On May 9, 2016 Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to treat cancer, reported financial results for the first quarter ended March 31, 2016, and also provided an overview of certain corporate developments (Press release, Verastem, MAY 9, 2016, View Source;p=RssLanding&cat=news&id=2166539 [SID:1234512196]).

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"To date in 2016, Verastem has announced two new clinical collaborations with world-class organizations, including Merck KGaA and Pfizer, and Washington University in St. Louis and Merck & Co., to further elucidate the potential of FAK inhibition to enhance the efficacy of PD-(L)1 inhibitors in patients with pancreatic and ovarian cancer," said Robert Forrester, President and Chief Executive Officer of Verastem. "The data generated from these trials will continue to inform the ongoing development of our anti-cancer therapeutics which reduce cancer stem cells and modulate the local tumor microenvironment to allow both cancer treatments and the immune system to do their job more efficiently. We’ve had a strong start to 2016 with the announcement of these clinical collaborations in addition to attracting key strategic hires on the development team, including Dr. Greg Berk as Chief Medical Officer and Dr. Toyin Shonukan as Vice President of Clinical Development, to oversee and execute on our ongoing and future studies. We are well financed with approximately $100 million in available capital and we look forward to keeping you updated in the coming quarters on our progress."

First Quarter 2016 and Recent Highlights:
Focal Adhesion Kinase Inhibition Program
Clinical Collaboration with Pfizer and Merck KGaA to Evaluate Combination of VS-6063 and Avelumab in Ovarian Cancer – In March 2016, the companies announced a clinical trial collaboration agreement to evaluate the combination of Verastem’s focal adhesion kinase (FAK) inhibitor VS-6063 and Pfizer and Merck KGaA’s anti-PD-L1 immunotherapy avelumab. Verastem has previously reported initial signs of clinical activity in patients with ovarian cancer when VS-6063 is used in combination with paclitaxel. Under the terms of the agreement, the parties will conduct a planned Phase 1/1b clinical trial evaluating escalating doses of the combination of VS-6063 and avelumab as a potential treatment option for patients with advanced ovarian cancer.

Washington University in St. Louis Initiated a Clinical Study of VS-6063 in Combination with Merck & Co.’s Pembrolizumab and Gemcitabine in Pancreatic Cancer – In January 2016, Verastem announced the initiation of a Phase 1 dose-escalation study at Washington University to evaluate its FAK inhibitor VS-6063 in combination with Merck & Co.’s anti-PD-1 immunotherapy pembrolizumab and gemcitabine in patients with pancreatic cancer. The trial builds upon preclinical research conducted by Dr. David Denardo, presented at several conferences in late 2015 and early 2016, demonstrating the ability of FAK inhibition to increase the efficacy of checkpoint inhibition in the reduction of tumor volume and overall survival in models of pancreatic cancer. This Phase 1 clinical trial is currently enrolling and is anticipated to enroll approximately 50 patients with advanced pancreatic cancer.

Presented Scientific Data Supporting FAK Inhibition in Combination with Immunotherapy at Key Medical Meetings – During the first quarter of 2016, Verastem presented data in support of its new development programs focused on advancing its FAK inhibitors in combination with immune-oncology agents and other current and emerging standard of care cancer treatments. Data were presented at several medical and scientific meetings, including the 2016 American Academy of Cancer Research (AACR) (Free AACR Whitepaper), the Society for Gynecologic Oncology’s 2016 Annual Meeting on Women’s Cancer, the Keystone Symposium on Cancer Pathology, the Keystone Symposium on Stem Cells and Cancer, and Immunotherapy World 2016.

Presented Clinical Data from the Window of Opportunity Study at iMig 2016 – In May 2016, the Company announced results from the ongoing open-label, single-center, neoadjuvant Window of Opportunity study evaluating tolerability, along with biomarker and tumor volume response to VS-6063 (400mg BID) following either 12 days (Cohort 1) or 35 days (Cohort 2) of treatment in surgically-eligible patients with malignant pleural mesothelioma. Data analysis from Cohort 1 and Cohort 2 showed that VS-6063 was generally well tolerated with early signs of tumor reduction observed, with six of the twenty patients demonstrating an encouraging tumor reduction after brief treatment with VS-6063.

Development of VS-4718 Continues in Solid Tumors – Dosing continues in a Phase 1 dose escalation trial evaluating single-agent VS-4718 and a Phase 1 clinical trial evaluating VS-4718 in combination with gemcitabine and Abraxane is currently ongoing. Following results from the dose escalation trial, an expansion cohort of VS-4718 + Gemcitabine/Abraxane vs Gemcitabine/Abraxane alone in patients with pancreatic cancer is planned.

Dual PI3K/mTORC1/2 Inhibition Program
Confirmatory Recommended Phase 2 Dose and Expansion Cohorts – The maximum tolerated dose of single-agent VS-5584 has been reached in a Phase 1 study, and the recommended Phase 2 dose (RP2D) is being confirmed. Reductions in pharmacodynamic markers of PI3K and mTOR activity and clinical activity has been observed in some tumor types.

Corporate
Gregory I. Berk, MD Named Chief Medical Officer – In April 2016, the Company announced the appointment of Gregory I. Berk, MD as Chief Medical Officer. Dr. Berk, a highly accomplished physician and a well-regarded oncology veteran with more than 25 years of both industry and academic experience, will be responsible for leading the Company’s global clinical development strategy and clinical operations.

