On January 11, 2016 Tensha Therapeutics, a privately-held company based in Cambridge, MA, reported it will be acquired by Roche (Press release, Tensha Therapeutics, JAN 11, 2016, View Source [SID1234518479]). Founded by James E. Bradner, MD, of the Dana-Farber Cancer Institute, and managed and funded by HealthCare Ventures, Tensha has developed a pioneering epigenetic technology that disrupts bromodomain and extra terminal domain (BET) proteins in order to develop potential treatments for cancer. The lead product, TEN-010, is a small molecule BET inhibitor that is currently in two Phase 1b clinical trials for the treatment of patients with cancer.

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"BET inhibitors function as targeted therapy in rare cancers with BET gene rearrangements (NUT midline carcinoma), and in common cancers as a means of inhibiting the function of the master growth control genes, such as MYC."
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Under the terms of the agreement, Tensha’s shareholders will receive an upfront cash payment of $115 million, plus additional contingent payments of up to $420 million based on the achievement of certain predetermined clinical and regulatory milestones. The transaction is subject to customary closing conditions and anticipated to close in the first quarter of 2016.

"BET proteins are a highly promising class of therapeutic targets in cancer," said James Bradner, MD, Founder of Tensha, Associate Professor at Harvard Medical School, and Investigator at the Dana-Farber Cancer Institute. "BET inhibitors function as targeted therapy in rare cancers with BET gene rearrangements (NUT midline carcinoma), and in common cancers as a means of inhibiting the function of the master growth control genes, such as MYC."

"We selected TEN-010 as a highly selective, potent BET inhibitor, and we moved rapidly and strategically to advance its development," said Steven Landau, MD, Chief Medical Officer of Tensha and Director of Clinical and Scientific Analysis for HealthCare Ventures. "Our initial clinical data demonstrating the potential of TEN-010 in patients with NUT midline carcinoma was presented at the AACR (Free AACR Whitepaper)/NCI/EORTC conference in November."

"We are very excited about this acquisition, as it moves TEN-010 into the pipeline of a world leader in the development of novel cancer therapeutics. With leadership in solid and hematological tumors and deep understanding of BET biology, Roche is the ideal company to explore the broad clinical potential of TEN-010," said Douglas E. Onsi, Chief Executive Officer of Tensha and Managing Director of HealthCare Ventures. "We appreciate the work of the Bradner lab, the Tensha and HealthCare Ventures employees, and the clinical teams at our trial sites for their important roles in bringing TEN-010 to studies in patients."

About BET Proteins

Bromodomain and extra terminal domain (BET) proteins are central mediators of gene control and cellular memory. In cancer, BET proteins activate growth and survival genes. Further, they contribute to cancer cell memory by binding to the genome as molecular bookmarks. Tensha’s Founder, James Bradner, MD, Associate Professor at the Dana-Farber Cancer Institute and Harvard Medical School, was the first to recognize the broad potential of BET inhibitors as anti-cancer agents. The Bradner lab first reported BET inhibitors in 2010 in Nature magazine, and established the potential of BET inhibitors in solid tumors and blood cancers. With chemist Jun Qi, Bradner thereafter invented TEN-010, a best-in-class BET inhibitor, for clinical development. BET inhibitors function as targeted therapy in rare cancers with BET gene rearrangements (NUT midline carcinoma), and in common cancers as a means of inhibiting the function of the master growth control genes, such as MYC. BET inhibition represents a new paradigm of targeting cellular memory, or epigenetics, in cancer, inflammation, and fibrosis.

On January 11, 2016 Cyclacel Pharmaceuticals, Inc. (Nasdaq:CYCC) (Nasdaq:CYCCP) (Cyclacel or the Company) reported 2015 achievements and provided an outline of the Company’s key business objectives for 2016 (Press release, Cyclacel, JAN 11, 2016, View Source [SID:1234508742]). These will be highlighted at the Company’s presentation during the Biotech Showcase 2016 Conference at 9:30 a.m. PST, Monday, January 11, 2016, at the Parc 55 Wyndham Hotel – Union Square at 55 Cyril Magnin Street in San Francisco.

