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Cyclacel Reviews 2015 Achievements and Announces Key Business Objectives for 2016

On January 11, 2016 Cyclacel Pharmaceuticals, Inc. (Nasdaq:CYCC) (Nasdaq:CYCCP) (Cyclacel or the Company) reported 2015 achievements and provided an outline of the Company’s key business objectives for 2016 (Press release, Cyclacel, JAN 11, 2016, View Source [SID:1234508742]). These will be highlighted at the Company’s presentation during the Biotech Showcase 2016 Conference at 9:30 a.m. PST, Monday, January 11, 2016, at the Parc 55 Wyndham Hotel – Union Square at 55 Cyril Magnin Street in San Francisco.

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"During 2015, we continued to follow-up patients in SEAMLESS, our Phase 3 clinical trial evaluating sapacitabine in the front-line treatment setting of elderly patients with acute myeloid leukemia, or AML," said Spiro Rombotis, President and Chief Executive Officer of Cyclacel. "Approximately 7% of prespecified events remain to be observed before we can unblind the randomization code and report top-line results. We anticipate this happening by the end of the first half of 2016. At that point, we will analyze available data and determine submissibility to regulatory authorities. We also continued to follow patients in our Phase 1 trial of sapacitabine and seliciclib in patients with advanced solid tumors. Based on observations to date, we are extending the trial into a selected population of patients with breast cancer who are positive for BRCA mutations. Finally, we advanced CYC065, our second-generation CDK2/9 inhibitor, into a Phase 1, first-in-human study. Based on our preclinical data, we have determined the mechanistic rationale for the clinical development of CYC065 in certain hematological and solid tumor indications. We believe that 2016 may prove to be an important year for Cyclacel and we look forward to keeping you apprised of developments as the year unfolds."

2015 Achievements

Drug Development

Sapacitabine in SEAMLESS, pivotal Phase 3 study as first-line treatment in elderly patients with AML:

Continued follow-up and treatment of patients of this fully enrolled study.
7% of events remain before reporting topline results and mature data analysis.
Submitted to the European Medicines Agency (EMA) a Paediatric Investigation Plan application for sapacitabine.

Sapacitabine and seliciclib in Phase 1 study in patients with advanced solid tumors

Continued to follow patients treated with the all-oral combination of the CDK2/9 inhibitor seliciclib and sapacitabine in a Phase 1 trial in patients with advanced solid tumors. A breast cancer patient with BRCA mutations has been administered more than 70 cycles of the combination and continues on treatment.

Cyclin Dependent Kinase (CDK) Inhibitor Programs

Dosed the first patients in a Phase 1 trial of CYC065, the Company’s second-generation CDK2/9 inhibitor, to evaluate the safety, tolerability and pharmacokinetic profile of CYC065 in solid tumor and lymphoma patients.

Presented preclinical data on the molecular rationale and therapeutic potential in both hematologic and solid tumors of CYC065 at several medical conferences including the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2015, the Society of Hematologic Oncology (SOHO) 2015 Annual Meeting, the AACR (Free AACR Whitepaper)-NCI-EORTC International Conference and the San Antonio Breast Cancer Symposium (SABCS). The data show that:
CYC065 may reverse drug resistance associated with addiction of cancer cells to cyclin E, the partner protein of CDK2.
CYC065 may also inhibit CDK9-dependent oncogenic and leukemogenic pathways, including malignancies driven by certain oncogene and MLL rearrangements. MLL gene status and levels of Bcl-2 family proteins correlated with sensitivity of AML cell lines to CYC065.

CYC065’s anticancer activity presents an opportunity for patient stratification and combinations with anti-leukemic agents.
CYC065 was also effective against uterine cancer cells including those resistant to chemotherapy and was especially potent in uterine cancer cells in which cyclin E was amplified or overexpressed.

CYC065 could be active in triple-negative breast cancer.

First patients dosed in a Phase 2 investigator-sponsored trial (IST) evaluating seliciclib in patients with Cushing’s disease.
Presented preclinical data at the 4th Neuroblastoma Symposium in Newcastle Upon Tyne, UK demonstrating that CYC065 prolongs survival in MYCN-addicted neuroblastoma models.

Corporate Developments

Raised gross proceeds of $10 million from a public offering of common stock.
Entered into a Controlled Equity OfferingSM Sales Agreement with Cantor Fitzgerald & Co., as sales agent ("Cantor"), under which the Company may, from time to time, sell shares of its common stock having an aggregate offering price of up to $8.35 million through Cantor.

