On May 4, 2017 Five Prime Therapeutics, Inc. (Nasdaq:FPRX), a clinical-stage biotechnology company focused on discovering and developing innovative immuno-oncology protein therapeutics, reported a corporate update and reported financial results for the quarter ending March 31, 2017 (Press release, Five Prime Therapeutics, MAY 4, 2017, View Source [SID1234518839]).

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"We had a productive quarter at Five Prime, in which we made notable progress in our clinical and preclinical programs," said Lewis T. "Rusty" Williams, M.D., Ph.D., president and chief executive officer of Five Prime. "We achieved an important milestone by completing enrollment in the Phase 2 part of the ongoing trial of cabiralizumab in patients with pigmented villonodular synovitis, or PVNS. Additionally, we have completed enrollment in some of the Phase 1b cohorts in the cabiralizumab immuno-oncology trial. Each of our three clinical-stage programs advanced and we look forward to announcing clinical trial data from these programs this year. Additionally, we continue to make progress in our pre-clinical programs and, beginning this year, are on track to file at least one IND application for a new molecule each year for the foreseeable future."

Business Highlights and Recent Developments

Clinical Pipeline:

Cabiralizumab (FPA008): an investigational antibody that inhibits CSF1R and has been shown to block the activation and survival of monocytes and macrophages. In the setting of advanced cancer, tumor-associated macrophages (TAMs) can inhibit the immune system’s ability to eradicate the disease. In the CSF-1-driven tumor diffuse tenosynovial giant cell tumor (dsTGCT), also known as pigmented villonodular synovitis (PVNS), the bulk of the tumor mass in joints is formed by the macrophages themselves. Five Prime and Bristol-Myers Squibb (BMS) have an exclusive worldwide collaboration agreement for the development and commercialization of cabiralizumab for these and potentially additional indications.

– Advanced the ongoing Phase 1 trial of cabiralizumab/OPDIVO in immuno-oncology.

– Five Prime and BMS are evaluating the safety, tolerability and preliminary efficacy of the immunotherapy combination of cabiralizumab with the PD-1 immune checkpoint inhibitor OPDIVO (nivolumab) in advanced solid tumors, including non-small cell lung cancer, squamous cell carcinoma of the head and neck, pancreatic cancer, glioblastoma, renal cell carcinoma and ovarian cancer. The companies are also evaluating cabiralizumab as monotherapy and in combination with OPDIVO in additional tumor settings.

– Five Prime and BMS are also assessing multiple tissue biomarkers on tumors, TAMs and T cells. These assessments are guiding optimal dose selection to maximize the probability of success in future development.

– Five Prime completed enrollment in some of the Phase 1b cohorts in the cabiralizumab immuno-oncology trial and expects to complete enrollment in all of the current Phase 1b trial cohorts in the second half of 2017.

– The company expects to announce initial clinical trial data from the cabiralizumab immuno-oncology trial in the second half of 2017.

– Advanced the ongoing Phase 1/2 trial of cabiralizumab in patients with PVNS.

– In April, Five Prime completed enrollment in the initially planned 30-patient cohort of the Phase 2 part of the ongoing clinical trial evaluating cabiralizumab in patients with PVNS, an aggressive tumor confined to the synovium. Five Prime is evaluating clinical measures, including response rate, pain and range of motion.

– Five Prime plans to seek regulatory guidance to initiate a pivotal trial studying cabiralizumab in PVNS to begin in 2018.

– The company also plans to disclose initial clinical data from the cabiralizumab PVNS trial at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2017 Annual Meeting.

FPA144: an isoform-selective antibody in development as a targeted immuno-therapy for tumors that overexpress FGFR2b, a splice variant of a receptor for some members of the fibroblast growth factor (FGF) family. FPA144 has been engineered for enhanced antibody-dependent cell-mediated cytotoxicity (ADCC) to increase direct tumor cell killing by recruiting natural killer (NK) cells.