Announced Key Executive Management Appointments and Changes – In April 2016, the Company strengthened its management team through the appointment and promotion of several key individuals. Jonathan Pachter, PhD was promoted to Chief Scientific Officer, David Weaver, PhD was appointed Vice President, Translational Medicine, Joe Chiapponi, Vice President, Finance, was named Treasurer, Principal Accounting and Financial Officer and Oluwatoyin (Toyin) Shonukan, MD, has been appointed Vice President, Clinical Development. Dr. Shonukan most recently served as Senior Medical Director, Oncology Clinical Development at Vertex Pharmaceuticals and has held previous senior appointments at Millennium: The Takeda Oncology Company, Novartis Oncology and Eli Lilly.

First Quarter 2016 Financial Results
Net loss for the first quarter ended March 31, 2016 (2016 Quarter) was $8.3 million, or $0.22 per share, as compared to a net loss of $15.2 million, or $0.46 per share, for the first quarter ended March 31, 2015 (2015 Quarter). Net loss for the 2016 Quarter and 2015 Quarter, excluding non-cash stock-based compensation expense of $1.7 million and $2.9 million, was $6.6 million and $12.3 million, respectively.

Research and development expense for the 2016 Quarter was $4.2 million compared to $10.5 million for the 2015 Quarter. The $6.3 million decrease from the 2015 Quarter to the 2016 Quarter was primarily related to a decrease of $4.2 million in contract research organization expense for outsourced biology, chemistry, development and clinical services, which includes our clinical trial costs, a decrease in personnel related costs of $1.4 million, a decrease of approximately $550,000 in stock-based compensation, and a decrease of approximately $441,000 in travel, facilities and other research and development costs. These decreases were partially offset by an increase of approximately $276,000 in consulting fees.

General and administrative expense for the 2016 Quarter was $4.3 million compared to $4.7 million for the 2015 Quarter. The decrease of approximately $400,000 from the 2015 Quarter to the 2016 Quarter primarily resulted from approximate decreases in stock-based compensation expense of $734,000 and $148,000 in personnel related costs. These decreases were offset by an increase of approximately $411,000 in consulting and professional fees.

As of March 31, 2016, Verastem had cash, cash equivalents and investments of $99.5 million compared to $110.3 million as of December 31, 2015. Verastem used $10.8 million for operating activities during the 2016 Quarter settling one-time compensation payments, severance payments and paying down accounts payable and accruals.
The number of outstanding common shares as of March 31, 2016, was 36,992,418.

Financial Guidance
Based on current operating plans, we expect to have sufficient cash, cash equivalents and short-term investments to fund our research and development programs and operations into 2018.

About Focal Adhesion Kinase
Focal Adhesion Kinase (FAK) is a non-receptor tyrosine kinase encoded by the PTK-2 gene that is involved in cellular adhesion and, in cancer, metastatic capability. VS-6063 (defactinib) and VS-4718 are orally available compounds that are potent inhibitors of FAK. VS-6063 and VS-4718 utilize a multi-faceted approach to treat cancer by reducing cancer stem cells, enhancing anti-tumor immunity, and modulating the local tumor microenvironment. VS-6063 and VS-4718 are currently being studied in multiple clinical trials for their ability to improve patient survival.

About VS-5584
VS-5584 is an orally available compound that has demonstrated potent and highly selective activity against class 1 PI3K enzymes and dual inhibitory actions against mTORC1 and mTORC2. In preclinical studies, VS-5584 has been shown to reduce the percentage of cancer stem cells and induce tumor regression in chemotherapy-resistant models. Verastem is currently conducting a dose escalation trial of VS-5584 in patients with advanced solid tumors.

On May 9, 2016 Novocure (NASDAQ: NVCR), a commercial stage oncology company pioneering a novel therapy for solid tumors, reported financial results for the first quarter ended March 31, 2016, highlighting strong growth in prescriptions, active patients and reported revenues (Press release, NovoCure, MAY 9, 2016, View Source [SID:1234512153]).

First quarter 2016 highlights include:
Three months ended March 31,
2016 2015 % change
Non-financial
Prescriptions received in period(1) 755 437 73 %
Active patients at period end(2) 797 372 114 %

Financial, in millions (unaudited)

Revenues(3) $ 13.1 $ 5.2 151 %

Net loss $ (35.4 ) $ (23.3 )

Cash and cash equivalents at the end of period $ 115.9 $ 74.2
Short-term investments at the end of period $ 119.8 $ 22.0

(1) A "prescription received" is a commercial order for Optune that is received from a physician certified to treat patients with Tumor Treating Fields (TTFields) therapy for a patient not previously on TTFields therapy. Orders to renew or extend treatment are not included in this total. In the future, Novocure may have regulatory approvals and commercial programs for multiple clinical indications, at which time Novocure will recognize a commercial order as a prescription for the same patient for each clinical indication treated. For example, in the future, a patient may have a prescription for the treatment of lung cancer and a prescription for the treatment of brain metastases from the lung cancer.
(2) An "active patient" is a patient who is on TTFields therapy under a commercial prescription order as of the measurement date, including patients who may be on a temporary break from treatment and who plan to resume treatment in less than 60 days.
(3) For the reported periods, all revenues were recognized when cash was collected and all other revenue recognition criteria had been met.

