On April 27, 2017 Novocure (NASDAQ: NVCR) reported financial results for the three months ended March 31, 2017, highlighting year-over-year and sequential growth in active patients and net revenues (Press release, NovoCure, APR 27, 2017, View Source [SID1234518736]). Novocure is an oncology company developing a profoundly different approach to cancer treatment utilizing a proprietary therapy called TTFields, the use of electric fields tuned to specific frequencies to disrupt solid tumor cancer cell division.

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First quarter 2017 highlights include:


Three months ended March 31,
2017 2016 % Change

Non-financial
Prescriptions received in period(1) 894 755 18%
Active patients at period end(2) 1,266 797 59%

Financial, in millions
Net revenues $ 34.9 $ 13.1 167%
Net loss $ (18.0 ) $ (35.4 ) 49%

Cash and cash equivalents at the end of period $ 84.6 $ 115.9
Short-term investments at the end of period $ 104.7 $ 119.8
(1) A "prescription received" is a commercial order for Optune that is received from a physician certified to treat patients with Optune for a patient not previously on Optune. Orders to renew or extend treatment are not included in this total.
(2) An "active patient" is a patient who is on Optune 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.
"Our growth continued in the first quarter 2017. It was the ninth consecutive quarter of active patient growth since the first presentation of our EF-14 data in newly diagnosed glioblastoma (GBM). At the end of the quarter, we had more than 1,260 active patients on therapy," said Asaf Danziger, Novocure’s Chief Executive Officer. "Our 167% year-over-year revenue growth was driven by an expanding global base of active patients as well as coverage and contracting success in the U.S."

"We are pleased with the progress we made this quarter and remain committed to bringing Optune to all the patients who may benefit from it," continued Mr. Danziger. "The final analyses of our EF-14 phase 3 pivotal trial presented at AACR (Free AACR Whitepaper) showed a consistent improvement in overall survival when Optune was added to standard temozolomide chemotherapy for the treatment of newly diagnosed GBM. The improvement in overall survival was maintained at two, three, four and five years. The four and five year survival rates of Optune-treated patients were more than double those of patients treated with temozolomide alone. We believe that Optune plus temozolomide is an essential combination treatment for patients with newly diagnosed GBM."

"Beyond GBM, our data continue to support that TTFields may be well suited for combination with standard of care treatments for a variety of additional solid tumor types," said William Doyle, Novocure’s Executive Chairman. "As committed as we are to bringing Optune to patients with GBM, we are equally focused on advancing TTFields for additional solid tumor indications."

First Quarter 2017 Operating Statistics and Financial Update

There were 1,266 active patients on Optune at March 31, 2017, an increase of 469 active patients, or 59 percent, compared to March 31, 2016. 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 and who typically have a longer duration of treatment with Optune. In the first quarter 2017, more than 55 percent of Optune prescriptions were for newly diagnosed GBM.

In the United States, there were 933 active patients on Optune at March 31, 2017, an increase of 234 active patients, or 33 percent, compared to March 31, 2016.
In Germany and other EMEA markets, there were 331 active patients on Optune at March 31, 2017, an increase of 233 active patients, or 238 percent, compared to March 31, 2016.
Additionally, 894 prescriptions were received in the quarter ended March 31, 2017, an increase of 139 prescriptions, or 18 percent, compared to the same period in 2016. The increase in prescriptions was driven primarily by commercial activities in our currently active markets.

In the United States, 685 prescriptions were received in the quarter ended March 31, 2017, an increase of 1 prescription compared to the same period in 2016.
In Germany and other EMEA markets, 206 prescriptions were received in the quarter ended March 31, 2017, an increase of 135 prescriptions, or 190 percent, compared to the same period in 2016.
In Japan, there were 3 prescriptions received in the quarter ended March 31, 2017.
We continued to work with payers in the United States to expand coverage of Optune for the treatment of both newly diagnosed and recurrent GBM. As of March 31, 2017, payers administering plans for more than 187 million lives had issued positive coverage policies stating that Optune is approved for the treatment of newly diagnosed or recurrent GBM, an increase of approximately 7 million lives since January 1, 2017, including new policies with Highmark Blue Cross Blue Shield, Blue Cross Blue Shield of Idaho, and Dean Health Plan.

For the three months ended March 31, 2017, net revenues increased to $34.9 million compared to $13.1 million for the same period in 2016, representing 167 percent growth. This growth was primarily driven by increased Optune adoption and the transition to accrual-based revenue recognition for a portion of our billings.