– Advanced the Phase 1 monotherapy trial of FPA144 in patients with gastric cancer. Enrollment continues in the expansion portion of the trial, evaluating the safety, PK and efficacy of biweekly 15 mg/kg infusions of FPA144 in patients with gastric cancer whose tumors overexpress FGFR2b.

– Plan to disclose updated gastric cancer clinical trial data from the FPA144 program at the ASCO (Free ASCO Whitepaper) 2017 Annual Meeting.

– Plan to seek regulatory guidance on a registrational path for FPA144 in combination with chemotherapy as a front-line gastric cancer therapy. Preclinical data suggest that the combination of FPA144 with standard-of-care chemotherapy is additive. Five Prime plans to initiate a combination trial of FPA144 with chemotherapy to advance into front-line therapy.

– Plan to launch a Phase 1 safety trial for FPA144 in patients with gastric cancer in Japan. The observed incidence of gastric cancer is higher in Asian populations than in other populations. Five Prime recently received clinical trial notification (CTN) approval from the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan and plans to initiate a Phase 1 monotherapy clinical trial in Japan in the third quarter of 2017.

– Advanced the Phase 1 monotherapy trial of FPA144 in patients with bladder cancer. Five Prime observed a complete response in a Stage 4 metastatic bladder cancer patient who received FPA144 in the dose escalation portion of the trial. That patient remains in remission and on treatment since April 15, 2015. The company opened an additional cohort in the Phase 1 clinical trial to test FPA144 as a treatment for bladder cancer patients whose tumors overexpress FGFR2b, as assessed by the company’s immunohistochemistry (IHC) test. Five Prime is adding sites that specialize in bladder cancer.

FP-1039: a protein drug designed to block FGF signaling. As a ligand trap, FP-1039 binds to and neutralizes a subset of FGF ligands (such as FGF2), preventing these growth-promoting and angiogenic proteins from reaching FGFR1 on the surface of tumor cells.

– Patients continue to be dosed with FP-1039 in the ongoing Phase 1b clinical trial in mesothelioma. Five Prime regained full rights to FP-1039 from GlaxoSmithKline (GSK) in September 2016. GSK is completing the ongoing Phase 1b trial combining FP-1039 with front-line pemetrexed and cisplatin in untreated, unresectable mesothelioma. GSK concluded trial recruitment with 25 patients enrolled at the 15 mg/kg dose in June 2016. Further clinical development of FP-1039 in mesothelioma will be determined after data are mature.

– Five Prime plans to disclose clinical data from the FP-1039 program at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2017 Congress.
Preclinical Research and Development:

Featured three preclinical research poster presentations during the 2017 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting that took place from April 1 – 5 in Washington, D.C.
– Preclinical results demonstrated that both anti-CSF-1R and anti-GITR synergize with anti-PD1 therapy.
– Highlighted Five Prime’s robust in vitro and in vivo platforms to discover new immuno-oncology targets.
Continues to advance three preclinical development candidates in IND-enabling studies
– FPA150 (anti-B7-H4)
– An antibody designed for two mechanisms of action: to block an inhibitory T cell checkpoint pathway and to enhance killing of B7-H4-expressing tumors by ADCC.
– Investigational New Drug (IND) application planned for the fourth quarter of 2017.

– FPA154 (GITR agonist antibody)
– A tetravalent agonist antibody designed for greater GITR activation versus conventional antibodies. Conventional GITR agonist antibodies have two GITR binding sites while FPA154 has four.
– IND application planned for the fourth quarter of 2017.

– FPT155 (CD80-Fc)
– A multi-targeting immune modulator that can stimulate T cell responses through three critical pathways: CTLA4 blockade, CD28 agonism (without superagonism) and PD-L1 blockade.
– IND application planned in 2018.
Progress in pre-clinical and research programs.
– Five Prime is on track to achieve the goal of filing at least one IND application for a new molecule each year for the foreseeable future, beginning this year.