"I am pleased to see commercial momentum building following the October 2015 FDA approval of Optune for the treatment of newly diagnosed GBM and the December 2015 publication of EF-14 phase 3 pivotal trial results in the Journal of the American Medical Association," commented Asaf Danziger, Chief Executive Officer. "Our sales and marketing teams are intensely focused on the education of oncologists as we introduce a completely new treatment modality to the market. I am extremely proud of the hard-earned progress we have made in the two quarters since approval." Mr. Danziger continued, "I believe that we are well positioned to deliver substantial year-over-year growth in 2016. We expect the incorporation of Optune for newly diagnosed GBM into treatment guidelines, the launch, pending FDA approval, of our second generation Optune system in the United States, and the continued expansion of our U.S. and German sales forces to provide additional catalysts to support our commercial execution. We are committed to bringing Optune to all GBM patients who may benefit from it." Wilco Groenhuysen, Chief Financial Officer, stated, "2015 started a transformational phase for Novocure with the October FDA approval of Optune for newly diagnosed GBM. I am pleased to report triple digit growth in both active patients and revenues in the first quarter of 2016 compared to the same period in 2015 and believe we will continue to see significant year-over-year growth in operating statistics and revenues throughout 2016." "As our commercial teams focus on execution within the glioblastoma indication, our R&D team continues to make progress toward developing TTFields for a variety of additional solid tumors," added William Doyle, Chairman. "We have five ongoing or completed phase 2 pilot trials in brain metastases, non-small cell lung cancer, ovarian cancer, pancreatic cancer and mesothelioma. We plan to open phase 3 pivotal trials in brain metastases and non-small cell lung cancer in 2016. In addition to our clinical development activities, our engineering team continues its work to improve all aspects of TTFields delivery to enhance ease-of-use for patients." 2016 First Quarter Operating Statistics and Financial Update Prescriptions in the quarter ended March 31, 2016, increased by 318 prescriptions, or 73 percent, compared to the same period in 2015. The increase in prescriptions was driven primarily by commercial activities in the United States after the October 2015 U.S. Food and Drug Administration (FDA) approval of Optune for the treatment of newly diagnosed glioblastoma (GBM), increased commercial activities in Germany, and enhanced awareness of Optune following the December 2015 publication of EF-14 phase 3 pivotal trial results in the Journal of the American Medical Association.
In the United States, 684 prescriptions were received in the quarter ended March 31, 2016, an increase of 273 prescriptions, or 66 percent, compared to the same period in 2015.
In Germany and other EMEA markets, 71 prescriptions were received in the quarter ended March 31, 2016, an increase of 45 prescriptions, or 173 percent, compared to the same period in 2015.
The conversion of prescriptions to new patients is driven by the prescription fill rate and the time to fill the prescription. The prescription fill rate for the 12 months ended March 31, 2016, was 75 percent. The increase or decrease in active patients in any given period reflects the number of new patients less the number of patients discontinuing therapy. The rate of patients discontinuing therapy is determined by the treatment duration for patients starting therapy in prior periods. Novocure expects average treatment duration of its total active patient population to increase as the ratio of newly diagnosed GBM to recurrent GBM active patients increases. The portion of Optune prescriptions for newly diagnosed GBM was more than 50 percent in the first quarter of 2016. There were 797 active patients on Optune therapy at March 31, 2016, an increase of 425 active patients, or 114 percent, compared to March 31, 2015. The increase in active patients was driven both by prescription growth and by an increase in the percentage of newly diagnosed GBM patients who started Optune in prior periods.
In the United States, there were 699 active patients on Optune therapy at March 31, 2016, an increase of 352 active patients, or 101 percent, compared to March 31, 2015.
In Germany and other EMEA markets, there were 98 active patients on Optune therapy at March 31, 2016, an increase of 73 active patients, or 292 percent, compared to March 31, 2015.
For the 12 months ended March 31, 2016, the average cash payment received was more than $14,000 per charged month in the United States. The difference between billed and paid amounts consists of patient financial assistance, charitable care, discounts, disputed underpayments and indirect taxes. Generally, Novocure’s average time to collect on billed charges ranges between four and five months in the United States. The "payment amount" and "average time to collect" metrics do not include Novocure’s experience with patients covered by the Medicare fee-for-service program, as Novocure has not received material payments from the Medicare fee-for-service program, or Novocure’s experience with patients outside of the United States. For the three months ended March 31, 2016, revenues increased to $13.1 million compared to $5.2 million for the same period in 2015, representing 151 percent growth. This growth was primarily driven by increased Optune adoption. For the three months ended March 31, 2016, cost of revenues increased to $8.0 million compared to $3.9 million for the same period in 2015, representing 105 percent growth. This was primarily driven by an increase in the cost of transducer array shipments due to an increase in active Optune patients as well as increased personnel costs to establish infrastructure necessary to support an increasing volume of shipments to patients. Research, development and clinical trials expenses for the three months ended March 31, 2016, were $11.4 million compared to $9.9 million for the same period in 2015, representing growth of 15 percent. This growth was primarily due to increased personnel costs and increased expenses related to clinical education and investigator-sponsored trials, partially offset by reduced expenses related to the development of the second generation Optune system. Sales and marketing expenses for the three months ended March 31, 2016, were $13.3 million compared to $6.4 million for the same period in 2015, representing growth of 109 percent. This growth was primarily due to increased personnel costs as well as increased marketing expenses to expand commercial operations in the United States and Germany and to