For the three months ended March 31, 2017, net revenues included $14.7 million in accrual-based net revenues. For the three months ended March 31, 2017, gross billings totaled $73.2 million, and 23% of first quarter 2017 gross billings qualified for accrual-based net revenue recognition. We continue to recognize revenue on a cash basis for payers with which we do not have contractual arrangements or sufficient history to reliably estimate their individual payment patterns and for which we cannot reliably estimate the amount that would ultimately be collected. We believe there will be an extended period of time during which our revenue will be a mix of cash-based and accrual-based revenue.

For the three months ended March 31, 2017, cost of revenues increased to $11.7 million compared to $8.0 million for the same period in 2016, representing an increase of 46 percent. This was primarily driven by an increase in active Optune patients, resulting in increased transducer array shipments and increased field equipment depreciation expenses, 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, 2017, were $9.4 million compared to $11.4 million for the same period in 2016, representing a decline of 18 percent. This was primarily due to a decrease in clinical trial expenses resulting from the conclusion of our EF-14 phase 3 pivotal trial in newly diagnosed GBM.

Sales and marketing expenses for the three months ended March 31, 2017, were $14.8 million compared to $13.3 million for the same period in 2016, representing an increase of 11 percent. This was primarily due to increased personnel costs, reflecting our expanding commercial operations in the U.S. and Germany, partially offset by a decrease in advertising and professional services related to the launch of Optune for newly diagnosed GBM.

General and administrative expenses for the three months ended March 31, 2017, were $12.4 million, representing an increase of 1 percent compared to the same period in 2016. This was primarily due to increased headcount partially offset by a decrease in professional services and other expenses.

Personnel costs for the three months ended March 31, 2017, included $4.6 million in non-cash share-based compensation expenses, comprised of $0.1 million in cost of revenues; $0.9 million in research, development and clinical trials; $0.7 million in sales and marketing; and $2.9 million in general and administrative expenses. Total non-cash share-based compensation expenses for the first quarter 2016 were $5.5 million.

Net losses for the three months ended March 31, 2017, were $18.0 million compared to net losses of $35.4 million for the same period in 2016.

At March 31, 2017, we had $84.6 million in cash and cash equivalents and $104.7 million in short-term investments, for a total balance of $189.3 million in cash, cash equivalents and short-term investments. At March 31, 2017, we had $100.0 million of principal indebtedness outstanding under our Loan and Security Agreement with Biopharma Secured Investments III Holdings Cayman LP.

Anticipated clinical milestones

Trial initiations:

Phase 3 pivotal trial in locally advanced pancreatic cancer (2H 2017)
Phase 3 pivotal trial in recurrent ovarian cancer (2018)
Top-line data readouts:

Phase 2 pilot STELLAR trial in mesothelioma (2018)
Phase 3 pivotal METIS trial in brain metastases (2020)
Phase 3 pivotal LUNAR trial in non-small cell lung cancer (2021)

On April 27, 2017 Syndax Pharmaceuticals, Inc. (NASDAQ:SNDX), reported the expansion of ENCORE 601/KEYNOTE 142, the ongoing Phase 2 clinical collaboration with a subsidiary of Merck, known as MSD outside the United States and Canada, to include a cohort of patients with microsatellite stable colorectal cancer (Press release, Syndax, APR 27, 2017, View Source [SID1234518726]). This trial is designed to evaluate the safety, tolerability and efficacy of Syndax’s entinostat, an oral, small molecule that targets immune regulatory cells, in combination with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 (programmed death receptor-1) therapy.

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"Microsatellite stable colorectal cancers comprise approximately 85% of colorectal cancers, have limited treatment options once the disease advances, and have been unresponsive to anti-PD-1 monotherapy," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "This expansion of ENCORE 601/KEYNOTE 142 is based in part on the previously communicated clinical responses we observed for the entinostat-KEYTRUDA combination in advanced melanoma patients that had progressed on prior anti-PD-1 therapy."

ENCORE 601 continues to evaluate the combination of entinostat and KEYTRUDA in patients with advanced melanoma or non-small cell lung cancer (NSCLC) who have experienced disease progression following treatment with an anti-PD-1 therapy, and patients with NSCLC who are naïve to treatment with a PD-1 or PD-L1 antagonist. In March 2017, Syndax announced that the melanoma cohort achieved the pre-specified criteria required to advance to the second stage of the trial, and re-opened enrollment of that cohort. The Company will present results from the first stage of the melanoma cohort at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June. A decision on whether the two NSCLC cohorts can proceed to the second stage of the trial is expected in 2Q17.

Financial and other terms of the initial agreement, as well as the amendment covering the expanded collaboration between Syndax and Merck, were not disclosed.

