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10-Q – Quarterly report [Sections 13 or 15(d)]

(Filing, 10-Q, Heat Biologics, OCT 30, 2015, View Source [SID:1234507853])

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FDA Grants Breakthrough Therapy Designation for Daiichi Sankyo and Plexxikon’s Investigational CSF-1R Inhibitor Pexidartinib (PLX3397) in Tenosynovial Giant Cell Tumor

On October 30, 2015 Daiichi Sankyo, Inc. and Plexxikon Inc., a member of the Daiichi Sankyo Group, reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to its investigational oral CSF-1R inhibitor pexidartinib (formerly PLX3397) for the treatment of tenosynovial giant cell tumor (TGCT) where surgical removal of the tumor would be associated with potentially worsening functional limitation or severe morbidity (Press release, Daiichi Sankyo, OCT 30, 2105, http://www.prnewswire.com/news-releases/fda-grants-breakthrough-therapy-designation-for-daiichi-sankyo-and-plexxikons-investigational-csf-1r-inhibitor-pexidartinib-plx3397-in-tenosynovial-giant-cell-tumor-300169556.html [SID1234515965]).

Breakthrough Therapy Designation is designed to expedite the development and review of medicines that may demonstrate substantial benefit over currently available treatments in order to ensure that patients with serious diseases have access to new treatments as soon as possible. Currently, there is no FDA-approved systemic therapy for the treatment of TGCT.

"Surgery is the primary treatment for TGCT, but for patients with a diffuse form of the condition, the tumor is more difficult to remove and has a high rate of recurrence, resulting in multiple complicated surgeries and even amputation in some patients," said Mahmoud Ghazzi, MD, PhD, Executive Vice President and Global Head of Development for Daiichi Sankyo. "We are pleased that the FDA recognizes the unmet need for the treatment of TGCT and we look forward to working closely with the Agency on the expedited development of this potential non-surgical treatment for patients with TGCT."

The Breakthrough Therapy Designation was granted based on results from an extension cohort of a single-arm, multi-center phase 1 study that assessed the safety and efficacy of pexidartinib. Results of this study were published in the July 30, 2015 issue of The New England Journal of Medicine.1 "The responses seen in our ongoing phase 1 study provided initial proof-of-concept that selective CSF-1R inhibition with pexidartinib may safely and effectively reduce tumor burden in patients with TGCT, providing the rationale to move directly into a phase 3 clinical trial," said Gideon Bollag, PhD, Chief Executive Officer of Plexxikon. "This Breakthrough Therapy Designation represents another significant milestone in our commitment to develop novel targeted agents that address unmet medical needs in rare conditions such as TGCT."

A pivotal, phase 3 study of pexidartinib called ENLIVEN is currently enrolling patients with symptomatic TGCT for whom surgical removal of the tumor would be associated with potentially worsening functional limitation or severe morbidity. More information about ENLIVEN is available at View Source

The most common treatment-related adverse events seen in the ongoing Phase 1 study of pexidartinib included fatigue, hair color changes, nausea, dysgeusia (abnormal taste), and periorbital edema (swelling around the eyes), which rarely led to drug discontinuation. Treatment-related severe adverse events included fatigue, diarrhea, anemia, hyponatremia, elevated liver enzymes and neutropenia.

About TGCT

Tenosynovial giant cell tumor (TGCT) – a group of neoplasms including pigmented villonodular synovitis (PVNS) and giant cell tumor of the tendon sheath (GCT-TS) – is a rare, usually non-metastatic tumor that affects the synovium-lined joints, bursae, and tendon sheaths, resulting in swelling, pain, stiffness and reduced mobility in the affected joint or limb.2 It is estimated that TGCT has an annual incidence of 11 cases per million.3 Patients are commonly diagnosed in their 20s to 50s,4 and depending on the type of TGCT, women can be up to twice as likely to develop a tumor as men.5

Primary treatment of TGCT includes surgery to remove the tumor, but in patients with a diffuse form where it can wrap around bone, tendons, ligaments and other parts of the joint, the tumor is more difficult to remove and may require multiple surgeries or joint replacement, eventually advancing to the point where surgery is no longer an option and amputation may be considered. It is estimated that the rate of recurrence in the diffuse form of the disease can be about 45 percent or higher in some case series.6

About Pexidartinib (PLX3397)

Pexidartinib is an investigational novel, oral small molecule that potently and selectively inhibits CSF-1R (colony stimulating factor-1 receptor), which is a primary growth driver of abnormal cells in the synovium that causes TGCT. Pexidartinib has not been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory authority for uses under investigation.

In addition to Breakthrough Therapy Designation, pexidartinib has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of PVNS and GCT-TS. Pexidartinib also has received Orphan Designation from the European Commission for the treatment of TGCT.