Entered into a license and supply agreement with ManRos Therapeutics regarding the development of oral seliciclib for the treatment of cystic fibrosis.

2016 Key Upcoming Business Objectives

Sapacitabine in SEAMLESS:

Continue follow-up of patients until the requisite number of events occur, which is anticipated by the end of the first half of 2016.
Report top-line results.

Following analysis of the mature data set determine submissibility to regulatory authorities for marketing approval.
Progress a Paediatric Investigation Plan for sapacitabine with the European Medicines Agency.
Sapacitabine in myelodysplastic syndromes (MDS):

Initiate a Phase 1/2 trial of sapacitabine in combination with other agents to determine safety and tolerability.
Plan a Phase 2 randomized controlled trial (RCT) of sapacitabine in combination with other agents following review of all relevant clinical data with mature follow-up.

Sapacitabine and seliciclib in Phase 1 study in patients with advanced solid tumors:

Initiate expansion of the Phase 1 study in a breast cancer patient population enriched for BRCA mutations.
Report updated Phase 1 data.

Cyclin Dependent Kinase (CDK) Inhibitor Programs

Report top-line results of the CYC065 Phase 1 trial in solid tumor and lymphoma patients.

Report data from seliciclib ISTs when available.

For the live and archived webcast of the Company’s presentation at the Biotech Showcase 2016 San Francisco conference, please visit the Corporate Presentations page on the Cyclacel website at www.cyclacel.com. The webcast will be archived for 90 days and the audio replay for seven days.

Celgene Corporation Announces 2016 Financial Outlook and Preliminary 2015 Results

On January 11, 2015 Celgene Corporation (NASDAQ:CELG) reported a business update as well as its preliminary 2015 results and financial outlook for 2016 at the 34th Annual J.P. Morgan Healthcare Conference (Press release, Celgene, JAN 11, 2016, View Source [SID:1234508741]). In 2016, net product sales are expected to be approximately $10.5 billion to $11.0 billion, a 17 percent increase year-over-year, based on the mid-point of the range. The negative impact of foreign exchange on net product sales is expected to be approximately $120 million in 2016. For the full-year 2016, REVLIMID net sales are expected to be in the range of $6.6 billion to $6.7 billion. Adjusted diluted earnings per share (EPS) for the full-year 2016 is expected to be in the range of $5.50 to $5.70, a 19 percent increase year-over-year, based on the mid-point of the range. Based on U.S. Generally Accepted Accounting Principles (GAAP), diluted EPS is expected to be in the range of $4.26 to $4.64.

"In 2015, we delivered a strong year operationally and commercially with eight regulatory approvals, the acquisition of Receptos and significant acceleration of our pipeline," said Bob Hugin, Celgene’s Chairman and Chief Executive Officer. "The momentum in our operations and the advancement of our pipeline gives us confidence in our 2020 targets and beyond."

Preliminary 2015 Financial Results Year-Over-Year (Unaudited)

Total net product sales are expected to be approximately $9,160 million, up 21 percent year-over-year. Fourth quarter of 2015 net product sales are expected to be approximately $2,540 million.
REVLIMID: $5,801 million, 16 percent year-over-year increase
ABRAXANE: $967 million, 14 percent year-over-year increase

POMALYST/IMNOVID: $983 million, 45 percent year-over-year increase
OTEZLA: $472 million in the first full year of sales

Adjusted operating margin is expected to be approximately 52 percent for the full year, up 140 basis points (bps) year-over-year. GAAP operating margin is expected to be approximately 24 percent, a decrease from 33 percent in the prior year, primarily due to increased upfront payments to collaboration partners in 2015

Adjusted diluted EPS is expected to be approximately $4.71, a 27 percent year-over-year increase, and reflects $0.14 dilution associated with the Receptos Inc. transaction. GAAP diluted EPS is expected to be in the range of $1.89 to $1.99, and reflects $0.40 dilution associated with the Receptos Inc. transaction

For the fourth quarter of 2015 adjusted diluted EPS is expected to be approximately $1.18, including a $0.07 impact from a $70 million milestone achieved by OncoMed Pharmaceuticals Inc. during the quarter. GAAP diluted EPS is expected to be in the range of $0.63 to $0.73

Certain activities involved in determining the audited results for the fiscal year ended December 31, 2015 are in process and could result in the final reported audited results being different from the unaudited results noted in this press release. Please see the attached Reconciliation of Estimated/Projected GAAP to Adjusted (Non-GAAP) Measures for further information.