establish commercial operations in Switzerland and Japan. General and administrative expenses for the three months ended March 31, 2016, were $12.3 million compared to $7.0 million for the same period in 2015, representing growth of 76 percent. This growth was primarily due to increased personnel costs as well as increased expenses related to professional services and activities associated with being a public company. Personnel costs for the three months ended March 31, 2016, included $5.5 million in non-cash share-based compensation expenses, comprised of $0.1 million in cost of revenues; $0.8 million in research, development and clinical trials; $1.3 million in sales and marketing; and $3.2 million in general and administrative expenses. Total non-cash share-based compensation expenses for Q1 2015 were $1.8 million. Net losses for the three months ended March 31, 2016, were $35.4 million compared to $23.3 million for the same period in 2015. As of March 31, 2016, we had $115.9 million in cash and cash equivalents and $119.8 million in short-term investments for a total balance of $235.8 million in cash, cash equivalents and short-term investments. Other first quarter 2016 corporate achievements
Expanded coverage: Novocure expanded coverage of Optune for the treatment of newly diagnosed and/or recurrent GBM to include more than 63 million additional lives through new policies with Anthem, Cigna, Humana, Regence Blue Cross Blue Shield, Preferred One, Univera Healthcare, and Asuris Northwest Health. This brought the total number of covered lives to more than 107 million in the United States as of March 31, 2016. Subsequent to the quarter end, positive coverage polices were issued by Group Health Cooperative Washington and Idaho, and Blue Cross Blue Shield of Michigan, bringing the total number of covered lives to more than 112 million in the United States as of May 1, 2016.
Second generation Optune system European launch: All Optune patients in Europe are now on the second generation of Optune, which is less than half the weight and half the size of the original commercial system.
PANOVA phase 2 pilot data in pancreatic cancer: Results from the first cohort of the PANOVA trial were presented at ASCO (Free ASCO Whitepaper) GI suggesting TTFields therapy plus chemotherapy may be safe as first-line treatment and improve survival of patients with advanced pancreatic cancer. Progression free survival and overall survival of patients treated with TTFields combined with gemcitabine were more than double those of gemcitabine-treated historical controls. Of the evaluable tumors, 30 percent had partial response and another 30 percent had stable disease.
Recognition as a clinical cancer advance by ASCO (Free ASCO Whitepaper): Phase 3 pivotal trial results of TTFields in combination with temozolomide for the treatment of newly diagnosed GBM were selected for inclusion in the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper)’s Clinical Cancer Advances 2016: Annual Report on Progress against Cancer. The report reviews the recent top advances and emerging trends in clinical cancer research that are leading to improved cancer treatments for patients.
Anticipated milestones over the next nine months
NCCN guidelines update: With the October 2015 FDA approval of Optune for the treatment of newly diagnosed GBM, Novocure expects that Optune should be added to the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for Central Nervous System Cancers for newly diagnosed GBM in 2016.
FDA approval for the second generation Optune system: In December 2015, Novocure filed a premarket approval (PMA) supplement application with the FDA seeking approval of the second generation of Optune for the approved indications. The company subsequently received and, in April 2016, responded to questions from the FDA on the PMA supplement application. Assuming no further FDA comments or requests for additional information, the company hopes to begin marketing the second generation Optune system in the United States in the third quarter of 2016.
PANOVA phase 2 pilot trial last patient in: Following the approval of nab-paclitaxel, a taxane-based chemotherapy, for the treatment of advanced pancreatic cancer, the PANOVA study was expanded to include 20 additional patients treated with TTFields in combination with nab-paclitaxel and gemcitabine. Novocure expects to finish enrollment of the second patient cohort in 2016 and, with an expected six months follow-up, anticipates phase 2 pilot data will be available for presentation in 2017.
INNOVATE phase 2 pilot trial last patient in: In October 2014, the INNOVATE trial opened to study TTFields together with weekly paclitaxel for patients with recurrent ovarian cancer. The trial is expected to finish enrollment in 2016 and, with an expected six months follow-up, Novocure anticipates phase 2 pilot data will be available for presentation in 2017.
METIS phase 3 pivotal trial first patient in: Based upon pre-clinical data showing TTFields can prevent metastatic seeding in vivo and the established safety and efficacy of TTFields in GBM, Novocure believes TTFields could be an effective treatment for patients with brain metastases. Novocure submitted a pre-submission package to the FDA for discussion and feedback in December 2015 and submitted a full investigational device exemption (IDE) application in April 2015. The open-label randomized study will test the effectiveness of TTFields following stereotactic radiosurgery (SRS) compared with watchful waiting alone in patients with brain metastases stemming from non-small cell lung cancer. Novocure plans to open the trial in mid-2016 subject to FDA approval of the IDE.
LUNAR phase 3 pivotal trial first patient in: Novocure has completed a phase 2 pilot trial in advanced non-small cell lung cancer and is planning a randomized phase 3 pivotal trial for the treatment of non-small cell lung cancer. Given recent preclinical research of TTFields in combination with PD-1 inhibitors, the company is currently reviewing the protocol design to possibly capitalize on the potential synergies with taxane-based chemotherapies and the potential additivity with PD-1 inhibitors. The company plans to open the trial in late 2016 subject to FDA approval of the IDE.
Japanese approval for newly diagnosed GBM: Novocure submitted a partial amendment application to the Japanese Pharmaceuticals and Medical Devices Agency for the treatment of newly diagnosed GBM patients in December 2015 and hopes to receive Japanese Ministry of Health, Labour and Welfare (MHLW) approval in late 2016. The MHLW approved the use of Optune in patients with recurrent GBM in March 2015. The company plans to wait until MHLW approval for newly diagnosed GBM before submitting an application for public reimbursement in Japan.