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"During 2015, we continued to follow-up patients in SEAMLESS, our Phase 3 clinical trial evaluating sapacitabine in the front-line treatment setting of elderly patients with acute myeloid leukemia, or AML," said Spiro Rombotis, President and Chief Executive Officer of Cyclacel. "Approximately 7% of prespecified events remain to be observed before we can unblind the randomization code and report top-line results. We anticipate this happening by the end of the first half of 2016. At that point, we will analyze available data and determine submissibility to regulatory authorities. We also continued to follow patients in our Phase 1 trial of sapacitabine and seliciclib in patients with advanced solid tumors. Based on observations to date, we are extending the trial into a selected population of patients with breast cancer who are positive for BRCA mutations. Finally, we advanced CYC065, our second-generation CDK2/9 inhibitor, into a Phase 1, first-in-human study. Based on our preclinical data, we have determined the mechanistic rationale for the clinical development of CYC065 in certain hematological and solid tumor indications. We believe that 2016 may prove to be an important year for Cyclacel and we look forward to keeping you apprised of developments as the year unfolds."

2015 Achievements

Drug Development

Sapacitabine in SEAMLESS, pivotal Phase 3 study as first-line treatment in elderly patients with AML:

Continued follow-up and treatment of patients of this fully enrolled study.
7% of events remain before reporting topline results and mature data analysis.
Submitted to the European Medicines Agency (EMA) a Paediatric Investigation Plan application for sapacitabine.

Sapacitabine and seliciclib in Phase 1 study in patients with advanced solid tumors

Continued to follow patients treated with the all-oral combination of the CDK2/9 inhibitor seliciclib and sapacitabine in a Phase 1 trial in patients with advanced solid tumors. A breast cancer patient with BRCA mutations has been administered more than 70 cycles of the combination and continues on treatment.

Cyclin Dependent Kinase (CDK) Inhibitor Programs

Dosed the first patients in a Phase 1 trial of CYC065, the Company’s second-generation CDK2/9 inhibitor, to evaluate the safety, tolerability and pharmacokinetic profile of CYC065 in solid tumor and lymphoma patients.

Presented preclinical data on the molecular rationale and therapeutic potential in both hematologic and solid tumors of CYC065 at several medical conferences including the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2015, the Society of Hematologic Oncology (SOHO) 2015 Annual Meeting, the AACR (Free AACR Whitepaper)-NCI-EORTC International Conference and the San Antonio Breast Cancer Symposium (SABCS). The data show that:
CYC065 may reverse drug resistance associated with addiction of cancer cells to cyclin E, the partner protein of CDK2.
CYC065 may also inhibit CDK9-dependent oncogenic and leukemogenic pathways, including malignancies driven by certain oncogene and MLL rearrangements. MLL gene status and levels of Bcl-2 family proteins correlated with sensitivity of AML cell lines to CYC065.

CYC065’s anticancer activity presents an opportunity for patient stratification and combinations with anti-leukemic agents.
CYC065 was also effective against uterine cancer cells including those resistant to chemotherapy and was especially potent in uterine cancer cells in which cyclin E was amplified or overexpressed.

CYC065 could be active in triple-negative breast cancer.

First patients dosed in a Phase 2 investigator-sponsored trial (IST) evaluating seliciclib in patients with Cushing’s disease.
Presented preclinical data at the 4th Neuroblastoma Symposium in Newcastle Upon Tyne, UK demonstrating that CYC065 prolongs survival in MYCN-addicted neuroblastoma models.

Corporate Developments

Raised gross proceeds of $10 million from a public offering of common stock.
Entered into a Controlled Equity OfferingSM Sales Agreement with Cantor Fitzgerald & Co., as sales agent ("Cantor"), under which the Company may, from time to time, sell shares of its common stock having an aggregate offering price of up to $8.35 million through Cantor.

Entered into a license and supply agreement with ManRos Therapeutics regarding the development of oral seliciclib for the treatment of cystic fibrosis.

2016 Key Upcoming Business Objectives

Sapacitabine in SEAMLESS:

Continue follow-up of patients until the requisite number of events occur, which is anticipated by the end of the first half of 2016.
Report top-line results.

Following analysis of the mature data set determine submissibility to regulatory authorities for marketing approval.
Progress a Paediatric Investigation Plan for sapacitabine with the European Medicines Agency.
Sapacitabine in myelodysplastic syndromes (MDS):

Initiate a Phase 1/2 trial of sapacitabine in combination with other agents to determine safety and tolerability.
Plan a Phase 2 randomized controlled trial (RCT) of sapacitabine in combination with other agents following review of all relevant clinical data with mature follow-up.

Sapacitabine and seliciclib in Phase 1 study in patients with advanced solid tumors:

Initiate expansion of the Phase 1 study in a breast cancer patient population enriched for BRCA mutations.
Report updated Phase 1 data.

Cyclin Dependent Kinase (CDK) Inhibitor Programs

Report top-line results of the CYC065 Phase 1 trial in solid tumor and lymphoma patients.