Celgene Expects Strong Product Sales and Earnings Growth in 2016

Total net product sales of $10.5 billion to $11.0 billion, an increase of 17 percent year-over-year based on the mid-point of the range and includes a negative impact from foreign exchange of approximately $120 million
REVLIMID net sales in the range of $6.6 billion to $6.7 billion, an increase of 15 percent year-over-year based on the mid-point of the range

Adjusted operating margin of approximately 53.5 percent after investments across the entire organization, a 150 bps improvement over 2015. GAAP operating margin is expected to be approximately 42 percent

Adjusted diluted EPS in the range of $5.50 to $5.70, an increase of approximately 19 percent year-over-year based on the mid-point of the range. GAAP diluted EPS is expected to be in the range of $4.26 to $4.64
Fully diluted share count in 2016 of approximately 825 million

Please see the attached Reconciliation of Estimated/Projected GAAP to Adjusted (Non-GAAP) Measures for further information.

Affirming Expected 2020 Long-term Financial Targets

2020 total net product sales to exceed $21 billion
Hematology franchise to exceed $14.8 billion
Oncology franchise to exceed $2.2 billion
I&I franchise to exceed $4 billion
Adjusted diluted EPS to exceed $13.00
Fully diluted share count of approximately 830 million
2016 Expected Operational Milestones

Hematology/Oncology

Regulatory Submissions/Decisions

Submission for REVLIMID as maintenance after stem-cell transplant in newly diagnosed multiple myeloma (NDMM) in the U.S. and Europe
Submission of renal impairment data for label update for POMALYST/IMNOVID in relapsed/refractory multiple myeloma (RRMM) in the U.S. and Europe
Submission of ABRAXANE for early-stage breast cancer in Europe
Decision by the Committee for Medicinal Products for Human Use (CHMP) on the submission of REVLIMID for relapsed/refractory mantle cell lymphoma in Europe

Clinical Data

Data from the phase III REMARC trial with REVLIMID as maintenance in diffuse large B-cell lymphoma (DLBCL)
Data from the phase III cooperative group ETNA trial with ABRAXANE as neoadjuvant therapy in HER2-negative high-risk breast cancer
Data from the CLL-002 CONTINUUM trial with REVLIMID as maintenance in relapsed/refractory chronic lymphocytic leukemia (CLL)

Data from a phase II trial with CC-122 in non-Hodgkin’s lymphoma (NHL)
Data from phase II trials with motolimod (VTX-2337) in squamous cell carcinoma of the head and neck and ovarian cancer in collaboration with partner VentiRx
Data from the phase II tnAcity trial with ABRAXANE in triple-negative breast cancer

Trial Enrollment

Complete enrollment in the phase III AUGMENT trial with REVLIMID in relapsed/refractory follicular lymphoma
Complete enrollment in the phase III apact (PANC-003) trial with ABRAXANE as adjuvant therapy in surgically resected pancreatic cancer
Complete enrollment in a phase II trial with CC-486 in combination with pembrolizumab in locally advanced or metastatic non-small cell lung cancer

Trial Initiations

Initiate enrollment in a phase I trial of B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T-cell therapy in RRMM in collaboration with partner bluebird bio
Initiate enrollment in six trials in the FUSIONTM program with durvalumab in NDMM, RRMM, NHL, myelodysplastic syndromes and acute myeloid leukemia with partner AstraZeneca/MedImmune
Initiate phase II combination studies with AG-221 and AG-120 in frontline AML in collaboration with partner Agios

I&I

Regulatory Submissions/Decisions

Submission of OTEZLA for psoriasis in Japan

Clinical Data

Long-term radiographic data from the POSTURE (AS-001) trial with OTEZLA in ankylosing spondylitis
Data from the phase III PSA-006 trial with OTEZLA in patients with active psoriatic arthritis who are biologic-naïve
Data from the phase III PSOR-011 trial with OTEZLA in Japanese patients with psoriasis
Data from a pharmacokinetic comparability study to support registration of the OTEZLA once-daily formulation
Data from a phase II trial with OTEZLA in atopic dermatitis
Data from a phase II trial with CC-220 in systemic lupus erythematosus
Data from a phase II trial with RPC-4046 in eosinophilic esophagitis