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On May 9, 2016 Kite Pharma, Inc. (Nasdaq: KITE), a clinical-stage biopharmaceutical company focused on developing engineered autologous cell therapy (eACT) products for the treatment of cancer, reported a corporate update and reported first quarter 2016 financial results for the period ended March 31, 2016 (Press release, Kite Pharma, MAY 9, 2016, View Source [SID:1234512151]).

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"Our ZUMA-1 update at AACR (Free AACR Whitepaper) last month highlighted the potential of KTE-C19, our breakthrough immunotherapy candidate, to put patients with chemorefractory aggressive non-Hodgkin lymphoma (NHL), a patient population burdened with a short life expectancy and limited treatment options, into a durable complete remission," noted Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer. "We remain on track to provide interim data from the pivotal phase 2 portion of ZUMA-1 later this year and plan to submit the KTE-C19 registration filing to the U.S. Food and Drug Administration (FDA) by the end of 2016."

"Our commitment to delivering a breakthrough personalized cell therapy to cancer patients now extends fully to our manufacturing and market planning areas. Validation and qualification activities at our state-of-the-art commercial manufacturing facility are underway. The commercial team is actively evaluating a broad range of market and payor strategies for making KTE-C19 available to patients with a significant unmet need."

First Quarter 2016 and Recent Highlights

At AACR (Free AACR Whitepaper), reported rapid and durable responses in patients with chemorefractory diffuse large B cell lymphoma treated with KTE-C19 in the phase 1 portion of ZUMA-1.
Ongoing complete responses were observed in 3 of 7 patients at 9-month study follow-up (1 patient) and 6-month study follow-up (2 patients).
KTE-C19-related adverse events consisted predominantly of cytokine release syndrome and neurotoxicity, which were generally reversible.
Partnered with Genentech to study KTE-C19 in combination with the checkpoint inhibitor atezolizumab. Kite expects to initiate a Phase 1b/2 combination study in patients with chemorefractory diffuse large B cell lymphoma in the second half of 2016.
Expanded our clinical and research partnership with the National Cancer Institute (NCI) by entering into a new Cooperative Research and Development Agreement (CRADA) with James (Jim) N. Kochenderfer, M.D., and the NCI’s Experimental Transplantation and Immunology Branch.
Phase 1 study of human anti-CD19 chimeric antigen receptor for treating B-cell malignancies currently ongoing.
Also at AACR (Free AACR Whitepaper), William Lu, Ph.D., a collaborator of Kite’s at the NCI, reported data from a Phase 1 study of a T cell receptor (TCR) product candidate targeting MAGE-A3 that was advanced under Kite’s CRADA with the Surgery Branch at the NCI.
Data reported at AACR (Free AACR Whitepaper) support Kite’s plan to file later this year an investigational new drug (IND) application for a TCR product candidate that targets a MAGE-A3 antigen expressed on solid tumors.
Entered into a research and license agreement with Leiden University Medical Center in the Netherlands to identify and develop additional TCR product candidates targeting solid tumors that are associated with the human papillomavirus (HPV) type 16 infection.
Appointed Tim Moore, a biopharma executive with more than 30 years of global operations experience, as Executive Vice President, Technical Operations, to lead product development, manufacturing, supply chain, quality assurance, and end-to-end process optimization for all aspects of Kite’s engineered T cell product candidates.
Augmented Kite’s commercial function, under the leadership of Chief Commercial Officer Shawn Tomasello, with the appointment of an integrated executive team responsible for all aspects of commercial and medical affairs strategy, planning, and analysis for the potential launch of KTE-C19.
First Quarter 2016 Financial Results

Revenue was $5.1 million for the first quarter of 2016.
Research and development expenses were $34.4 million for the first quarter of 2016, and include $8.5 million of non-cash stock-based compensation expense.
General and administrative expenses were $16.5 million for the first quarter of 2016, and include $6.4 million of non-cash stock-based compensation expense.
Net loss was $43.9 million, or $0.90 per share, for the first quarter of 2016.
Non-GAAP net loss for the first quarter of 2016 was $29.1 million, or $0.60 per share, which excludes non-cash stock-based compensation of $14.9 million.
As of March 31, 2016, Kite had $577.4 million in cash, cash equivalents, and marketable securities.
Kite continues to expect the full year 2016 net cash burn to be $235 to $250 million dollars, which includes approximately $20 million in capital expenditures, but excludes any inflows or outflows from business development activities. The estimated full year 2016 net cash burn is primarily driven by an estimated net loss of $295 to $310 million, which includes an estimated $80 million of non-cash stock-based compensation expense.

On May 9, 2016 Dicerna Pharmaceuticals, Inc. (Nasdaq:DRNA), a leading developer of investigational RNA interference (RNAi) therapeutics, reported financial and operational results for the first quarter ended March 31, 2016 (Press release, Dicerna, MAY 9, 2016, View Source [SID:1234512150]).

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"We continue to achieve milestones across our portfolio of opportunities, including program launches with our DsiRNA-EX Conjugate technology, as well as our DCR-PH1 and DCR-MYC clinical programs," said Douglas M. Fambrough, Ph.D., president and chief executive officer of Dicerna. "We look forward to sharing new preclinical data for our conjugate programs as well as further updates on our clinical programs at our upcoming Investor Day."

