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8-K – Current report

On March 3, 2016 Bio-Path Holdings, Inc., (NASDAQ: BPTH) ("Bio-Path"), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, reported positive results from the eighth and final cohort of the safety segment of Bio-Path’s Phase II trial assessing the toxicity of the Company’s lead product candidate, BP1001 (Liposomal Grb2 antisense), in combination with low-dose cytarabine (LDAC) chemotherapy in patients with advanced acute myeloid leukemia (AML) (Filing, 8-K, Bio-Path Holdings, MAR 3, 2016, View Source [SID:1234509382]).

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Patients in Cohort 8 were treated with 90 mg/m2 of BP1001 twice a week over a four-week period, in combination with a standard regimen of frontline LDAC. Results were consistent with those seen in previous cohorts, demonstrating BP1001 to be safe and well tolerated, with signs of anti-leukemia activity. The Company saw no adverse events attributable to BP1001 treatment. In Cohort 8, two AML patients achieved partial remission. One patient continues to receive additional treatments.

"The encouraging results from the eighth and final cohort of the safety portion of this trial are exciting not just for Bio-Path, but also for patients suffering from blood cancers like AML," said Peter Nielsen, President and Chief Executive Officer of Bio-Path. "These results reinforce our confidence in BP1001 as a safe and effective treatment for AML in combination with frontline chemotherapy. We look forward to assessing the efficacy of BP1001 in the Phase II clinical trial."

With the closing of Cohort 8, the Phase II safety assessment of BP1001 in combination with low-dose cytarabine is complete. The safety assessment included two cohorts, which assessed six patients evaluated with 60 mg/m2 and 90 mg/m2 of BP1001 in combination with low-dose cytarabine. No toxic side effects attributable to BP1001 treatment were observed at either dose. Of the six evaluable patients included in both cohorts of the safety segment, two achieved complete remission, while two others achieved partial remission. Of the patients responding, their blood properties show improvement, suggesting BP1001’s potential efficacy as a combination therapy.

The Company is in the process of submitting these results to the U.S. Food and Drug Administration and is planning to open the efficacy portion of the Phase II trial of BP1001 in the second quarter of 2016.

Onconova Therapeutics, Inc. Receives Notice of Termination for Convenience from Baxalta

On March 03, 2016 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer,reported that it has received notice from Baxalta US Inc. of Baxalta’s termination of the September 2012 development and license agreement between Baxalta and Onconova for rigosertib, Onconova’s lead development candidate (Press release, Onconova, MAR 3, 2016, View Source [SID:1234509375]).

Onconova, following prior consultation with Baxalta, began enrolling the first of approximately 225 patients in December 2015 for the INSPIRE trial for IV rigosertib as a treatment for higher-risk myelodysplastic syndromes (HR-MDS) after failure of hypomethylating agent (HMA) therapy.

In accordance with the terms of the Baxalta agreement, upon termination, the rights that Onconova has licensed to Baxalta, in particular the exclusive right to commercialize rigosertib for specified indications in Europe, will revert to Onconova at no cost to Onconova.

Dr. Ramesh Kumar, Onconova’s President and CEO, stated that "Onconova is deeply disappointed by the timing of Baxalta’s decision, given that patient enrollment in this pivotal trial for patients with short life spans and no alternative available therapies commenced only three months ago." Baxalta stated in its termination letter that its continuing support for the INSPIRE trial did "not align with Baxalta’s strategic priorities" and, on that basis, Baxalta terminated the agreement for convenience, effective August 30, 2016.

The INSPIRE trial is a global, multi-center, randomized controlled study to assess the efficacy and safety of IV rigosertib in HR-MDS patients under 80 years of age who had progressed on, or failed to respond to, or relapsed after previous treatment with HMAs. For these patients who have failed treatment with an HMA, there is a significant unmet medical need because there is no alternative available therapy. The INSPIRE trial is now enrolling higher-risk MDS patients who have failed all approved therapies, at multiple U.S. sites, and the Company is initiating additional clinical centers in the U.S. and abroad.

Until the August 30, 2016 termination date, Baxalta is obligated to provide various financial contributions, including 50 percent of the clinical trial costs for the INSPIRE trial up to a specified cap. Onconova is currently in discussions with Baxalta regarding the amount of financial support sufficient to complete the INSPIRE trial that was commenced in December following consultation with Baxalta.

Heron Therapeutics Provides Update on SUSTOL® NDA

On March 3, 2016 Heron Therapeutics, Inc. (NASDAQ:HRTX), reported that the U.S. Food and Drug Administration (FDA) has informed the Company that it anticipates concluding its review of the New Drug Application (NDA) of SUSTOL (granisetron) Injection, extended release, by early April 2016 (Press release, Heron Therapeutics, MAR 3, 2016, View Source [SID:1234509360]).