Trial Enrollment

Complete enrollment in the phase III RELIEFTM (BCT-002) trial with OTEZLA in active Behçet’s disease
Complete enrollment in a phase II trial with OTEZLA in ulcerative colitis
Complete enrollment in the phase II STEPSTONE trial with ozanimod in Crohn’s disease
Research and Early Development

File eight Investigational New Drug (IND) applications
Advance at least two compounds to mid-to-late stage development

Management Changes

On January 8, the Company announced changes to our Research & Early Development organizational structure, which will leverage our strong foundation and drive the rapid advancement of next generation landmark therapies. Tom Daniel, MD, President, Research and Early Development is appointed to Chairman of Celgene Research. Tom, who has been instrumental in building our broad and deep development engine, will oversee strategic initiatives and investments in our research and collaboration portfolio. Rupert Vessey, MA, BM BCh, FRCP, DPhil succeeds Tom Daniel as President, Research and Early Development. Rupert joined Celgene a year ago bringing great insight and leadership to the evolution of our discovery and development strategies in inflammation and immunology as well as in hematology and oncology. Rupert is responsible for leading the execution of discovery and development programs across our portfolio ensuring Celgene capitalizes on the potential of its internal and partnered opportunities. Rob Hershberg, MD, PhD is promoted to Chief Scientific Officer (CSO) and will lead the expansion of our scientific platforms and discovery capabilities. Rob joined Celgene in August 2014 and has led the development of our immuno-oncology efforts, helping to engineer several key collaborations and partnerships.

Q4 and Full year 2015 Conference Call and Webcast Information

Celgene will host a conference call to discuss the fourth quarter and full-year of 2015 operational and financial performance on Thursday, January 28, 2016, at 9 a.m. ET. The conference call will be available by webcast at www.celgene.com. An audio replay of the call will be available from noon January 28, 2016, until midnight ET February 4, 2016. To access the replay in the U.S., dial 1-855-859-2056; outside the U.S. dial 404-537-3406. The participant passcode is 11177982.

Celator® Pharmaceuticals Announces Participation in Cardiff University Clinical Trial in Adults with Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome (AML19)

On January 11, 2016 Celator Pharmaceuticals, Inc. (Nasdaq:CPXX) reported that CPX-351 (also known as VYXEOS) has been selected for participation in a Cardiff University-sponsored clinical trial in adult patients with newly diagnosed acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) (Press release, Celator Pharmaceuticals, JAN 11, 2016, View Source [SID:1234508740]).

"This study is designed to use a risk-based approach to treat younger patients with AML," said Professor Nigel Russell, Centre for Clinical Haematology, Nottingham University Hospital, the trial’s chief investigator. "CPX-351 has demonstrated encouraging activity in high-risk patients. We are excited to study the product in those patients with the highest risk disease."

Some of the anticipated findings from the trial related to CPX-351:

Evaluating CPX-351 in younger patients ( < 60 years of age) in a front line setting
For poor risk patients, to compare CPX-351 versus FLAG-Ida
In patients who fail following FLAG-Ida (two courses) to evaluate a combination of CPX-351 and fludarabine
Evaluating the clinical activity of CPX-351 in patients following treatment with an immunoconjugate
Evaluating the relevance of detecting minimal residual disease using one of two methods (molecular and immunophenotypic)
Health resource utilization
Clinical trials with CPX-351 have shown improved efficacy and reduced early mortality in the majority of patients studied directly supporting evaluating CPX-351 in these patient populations.

"We are pleased that Professor Russell and the trial sponsors chose to study CPX-351 in these patient populations," said Arthur Louie, Chief Medical Officer of Celator Pharmaceuticals. "As we await overall survival data from our CPX-351 Phase 3 trial in patients with high-risk AML, we believe there is significant potential for CPX-351 in other AML populations as well as other blood cancers such as high-risk MDS, and we believe it important to continue to evaluate these opportunities."

About the AML 19 clinical trial

The trial is open to all patients aged 18 to 60 years, and also to patients aged 60 years or over for whom intensive therapy is considered appropriate. In total, across all arms of the study, approximately 3,000 patients are expected to be recruited. The clinical trial has started enrolling patients.

Patients with known adverse cytogenetics will be randomized to receive either FLAG-Ida (fludarabine/cytarabine/G-CSF and idarubicin) or CPX-351 (cytarabine:daunorubicin) liposome injection as induction. Non-adverse cytogenetic patients will be randomized to compare two courses of FLAG-Ida with DA (daunorubicin/cytarabine) chemotherapy each in combination with one of two doses of the immunoconjugate Mylotarg (gemtuzumab ozogamicin).

