On May 1, 2017 Sunesis Pharmaceuticals, Inc. (Nasdaq:SNSS) reported that the Company will withdraw its European Marketing Authorization Application (MAA) for vosaroxin as a treatment for relapsed/refractory acute myeloid leukemia (AML) in patients aged 60 years and older (Press release, Sunesis, MAY 1, 2017, View Source [SID1234518766]). The decision follows recent interactions with the European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), during which the Company learned that the committee was likely to formally adopt a negative opinion in its evaluation of the application.

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"We are disappointed to not achieve approval for vosaroxin’s MAA given its reported efficacy in a patient population with such poor outcomes. Although we did not receive a definitive CHMP opinion, we believed that a positive opinion was unlikely," said Daniel Swisher, President and Chief Executive Officer of Sunesis. "Following our appearances before the committee’s Scientific Advisory Group Oncology Division and CHMP, we carefully considered feedback from our rapporteurs and input from retained regulatory experts to make our decision to notify EMA to withdraw vosaroxin’s MAA as our assessment concluded it was unlikely we could achieve a majority vote of CHMP members at this time or upon an immediate re-examination for our proposed indication based on VALOR data from a sub-group of a single pivotal trial that had missed reaching full statistical significance in its primary analysis."

Mr. Swisher added: "In light of this, we are significantly reducing our investment in the AML program and shifting an increasing portion of resources to our kinase inhibitor pipeline, including lead asset SNS-062, our non-covalent reversible BTK-inhibitor, which will begin dosing this quarter in a Phase 1b/2 trial in cancer patients with B-cell malignancies. We expect to continue to advance the development of vosaroxin through a modest investment in investigator-sponsored group trials, and will carefully assess business development alternatives to support the conduct of another pivotal trial to achieve future regulatory approval of vosaroxin. We expect that our current cash resources are sufficient to fund the company beyond Q1 2018."

About SNS-062
SNS-062 is a novel, second-generation BTK inhibitor, a class of kinase inhibitors that selectively inhibits the enzyme Bruton’s tyrosine kinase (BTK). This target mediates signaling through the B-cell receptor, which is critical for adhesion, migration, proliferation and survival of normal and malignant B-lineage lymphoid cells. Unlike other drugs in its class, SNS-062 has a distinct kinase selectivity profile and binds non-covalently to the BTK enzyme. This alternate binding site potentially provides an opportunity to address the leading resistance mechanism, a mutation in the enzyme’s binding site required for covalent binding. In preclinical studies, SNS-062 demonstrated potent activity against Cys-481S mutated B-cell malignancies, and has been studied in healthy subjects in a Phase 1A, randomized, double-blind, placebo-controlled dose-ranging study to investigate the drug’s safety, pharmacokinetics, and pharmacodynamics. With the reported successful study outcome, SNS-062 is proceeding to a Phase 1B/2 study in patients with B-cell malignancies.

About QINPREZO (vosaroxin)
QINPREZO (vosaroxin) is an anti-cancer quinolone derivative (AQD), a class of compounds that has not been used previously for the treatment of cancer. Preclinical data demonstrate that vosaroxin both intercalates DNA and inhibits topoisomerase II, resulting in replication-dependent, site-selective DNA damage, G2 arrest and apoptosis. Both the U.S. Food and Drug Administration (FDA) and European Commission have granted orphan drug designation to vosaroxin for the treatment of AML. Additionally, vosaroxin has been granted fast track designation by the FDA for the potential treatment of relapsed/refractory AML in combination with cytarabine. Vosaroxin is an investigational drug that has not been approved for use in any jurisdiction.
The trademark name QINPREZO is conditionally accepted by the FDA and the EMA as the proprietary name for the vosaroxin drug product candidate.

On May 1, 2017 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, reported one oral and one poster presentation at the 14th International Symposium on Myelodysplastic Syndromes taking place May 3-6, 2017 at the Palacios de Congresos de Valencia in Valencia, Spain (Press release, Onconova, MAY 1, 2017, View Source [SID1234518754]). These presentations will be made by the Company’s collaborators from the Mount Sinai School of Medicine and the Cleveland Clinic.