Report data from seliciclib ISTs when available.

For the live and archived webcast of the Company’s presentation at the Biotech Showcase 2016 San Francisco conference, please visit the Corporate Presentations page on the Cyclacel website at www.cyclacel.com. The webcast will be archived for 90 days and the audio replay for seven days.

On January 11, 2016 Advanced Accelerator Applications S.A. (NASDAQ:AAAP) ("AAA" or "the Company"), an international specialist in molecular nuclear medicine, reported an exclusive license agreement with Johns Hopkins University in Baltimore, Maryland to develop and market PSMA-SR6, a receptor ligand of Prostate-Specific Membrane Antigen (PSMA) for clinical therapeutic and diagnostic purposes (Press release, Johns Hopkins University, JAN 11, 2016, View Source [SID1234524452]). AAA will focus on developing this treatment and its companion diagnostic for prostate cancer through novel molecular nuclear medicine techniques similar to those implemented for the development of Lutathera and Somakit. Prostate cancer affects nearly 1 in 7 men during their lifetime worldwide.

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The terms of the agreement include payment to Johns Hopkins of an upfront licensing fee, as well as certain milestone and royalty payments.

"This licensing agreement is the first step toward broadening our development pipeline by leveraging a formula that we have already successfully applied to develop our lead therapeutic and diagnostic candidates Lutathera and Somakit. We plan to radiolabel PSMA-SR6, to develop a 177Lu-PSMA-SR6 to treat and monitor prostate cancer and a 68Ga-PSMA-SR6, which will help to diagnose and stage disease. The PSMA expression pathway has been widely investigated with labelled antibodies, but we believe that a small molecule, with very high specificity and rapid uptake into tumors and clearance from non-targeted organs could be the ideal candidate for a full theragnostic approach. We are very pleased to partner with the Johns Hopkins University as they have been pioneering and leading this new field for many years," says Stefano Buono, Chief Executive Officer of AAA.

"Our license agreement with AAA extends Johns Hopkins University’s research leadership in PSMA to benefit patients," says Neil Veloso, Executive Director of Johns Hopkins Technology Ventures. "We are very pleased that AAA has selected PSMA-SR6 for full development for commercial applications in an area of significant patient need."

AAA is planning to support a proof-of-concept study in humans that may start in 2016 for both diagnostic and therapeutic applications of PSMA-SR6.

PSMA-SR6 is a unique second-generation selective prostate cancer PSMA receptor ligand developed by Dr Martin Pomper at Johns Hopkins University. PSMA-SR6 has a unique structure and is selective for PSMA expressed on prostate cancer tumor cells. It belongs to a new class of PSMA receptor ligands with high potential as diagnostic and therapeutic markers for prostate cancer. Studies have consistently demonstrated PSMA expression in all types of prostate tissue and an increased PSMA expression in cancer tissue.

On January 11, 2016 Foundation Medicine (NASDAQ:FMI) reported preliminary unaudited total revenue of approximately $26.1 million in the fourth quarter of 2015 and $93.2 million for the full year ended December 31, 2015, a 39% and 53% increase from the $18.7 million and $61.1 million recorded in the fourth quarter and full year ended December 31, 2014, respectively (Press release, Foundation Medicine, JAN 11, 2016, View Source [SID:1234508744]). The company reported 8,286 clinical tests to ordering physicians in the fourth quarter of 2015, compared to a total of 7,233 tests reported during the fourth quarter of 2014. A total of 32,998 clinical tests were reported to ordering physicians for the full year ended December 31, 2015, compared to 24,271 clinical tests reported in 2014. Cash, cash equivalents and marketable securities at December 31, 2015, was approximately $232 million.

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"Foundation Medicine achieved significant strategic and operational advances across all aspects of our molecular information business in 2015 that position the Company for continued growth in 2016 and beyond," stated Michael J. Pellini, M.D., chief executive officer of Foundation Medicine. "Specifically, we grew our commercial business and our molecular information platform to more than 68,000 cases, expanded our biopharmaceutical business both in terms of the number of partners and revenue, strengthened our leadership position through our collaboration with Roche, and secured our first national payer contract for metastatic non-small cell lung cancer with United Healthcare."

2015 Enterprise Highlights:

Completed a strategic transaction with Roche to further advance Foundation Medicine’s market-leading position in molecular information and expand access to the company’s products globally.

Grew biopharmaceutical revenue by approximately 80% in 2015 and added new molecular information and companion diagnostic collaborations with Roche, H3 Biomedicine, Mirati Therapeutics, and several other partners.