On April 27, 2017 Mateon Therapeutics, Inc. (OTCQX:MATN), a biopharmaceutical company developing vascular disrupting agents (VDAs) for the treatment of orphan oncology indications, reported that four presentations regarding the company’s preclinical drug development programs will be presented at the Tumor Microenvironment Workshop being held from April 27-29, 2017 at the Palms Hotel in Miami, Florida (Press release, Mateon Therapeutics, APR 27, 2017, View Source [SID1234518725]).

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"These presentations highlight new potential applications for our vascular disrupting agents, which are currently in clinical development for other indications," stated William D. Schwieterman, M.D., Mateon’s President and Chief Executive Officer. "The workshop will also show selected potential applications for our earlier-stage research programs."

Titles of the presentations by Mateon and/or its collaborators include the following:

Combining Vascular Disrupting Agents with Checkpoint Inhibitors to Improve Tumour Immunogenicity. Pernille B. Elming, M.D., Thomas R. Wittenborn, Ph.D., and Michael R. Horsman, Ph.D., Department of Experimental Clinical Oncology, Aarhus University Hospital, Aarhus, Denmark .

Targeting Tumor-Associated Hypoxia with Bioreductively Activatable Prodrug Conjugates of Small-Molecule Inhibitors of Tubulin Polymerization. Blake A. Winn, Ph.D.,1 Laxman Devkota, Ph.D.,1 Yifan Wang, M.S.,1 Tracy E. Strecker, Ph.D.,1 Jeni Gerberich, B.S.,2 Ralph P. Mason, Ph.D.,2 Mary Lynn Trawick, Ph.D.,1 Kevin G. Pinney, Ph.D.1

1Department of Chemistry and Biochemistry, Baylor University, Waco, Texas
2Department of Radiology, University of Texas Southwestern Medical Center, Dallas, Texas

Cathepsin inhibition impairs M2 macrophage pro-tumor functions. Samantha S. Dykes, Ph.D. and Dietmar W. Siemann, Ph.D., Department of Radiation Oncology, University of Florida, Gainesville, FL

The small molecule Cathepsin L and K inhibitor KGP-94 impairs the Tumor-Associated Macrophage-mediated metastatic phenotype of osteosarcoma under hypoxia. Henrietta Fasanya and Dietmar Siemann, Ph.D., Department of Radiation Oncology, University of Florida, Gainesville, Florida

On April 27, 2017 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported that following the recent identification of a fixed dose and patient-centric dosing schedule, the ongoing clinical trial of TSR-042 has been expanded to enroll patients with metastatic microsatellite instability high (MSI-H) endometrial cancer who have progressed following one or two prior chemotherapy treatments (Press release, TESARO, APR 27, 2017, View Source [SID1234518720]).

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During the first 12 weeks of treatment, TSR-042 is administered once every three weeks, followed by a single dose administration every six weeks until disease progression. The intent of this study is to support a request for accelerated approval and Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA). The primary endpoints of this trial are overall response rate (ORR) and duration of response, and secondary endpoints include disease control rate, progression free survival (PFS), and overall survival (OS). The addition of cohorts for patients with other tumor types is also planned. This is the first clinical development program within a broader plan that includes potential label expansion trials of TSR-042 in multiple cancers in combination with ZEJULA, TSR-022, TESARO’s anti-TIM-3 antibody, and TSR-033, TESARO’s anti-LAG-3 antibody.

"TSR-042 was the first antibody from our immuno-oncology portfolio to enter clinical trials, and following identification of a fixed dose and schedule, we are pleased to be advancing TSR-042 into a registration program," said Mary Lynne Hedley, Ph.D., President and COO of TESARO. "With the recent approval of ZEJULA in the U.S., the initiation of this development program furthers our commitment to women with gynecologic tumors, including ovarian, fallopian tube, and peritoneal cancer. We intend to continue our efforts with future combination studies of TSR-042 and ZEJULA, and to someday eliminate these terrible diseases and benefit women who were until recently largely underserved by new therapeutic options."

About TSR-042

TSR-042 is a monoclonal antibody targeting PD-1 and was developed as part of the collaboration between TESARO and AnaptysBio, Inc. This collaboration was initiated in March of 2014, and is focused on the development of monospecific antibody drugs targeting PD-1, TIM-3 (TSR-022), and LAG-3 (TSR-033), in addition to a bi-specific antibody drug candidate targeting PD-1/LAG-3.

