On May 4, 2017 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a clinical stage oncology biotechnology company, reported financial results for the first quarter 2017 and provided an update on its product development programs (Press release, Mirati, MAY 4, 2017, View Source [SID1234518877]).

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"As anticipated, 2017 will be an important and defining year for Mirati. Our single agent precision medicine programs and immuno-oncology combination programs are advancing and we remain on track to report key data in the second half of the year," said Charles M. Baum, M.D., Ph.D., President and Chief Executive Officer. "We have made significant progress in our pre-clinical KRAS and LSD-1 programs and we are very encouraged by the data from these programs".


Single Agent Programs
Glesatinib (MGCD265)
Mirati is enrolling patients in its registration-enabling Phase 2 NSCLC AMETHYST clinical trial, which is evaluating single agent glesatinib for the treatment of NSCLC patients with MET driver mutations. The Company expects to provide an update on efficacy data from the AMETHYST trial in the second half of 2017.

Sitravatinib (MGCD516)
The Phase 1b expansion clinical trial of sitravatinib is enrolling NSCLC patients with RET, CHR4q12 and CBL genetic alterations. The Company expects to provide an update on efficacy data in the third quarter of 2017.


Immuno-oncology Combination Programs
Sitravatinib plus nivolumab
The multicenter Phase 2 NSCLC clinical trial is evaluating sitravatinib in combination with nivolumab, a checkpoint inhibitor approved for the treatment of patients with a variety of solid tumors including NSCLC. The trial is enrolling patients who have relapsed after treatment with a checkpoint inhibitor. Sitravatinib is a potent inhibitor of the TAM (Tyro, Axl, Mer) and split (KDR, KIT) tyrosine kinase families which regulate multiple aspects of the immune system thought to enhance anti-tumor immunity. The Company expects to provide an initial update on this combination trial in the second half of 2017.

Mocetinostat (MGCD103) plus durvalumab
Mirati is collaborating with Medimmune/Astra Zeneca on a Phase 2 clinical trial combining mocetinostat, an orally administered spectrum-selective Class 1 HDAC inhibitor, and durvalumab, MedImmune’s monoclonal antibody inhibiting PD-L1. The combination trial is exploring the potential of mocetinostat to enhance the effectiveness of checkpoint inhibitors in NSCLC and the Company expects to provide an update in mid 2017.




Preclinical Programs
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KRAS Inhibitor Program: A mutant-selective (G12C) KRAS inhibitor program is advancing rapidly. Potent and selective inhibitors have been identified and have demonstrated marked tumor regression in KRAS mutant xenograft tumor models. An IND candidate selection is anticipated by second half of 2017.

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LSD1 Inhibitor Program: A highly-potent and potentially best-in-class LSD1 inhibitor has been identified with potential for rapid clinical proof-of-concept in small cell lung cancer or acute myeloid leukemia. An investigational new drug (IND) submission is planned for this compound in the fourth quarter 2017.


First Quarter 2017 Financial Results

In January 2017, we completed a public offering of our common stock and pre-funded common stock warrants that generated net proceeds of $66.8 million. Cash, cash equivalents, and short-term investments were $105.5 million at March 31, 2017, compared to $56.7 million at December 31, 2016. We continue to expect that our currently available cash, cash equivalents and short-term investments are sufficient to fund operations into late 2018.

Research and development expenses for the first quarter of 2017 were $14.4 million, compared to $18.0 million for the same period in 2016. The decrease in research and development expenses is primarily driven by a decrease in manufacturing expenses associated with glesatinib, offset by an increase in expenses associated with our ongoing Phase 1b clinical trial of sitravatinib.

General and administrative expenses for the first quarter of 2017 were $3.7 million, compared to $4.1 million for the same period in 2016. The decrease in general and administrative expense is largely the result of a decrease in non-cash share-based compensation expense, which is due to lower exercise prices for options granted during the last half of 2016 and first quarter of 2017.

Net loss for the first quarter of 2017 was $17.8 million, or $0.73 per share basic and diluted, compared to net loss of $21.9 million, or $1.13 per share basic and diluted for the same period in 2016.