Continues to conduct multiple immuno-oncology research screens to identify new targets and drug candidates.
– Five Prime initiated antibody campaigns for new targets.
– Five Prime’s research team previously conducted in vivo screens of approximately 700 immune-related cell surface proteins to find immune proteins that could be new targets for validation as potential novel immuno-oncology therapeutics.
Summary of Financial Results and Guidance:

Cash Position. Cash, cash equivalents and marketable securities totaled $380.3 million on March 31, 2017, compared to $421.7 million on December 31, 2016. The decrease in cash was primarily attributable to cash used in operations to advance the FPA144 clinical trial, the cabiralizumab Phase 2 clinical trial in PVNS and preclinical development programs.
Revenue. Collaboration revenue for the first quarter of 2017 increased by $3.6 million to $10.1 million from $6.5 million in the first quarter of 2016. This increase was primarily due to revenue recognized under the 2015 cabiralizumab collaboration agreement with BMS, under which Five Prime is reimbursed for the expenses from the cabiralizumab immuno-oncology trial.
R&D Expenses. Research and development expenses for the first quarter of 2017 increased by $14.9 million to $33.8 million from $18.9 million in the first quarter of 2016. This increase was primarily related to advancing cabiralizumab in the Phase 2 clinical trial in PVNS and the Phase 1a/1b clinical trial in immuno-oncology, advancing the FPA144 Phase 1 clinical trial and further advancing preclinical development programs.
G&A Expenses. General and administrative expenses for the first quarter of 2017 increased by $2.4 million to $10.5 million from $8.1 million in the first quarter of 2016. This increase was primarily due to increases in payroll and stock-based compensation expenses.
Net Loss. Net loss for the first quarter of 2017 was $33.4 million, or $1.21 per basic and diluted share, compared to a net loss of $13.0 million, or $0.49 per basic and diluted share, for the first quarter of 2016.

Shares Outstanding. Total shares outstanding were 28.6 million as of March 31, 2017.
Cash Guidance. Five Prime expects full-year 2017 net cash used in operating activities to be less than $120 million. The company estimates ending 2017 with approximately $300 million in cash, cash equivalents and marketable securities.

On May 4, 2017 Epizyme, Inc. (NASDAQ:EPZM), a clinical-stage biopharmaceutical company creating novel epigenetic therapies, reported it has earned a $10 million milestone payment from GlaxoSmithKline (GSK) (Press release, Epizyme, MAY 4, 2017, View Source [SID1234518838]). The milestone payment follows GSK’s initiation of GLP toxicology studies for a first-in-class methyltransferase inhibitor discovered by Epizyme and licensed to GSK.

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"We are pleased to achieve another milestone under our collaboration with GSK, further validating the strength of our epigenetic approach and drug discovery platform, as well our success in collaborating with industry leaders like GSK," said Susan Graf, Chief Business Officer of Epizyme. "We are encouraged that this investigational medicine continues to advance toward the clinic."

About the Epizyme-GSK Collaboration
Under the terms of its collaboration and license agreement with GSK, Epizyme granted GSK exclusive worldwide license rights to methyltransferase inhibitors directed to three targets. During the research term of the collaboration, which is now complete, Epizyme was primarily responsible for preclinical research, and now GSK is responsible for subsequent research, development and commercialization of the three programs. Using its proprietary drug discovery platform, Epizyme discovered and optimized compounds targeting three methyltransferases. GSK3326595 (formerly EPZ015938), a first-in-class PRMT5 inhibitor, was the first of these to enter the clinic in September 2016. GSK holds worldwide rights to all three programs. Epizyme has earned $69 million in up-front, research, and milestone payments to date, and may receive up to an additional $607 million from GSK if all milestones are met for all three programs. Epizyme is eligible to receive up to double-digit royalties on worldwide net sales of collaboration products.

On May 4, 2017 Corvus Pharmaceuticals, Inc. (NASDAQ:CRVS), a clinical-stage biopharmaceutical company focused on the development and commercialization of novel immuno-oncology therapies, reported financial results for the first quarter ended March 31, 2017, and provided a business update (Press release, Corvus Pharmaceuticals, MAY 4, 2017, View Source [SID1234518834]).

