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Five Prime Therapeutics Reports Second Quarter 2019 Results

On august 7, 2019 Five Prime Therapeutics, Inc. (NASDAQ: FPRX), a clinical-stage biotechnology company focused on discovering and developing innovative immuno-oncology protein therapeutics, reported its results for the second quarter and provided an update on the company’s recent activities (Press release, Five Prime Therapeutics, AUG 7, 2019, View Source [SID1234538338]).

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"We are pleased that enrollment in our Phase 3 FIGHT trial continues to exceed expectations due to FGFR2b biomarker prevalence above 30% and continued strong investigator support. We are enthusiastic about the potential of bemarituzumab to be an important new medicine for patients with previously untreated, advanced gastric cancer," said Aron Knickerbocker, Chief Executive Officer of Five Prime Therapeutics. "We estimate that the FIGHT trial will reach sufficient enrollment in the coming months to support the planned, event-driven futility analysis in the first half of 2020, allowing us to pause enrollment in the fourth quarter of this year. This is consistent with our portfolio prioritization, which is currently focused on conducting the FIGHT trial, advancing the most promising FPA150 opportunities, as well as assessing safety and dose finding for FPT155."

Second Quarter 2019 Business Highlights and Milestones

Clinical Pipeline:

Bemarituzumab (anti-FGFR2b) is a first-in-class isoform-selective antibody with enhanced antibody-dependent cell-mediated cytotoxicity (ADCC) in development as a targeted immunotherapy for tumors that overexpress FGFR2b. Bemarituzumab and mFOLFOX6 are being evaluated in combination with mFOLFOX6 in the Phase 3 FIGHT (FGFR2b Inhibition in Gastric and Gastroesophageal Junction Cancer Treatment) trial.

Enrollment in the FIGHT trial continues ahead of projections due to FGFR2b biomarker prevalence that has remained steady at more than 30%, strong clinical trial execution, and continued support and enthusiasm from clinical investigators.
The company plans to conduct an early futility analysis for the FIGHT trial during the first half of 2020. The purpose of the futility analysis is to ensure the trial is adequately powered to detect an overall survival benefit at full enrollment.
Consistent with its portfolio prioritization and given the higher than expected enrollment rate, the company will pause enrollment in the FIGHT trial when sufficient patients have been enrolled in the trial to support the planned futility analysis. The company expects the pause in enrollment to occur during the fourth quarter of 2019.
FPA150 (anti-B7-H4) is a first-in-class B7-H4 antibody designed to target tumor cells by enhancing killing of B7-H4 overexpressing tumors through ADCC and by blocking B7-H4 from sending an inhibitory signal to CD8 T cells. B7-H4 is frequently overexpressed in breast, ovarian and endometrial cancers.

Five Prime will present a poster at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress that will include preliminary FPA150 data from the monotherapy Phase 1b expansion cohorts at the 20 mg/kg dose in patients with breast, ovarian or endometrial cancers that overexpress B7-H4 and early safety data from the Phase 1a lead-in testing FPA150 in combination with Keytruda.
The company presented preliminary monotherapy safety data from the dose escalation and exploration portions of the Phase 1a/1b trial of FPA150 in patients with advanced solid tumors at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
FPT155 (CD80-Fc) is a first-in-class CD80-Fc fusion protein that uses the binding interactions of soluble CD80 to directly engage CD28 to enhance its co-stimulatory T cell activity without inducing super agonism and to block CTLA-4 from competing for endogenous CD80, allowing CD28 signaling to prevail in T cell activation in the tumor microenvironment.

Enrollment is proceeding according to plan in the dose escalation portion of the Phase 1a/1b trial, with six dose level cohorts enrolled and dose escalation continuing.
An abstract was submitted for the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting, where the company expects to present safety and pharmacokinetic data from the Phase 1a dose escalation portion of the ongoing Phase 1a/1b trial.
Cabiralizumab (anti-CSF1R) is an antibody that inhibits CSF1R and has been shown to block the activation and survival of tumor-associated macrophages. Pursuant to a worldwide collaboration agreement, Bristol-Myers Squibb (BMS) has an exclusive worldwide license for the development and commercialization of cabiralizumab, and Five Prime retains the rights to a U.S. co-promotion option.

