the trial and anticipates Phase 1 data in 2020. The study is designed to assess the safety and tolerability of a single infusion of RP-A501. Pediatric dosing will initiate pending determination of safety in a patient population comprised of older adolescents and young adults.
Commencement of Phase 1/2 trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). The Phase 1 portion of the registrational trial is underway with the first patient’s cells harvested in the second quarter. The Phase 1 portion of the trial is expected to enroll two patients and will assess the safety and tolerability of RP-L201. Preliminary data will be presented in the fourth quarter.
Anticipated Milestones

FA (RP-L102)
End-of-Phase 1 Meeting with FDA (3Q19)
Commencement of E.U. Phase 2 study (3Q19)
Commencement of U.S. Phase 2 study (4Q19)
Additional data for "Process A" (4Q19)
Initial Phase 1 data for "Process B" (4Q19)
Danon Disease (RP-A501)
Phase 1 data (2020)
LAD-I (RP-L201)
Initial Data from RP-L201 (4Q19)
PKD (RP-L301)
IMPD clearance (3Q19)
Initiation of Phase 1 study (4Q19)
IMO (RP-L401)
Initiation of clinical study (2020)
Upcoming Investor Conferences

Leerink’s Spotlight Series on Rare & Genetic Diseases—August 7, 2019 in Boston, M.A.
Citi’s 14th Annual Biotech Conference—September 4, 2019 in Boston, M.A.
Baird’s 2019 Global Healthcare Conference—September 5, 2019 in New York, N.Y.
Morgan Stanley’s 17th Annual Global Healthcare Conference—September 11, 2019 in New York, N.Y.
Oppenheimer’s Fall Summit Focused on Specialty Pharma and Rare Disease Companies—September 23, 2019 in New York, N.Y.
Ladenburg Thalmann’s 2019 Healthcare Conference—September 24, 2019 in New York, N.Y.
Second Quarter 2019 Financial Results

Cash position. Cash, cash equivalents and investments as of June 30, 2019, were $257.8 million.
Debt. Our balance sheet includes a $52.0 million fully convertible debenture which matures in August of 2021.
R&D expenses. Research and development expenses were $14.0 million for the three months ended June 30, 2019, compared to $10.8 million for the three months ended June 30, 2018. The increase was primarily driven by an increase in clinical trial expenses of $2.1 million as the Phase 1 clinical trials commenced for Fanconi Anemia and Danon disease and an increase in license expense of $1.1 million.
G&A expenses. General and administrative expenses were $4.4 million for the three months ended June 30, 2019, compared to $4.1 million for the three months ended June 30, 2018.
Net loss. Net loss was $18.7 million or $0.38 per share (basic and diluted) for the three months ended June 30, 2019, compared to $15.8 million or $0.40 per share (basic and diluted) for the three months ended June 30, 2018.
Shares outstanding. 50,332,435 shares of common stock were outstanding as of June 30, 2019.
Financial Guidance

Cash position. As of June 30, 2019, we had cash, cash equivalents and investments of $257.8 million. Rocket expects such resources will be sufficient to fund its operations into the first half of 2021.

On August 7, 2019 Cambrex Corporation (NYSE: CBM), the leading small molecule company providing drug substance, drug product and analytical services across the entire drug lifecycle, reported that it will not hold a second quarter 2019 earnings conference call and will not update previously provided financial guidance given the pending acquisition by an affiliate of the Permira funds (Press release, Cambrex, AUG 7, 2019, View Source [SID1234538333]).

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