On May 4, 2017 Progenics Pharmaceuticals, Inc. (Nasdaq:PGNX) reported financial results and provided a business update for the first quarter of 2017 (Press release, Progenics Pharmaceuticals, MAY 4, 2017, View Source [SID1234518857]).

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"This first quarter of 2017 was marked by major developments within our clinical pipeline, particularly the announcement of positive topline data from our registrational Phase 2b study of AZEDRA for the treatment of pheochromocytoma and paraganglioma," said Mark Baker, Chief Executive Officer of Progenics. "The study not only achieved its primary endpoint by a significant margin, but also showed that treatment with AZEDRA resulted in favorable anti-tumor activity, a key secondary endpoint. We are now working diligently to complete our New Drug Application for this important new therapy, which we remain on track to submit by the middle of this year."

Mr. Baker continued, "With our portfolio of PSMA-targeted therapeutics and imaging agents, we continue to leverage our Find Fight and Follow strategy that provides us with multiple opportunities to improve the lives of patients who have or are at risk of developing prostate cancer. During the quarter, we continued to advance clinical trials of our three prostate cancer theranostic candidates, 1404, PyL, and 1095, reported positive data regarding our Automated Bone Scan Index (aBSI), and look forward to providing further updates on our progress throughout the year."

First Quarter and Recent Key Business Highlights

AZEDRA, Ultra-orphan radiotherapeutic candidate

Positive Topline Results for AZEDRA Announced in the First Quarter 2017. In March 2017, Progenics reported positive topline data from its Phase 2b registrational trial of AZEDRA for the treatment of rare and difficult-to-treat neuroendocrine tumors, pheochromocytoma and paraganglioma. The primary endpoint evaluated a proportion of patients who achieved a 50% or greater reduction in all antihypertensive medication for at least six months. Under the study protocol agreed to with the U.S. Food and Drug Administration (FDA), the primary endpoint was to be achieved if the lower limit of the two-sided 95% confidence interval was above 10%. In order to achieve this primary endpoint, a minimum of 12 of the total 68 evaluable patients must have a 50% or greater reduction of all antihypertensive medication for at least 6 months.

The trial met the primary endpoint with 17 of the 68 evaluable patients experiencing at least a 50% reduction in all antihypertensive medication for at least six months, with a 95% confidence interval lower limit of 16.15%. In addition, favorable data was reported for a key secondary endpoint, the proportion of patients with overall tumor response as measured by Response Evaluation Criteria in Solid Tumors (RECIST) criteria. Of the 64 RECIST evaluable patients, 92.2% of patients showed a best response of partial response or stable disease.

AZEDRA was generally well tolerated. The most common treatment emergent adverse events were nausea, thrombocytopenia, anemia, fatigue, leukopenia, and neutropenia. These events are consistent with those observed in prior AZEDRA studies.

Based on these data, the Company has already submitted non-clinical sections of the New Drug Application to the FDA and expects to complete the submission by mid-2017.
PSMA-Targeted Prostate Cancer Pipeline

Data Presentations Highlighting Utility of Automated Bone Scan Index (aBSI). In February 2017, data was presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary (ASCO GU) Meeting demonstrating the utility of Progenics’ aBSI to quantitatively assess total tumor burden during the course of disease progression as called for by the Prostate Cancer Working Group (PCWG). The data will also be presented at the upcoming ASCO (Free ASCO Whitepaper) 2017 meeting. Also at ASCO (Free ASCO Whitepaper), a prognostic analysis of aBSI in men with metastatic castration-resistant prostate cancer (mCRPC) using data from a Phase 3 trial of a prostate cancer therapeutic will be discussed in an oral presentation.

Phase 3 Study of 1404 Remains On-Track; Phase 2 Data Published in the Journal of Nuclear Medicine. Progenics continues to enroll patients in a Phase 3 study of 1404. The trial is designed to evaluate the specificity of the imaging agent in identifying patients without clinically significant prostate cancer as well as its sensitivity to identify patients with clinically significant disease, and will include approximately 450 patients. Progenics still expects to complete enrollment by the end of 2017.