Technology Update

Subcutaneous delivery to the liver with DsiRNA-EX Conjugates: Dicerna’s Dicer substrate short interfering RNA extended (DsiRNA-EX) Conjugates are proprietary RNAi therapeutic candidates rationally designed to enable convenient subcutaneous delivery for Dicerna’s emerging pipeline of liver-targeted RNAi investigational therapies. These conjugates do not involve lipid nanoparticles and are built on the DsiRNA-EX platform, using an extension to one end of the double-stranded DsiRNA molecule. These extensions are unique to Dicerna and utilize proprietary RNA structures that enable a differentiated and independent approach to subcutaneous delivery of RNAi-inducing therapeutics.

Dicerna is enhancing and extending the potency and reach of its DsiRNA-EX Conjugate technology, and has now achieved an IC50 level as low as 0.1 mg/kg in mice, generating greater than 90% gene knockdown at a dose of 1 mg/kg. Dicerna believes that potency in this range will translate into simple, infrequent single-shot dosing regimens in patients.
Dicerna will present extensive preclinical data for its DsiRNA-EX Conjugate programs, including non-human primate data against multiple gene targets, at the Company’s first Investor Day on June 29, 2016 in New York City.
Dicerna is on track to launch three DsiRNA-EX Conjugate programs in 2016, including an ongoing subcutaneous program for the treatment of primary hyperoxaluria that is advancing into IND-enabling studies.
Dicerna’s emerging library of DsiRNA-EX Conjugate molecules encompasses subcutaneously administered inhibitors for more than a dozen liver disease gene targets at various levels of optimization. These gene targets are in the fields of rare diseases as well as chronic liver disease, cardiovascular disease, and viral infectious disease. We expect these emerging programs will enable Dicerna to build a deep and broad pipeline of liver-targeted therapies, and create extensive opportunity for partnership and collaboration leading to additional program launches.
Rare Disease Program Update

DCR-PH1: DCR-PH1 is an intravenously infused DsiRNA-EX-based therapeutic candidate for primary hyperoxaluria type 1 (PH1), a severe, rare genetic disease of liver metabolism that often results in life-threatening damage to the kidneys. In a genetic mouse model of PH1, DCR-PH1 knocked down the activity of the HAO1 gene transcript that encodes for the enzyme glycolate oxidase, thereby significantly reducing the production of oxalate, the key mediator of disease pathology in PH1. Similar results, if obtained in PH1 patients, may have significant clinical benefit. In non-human primate studies, a single dose of DCR-PH1 led to an average of 84% knockdown of the HAO1 gene transcript. The DCR-PH1 clinical program consists of the following studies:

PHYOS: During the fourth quarter of 2015, Dicerna initiated PHYOS (Primary Hyperoxaluria Observational Study), an international, multicenter, observational study in patients with a genetically confirmed diagnosis of PH1. PHYOS is designed to measure biomarkers implicated in PH1 and to identify patients who may be eligible for the Phase 1 trial of DCR-PH1.
Healthy Volunteer Study: Dicerna continues to enroll in DCR-PH1-102, a Phase 1 dose escalation trial of DCR-PH1 in healthy volunteers. The primary objective of this study is to determine the safety profile of DCR-PH1 in healthy volunteers in order to support dosing of PH1 patients in the United States. This trial was initiated in the fourth quarter of 2015.
PH1 Patient Study: During the second quarter of 2016, Dicerna initiated DCR-PH1-101, a Phase 1 dose escalation trial in PH1 patients, in Germany. Dicerna anticipates initiating additional sites in Europe and the United States during the second-half of 2016. The primary objective of this study is to determine the safety and tolerability of single ascending doses of DCR-PH1, and secondary objectives include measurement of the drug’s PK properties, and corresponding PD measurements of oxalate (associated with disease progression) and glycolate (a marker of DCR-PH1 activity). Dicerna expects to complete this trial in the first half of 2017.
Oncology Program Update

DCR-MYC: DCR-MYC is a potent and specific inhibitor of MYC, an oncogene frequently amplified or overexpressed in a wide variety of tumor types, including hepatocellular carcinoma (HCC). MYC has long been considered "undruggable" with small molecule and antibody technologies. DCR-MYC is a Dicer substrate short interfering RNA (DsiRNA)-based therapeutic formulated in Dicerna’s EnCore lipid nanoparticle for delivery to solid tumors. In preclinical studies, DCR-MYC knocked down MYC transcript levels and significantly reduced tumor volume in multiple mouse tumor models, including models of HCC. DCR-MYC is currently being tested in two ongoing clinical trials.

Phase 1 DCR-MYC Trial in Solid Tumors

Having established safety at 1.0 mg/kg, Dicerna has initiated two expansion cohorts of this Phase 1 clinical trial. The first expansion cohort is enrolling patients with low-to-intermediate grade pancreatic neuroendocrine tumors (PNETs) having failed one or two lines of prior therapy. The second expansion cohort will enroll patients who will undergo pre- and post-treatment biopsies in order to directly assess molecular markers of RNAi activity against the MYC transcript. Direct observation of RNAi activity of DCR-MYC, combined with observations both of anti-tumor activity and inhibition of FDG uptake in tumors, will establish proof-of-concept for the RNAi-based mechanism of action of DCR-MYC. The Company expects to have these proof-of-concept data in 2016.
Phase 1 DCR-MYC Trial in Hepatocellular Carcinoma (HCC)

In December 2014 Dicerna initiated a Phase 1b/2 clinical trial of DCR-MYC in patients with advanced HCC. As of April 26, 2016, 19 patients have been treated with DCR-MYC in six cohorts. The current dosing level is 0.85 mg/kg. While an objective response based on modified Response Evaluation Criteria in Solid Tumors (mRECIST) has not been observed, a reduction in circulating alpha-fetoprotein level, a marker associated with anti-tumor activity, has been observed. The trial continues with dose escalation. Dicerna expects to have preliminary data from the HCC trial by the end of 2016.
Corporate Update

Dicerna announces today that it plans to host an Investor Day on June 29, 2016 in New York City to present new preclinical data for its emerging pipeline of DsiRNA-EX Conjugate programs, as well as an update on its PH1 and oncology clinical programs.