Improved patient access to comprehensive genomic profiling by signing a national agreement with United Healthcare for FoundationOne in metastatic non-small cell lung cancer. Additionally, Palmetto GBA, a Medicare Administrative Contractor (MAC), announced a final local coverage determination for comprehensive genomic profiling in a subset of patients with non-small cell lung cancer.

Expanded its molecular information platform to more than 68,000 cases.

Advanced its circulating tumor DNA (ctDNA) assay program by initiating a large, multi-center clinical study to support the anticipated commercial launch of the assay in 2016. The ctDNA assay was launched to biopharmaceutical partners in 2015, as planned.

Launched the Precision Medicine Exchange Consortium (PMEC) with leading academic medical centers and community oncology networks to advance the integration of molecular information into clinical oncology and accelerate the adoption of precision care.
Launched decision support tools to help improve utilization of molecular information by oncologists and pathologists, including enhancements to FoundationICE, such as PatientMatchTM and the introduction of GeneKitTM, a genomics solutions portal for pathologists.

Published 56 peer-reviewed manuscripts in top medical and scientific journals and presented 101 podium talks and posters at scientific and medical meetings.

2016 Outlook

The company expects revenue in 2016 will be in the range of $110 to $120 million and operating expenses will be in the range of $175 and $185 million. The company expects to deliver between 37,000 and 40,000 FoundationOne and FoundationOne Heme clinical tests in 2016. The company also expects to expand upon progress made in 2015 with Palmetto and commercial payers and drive additional coverage decisions.

As part of its commitment to providing healthcare practitioners with a full suite of analytically validated genomic profiling assays to support precision medicine in oncology, the company plans to expand its offering of molecular information products with the commercial launch of its ctDNA assay in the first quarter 2016. The assay is being rigorously evaluated as part of an ongoing, multi-center study to identify patients who are most likely to benefit from liquid-based genomic profiling. The study will also provide the analytic validation mandated for clinical use of a commercial ctDNA assay. To further support the commercial launch of the assay, the laboratory will have expansion capability for a QSR-compliant version of the assay to support companion diagnostics for potential FDA approval.

Complete quarterly and full year financial results will be announced during the company’s fourth quarter and fiscal year 2015 financial results conference call in February. This press release contains certain unaudited financial results for the company. These unaudited results could change as a result of further review by the company’s management and its independent auditors.

Dr. Pellini is scheduled to present at the 34th Annual J.P. Morgan Healthcare Conference on Wednesday, January 13, 2016, at 9:30 a.m. PST, in San Francisco. A live, listen-only webcast of the presentation and breakout session may be accessed by visiting the investors section of the company’s website at investors.foundationmedicine.com. A replay of the webcast will be available shortly after the conclusion of the presentation and breakout session and will be archived on the company’s website for two weeks.

On January 11, 2016 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported key program updates and its Annual financial guidance at the 34th annual JP Morgan Healthcare Conference (Press release, Halozyme, JAN 11, 2016, View Source [SID:1234508747]).

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"We enter the year with great momentum in our expanded PEGPH20 clinical program and with our collaboration partners, including achieving targeted enrollment in our Phase 2 study in pancreatic cancer patients and signing our sixth ENHANZE global collaboration and licensing agreement through the Lilly relationship we announced last month," said Dr. Helen Torley, president and chief executive officer. "In 2016, we will continue to invest to advance our study of the pan-tumor potential of PEGPH20, and to further expand the value of our growing ENHANZE franchise."

Program updates and recent highlights include:

Achieving target enrollment in Stage 2 of Halozyme Study 202 of investigational new drug PEGPH20 in metastatic pancreatic ductal adenocarcinoma patients. Halozyme has enrolled approximately 120 patients through the end of 2015. The company remains blinded to the efficacy results and projects presentation of mature progression-free survival data in the event driven study in the fourth quarter of 2016.

Reporting a continued reduction in the rate of thromboembolic (TE) events in the PEGPH20 treatment arm in Stage 2 of Study 202. With patient data through Dec. 15, 2015, Halozyme reported a TE event rate in the PEGPH20 arm of 12 percent (9 out of 73 patients) compared to the previously reported 42 percent (31 out of 74 patients) in Stage 1 of the study. Halozyme amended the study protocol in 2014, including the addition of prophylactic administration of low molecular weight heparin (enoxaparin) in both treatment arms based on a reported potential imbalance of TE events in the study.

