On August 21, 2018 Y-mAbs Therapeutics, Inc. (YmAbs), an immunotherapy company discovering and developing innovative treatments for patients with cancer, reported that the Company has received a Breakthrough Therapy designation for naxitamab, in combination with GM-CSF, for the treatment of high risk neuroblastoma refractory to initial therapy or with incomplete response to salvage therapy in patients older than 12 months of age with persistent, refractory disease limited to bone marrow with or without evidence of concurrent bone involvement (Press release, Y-mAbs Therapeutics, AUG 21, 2018, View Source [SID1234529164]).

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YmAbs Founder, President and Head of Business Development and Strategy, Thomas Gad said, "We are very pleased that the FDA has granted the Breakthrough Therapy designation to naxitamab and we look forward to continuing to work with the FDA to make this therapy potentially available to children facing an unmet medical need. We believe that Naxitamab provides a new opportunity for pediatric patients otherwise faced with little or no options. This is an important milestone achievement for YmAbs, and we continue to work with the regulatory authorities to advance naxitamab to patients suffering from high risk neuroblastoma as quickly as possible."

Dr. Claus Møller, Chief Executive Officer further notes, "This is the first time naxitamab has earned the distinction of a Breakthrough Therapy Designation. We are pleased that the FDA continues to recognize the potential of naxitamab to help patients with high risk neuroblastoma."

About Breakthrough Therapy Designation:

The Breakthrough Therapy Designation was enacted as part of the 2012 FDA Safety and Innovation Act (FDASIA) and is intended to expedite development of drugs to treat serious and life-threatening medical conditions when preliminary clinical evidence demonstrates that the drug may have substantial improvement on at least one clinically significant endpoint over available therapies. Breakthrough Therapy Designation includes all the features of the Fast Track Designation, as well as more intensive guidance from the FDA on a drug’s clinical development program.

On August 21, 2018 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, with a primary focus on myelodysplastic syndromes (MDS), reported that its commercial partner, Pint Pharma GmbH, will assist in expanding access to clinical trials studying rigosertib, a novel and targeted anti-cancer compound currently in a Phase 3 study for the treatment of MDS, to several selected sites across South America (Press release, Onconova, AUG 21, 2018, View Source [SID1234529071]). Pint Pharma is a European-based pharmaceutical company focused on the development, registration and commercialization of specialty-based treatments for the Latin American market and has successfully participated in clinical trials for hematological cancers.

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"This assistance will help make rigosertib available to cancer patients on a fifth continent, following our ongoing clinical trials in North America, Europe, Asia and Australia. We are delighted to partner with Pint Pharma, which has a wide footprint in South and Central America, with first-hand experience in running clinical trials for malignant hematological disorders," said Dr. Ramesh Kumar, CEO of Onconova Therapeutics, Inc.

"We are excited to start helping Onconova open new clinical sites in Latin America. We are hopeful that Onconova will be able to start recruiting patients as soon as possible to continue the development of IV and Oral Rigosertib," said David Munoz, Chief Executive Officer of Pint Pharma. He added, "Rigosertib is highly complementary to our comprehensive hematology oncology portfolio, and will further strengthen our mission to enable the Latin American population with life-threatening conditions to live better lives by providing early and efficient access to innovative technologies."

Dr. Steven Fruchtman, President and Chief Medical Officer of Onconova, is working closely with Dr. Valnei Canutti, General Manager, Brazil, and Chief Medical Officer of Pint. Dr. Fruchtman commented, "Completion of the INSPIRE Trial and expanding the potential utility of rigosertib for cancer patients are our core objectives and we are delighted that our commercial partner will assist us in recruiting patients in the INSPIRE trial. There is a great medical need and interest to conduct studies in patients with higher risk MDS in this geographical region."

Dr. Canutti added, "We are looking forward to working with Dr. Fruchtman on this important initiative. My prior experience in MDS and our connections with Key Opinion Leaders across this continent will be greatly helpful as we collaborate with Onconova."

On August 21, 2018 Humanigen, Inc presented the Investor Presentation (Presentation, Humanigen, AUG 21, 2018, View Source [SID1234529049]).

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On August 21, 2018 Rexahn Pharmaceuticals, Inc. (NYSE American: RNN), a clinical stage biopharmaceutical company developing innovative, targeted therapeutics for the treatment of cancer, reported that it has entered into a clinical trial collaboration agreement with Merck (known as MSD outside the United States and Canada) to evaluate the combination of Rexahn’s RX-5902 and Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in a Phase 2 trial in patients with metastatic triple negative breast cancer (TNBC) (Press release, Rexahn, AUG 21, 2018, View Source [SID1234529020]).