After one course of treatment, patients who are at high risk of relapse based on the AML Risk Score, or who are deemed refractory ( < 50% reduction in blast percentage, and resistant disease) will be eligible to enter a randomized study of FLAG-Ida compared to CPX-351, with the aim of allowing patients to proceed to allogeneic transplant.

Patients who are not in remission after two courses of FLAG-Ida or in relapse within 6 months after FLAG-Ida, are eligible to receive CPX-351 in combination with fludarabine, for induction and consolidation.

AstraZeneca and Incyte announce new lung cancer clinical trial collaboration

On January 11, 2016 AstraZeneca and Incyte Corporation reported a new collaboration to evaluate the efficacy and safety of Incyte’s Janus-associated kinase (JAK) 1 inhibitor, INCB39110, in combination with AstraZeneca’s next generation epidermal growth factor receptor (EGFR) inhibitor, Tagrisso (osimertinib) (Press release, AstraZeneca, JAN 11, 2016, View Source [SID:1234508733]). The combination will be assessed as a second line treatment for patients with EGFR mutation-positive non-small cell lung cancer (NSCLC), who have been treated with a first generation EGFR tyrosine kinase inhibitor (TKI) and subsequently developed the T790M resistance mutation.

There is increasing evidence that signalling through the JAK-STAT (signal transducer and activator of transcription) pathway could be a contributing factor in resistance to EGFR TKI treatment in patients with EGFR mutation NSCLC. Blocking both JAK and EGFR activity may therefore offer an improved targeted treatment benefit in some patients.

Under the terms of the agreement, AstraZeneca and Incyte will collaborate on a Phase I/II study, to be conducted by Incyte. The Phase I part of the trial is expected to establish a recommended dose regimen for the combination of INCB39110 and Tagrisso, while the Phase II part of the study will assess the safety and efficacy profile. Results from the study will be used to determine whether further clinical development of this combination is warranted.

Antoine Yver, Head of Oncology, Global Medicines Development at AstraZeneca said: "We are pleased to be building on our existing relationship with Incyte and exploring a potentially exciting combination for lung cancer patients who have developed a resistance to first generation EGFR inhibitor treatment. This collaboration allows us to explore further ways in which Tagrisso, our first in class T790M-directed tyrosine kinase inhibitor, can help meet urgent unmet patient need, following its accelerated approval in the US and the recent positive CHMP opinion, recommending approval in Europe."

Rich Levy, MD, Chief Drug Development Officer of Incyte said: "The expansion of our research collaboration with AstraZeneca will allow us to further our understanding of these two compounds and explore their potential synergies which support our goal of delivering innovative medicines that will benefit patients with cancer or other diseases. We look forward to adding to our ongoing clinical research for INCB39110 and exploring the potential of this combination."

This agreement builds on an existing collaboration between the two companies, announced in May 2014, to explore AstraZeneca’s anti-PD-L1 immune checkpoint inhibitor, durvalumab, in combination with Incyte’s oral indoleamine dioxygenase-1 (IDO1) inhibitor, epacadostat (INCB24360).

NOTES TO EDITORS

About Tagrisso (osimertinib)

Tagrisso (osimertinib) is the only approved medicine indicated for adult patients with metastatic EGFR T790M mutation-positive non-small cell lung cancer. This indication is approved under the FDA’s accelerated approval process based on tumour response rate and duration of response (DoR). Osimertinib is being compared with platinum-based doublet chemotherapy in the confirmatory AURA3 Phase III study in patients with EGFR T790M-positive, locally advanced, or metastatic NSCLC who have progressed after EGFR-TKI therapy. It is also being investigated in the adjuvant and metastatic first-line settings, including in patients with brain metastases, and in combination with other compounds.

Non-clinical in vitro studies have demonstrated that osimertinib has high potency and inhibitory activity against mutant EGFR phosphorylation across the range of clinically relevant EGFRm and T790M mutant NSCLC cell lines, with significantly less activity against EGFR in wild-type cell lines.