In March 2017, data from a Phase 2 study of 1404 were published in the online edition of the Journal of Nuclear Medicine. The data reviewed previously reported results demonstrating the sensitivity of 1404 to detect prostate cancer, potentially enabling better assessment of the stage and extent of a patient’s prostate cancer versus biopsy.
Advancing Phase 2/3 Study of PyL. Progenics’ study evaluating the diagnostic accuracy of PyL PET/CT imaging in patients with metastatic prostate cancer continues to enroll patients. The Phase 2/3 study will enroll approximately 300 patients with recurrent and/or metastatic prostate cancer in the United States and Canada.

Initiated Phase 1 Trial of 1095. Dosing has commenced in the Phase 1 open-label dose escalation study of 1095. The study, which is being conducted at Memorial Sloan Kettering, is expected to enroll approximately 30 patients with mCRPC who have demonstrated tumor avidity to 1095. The study’s primary objectives are to determine the maximum tolerated dose, safety and tolerability, biodistribution, and efficacy.
RELISTOR, treatment for opioid-induced constipation (partnered with Valeant Pharmaceuticals International, Inc.)

First Quarter 2017 RELISTOR Net Sales of $14.1 Million. The first quarter 2017 sales, as reported to Progenics by its partner Valeant, translated to $2.1 million in royalty revenue for Progenics for the quarter.
First Quarter 2017 Financial Results

First quarter revenue totaled $2.3 million, down from $2.5 million in the first quarter of 2016, reflecting RELISTOR royalty income of $2.1 million compared to $2.2 million in the corresponding period of 2016.

First quarter research and development expenses increased by $0.9 million compared to the corresponding prior year period, resulting primarily from higher clinical trial and contract manufacturing expenses for PyL, partially offset by lower compensation expenses. First quarter general and administrative expenses decreased by $0.1 million compared to the corresponding prior year period, primarily attributable to lower depreciation and compensation expenses, partially offset by higher costs associated with pre-launch activities for AZEDRA. Progenics also recorded a non-cash charge of $1.9 million in the first quarter related to an increase in the fair value estimate of the contingent consideration liability primarily resulting from an increase in the probability of success of AZEDRA used to calculate the potential milestone payments to former Molecular Insight Pharmaceuticals, Inc. stockholders. For the three months ended March 31, 2017, Progenics recognized interest expense, including amortization of the debt discount, related to the RELISTOR royalty-backed loan, of $1.3 million.

Net loss attributable to Progenics for the quarter was $16.4 million or $0.23 per diluted share, compared to a net loss of $12.7 million or $0.18 per diluted share in the corresponding 2016 period. Progenics ended the quarter with cash and cash equivalents of $126.3 million, a decrease of $12.6 million compared to cash and cash equivalents as of December 31, 2016.

MEI Pharma, Inc. (Nasdaq: MEIP), an oncology company focused on the clinical development of novel therapies for cancer, reported financial results for its third quarter ended March 31, 2017 (Press release, MEI Pharma, MAY 4, 2017, View Source [SID1234518852]). The Company also outlined a number of key upcoming milestones.

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"I am proud of the progress we have made over the past quarter, with a steadfast focus on clinical study planning and execution, while maintaining a healthy cash position," said Daniel P. Gold, Ph.D., President and Chief Executive Officer of MEI Pharma. "We have been working diligently with our partners at Helsinn on the design and implementation of a large, international Phase 3 study of Pracinostat in acute myeloid leukemia (AML) and a Phase 2 dose-optimization study in myelodysplastic syndrome (MDS), and expect to enroll the first patients next month. We also look forward to presenting new genetic analysis data from our Phase 2 study in AML at ASCO (Free ASCO Whitepaper), which increases our understanding of the study’s patient demographics.

"Finally," Dr. Gold continued, "we look forward to initial safety and efficacy data from our clinical study of ME-401 in relapsed/refractory chronic lymphocytic leukemia (CLL) and follicular lymphoma next month. ME-401 is a differentiated oral PI3K delta inhibitor that is predicted to have a wide therapeutic window which may lead to safer treatment options for patients with lymphomas. We are very encouraged by the early results from our ongoing study and look forward to presenting them in more detail. While much of our recent progress has occurred behind the scenes, the stage is now set for what should be an exciting remainder of the year."