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Abstract Details:

Poster Presentation:
Date: May 4th through May 6th
Time: 8:00 am
Location: Poster Presentation Section
Presenter: Dr. Aziz Nazha – Cleveland Clinic, Cleveland, Ohio

A Validation of a Post-Hypomethylating Agent Failure (HMAF) Prognostic Model in Myelodysplastic Syndromes (MDS) Patients Treated with Rigosertib versus Best Supportive Care (BSC) in a Randomized Controlled Phase III trial

Oral Presentation:
Date: Saturday May 6th
Time: 8:30 am-10 am
Location: Auditorium 1
Presenter: Dr. Shyamala Navada – Mount Sinai School of Medicine, NYC

Combination of Oral Rigosertib and Injectable Azacitidine In Patients with Myelodysplastic Syndromes (MDS)

Additional details and content from these presentations will be available on the Company’s website on the day of the presentations.

On May 1, 2017 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported the submission of an Investigational New Drug (IND) Application for TSR-033 to the U.S. Food and Drug Administration. TSR-033 is a monoclonal antibody targeting LAG-3 (Press release, TESARO, MAY 1, 2017, View Source [SID1234518748]).

“The IND for TSR-033 is the third application from our immuno-oncology franchise to be submitted to the FDA within the past 17 months,” said Mary Lynne Hedley, Ph.D., President and COO of TESARO. “Our vision is that immuno-oncology candidates such as TSR-033, TSR-042, our anti-PD-1 antibody, and TSR-022, our anti-TIM-3 antibody, could become a foundation of cancer therapy regimens across a variety of tumor types. A Phase 1 clinical study of TSR-033 is planned to begin in mid-2017.”

About TSR-033
TSR-033 is a monoclonal antibody candidate targeting LAG-3 developed as part of 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.

On May 1, 2017 ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported that the results from a Phase 1 dose-escalation study evaluating mirvetuximab soravtansine (IMGN853) in patients with folate receptor alpha (FRα)-positive solid tumors were published in the journal Cancer (Press release, ImmunoGen, MAY 1, 2017, View Source [SID1234518747]). The previously disclosed data demonstrated encouraging clinical activity and a manageable safety profile for mirvetuximab soravtansine (IMGN853), and informed the dose that was used in Phase 1 expansion cohorts and the ongoing Phase 3 FORWARD I trial of patients with platinum-resistant ovarian cancer.

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"These Phase 1 results have played an important role in determining the appropriate dose for mirvetuximab soravtansine in the recently initiated Phase 3 FORWARD I trial of patients with platinum-resistant ovarian cancer," said Kathleen Moore, M.D., Associate Professor, Department of Obstetrics and Gynecology at the Stephenson Cancer Center at the University of Oklahoma. "The combination of these data and the recent data published in the Journal of Clinical Oncology further support the dose that has been chosen and patients who have been selected for FORWARD I."

The open-label, Phase 1 dose-escalation study treated a total of 44 patients with recurrent ovarian (52%) or endometrial cancer (25%), along with renal cell carcinoma and non-small cell lung cancer (11% and 9%, respectively). Patients received mirvetuximab soravtansine on day 1 of a 21-day cycle (Q3W dosing) with cycles repeated until dose-limited toxicity or progression, concluding the recommended dose for future trials is 6.0 mg/kg of mirvetuximab (based on adjusted ideal body weight) dosed once every three weeks. On the basis of the study findings, and additional data that demonstrated the importance of FRα expression levels for optimal mirvetuximab sorvatansine activity, the Company designed the Phase 3 FORWARD I trial utilizing this dose in patients with platinum-resistant ovarian cancer, along with a Phase 1b trial evaluating mirvetuximab in combination with standard-of-care chemotherapy and targeted agents.1

Mirvetuximab soravtansine exhibited a manageable safety profile and encouraging preliminary clinical activity. Adverse events (AEs) were generally mild with the majority being grade 1 or grade 2 (least severe grades). The most commonly observed AEs were fatigue, blurred vision and diarrhea.1

The publication, "Phase I dose-escalation study of mirvetuximab soravtansine (IMGN853), a folate receptor alpha-targeting antibody-drug conjugate, in patients with solid tumors," is available on the Cancer website.