The Company is working closely with the FDA to facilitate the completion of its review.

"We remain committed to SUSTOL and the benefit it may provide to patients suffering from chemotherapy-induced nausea and vomiting and continue to be optimistic regarding the FDA’s review of the SUSTOL NDA," commented Barry D. Quart, Pharm.D., Chief Executive Officer of Heron Therapeutics. "We continue to be ready for the commercial launch of SUSTOL in the second quarter of 2016, if approved."

About SUSTOL (granisetron) Injection, extended release
SUSTOL is a long-acting formulation of the FDA-approved 5-hydroxytryptamine type 3 (5-HT3) receptor antagonist granisetron being developed for the prevention of both acute and delayed chemotherapy-induced nausea and vomiting (CINV) associated with moderately emetogenic chemotherapy (MEC) or highly emetogenic chemotherapy (HEC). SUSTOL is formulated utilizing Heron’s proprietary Biochronomer drug delivery technology, and has been shown to maintain therapeutic drug levels of granisetron for at least five days with a single subcutaneous injection.

Merck, Pfizer and Verastem Announce Combination Trial of Avelumab and VS-6063 in Ovarian Cancer

On March 3, 2016 Merck, Pfizer and Verastem reported that they have entered into an agreement to evaluate avelumab*, an investigational fully human anti-PD-L1 IgG1 monoclonal antibody, in combination with Verastem’s VS-6063**, an investigational focal adhesion kinase (FAK) inhibitor, in patients with advanced ovarian cancer (Press release, Merck KGaA, MAR 3, 2016, View Source [SID:1234509356]). Avelumab is currently under clinical investigation across a broad range of tumor types. The Phase I/Ib clinical trial is expected to begin in the second half of 2016. Financial terms of the agreement have not been disclosed.

"Combination strategies in immuno-oncology offer significant promise for patients in need. Through our collaboration with Verastem, we hope to accelerate our understanding of avelumab and its potential as a combination therapy with FAK inhibition for patients fighting ovarian cancer," said Dr. Alise Reicin, Head of Global Clinical Development at Merck’s biopharma business.

"Through this collaboration, we hope to advance our understanding of how FAK inhibition may complement our development program for avelumab, with the ultimate goal of potentially achieving better outcomes for women with ovarian cancer," said Chris Boshoff, Vice President and Head of Early Development, Translational and Immuno-Oncology at Pfizer Oncology.

"Recent research shows that FAK inhibitors could be beneficial in combination with immuno-oncology agents.1 We are excited to be working with Merck and Pfizer to build upon the early clinical signals observed in patients with ovarian cancer receiving combination therapy with VS-6063," said Robert Forrester, Verastem President and Chief Executive Officer.

FAK is a protein which is often overproduced in tumors, enabling cancer cells to evade attack by the immune system. As reported in the September 24, 2015, edition of Cell, pre-clinical research shows that FAK inhibition can modulate the balance of immune cells in the tumor, increasing the presence of cytotoxic T cells in the tumor and decreasing the presence of immunosuppressive T regulatory cells.1

*Avelumab is the proposed International Non-proprietary Name for the anti-PD-L1 IgG1 monoclonal antibody (MSB0010718C). Avelumab is under clinical investigation and has not been proven to be safe and effective. There is no guarantee any product will be approved in the sought-after indication by any health authority worldwide.

**VS-6063 (defactinib) is under clinical investigation and has not be proven to be safe and effective. There is no guarantee any product will be be approved in the sought-after indication by any health authority worldwide.

References
1. Serrels A et al. FAK Controls Chemokine Transcription, Tregs, and Evasion of Anti-tumor Immunity. Cell 2015; 163 (1): 160-73
2. Ferlay J et al. GLOBOCAN 2012 v1.0, Cancer Incidence and Mortality Worldwide: IARC CancerBase No. 11 [Internet]. Lyon, France: International Agency for Research on Cancer; 2013. Available from: View Source Accessed December 2015.
3. World Cancer Research Fund International. Ovarian Cancer Statistics. Available from: View Source Accessed November 2015.
4. NICE. Ovarian Cancer: Recognition and Initial Management. Available from:
View Source Accessed November 2015.
5. National Cancer Institute. Ovarian Epithelial, Fallopian Tube, and Primary Peritoneal Cancer Treatment (PDQ). Available from:View Source Accessed November 2015.
6. Ledermann JA et al. Newly diagnosed and relapsed epithelial ovarian carcinoma: ESMO (Free ESMO Whitepaper) clinical practice guidelines for diagnosis, treatment and follow-up. Ann Oncol 2013; 24(Suppl 6): vi24-32.
7. Ojalvo LS et al. Emerging immunotherapies in ovarian cancer. Discov Med 2015; 20(109): 97-109.