About Endometrial Cancer
Endometrial cancer is the most common type of uterine cancer, accounting for more than 95 percent of cases. Endometrial cancer develops in the lining of the uterus, called the endometrium. The annual number of new cases of endometrial cancer is estimated at 325,000 worldwide. The most common histologic form is endometrioid adenocarcinoma originating in the glandular tissue, which represents about 75-80% of diagnosed cases. In 2017, SEER1 estimates 61,380 patients will be diagnosed with endometrial cancer, with approximately 30% or 18,414 being stage III/IV patients. Based on genomic characterization studies of endometrial cancer, 20-25% of patients have tumors with a microsatellite instability phenotype (MSI-H)2. Microsatellite instability arises from a failure to repair replication-associated errors due to a defective DNA mismatch repair system. This failure allows persistence of mismatch mutations all over the genome, but especially in regions of repetitive DNA known as microsatellites, leading to increased mutational load that has been demonstrated to improve responses to anti-PD-1 therapies.3,4

On April 27, 2017 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported a comprehensive program update on entrectinib – an investigational, orally available, CNS-active tyrosine kinase inhibitor targeting tumors that harbor TRK, ROS1 or ALK fusions – currently being studied in a registration-enabling Phase 2 clinical trial known as STARTRK-2 (Press release, Ignyta, APR 27, 2017, View Source [SID1234518716]).

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"Today’s update is the result of our team’s diligent efforts to advance entrectinib through the clinic for the benefit of patients with TRK and ROS1 fusion-positive cancers," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "We are proud to announce substantial progress towards dual entrectinib NDA submissions for both TRK and ROS1, and are excited by the opportunity to serve two distinct groups of patients with cancer."

Entrectinib development highlights:

TRK –

Based on written feedback from FDA, Ignyta intends to pursue an NDA submission for entrectinib to support a TRK fusion-positive, tissue agnostic indication.
Ignyta’s entrectinib program is more than 85% enrolled to goal for the primary efficacy analysis for a TRK tissue agnostic NDA submission, and is on track to complete enrollment by the 3rd quarter of 2017.
Within STARTRK-2, several dozens of patients with TRK fusions have been enrolled, across more than 15 different tumor types; and investigators in approximately a dozen different countries on four continents currently have experience treating patients with entrectinib.
Ignyta plans to announce top-line NDA registration-enabling data from STARTRK-2, after consultation with FDA, in the first half of 2018.
ROS1 –

Ignyta is simultaneously pursuing an NDA submission for entrectinib to support a ROS1 fusion-positive NSCLC indication.
More than 50 ROS1 fusion-positive NSCLC patients have been enrolled to support a potential ROS1 NSCLC NDA for entrectinib, greater than the number that formed the basis of crizotinib’s approval in this setting.
Ignyta reported interim data from 32 patients with ROS1 fusion-positive NSCLC, including new patients from STARTRK-2 who were enrolled as of December 16, 2016.
24 out of the 32 patients had confirmed RECIST responses, for an objective response rate (ORR) of 75%.
The median duration of response (DOR) was 17.2 months among the responder patients, and the median progression free survival (PFS) was 19.1 months among all patients.
11 out of the 32 patients, or 34%, had CNS metastases at baseline.
Of these 11 patients with CNS disease, 7 had confirmed RECIST responses, for an ORR of 64% in ROS1 NSCLC patients with CNS metastases.
When aggregated with Ignyta’s Phase 1 data set of patients with primary and secondary CNS disease, a total of 10 out of 16 patients had confirmed RECIST responses, for an ORR of 63% in TRK, ROS1 or ALK patients with CNS disease.
Entrectinib commercial highlights:

Based upon analysis of more than 120,000 cancer patient samples from internal and external sources, Ignyta believes that in the US each year there are approximately 1,000-2,000 new patients with TRK fusion-positive advanced solid tumors, and 2,000 new patients with ROS1 fusion-positive NSCLC.
Entrectinib is simultaneously tracking towards TRK tissue agnostic NDA and ROS1 NSCLC NDA submissions in 2018, with anticipated US launch in 2019.
Based upon these dual indications, Ignyta believes the total addressable market for entrectinib could be in excess of $1B annually in the US alone.
A conference call and webcast is being held on April 27, 2017 at 4:30 p.m. Eastern/1:30 p.m. Pacific to discuss the entrectinib program update. To participate in the conference call, please dial (877) 548-7906 (U.S.) or (719) 325-4765 (International) and provide Conference ID 2028635. To access the live webcast, go to View Source." target="_blank" title="View Source." rel="nofollow">View Source

A replay of the presentation will be available shortly after the conclusion of the live call in the Investors section of the company’s website at View Source, and will be archived and available at that site for 14 days.