Financial Results

Cash Position – As of March 31, 2016, the Company had $80.6 million in cash and cash equivalents and held-to-maturity investments as compared to $94.6 million in cash and cash equivalents and held-to-maturity investments as of December 31, 2015. In addition, the Company had $1.1 million of restricted cash, which reflects collateral securing its lease obligations.
R&D Expenses – Research and development (R&D) expenses for the first quarter were $11.3 million, compared to $8.7 million for the same period in 2015. The increase in R&D expenses was due primarily to increased expenses related to discovery and early development of future programs including development of the DsiRNA-EX Conjugate delivery platform, increases in clinical activities from initiating additional studies in PH1, increased employee-related expenses primarily due to additional hiring during the period, along with stock-based compensation, and increased occupancy costs.
G&A Expenses – General and administrative (G&A) expenses for the first quarter were $4.5 million, compared to $5.4 million for the same period in 2015. The decrease in these costs was primarily due to stock-based compensation and payroll related cost savings, and a decrease in costs related to the facility move incurred in the first quarter of 2015.
Net Loss – Net loss for the first quarter was $15.7 million compared to a net loss of $14.1 million for the same period in 2015.
For more detailed information and analysis see the Company’s Quarterly Report on Form 10-Q, which was filed with the Securities and Exchange Commission (SEC) on May 9, 2016.

Guidance

Based on Dicerna’s current cash position and operating plan, the Company reiterates its expectation that it has sufficient cash to fund operations for at least the next 12 months. This estimate assumes no additional funding from new partnership agreements or debt or equity financing events.

On May 9, 2016 Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to treat cancer, reported financial results for the first quarter ended March 31, 2016, and also provided an overview of certain corporate developments (Press release, Verastem, MAY 9, 2016, View Source;p=irol-newsArticle&ID=2166539 [SID:1234512149]).

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"To date in 2016, Verastem has announced two new clinical collaborations with world-class organizations, including Merck KGaA and Pfizer, and Washington University in St. Louis and Merck & Co., to further elucidate the potential of FAK inhibition to enhance the efficacy of PD-(L)1 inhibitors in patients with pancreatic and ovarian cancer," said Robert Forrester, President and Chief Executive Officer of Verastem. "The data generated from these trials will continue to inform the ongoing development of our anti-cancer therapeutics which reduce cancer stem cells and modulate the local tumor microenvironment to allow both cancer treatments and the immune system to do their job more efficiently. We’ve had a strong start to 2016 with the announcement of these clinical collaborations in addition to attracting key strategic hires on the development team, including Dr. Greg Berk as Chief Medical Officer and Dr. Toyin Shonukan as Vice President of Clinical Development, to oversee and execute on our ongoing and future studies. We are well financed with approximately $100 million in available capital and we look forward to keeping you updated in the coming quarters on our progress."

First Quarter 2016 and Recent Highlights:
Focal Adhesion Kinase Inhibition Program
Clinical Collaboration with Pfizer and Merck KGaA to Evaluate Combination of VS-6063 and Avelumab in Ovarian Cancer – In March 2016, the companies announced a clinical trial collaboration agreement to evaluate the combination of Verastem’s focal adhesion kinase (FAK) inhibitor VS-6063 and Pfizer and Merck KGaA’s anti-PD-L1 immunotherapy avelumab. Verastem has previously reported initial signs of clinical activity in patients with ovarian cancer when VS-6063 is used in combination with paclitaxel. Under the terms of the agreement, the parties will conduct a planned Phase 1/1b clinical trial evaluating escalating doses of the combination of VS-6063 and avelumab as a potential treatment option for patients with advanced ovarian cancer.

Washington University in St. Louis Initiated a Clinical Study of VS-6063 in Combination with Merck & Co.’s Pembrolizumab and Gemcitabine in Pancreatic Cancer – In January 2016, Verastem announced the initiation of a Phase 1 dose-escalation study at Washington University to evaluate its FAK inhibitor VS-6063 in combination with Merck & Co.’s anti-PD-1 immunotherapy pembrolizumab and gemcitabine in patients with pancreatic cancer. The trial builds upon preclinical research conducted by Dr. David Denardo, presented at several conferences in late 2015 and early 2016, demonstrating the ability of FAK inhibition to increase the efficacy of checkpoint inhibition in the reduction of tumor volume and overall survival in models of pancreatic cancer. This Phase 1 clinical trial is currently enrolling and is anticipated to enroll approximately 50 patients with advanced pancreatic cancer.

Presented Scientific Data Supporting FAK Inhibition in Combination with Immunotherapy at Key Medical Meetings – During the first quarter of 2016, Verastem presented data in support of its new development programs focused on advancing its FAK inhibitors in combination with immune-oncology agents and other current and emerging standard of care cancer treatments. Data were presented at several medical and scientific meetings, including the 2016 American Academy of Cancer Research (AACR) (Free AACR Whitepaper), the Society for Gynecologic Oncology’s 2016 Annual Meeting on Women’s Cancer, the Keystone Symposium on Cancer Pathology, the Keystone Symposium on Stem Cells and Cancer, and Immunotherapy World 2016.