About INCB39110

INCB39110 is an orally bioavailable, isoform-selective inhibitor of Janus-associated kinase 1 (JAK1). JAK1 activity is believed to play an important role in both autoimmune and oncologic diseases. JAK1 forms heterodimeric complexes with JAK2, JAK3 or TYK2 and functions as an immunomodulatory and inflammatory signalling kinase. Selective JAK1 inhibition prevents STAT signalling downstream of a number of cytokines, including IL-6, IL-10 and interferon-gamma. Consistent with the dominant role for JAK1 in mediating heterodimeric JAK/STAT signalling, JAK1 inhibition has been shown to result in equivalent efficacy compared to balanced JAK1/JAK2 modulation in a variety of preclinical solid and liquid tumor models. INCB39110 will be investigated in clinical trials as monotherapy in graft versus host disease (GvHD) and in several combination-based therapeutic regimens, including with PI3Kδ (INCB50465), IDO1 (epacadostat) and EGFR (Tagrisso) inhibitors.

Five Prime Therapeutics Provides Update on FP-1039 Clinical Program

On January 11, 2016 Five Prime Therapeutics, Inc. (Nasdaq:FPRX), a clinical-stage biotechnology company focused on discovering and developing novel protein therapeutics for cancer and inflammatory diseases, reported an update on GlaxoSmithKline’s (GSK) ongoing Phase 1b clinical trial of FP-1039/GSK3052230, an FGF ligand trap, in patients with squamous non-small cell lung cancer (sqNSCLC) and mesothelioma (Press release, Five Prime Therapeutics, JAN 11, 2016, View Source [SID:1234508729]).

GSK presented preliminary clinical safety and efficacy data from the Phase 1b trial at the World Conference on Lung Cancer on September 9, 2015. At the time, data from Arm A in treatment-naïve sqNSCLC patients were encouraging, Arm B in second line sqNSCLC had few patients enrolled, and data from Arm C were immature because few mesothelioma patients were then evaluable. Based on preliminary data, GSK and Five Prime have agreed to continue to enroll up to 30 mesothelioma patients at the expansion dose of 15 mg/kg in Arm C of the trial. GSK plans to submit data for presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2016 Annual Meeting in June. The companies have also agreed to stop enrolling the sqNSCLC patient study cohorts given the change in treatment paradigms following approvals of immuno-oncology agents and the increasingly competitive landscape.

"We are encouraged by the preliminary data we have recently reviewed from the mesothelioma arm of the study and look forward to seeing the results as additional patients are enrolled and followed," said Lewis T. "Rusty" Williams, M.D., Ph.D., president and chief executive officer of Five Prime. "The majority of mesothelioma tumors express high levels of FGF-2, so our FGF ligand trap represents a rational therapeutic approach, and patients currently have limited treatment options. If we see similar results once we have full, mature data for Arm C, we will seek to regain rights for FP-1039 in the U.S., Canada and the E.U. from GSK, as mesothelioma could represent a potentially attractive market opportunity for Five Prime."

About the Phase 1b Trial

The Phase 1b clinical trial of FP-1039 is being conducted by GSK and is designed as a three-arm, multicenter, non-randomized, parallel-group, uncontrolled, open-label trial. This clinical trial was designed to evaluate the safety, tolerability, dosage, response rate and duration of response of FP-1039:

in combination with paclitaxel and carboplatin in previously untreated metastatic sqNSCLC (Arm A);
in combination with docetaxel in metastatic sqNSCLC that has progressed after previous therapy (Arm B); or
in combination with front-line pemetrexed and cisplatin in mesothelioma (Arm C), a tumor in which the FGF-2 ligand is overexpressed.

About FP-1039

FP-1039 is a protein drug designed to intervene in FGF signaling. As a ligand trap, FP-1039 binds to FGF ligands circulating in the extracellular space (such as FGF-2), preventing these signaling proteins from reaching FGFR1 on the surface of tumor cells where they would otherwise stimulate cancer cell division and/or angiogenesis. However, FP-1039 does not bind to certain "hormonal" FGFs, including FGF-23, which regulates phosphate levels in the blood. As a result, treatment with FP-1039 treatment has not been shown to cause hyperphosphatemia, a side effect seen with small molecule inhibitors of FGF receptors, which block the activity of both cancer-associated FGFs and FGF-23.

About Mesothelioma

Mesothelioma is a disease with high unmet medical need and high mortality rate. A majority of mesothelioma patients have tumors with abnormally high levels of FGF-2. In preclinical testing, Five Prime observed inhibition of mesothelioma tumor growth with single-agent FP-1039. Mesothelioma is an orphan indication in the United States with a prevalence of about 4,000 patients and incidence of about 3,000 patients. Worldwide, there are a total of approximately 14,000 cases of mesothelioma diagnosed each year.