The TE event rate in both stages and arms of the study are:

Progressing toward HALO-301 | Pancreatic study start, the company provided an update on the Phase 3 trial design selecting approximately 200 sites in 20 countries concentrated in North America, Europe, South America and Asia Pacific. The protocol and statistical design have been reviewed by the majority of participating countries, including the U.S. and multiple E.U. member states through the voluntary harmonization procedure (VHP).

The trial is powered for two primary endpoints, progression-free survival (PFS) and overall survival. Statistical powering to support PFS is based on achieving a hazard ratio of 0.59

Advancing development of the companion diagnostic test to prospectively identify patients with high levels of hyaluronan, or HA.
Halozyme has partnered with Ventana to develop the companion diagnostic and reported the methodology and scoring algorithm have been finalized. Based on the cutpoint for the Ventana diagnostic, Halozyme now expects approximately 35 to 40 percent of metastatic pancreatic cancer patients to have high-HA tumors, similar to the previously reported interim results from Stage 1 of its Phase 2 study using the Halozyme prototype assay.

Analysis of biopsy samples from patients in Stage 1 of Study 202 with the new diagnostic show a PFS benefit in the PEGPH20 arm with a hazard ratio of 0.48.

Signing last month a global collaboration and licensing agreement with Eli Lilly and Company to develop and commercialize products combining proprietary Lilly compounds with Halozyme’s ENHANZE platform. The agreement – Halozyme’s third in 12 months – is for up to 5 collaboration targets using Halozyme’s proprietary ENHANZE technology platform and is valued at up to $160 million for each target. The agreement included an upfront payment of $25 million.

Securing $150 million in non-dilutive financing through a royalty-backed debt transaction announced January 4.

Halozyme also provided financial guidance today for 2016, including:

Revenue of $110 million to $125 million, excluding revenue from any new ENHANZE global collaboration and licensing agreements that may be signed during the year. In 2015, Halozyme recorded $48 million for new ENHANZE partner upfront payments that was not included in 2015 guidance issued at the beginning of the year from initiation of agreements with AbbVie in June and Lilly in December.

Operating Expenses of $240 million to $260 million, supporting the initiation of the Phase 3 study in metastatic pancreatic cancer patients and the continued execution of clinical programs to study the pan-tumor potential of PEGPH20 in non-small cell lung cancer, gastric and breast cancers.

Cash flow of $35 million to $55 million, which assumes receipt in January of $25 million upfront payment from Lilly and $150M from the Royalty Backed Debt Financing.

Year-end cash balance of $140 million to $160 million.

Halozyme will present at 4 p.m. PST on Tuesday, Jan. 12 at the JP Morgan Healthcare Conference. The presentation will be webcast through the "Investors" section of Halozyme’s corporate website at www.halozyme.com, and a recording will be made available for 90 days following the event. To access the live webcast, please log on to Halozyme’s website approximately fifteen minutes prior to the presentation to register and download any necessary audio software.

About Study 202

Study 202 (Halo 109-202) is a phase 2 multi-center, randomized clinical trial evaluating investigational new drug PEGPH20 as a first-line therapy for potential treatment of patients with metastatic pancreatic cancer. The primary outcome of the trial is to measure improvement in progression-free survival in patients receiving investigational new drug PEGPH20 in combination with gemcitabine and ABRAXANE (nab-paclitaxel) compared to gemcitabine and ABRAXANE alone. A second primary endpoint will assess the TE event rate in the PEGPH20 treatment arm. Secondary endpoints also include objective response rate and overall survival. More information may be found at: View Source

About PEGPH20

PEGPH20 is an investigational PEGylated form of Halozyme’s proprietary recombinant human hyaluronidase under clinical development for the potential systemic treatment of tumors that accumulate hyaluronan.

FDA granted orphan drug designation to PEGPH20 for treatment of pancreatic cancer and fast track for PEGPH20 in combination with gemcitabine and nab-paclitaxel for the treatment of metastatic pancreatic cancer. Additionally, the European Commission, acting on the recommendation from the Committee for Orphan Medicinal Products of the European Medicines Agency, designated investigational drug PEGPH20 an orphan medicinal product for the treatment of pancreatic cancer.

About ENHANZE

ENHANZE is Halozyme’s proprietary drug delivery platform based on its patented, FDA-approved recombinant human hyaluronidase enzyme (rHuPH20) injection. The ENHANZE platform enables rHuPH20 to be co-administered with other biologics for both research and commercialization purposes. The combination of ENHANZE with some intravenously administered biologics and compounds offers the potential benefit of enabling subcutaneous delivery. In other cases, combining with ENHANZE may reduce the number of injections required for some subcutaneously administered biologics. For approved prescribing information for approved rHuPH20, visit www.hylenex.com.