About Ovarian Cancer

Globally, ovarian cancer is the seventh most common cancer in women.2 Annually, nearly 239,000 cases are diagnosed worldwide.3 Ovarian cancer may be difficult to diagnose, as symptoms may appear only in the later stages, when the disease has spread beyond the ovaries.3 Outcomes for women with ovarian cancer are generally poor due to most patients presenting with advanced disease.4 The 5-year prevalence of women globally living with ovarian cancer is 22.6 per 100,000.3 Current treatment options for epithelial ovarian cancer may include surgery, radiotherapy, chemotherapy and targeted therapies.5 Women who are unable to undergo treatment with platinum-based chemotherapy, due to resistance or refractory disease, currently have very limited treatment options. Platinum-resistant ovarian cancer is defined as ovarian cancer that recurs within six months of completing primary chemotherapy with a platinum-based medication.6 Platinum-refractory ovarian cancer is defined as ovarian cancer that progresses during treatment with a platinum-based chemotherapy regimen.6 There is still a clear unmet need in ovarian cancer in relation to general disease awareness,3 improving initial investigations in primary and secondary care and novel therapies with demonstrable efficacy.7

About Avelumab
Avelumab (also known as MSB0010718C) is an investigational fully human anti-PD-L1 IgG1 monoclonal antibody. By inhibiting PD-L1 interactions, avelumab is thought to enable the activation of T-cells and the adaptive immune system. By retaining a native Fc-region, avelumab is thought to potentially engage the innate immune system and induce antibody-dependent cell-mediated cytotoxicity (ADCC). In November 2014, Merck and Pfizer announced a strategic alliance to co-develop and co-commercialize avelumab.

Autolus Limited secures £40 million funding

On March 3, 2016 UCLB spinout, Autolus Limited, a biopharmaceutical company focused on the development and commercialisation of next-generation engineered T-cell therapies for haematological and solid tumours, reported that it has raised £40 million of new capital in a Series B financing round (Press release, UCLB, MAR 3, 2016, View Source [SID:1234509353]).

Woodford Investment Management LLP ("Woodford") and Perceptive Bioscience Investments Ltd ("Perceptive Bioscience") participated in the new investment, which augments the previous £30 million Seed and Series A investment from founding investor Syncona LLP ("Syncona").

The funds will enable Autolus to develop its proprietary pipeline of engineered T-cell products, and to further implement its industry-leading platform of T-cell programming technologies. In parallel with the financing, the Company’s technology platform was enhanced by a licence to additional technologies from UCLB, UCL’s technology transfer company.

In association with the financing, Dr Joe Anderson, Chief Executive Officer of Perceptive Bioscience, has joined the board of directors of Autolus.

Dr Christian Itin, Chairman of Autolus, said:
"We are pleased to welcome investors of the calibre of Woodford Investment Management and Perceptive Bioscience, which support our goal of building Autolus into a leading engineered T-cell company. The quality of Autolus’ technology and pipeline has allowed the company to raise £70m since its foundation in September 2014, and positions us to take multiple programmes into the clinic. We have also expanded the scope of our licence with UCLB to bring additional inventions from founder Dr Martin Pule’s group into the company adding to the suite of Autolus’ T-cell programming technologies."

Dr Joe Anderson, CEO of Perceptive Bioscience, added:
"We are delighted that Perceptive Bioscience’s first investment is in a company with the potential to transform cancer therapy. Autolus is at the cutting-edge of T-cell engineering to create a new generation of programmed T-cells acting as agents to kill tumour cells. Our investment in Autolus underlines our commitment to supporting emerging, innovative companies. Our flexible investment platform enables us to assist companies with finance and practical support whatever their stage of development."

Dr Martin Murphy, CEO of Syncona, said:
"We are excited that Woodford and Perceptive have invested in Autolus. The company is now funded by a group of investors that share our vision of creating sustainable standalone businesses that will develop products designed to deliver exceptional benefit to patients in areas of high unmet need."

Cengiz Tarhan, Managing Director of UCLB, commented:
"We are delighted to support this significant milestone in Autolus’ development. The licence and the commitment from Syncona, Woodford and Perceptive Bioscience provide a solid foundation to translate the licensed UCL technologies into healthcare benefits for patients."

Autolus is founded upon the work of Dr Martin Pule, an academic clinical haematologist at the UCL Cancer Institute and NIHR University College London Hospitals Biomedical Research Centre and a thought-leader in T-cell engineering. It is a next-generation engineered T-cell company, developing a series of T-cell products based on its proprietary targets, constructs and technologies.