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"Rexahn is excited to announce this collaboration with Merck, an established leader in the field of immuno-oncology," said Peter D. Suzdak, Ph.D., chief executive officer of Rexahn. "RX-5902 has both antitumor and immune-modulatory effects and augments the efficacy of checkpoint inhibitors in animal models. Based on the mechanism of action of RX-5902 and our observations in preclinical studies, we are optimistic that the combination of RX-5902 with KEYTRUDA may provide meaningful clinical benefit in patients with metastatic triple negative breast cancer – a cancer that is notoriously difficult to treat".

The study will evaluate the safety and efficacy of the combination of RX-5902 and KEYTRUDA in patients with metastatic TNBC who have progressed following at least one prior treatment. Under the terms of the agreement, Rexahn will sponsor the RX-5902 and KEYTRUDA study.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.

About RX-5902

RX-5902 (Supinoxin) is an orally administered, potential first-in-class, small molecule inhibitor of phosphorylated-p68 (P-p68). P-p68, which is selectively overexpressed in cancer cells and is absent in normal tissue, modulates the activity of the β-catenin/Wnt pathway and plays a role in tumor progression, metastasis and tumor immunogenicity.

In preclinical studies, RX-5902 has been shown to inhibit the growth and proliferation of multiple human cancer cell lines (including triple negative breast cancer), decrease tumor growth in patient derived xenograft models and potentiate the activity of immune checkpoint inhibitors and other anti-tumor agents. RX-5902 is currently being evaluated as monotherapy in a Phase 2 clinical trial in patients with metastatic TNBC. Preliminary data was presented at ASCO (Free ASCO Whitepaper) (American Society for Clinical Oncology) Annual Meeting in June 2018. Additional information on RX-5902 can be found at: View Source

On August 21, 2018 Myriad Genetics, Inc. (NASDAQ: MYGN, "Myriad" or the "Company"), a global leader in molecular diagnostics and personalized medicine, reported financial results for its fiscal fourth-quarter and full-year 2018, provided an update on recent business highlights and issued its fiscal year and first-quarter 2019 financial guidance (Press release, Myriad Genetics, AUG 21, 2018, View Source [SID1234529019]).

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"Fiscal year 2018 was an excellent year for Myriad as record-setting growth in new products with increasing reimbursement added to a solid hereditary cancer business and a re-engineered cost structure," said Mark C. Capone, president and CEO, Myriad Genetics. "Based upon our operational momentum and the recent completion of the Counsyl acquisition, we are confident in our strategy to transform Myriad into the global leader in personalized medicine."

Financial Highlights

The following table summarizes the financial results for the fiscal fourth-quarter and fiscal full-year 2018

Revisions of Previously-Issued Financial Statements: During the financial close for fiscal year 2018, the Company determined that it had not fully reserved for its sales allowance for the financial periods from fiscal year 2015 through fiscal year 2018. These errors which represented less than one percent of total revenue during that time frame were determined to be. The financial information for prior periods has been restated to reflect these adjustments.

Business Highlights

Hereditary Cancer

Achieved sixth consecutive quarter of year-over-year hereditary cancer testing volume growth.

Announced the second major clinical validation study for riskScore at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting. The study evaluated 518 women and found that riskScore is a highly statistically significant predictor of the 5-year and lifetime risk of breast cancer (p=2.6×10-12 and p=2.5×10-12, respectively). Moreover, riskScore was statistically significantly superior to Tyrer-Cuzick alone for both 5-year and lifetime risk of breast cancer (1.9×10-8 and p=2.4×10-8, respectively), underscoring the independent contribution of the combined test score.

Based upon recent changes to the National Comprehensive Cancer Network professional treatment guidelines for prostate, pancreatic and colon cancer, we estimate 121,000 additional cancer patients now qualify for hereditary cancer testing in the United States every year.

GeneSight

Fiscal fourth-quarter revenue increased 33 percent year-over-year to $33.9 million with record volumes in the quarter.

A record 15,000 physicians, including almost 3,000 new ordering doctors, ordered a GeneSight test in the fiscal fourth-quarter.