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"We are continuing to make significant progress in our Phase 1/1b trial, which is designed to rapidly identify the diseases where our lead product candidate, CPI-444, has the greatest potential both as a single agent and in combination with atezolizumab," said Richard A. Miller, M.D., co-founder, president and chief executive officer of Corvus. "This strategic design has allowed us to identify four cohorts for expansion based on the demonstration of anti-tumor activity in renal cell cancer and non-small cell lung cancer, especially in patients that are resistant or refractory to prior treatment with anti-PD(L)-1 antibodies, and whose tumors are PDL-1 negative, an extremely difficult to treat patient population. We look forward to presenting additional clinical data from these cohorts in an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2017."

RECENT ACHIEVEMENTS AND UPCOMING MILESTONES
Clinical & Preclinical Development

Expanded four cohorts from 14 to 26 patients in the ongoing disease-specific expansion part of the Phase 1/1b clinical study of the Company’s lead oral checkpoint inhibitor, CPI-444. The expanded cohorts include treatment with both CPI-444 as a single agent and in combination with atezolizumab (Tecentriq), an anti-PD-L1 antibody, in renal cell cancer (RCC) and non-small cell lung cancer (NSCLC).
Presented interim safety data on 113 patients and efficacy data for 96 patients enrolled the Company’s Phase 1/1b study at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2017. The data showed that treatment with CPI-444 was well tolerated, provided disease control and induced tumor regression in a number of patients with extensive disease, especially in patients who were resistant/refractory to prior treatment with anti-PD(L)-1 antibodies.
Plan to present clinical data from the four disease expansion cohorts in both RCC and NSCLC at the ASCO (Free ASCO Whitepaper) Annual Meeting in June 2017.
Continued to progress anti-CD73 antibody and ITK inhibitor programs toward Phase 1 study initiation in 2018.
CORPORATE DEVELOPMENT

On May 1, 2017, Corvus expanded its collaboration agreement with Genentech, a member of the Roche Group. Under the new agreement, CPI-444 administered in combination with atezolizumab (Tecentriq) will be evaluated in a Phase 1b/2 randomized, controlled clinical study as second-line therapy in patients with non-small cell lung cancer (NSCLC) who are resistant/refractory to prior therapy with an anti PD(L)-1 antibody. It is anticipated that the study will enroll up to 65 patients in the treatment arm. Genentech will manage study operations for the Phase 1b/2 trial, which is expected to begin enrolling patients in the second half of 2017. Corvus retains global development and commercialization rights to CPI-444.
FINANCIAL RESULTS

At March 31, 2017, Corvus had cash, cash equivalents and marketable securities totaling $122.1 million. This compared to cash, cash equivalents and marketable securities of $134.9 million at December 31, 2016.

Research and development expenses for the three months ended March 31, 2017 totaled $13.5 million compared to $5.4 million for the same period in 2016. The increase of $8.1 million was primarily due to an increase of $2.1 million in outside costs for the Phase 1/1b clinical trial for CPI-444, an increase of $1.7 million in drug manufacturing costs for our anti-CD73 antibody program, an increase of $0.8 million in personnel and related costs associated with higher headcount and a $3.0 million milestone payment made to Vernalis plc pursuant to our license agreement.

General and administrative expenses for the three months ended March 31, 2017 totaled $2.7 million compared to $1.0 million for the same period in 2016. The increase of $1.7 million was primarily due to an increase of $0.9 million in personnel and associated costs, primarily due to an increase in headcount and a $0.5 million increase in legal and accounting costs.

The net loss for the three months ended March 31, 2017 was $16.0 million compared to $6.4 million for the same period in 2016. Total stock compensation expense for the three months ended March 31, 2017 was $1.5 million compared to $0.4 million for the same period in 2016.