Presented Clinical Data from the Window of Opportunity Study at iMig 2016 – In May 2016, the Company announced results from the ongoing open-label, single-center, neoadjuvant Window of Opportunity study evaluating tolerability, along with biomarker and tumor volume response to VS-6063 (400mg BID) following either 12 days (Cohort 1) or 35 days (Cohort 2) of treatment in surgically-eligible patients with malignant pleural mesothelioma. Data analysis from Cohort 1 and Cohort 2 showed that VS-6063 was generally well tolerated with early signs of tumor reduction observed, with six of the twenty patients demonstrating an encouraging tumor reduction after brief treatment with VS-6063.

Development of VS-4718 Continues in Solid Tumors – Dosing continues in a Phase 1 dose escalation trial evaluating single-agent VS-4718 and a Phase 1 clinical trial evaluating VS-4718 in combination with gemcitabine and Abraxane is currently ongoing. Following results from the dose escalation trial, an expansion cohort of VS-4718 + Gemcitabine/Abraxane vs Gemcitabine/Abraxane alone in patients with pancreatic cancer is planned.

Dual PI3K/mTORC1/2 Inhibition Program
Confirmatory Recommended Phase 2 Dose and Expansion Cohorts – The maximum tolerated dose of single-agent VS-5584 has been reached in a Phase 1 study, and the recommended Phase 2 dose (RP2D) is being confirmed. Reductions in pharmacodynamic markers of PI3K and mTOR activity and clinical activity has been observed in some tumor types.

Corporate
Gregory I. Berk, MD Named Chief Medical Officer – In April 2016, the Company announced the appointment of Gregory I. Berk, MD as Chief Medical Officer. Dr. Berk, a highly accomplished physician and a well-regarded oncology veteran with more than 25 years of both industry and academic experience, will be responsible for leading the Company’s global clinical development strategy and clinical operations.

Announced Key Executive Management Appointments and Changes – In April 2016, the Company strengthened its management team through the appointment and promotion of several key individuals. Jonathan Pachter, PhD was promoted to Chief Scientific Officer, David Weaver, PhD was appointed Vice President, Translational Medicine, Joe Chiapponi, Vice President, Finance, was named Treasurer, Principal Accounting and Financial Officer and Oluwatoyin (Toyin) Shonukan, MD, has been appointed Vice President, Clinical Development. Dr. Shonukan most recently served as Senior Medical Director, Oncology Clinical Development at Vertex Pharmaceuticals and has held previous senior appointments at Millennium: The Takeda Oncology Company, Novartis Oncology and Eli Lilly.

First Quarter 2016 Financial Results
Net loss for the first quarter ended March 31, 2016 (2016 Quarter) was $8.3 million, or $0.22 per share, as compared to a net loss of $15.2 million, or $0.46 per share, for the first quarter ended March 31, 2015 (2015 Quarter). Net loss for the 2016 Quarter and 2015 Quarter, excluding non-cash stock-based compensation expense of $1.7 million and $2.9 million, was $6.6 million and $12.3 million, respectively.

Research and development expense for the 2016 Quarter was $4.2 million compared to $10.5 million for the 2015 Quarter. The $6.3 million decrease from the 2015 Quarter to the 2016 Quarter was primarily related to a decrease of $4.2 million in contract research organization expense for outsourced biology, chemistry, development and clinical services, which includes our clinical trial costs, a decrease in personnel related costs of $1.4 million, a decrease of approximately $550,000 in stock-based compensation, and a decrease of approximately $441,000 in travel, facilities and other research and development costs. These decreases were partially offset by an increase of approximately $276,000 in consulting fees.

General and administrative expense for the 2016 Quarter was $4.3 million compared to $4.7 million for the 2015 Quarter. The decrease of approximately $400,000 from the 2015 Quarter to the 2016 Quarter primarily resulted from approximate decreases in stock-based compensation expense of $734,000 and $148,000 in personnel related costs. These decreases were offset by an increase of approximately $411,000 in consulting and professional fees.

As of March 31, 2016, Verastem had cash, cash equivalents and investments of $99.5 million compared to $110.3 million as of December 31, 2015. Verastem used $10.8 million for operating activities during the 2016 Quarter settling one-time compensation payments, severance payments and paying down accounts payable and accruals.
The number of outstanding common shares as of March 31, 2016, was 36,992,418.

Financial Guidance
Based on current operating plans, we expect to have sufficient cash, cash equivalents and short-term investments to fund our research and development programs and operations into 2018.

About Focal Adhesion Kinase
Focal Adhesion Kinase (FAK) is a non-receptor tyrosine kinase encoded by the PTK-2 gene that is involved in cellular adhesion and, in cancer, metastatic capability. VS-6063 (defactinib) and VS-4718 are orally available compounds that are potent inhibitors of FAK. VS-6063 and VS-4718 utilize a multi-faceted approach to treat cancer by reducing cancer stem cells, enhancing anti-tumor immunity, and modulating the local tumor microenvironment. VS-6063 and VS-4718 are currently being studied in multiple clinical trials for their ability to improve patient survival.

About VS-5584
VS-5584 is an orally available compound that has demonstrated potent and highly selective activity against class 1 PI3K enzymes and dual inhibitory actions against mTORC1 and mTORC2. In preclinical studies, VS-5584 has been shown to reduce the percentage of cancer stem cells and induce tumor regression in chemotherapy-resistant models. Verastem is currently conducting a dose escalation trial of VS-5584 in patients with advanced solid tumors