About Mirvetuximab Soravtansine

Mirvetuximab soravtansine (IMGN853) is the first FRα-targeting ADC. It uses a FRα-binding antibody to target the ADC specifically to FRα-expressing cancer cells and a potent anti-tumor agent, DM4, to kill the targeted cancer cells.

Mirvetuximab soravtansine is ImmunoGen’s lead program and is in Phase 3 testing as a single agent for the treatment of platinum-resistant ovarian cancer. The candidate is also being assessed in combination regimens for both platinum-resistant and platinum-sensitive disease in Phase 1b/2 FORWARD II trial.

About Ovarian Cancer and FRα

In 2016, approximately 22,300 new cases of ovarian cancer will be diagnosed in the U.S. and more than 14,200 women will die from the disease.2 ImmunoGen estimates that 60% of ovarian cancer cases have medium or high FRα expression.

Standard first-line therapy for ovarian cancer is a platinum-based regimen. Once the cancer becomes platinum-resistant, treatment options include single-agent cytotoxic therapies such as pegylated liposomal doxorubicin, paclitaxel, or topotecan.

On May 1, 2017 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq:RDUS), a fully integrated science-driven biopharmaceutical company that is committed to developing innovative therapeutics in the areas of osteoporosis, oncology and endocrine diseases, reported its financial results for the first quarter ended March 31, 2017, and provided a business update (Press release, Radius, MAY 1, 2017, View Source [SID1234518744]). As of March 31, 2017, Radius had $282.1 million in cash, cash equivalents and marketable securities.

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TYMLOS (abaloparatide)

On April 28, 2017, Radius announced that the U.S. Food and Drug Administration (FDA) approved TYMLOS (abaloparatide) injection for the treatment of postmenopausal women with osteoporosis at high risk for fracture defined as history of osteoporotic fracture, multiple risk factors for fracture, or patients who have failed or are intolerant to other available osteoporosis therapy. TYMLOS is the first new bone building therapy approved in nearly 15 years.

On February 1, 2017, results from the first six months of the recently completed 24- month ACTIVExtend trial were published in the Mayo Clinic Proceedings under the title of "Eighteen Months of Treatment With Subcutaneous Abaloparatide Followed by 6 Months of Treatment With Alendronate in Postmenopausal Women With Osteoporosis: Results of the ACTIVExtend Trial". The 24-month ACTIVExtend clinical trial has been completed and Radius expects to report the top-line results in the second quarter of 2017.

Pipeline Updates

Abaloparatide-SC

Radius’ marketing authorisation application (MAA) to the European Medicines Agency (EMA) for the treatment of postmenopausal women with osteoporosis is currently undergoing regulatory review, and we anticipate receiving an opinion from the Committee for Medicinal Products for Human Use (CHMP) in 2017.

Abaloparatide-TD

In September 2016, at the annual meeting of the American Association for Bone Mineral Research (ASBMR), we presented the positive results from a human replicative clinical evaluation of an optimized abaloparatide transdermal patch. These results established an important demonstration of how we have changed the pharmacokinetic profile in our program to develop a bioequivalent transdermal patch. Currently, we are focused on completing the manufacturing, scale-up, and other required activities needed to initiate a pivotal study to evaluate bioequivalence to TYMLOS. We believe that the transdermal patch program has the potential to allow physicians who treat osteoporosis, but rarely use injectable drugs, an opportunity to expand their practices to include the use of anabolic therapy.

Elacestrant (RAD1901)

We recently completed enrollment in both of our ongoing Phase 1 studies of elacestrant in advanced metastatic breast cancer. In the first half of 2017, we plan to engage with regulatory agencies to gain alignment on defining the next steps for the elacestrant breast cancer program, which would include the design of a Phase 2 trial. We expect to complete and report results from our elacestrant Phase 2b vasomotor trial in mid-2017.