On May 4, 2017 Eleven Biotherapeutics, Inc. (NASDAQ:EBIO), a late-stage clinical oncology company advancing a broad pipeline of novel product candidates based on its Targeted Protein Therapeutics (TPTs) platform, reported financial results for the quarter ended March 31, 2017, and provided a corporate update (Press release, Eleven Biotherapeutics, MAY 4, 2017, View Source [SID1234518830]).

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"During our first quarter of 2017, we made meaningful progress, advancing our Phase 3 registration clinical trial of Vicinium and continuing development efforts with Proxinium and VB6-845d," said Stephen Hurly, President and Chief Executive Officer of Eleven Biotherapeutics. "Importantly, we also presented new preclinical data supporting the potential of our locally- and systemically-administered drug candidates not just as monotherapies, but also in combination with immuno-oncology products, including checkpoint inhibitors. These results accord with our clinical development strategies, and we look forward to further exploring TPTs as new medicines with the potential to offer considerable improvements over existing options."

First Quarter and Recent Business Highlights and Anticipated Upcoming Milestones:

Vicinium: Vicinium is a single protein anti-epithelial cell adhesion molecule (anti-EpCAM) antibody fragment fused with Pseudomonas Exotoxin A (ETA) that is designed to specifically target and deliver a potent anti-cancer payload directly into tumor cells. Vicinium is currently in a Phase 3 registration clinical trial for the treatment of high-grade non-muscle invasive bladder cancer (NMIBC) in subjects who have previously received two courses of Bacillus Calmette-Guérin (BCG) and whose disease is now BCG-unresponsive.

Complete enrollment for Phase 3 registration clinical trial expected in second half of 2017
Topline data from Phase 3 registration clinical trial expected in 2018
Proxinium: Proxinium is a single protein anti-EpCAM antibody fragment fused with ETA for the treatment of late-stage squamous cell carcinoma of the head and neck (SCCHN). Proxinium has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and Fast Track designation from the FDA. Proxinium has demonstrated anti-tumor activity in prior Phase 1 and 2 clinical trials.

Initiation of Phase 1/2a clinical trial evaluating Proxinium in combination with a checkpoint inhibitor expected in second half of 2017
At the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April, Eleven presented new preclinical results with VB4-845, the active pharmaceutical ingredient used to formulate both Vicinium and Proxinium. Data suggest that VB4-845 induces the expression of HMGB1 in tumor treated cells; HMGB1 is one of three damage-associated molecular patterns (DAMPs) indicative of immunogenic cell death (ICD). Eleven has previously disclosed observations of the other two DAMPs markers – cell surface expression of calreticulin and extracellular release of ATP – following treatment with VB4-845. Together, these results suggest that product candidates formulated with VB4-845 are capable of driving host anti-tumor immune responses that can potentiate the activity of immuno-oncology agents.

As part of the same poster presentation, Eleven shared data from a preclinical model in patient-derived xenograft tumor-bearing mice reconstituted with a human immune system, which was used to assess the combination of intratumoral injection of VB4-845 with the anti-PD1 antibody, nivolumab. Treatment with VB4-845 alone suppressed the growth of injected tumors, while monotherapy nivolumab had little effect. In contralateral, non-injected tumors, responses from mice treated with both VB4-845 and nivolumab were more pronounced than responses in mice treated with either product as a monotherapy. Based on these results, Eleven believes that VB4-845 killing of tumor cells could facilitate and augment checkpoint inhibitor anti-tumor activity.

Systemically-administered TPT Pipeline: Eleven’s initial systemically-administered TPTs leverage a proprietary, highly potent, de-immunized, plant toxin, deBouganin. DeBouganin has picomolar killing of cancer cells and may be effective against cancer stem cells. In preclinical studies, deBouganin demonstrated the ability to avoid multi-drug resistance mechanisms that can decrease the efficacy of small molecule payloads. Based on these results, Eleven believes that deBouganin-based therapies may be effective against a wide spectrum of cancers.