Presented the results from the GeneSight GUIDED randomized controlled trial at the American Psychiatric Association annual meeting. The landmark study showed that patients receiving GeneSight had significantly better outcomes with a 50 percent increase in remission rates and a 30 percent increase in response rates relative to standard-of-care therapy.

Announced the results of the IMPACT study at the American Society of Clinical Psychopharmacology annual meeting. In the study, patients treated by primary care physicians had 33 percent greater symptom improvement, 34 percent increased response and 57 percent greater remission than those treated by psychiatrists.

CareFirst, the 15th largest commercial payer in the United States, announced a favorable coverage policy for GeneSight taking effect August 1, 2018 which included all physician specialties.

Announced a new coverage decision for GeneSight with a major U.S. company with 30,000 employees.

In late-stage negotiations with Kroger Prescription Plans on a potential favorable coverage decision for GeneSight.

Vectra DA

Fiscal fourth-quarter revenue increased 47 percent year-over-year to $15.1 million.

Announced a favorable coverage decision for Vectra DA from Kroger Prescription Plans, the pharmacy benefits manager for Kroger and other employers. Kroger is the fourth largest employer in the United States.

Prolaris

Fiscal fourth-quarter revenue increased 133 percent year-over-year to $7.0 million with record volumes in the quarter.

Received positive medical policy recommendations on Prolaris from eight commercial insurers, including a top-10 commercial insurer, and seven others, totaling over 20 million covered lives or 12 percent of total commercial lives in the United States. Prolaris is now covered for approximately 55 percent of prostate cancer patients in the United States.

EndoPredict

Fiscal fourth-quarter revenue increased 40 percent year-over-year to $2.8 million.

Received positive recommendation from the National Institute for Health and Care Excellence (NICE) in the United Kingdom to cover the EndoPredict test.

myPath Melanoma

Received positive medical policy recommendations on myPath Melanoma from eight commercial insurers.

Companion Diagnostics

Metastatic breast cancer patients tested by Myriad increased 13 percent sequentially based upon the recent launch of BRACAnalysis CDx as a companion diagnostic for Lynparza.

Received pre-market approval from the Japanese Ministry of Health, Labor, and Welfare for our BRACAnalysis CDx test for HER2- metastatic breast cancer.

Announced positive results from Phase III, SOLO-1 study which evaluated patients with advanced ovarian cancer treated in the first-line setting who tested positive with Myriad’s BRACAnalysis CDx test. Myriad intends to submit a supplementary pre-market approval (sPMA) to the U.S. Food and Drug Administration (FDA) for this indication.

Announced that the FDA has accepted Myriad’s sPMA application for BRACAnalysis CDx to be used as a companion diagnostic with Pfizer’s PARP inhibitor, talazoparib, in HER2- metastatic breast cancer. The New Drug Application for talazoparib has been granted priority review by the U.S. FDA and has a Prescription Drug User Fee Act goal date of December 2018.

Closing of Counsyl Acquisition

Myriad signed a definitive agreement to acquire Counsyl, Inc., a global leader in reproductive genetic testing, which closed on July 31, 2018. The acquisition of Counsyl provides Myriad with two new products, ForeSightTM and PreludeTM, in the expanded carrier screening and non-invasive prenatal screening markets respectively. Myriad

estimates that these markets will grow to approximately 3 million tests performed annually in the United States and $1.5 billion of revenue over the next five years.

Fiscal Year 2019 and Fiscal First-Quarter 2019 Financial Guidance

Myriad’s fiscal year 2019 and fiscal first-quarter 2019 adjusted earnings per share guidance excludes the impact of stock based compensation expense, non-cash amortization associated with acquisitions and certain non-recurring expenses. These projections are forward-looking statements and are subject to the risks summarized in the safe harbor statement at the end of this press release. The Company will provide further details on its business outlook during the conference call today and discuss the fiscal fourth-quarter financial results and fiscal year 2019 financial guidance.

Conference Call and Webcast

A conference call will be held today, Tuesday, August 21, 2018, at 4:30 p.m. EDT to discuss Myriad’s financial results for the fiscal fourth-quarter, business developments and financial guidance. The dial-in number for domestic callers is 1-800-616-4021. International callers may dial 1-303-223-2682. All callers will be asked to reference reservation number 21892392. An archived replay of the call will be available for seven days by dialing (800) 633-8284 and entering the reservation number above. The conference call along with a slide presentation will also will be available through a live webcast at www.myriad.com.