Upcoming Milestones

Initiation of global Phase 3 study of Pracinostat in AML. In August 2016, the Company entered into an exclusive license, development and commercialization agreement with Helsinn Healthcare SA, a Swiss pharmaceutical company, for the investigational drug candidate Pracinostat in AML and other potential indications (Helsinn License Agreement). Under the terms of the agreement, Helsinn is granted a worldwide exclusive license to develop, manufacture and commercialize Pracinostat, and is primarily responsible for funding its global development and commercialization. Site recruitment for the Phase 3 study of Pracinostat and azacitidine in newly diagnosed AML patients who are ≥75 years of age or unfit for intensive induction chemotherapy is ongoing.
First patient in Phase 2 dose-optimization study of Pracinostat in MDS. As part of the Helsinn License Agreement, the Company will work with Helsinn to determine an optimal dosing regimen of Pracinostat in combination with azacitidine for the treatment of high and very high risk MDS. The cost of this study will be shared by Helsinn and the Company, and enrollment is anticipated to commence in June 2017.
Gene mutation data from Phase 2 study of Pracinostat in AML at ASCO (Free ASCO Whitepaper). Data from a post hoc analysis of a Phase 2 clinical study of Pracinostat and azacitidine in elderly patients with AML who were not eligible for induction chemotherapy were accepted for presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago on Monday, June 5, 2017. The abstract, entitled "Correlation between Mutation Clearance and Clinical Response in Elderly Patients with Acute Myeloid Leukemia (AML) Treated with Azacitidine and Pracinostat," will be released on abstracts.asco.org at 5:00 pm EDT on May 17, 2017.
Interim data from Phase 1b study of ME-401 in CLL and follicular lymphoma. Interim safety and efficacy data from the first cohort in a Phase 1b clinical study of ME-401 in patients with relapsed/refractory CLL or follicular lymphoma are expected in June. ME-401 is a highly differentiated oral PI3K delta inhibitor that has a distinct chemical structure from other drugs in its class, including idelalisib (marketed as Zydelig). Results from a first-in-human study of ME-401 showed levels of drug exposure that support the potential for an improved therapeutic window compared to idelalisib, with a half-life that supports once-daily dosing.
Financial Highlights

In March 2017, the Company received a $5 million payment from Helsinn in accordance with the Helsinn License Agreement. The Company is also eligible to receive up to $444 million in potential regulatory and sales-based milestones, along with royalty payments on the net sales of Pracinostat.
As of March 31, 2017, the Company had $56.8 million in cash, cash equivalents and short-term investments, compared to $55.2 million as of December 31, 2016, with no outstanding debt. The Company believes its cash position will be sufficient to fund operations through at least the end of calendar year 2018.
Research and development expenses were $1.9 million for the three months ended March 31, 2017, and $5.2 million for the nine months ended March 31, 2017. This compares with research and development expenses of $3.4 million for the three months ended March 31, 2016, and $9.4 million for the nine months ended March 31, 2016. The decrease was primarily due to a reduction in expenses related to Pracinostat pursuant to the Helsinn License Agreement.
General and administrative expenses were $2.2 million for the three months ended March 31, 2017, and $6.8 million for the nine months ended March 31, 2017, compared to $2.0 million and $5.8 million, respectively, for the same periods in 2016. The increase was primarily due to professional service costs.
Revenues were $4.5 million during the three months ended March 31, 2017, and $22.8 million during the nine months ended March 31, 2017, related to the Helsinn License Agreement. During the three and nine months ended March 31, 2017, the cost of research and development revenue was $1.1 million and $4.0 million, respectively. Cost of research and development revenue is comprised primarily of reimbursable third-party pass-through costs.
Net loss was $0.6 million, or $0.02 per share, for the three months ended March 31, 2017, compared to $5.4 million, or $0.16 per share, for the quarter ended March 31, 2016. Net income was $7.0 million, or $0.19 per share, for the nine months ended March 31, 2017, compared to a net loss of $15.1 million, or $0.44 per share for the same period in 2016.