Investigational New Drug Application (IND) submission for VB6-845d planned for first quarter of 2018
At the AACR (Free AACR Whitepaper) Annual Meeting, Eleven presented preclinical data suggesting that its deBouganin payload is capable of effectively killing tumor cells that are resistant to treatment with antibody drug conjugates (ADCs) composed of DM-1 and MMAE payloads when conjugated to the same monoclonal antibody, trastuzumab. The Company believes this is due, in part, to deBouganin’s lack of sensitivity to both the multidrug resistance pumps and the changes in phosphorylation status of proteins involved in cell proliferation and survival that allow some cancers to escape the action of anti-mitotic ADCs.

Corporate: Eleven further expanded its internal clinical development team with two key hires: Gary Conboy as Executive Director, Clinical Sciences and Mary Rohrer as Associate Director, Clinical Operations.

First Quarter 2017 Financial Results:

Cash Position: Cash and cash equivalents were $20.3 million as of March 31, 2017, compared to $25.3 million as of December 31, 2016.
Revenue: Revenue was $0.4 million for the three months ended March 31, 2017, compared to $0.2 million for the same period in 2016. This increase was due to revenue recognized from the License Agreement (License Agreement) with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. (Roche).
R&D Expenses: Research and development expenses were $2.9 million for the three months ended March 31, 2017, compared to $4.6 million for the same period in 2016. The decrease was due primarily to a reduction in isunakinra and EBI-031 related development expenses, partially offset by an increase in Vicinium related development expenses.
G&A Expenses: General and administrative expenses were $2.2 million for the three months ended March 31, 2017, compared to $2.1 million for the same period in 2016.
Net Loss: Net loss was $6.1 million, or $0.25 per share, for the three months ended March 31, 2017, compared to net loss of $7.6 million, or $0.39 per share, for the same period in 2016.
Financial Guidance: Based on current operating plans, Eleven expects to have cash to fund research and development programs and operations into early 2018.
Upcoming Events and Presentations:

American Urological Association 2017 Annual Meeting, May 12-16, 2017 in Boston, Massachusetts

On May 4, 2017 Novocure (NASDAQ:NVCR) reported two pilot trials are open and actively recruiting pediatric patients with high grade gliomas to evaluate the safety and feasibility of Tumor Treating Fields (TTFields) in this population (Press release, NovoCure, MAY 4, 2017, View Source [SID1234518829]). The trials are sponsored by the Pediatric Brain Tumor Consortium and Hackensack University Medical Center.

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"Although solid tumor cancers are generally rare in children, brain and spinal cord tumors are the third most common type of childhood cancer," said Dr. Stewart Goldman, Professor of Pediatrics at the Ann & Robert H. Lurie Children Hospital of Chicago. "In order to make a meaningful impact in the lives of these children, we need ongoing research to test the safety and feasibility of potential treatments. We are pleased to see trials testing TTFields in this underserved population."

The first trial, sponsored by the Pediatric Brain Tumor Consortium in collaboration with the National Cancer Institute, will study the feasibility of using TTFields for children with recurrent or progressive suptratentorial high-grade glioma and ependymoma. This study will primarily assess patients’ compliance and number of treatment-related toxicities with secondary outcome measures including the response rate and event-free survival. This trial is actively recruiting an estimated 25 patients.

In the second study, the safety, tolerability and preliminary efficacy of TTFields will be tested in pediatric high-grade gliomas in two cohorts. The initial cohort of patients will receive TTFields alone and will consist of children with recurrent high-grade gliomas. The second cohort of patients will receive TTFields in combination with temozolomide and bevacizumab and is open to children with both newly diagnosed and recurrent gliomas. The trial, sponsored by Hackensack University Medical Center, is actively recruiting and is estimated to enroll 12 patients.

For more information on the trial designs, visit clinicaltrials.gov and reference NCT03033992 and NCT03128047. Treatment with TTFields is not approved for the treatment of pediatric gliomas by the U.S. Food and Drug Administration. The safety and effectiveness of TTFields therapy for pediatric gliomas has not been established.