The next anticipated event is completion of enrollment in the Phase 2 trial testing the combination of cabiralizumab with Opdivo (nivolumab) with and without chemotherapy in approximately 160 patients with locally advanced or metastatic pancreatic cancer that has progressed during or after one line of chemotherapy.
BMS-986258 (anti-TIM-3) is a fully-human monoclonal antibody targeting TIM-3 (T cell immunoglobulin and mucin domain-3), an immune checkpoint receptor that may limit the duration and magnitude of T cell responses. This is the first clinical candidate from the discovery collaboration between Five Prime and BMS that includes targets in three immune checkpoint pathways.

The Phase 1/2 clinical trial continues to progress, and, in July, the expected size of the trial was increased from 308 to 383 patients.
Corporate Highlights

During the second quarter, the company announced the appointment of Carol Schafer and Lori Lyons-Williams to its board of directors. The company also announced the departure of Dr. Sheila Gujrathi from the board.
Summary of Financial Results and Guidance:

Cash Position: Cash, cash equivalents and marketable securities totaled $214.1 million as of June 30, 2019, compared to $237.0 million as of March 31, 2019. The decrease in cash, cash equivalents and marketable securities was primarily attributable to quarterly operating expenses that exceeded quarterly revenues.

Revenue: Collaboration and license revenue for the second quarter of 2019 decreased by $4.3 million, or 56.6%, to $3.3 million from $7.6 million for the second quarter of 2018. This decrease was primarily related to lower collaboration revenues from BMS due to a reduction in activities in the Phase 1a/1b trial of the combination of cabiralizumab and nivolumab and the completion of the research term under the immuno-oncology research collaboration in March 2019.

R&D Expenses: Research and development expenses for the second quarter of 2019 decreased by $4.0 million, or 12.0%, to $29.4 million from $33.4 million for the second quarter of 2018. This decrease was primarily due to lower compensation costs, as well lower manufacturing costs related to FPT155 drug production and lower diagnostic costs related to the FIGHT trial. These cost reductions were partially offset by higher CRO costs that were related to strong patient enrollment and the opening of new clinical trial sites.

G&A Expenses: General and administrative expenses for the second quarter of 2019 decreased by $0.1 million, or 1%, to $9.7 million from $9.8 million for the second quarter of 2018. The decrease was primarily due to decreased use of consultants in a number of functions across the company.

Net Loss: Net loss for the second quarter of 2019 was $34.4 million, or $0.99 per basic and diluted share, compared to a net loss of $34.1 million, or $0.99 per basic and diluted share, for the second quarter of 2018.

Shares Outstanding: Weighted average shares outstanding for the second quarter of 2019 was 34,909,479 as of June 30, 2019.

Cash Guidance: Five Prime expects full-year 2019 net cash used in operating activities to be between $117 and $122 million and estimates ending 2019 with cash, cash equivalents and marketable securities between $148 and $153 million.

Conference Call Information

Five Prime will host a conference call and live audio webcast today at 4:30 p.m. (ET) / 1:30 p.m. (PT) to discuss its financial results and provide a corporate update. To participate in the conference call, please dial (877) 878-2269 (domestic) or (253) 237-1188 (international) and refer to conference ID 3575436. To access the live webcast please visit the "Events & Presentations" page under the "Investors" tab on Five Prime’s website at www.fiveprime.com. An archived copy of the webcast will be available on Five Prime’s website beginning approximately two hours after the conference call. Five Prime will maintain an archived replay of the webcast on its website for at least 30 days after the conference call.

TURALIO™ (pexidartinib), FDA Approved Treatment of TGCT, Available at Biologics by McKesson

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]).

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

Vor Biopharma Appoints Veteran Biotech Executive Dr. Robert Ang as President and Chief Executive Officer

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.

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.

Personalis, Inc. to Provide Comprehensive Tumor Immunogenomic Profiling to the New Mexico Cancer Care Alliance for Clinical Study

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.

"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.

Rocket Pharmaceuticals Reports Second Quarter 2019 Financial Results and Operational Highlights

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]).

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.