RAD140

An investigational new drug application, or IND, submitted to the FDA for RAD140, a selective androgen receptor modulator, has been accepted. We expect to initiate a first-in-human Phase 1 clinical trial in women with hormone receptor positive breast cancer in 2017.

Radius Expects the Following Upcoming Milestones

Abaloparatide-SC
Receive a CHMP opinion regarding the EMA’s review of the abaloparatide-SC MAA in 2017
Enter into a partnership for the potential commercialization of abaloparatide-SC prior to commercial launch
Report top-line results from the recently completed 24-month ACTIVExtend clinical trial in the second quarter of 2017
Elacestrant
Complete ongoing Phase 1 breast cancer clinical trials
Engage with regulatory authorities in 2Q 2017 to gain alignment on defining next steps for the program, which would include the design of a Phase 2 breast cancer trial
Present a poster on June 4, 2017 at the American Society of Oncology Annual Meeting (ASCO) (Free ASCO Whitepaper)
Complete and report results from our ongoing Phase 2b vasomotor trial in mid-2017
RAD140
Initiate a first-in-human Phase 1 study in 2017 in women with hormone receptor positive breast cancer
Radius Expects To Make Presentations at the Following Upcoming Conferences

On May 4, 2017, Radius President and CEO, Robert Ward will make a presentation and host one-on-ones at the 42nd Annual Deutsche Bank Healthcare Conference in Boston
On May 16-17, 2017, Radius President and CEO, Robert Ward will make a presentation and will host one-on-ones at the Bank of America Merrill Lynch 2017 Healthcare Conference in Las Vegas
On June 4, 2017, at the 2017 ASCO (Free ASCO Whitepaper) Annual Meeting, the following abstract will be presented as a poster and as part of the Poster Discussion Session: Abstract 1014
"Evaluation of Elacestrant (RAD1901), a novel investigational, selective estrogen receptor degrader (SERD), for the treatment of ER-positive (ER+) advanced breast cancer" Abstract 1014, 8:00 AM – 11:30 AM, Hall A, Poster Board #6, POSTER SESSION, Breast Cancer-Metastatic
Discussed at the Poster Discussion Session, 4:45 PM – 6:00 PM, Hall B1, Aditya Bardia, MD, MPH – First Author, Massachusetts General Hospital Cancer Center and Harvard Medical School
On June 13-14, 2017, Radius President and CEO, Robert Ward will make a presentation and will host one-on-ones at the Goldman Sachs 38th Annual Global Healthcare Conference in Palos Verdes, California
First Quarter 2017 Financial Results

For the three months ended March 31, 2017, Radius reported a net loss of $56.9 million, or $1.32 per share, as compared to a net loss of $40.5 million, or $0.94 per share, for the three months ended March 31, 2016. The increase in net loss for the three months ended March 31, 2017 as compared to the same period in 2016 was primarily due to an increase in general and administrative expenses, including the completion of the build out of our commercial organization in the first quarter of 2017, partially offset by a decrease in research and development expenses.

Research and development expenses for the three months ended March 31, 2017 were $19.5 million, compared to $27.5 million for the same period in 2016. This decrease was primarily driven by a decrease in TYMLOS/abaloparatide-SC, abaloparatide-TD and elacestrant program development costs, partially offset by an increase in RAD140 development costs.

General and administrative expenses for the three months ended March 31, 2017 were $38.1 million, compared to $13.6 million for the same period in 2016. This increase was primarily attributable to an increase in professional support costs, including the costs associated with increasing headcount in preparation for the commercialization of TYMLOS, including the completion of our build out of our commercial organization in the first quarter of 2017. This increase was also driven by an increase in compensation expense, including stock-based compensation, due to an increase in headcount from March 31, 2016 to March 31, 2017.

As of March 31, 2017, Radius had $282.1 million in cash, cash equivalents and marketable securities. Based upon Radius’ cash, cash equivalents and marketable securities balance, Radius believes that, prior to the consideration of revenue from the potential future sales, subject to favorable regulatory review, of any of its investigational products, it has sufficient capital to fund its development plans, U.S. commercial scale-up and other operational activities for not less than twelve months from the date of this press release and into 2018.