Pexidartinib is being evaluated in several other potential clinical indications including glioblastoma, melanoma, ovarian and breast cancer as well as in combination with anti-PD-1 therapy, pembrolizumab, for advanced melanoma and other multiple solid tumors. Pexidartinib also is being studied in the I-SPY 2 TRIAL, a collaborative research effort studying the effects of adding specific investigational drugs to standard chemotherapy prior to surgery in women with newly diagnosed, locally advanced breast cancer.

Takeda reports results for the first half of FY2015 Confirms management guidance for the full year

First half results aligned with management guidance
Underlying revenue +3.8% year-to-year (reported revenue growing +6.2% to 904 billion yen)
Underlying Core Earnings +3.7% year-to-year (operating profit -5.4% to 110.4 billion yen)

Continued performance of Takeda’s strategic growth drivers in the first half
Gastroenterology underlying revenue +28.7%, oncology up +1.6% year-to-year
Emerging markets underlying revenue +7.1% year-to-year, with particularly strong contributions from Russia and China
Consolidated underlying revenue of gastroenterology, oncology and emerging markets – Takeda’s growth drivers – is +10.0% year-to-year

First half growth supported by innovative new products
Entyvio success continues to support more than $2 billion peak sales target
Brintellix and Adcetris show steady growth
Azilva and Lotriga continue to grow strongly in Japan

Regional performance
Year-to-year underlying revenue growth in U.S. +12.0% (driven by Entyvio and Brintellix), Europe and Canada +1.5% (Entyvio and Adcetris), and emerging markets +7.1% (Russia and China)
Strong growth of new products in Japan. Underlying revenue in Japan decreased by 3.4% year-to-year as a result of increasing generic penetration

Efficiency gains continue
Project Summit achieved 11 billion yen of cost savings in the first half of FY2015
On track to deliver more than 20 billion yen savings this fiscal year

Affirming management guidance for profitable growth in FY2015
Low single-digit underlying revenue growth in FY2015, with underlying core earnings growth higher than underlying revenue growth, and underlying core EPS growth higher than underlying core earnings growth

On October 30, 2015 Christophe Weber, President and Chief Executive Officer of Takeda, reported:
"In the first half of FY2015, Takeda continued its turnaround with underlying revenue growth of 3.8%, led by our growth drivers of gastroenterology, oncology and emerging markets. Operating profit was down in the first half of the year, mainly due to an increase in expenses for several recent launches and in R&D, as planned, but underlying core earnings increased by 3.7%, getting closer to underlying revenue growth (Press release, Takeda, OCT 30, 2015, View Source [SID:1234507916]). On the basis of our first-half results, we confirm our full-year management guidance."

Underlying revenue growth in the first half was +3.8%, Underlying Core Earnings was +3.7% and Underlying Core EPS was +7.9%, with no changes in our management guidance for FY2015. Reported revenue grew +6.2% to 904.0 billion yen. An increase in expenses for new launch products, as well as a decrease in other income related to sale of real estate in 2014, resulted in a year-to-year decline in Operating Profit, Net Profit and EPS, by 6.2 billion yen, 7.1 billion yen and 9 yen, respectively. These measures have improved in the second quarter compared to the first quarter.

Underlying revenue growth was mainly driven by Takeda’s growth drivers, which are gastroenterology, oncology and emerging markets. Gastroenterology grew by +28.7% year-to-year, driven by Entyvio. Oncology revenue, including Velcade and Adcetris, increased by +1.6%. Emerging markets revenue grew by +7.1%, led by Value Brands (branded generics and OTC), with strong growth in Russia and China. Performance in the U.S. (+12.0% underlying revenue growth) and Europe and Canada (+1.5%) also contributed to revenue growth. In Japan, 　products such as Azilva and Lotriga contributed to revenue growth, but as a result of increasing generic competition, total revenue declined -3.4%.

Project Summit – a company-wide strategic initiative to increase efficiency – continued to produce results, with 11 billion yen savings in the first half. Execution of Summit initiatives in recent months include business restructuring programs, further optimization of production capacity utilization, and continued operational efficiency. A new global procurement organization also contributed by leveraging effective external spending. Efficiency initiatives as part of Project Summit remain on track, and Takeda is committed to continued operational efficiency.

Entyvio is expected to be a key global contributor to Takeda’s sales growth over the next few years, and Azilva, Lotriga, and Takecab are the products that are expected to enable Takeda to maintain a leading position in Japan. As part of the ongoing efforts of the R&D organization, ixazomib for relapsed/refractory multiple myeloma was granted accelerated assessment status in Europe in July, and Priority Review status in the U.S. in September 2015. In Japan, Takeda submitted a New Drug Application for the fixed-dose combination tablet of Nesina and metformin, and received approval for Leuplin 24 week depot for the treatment of prostate cancer and premenopausal breast cancer and for Copaxone for the treatment of multiple sclerosis in September 2015.

Takeda confirmed its management guidance for FY2015, leading to long-term sales and profit growth.

Adaptimmune Announces Upcoming Data Presentation at the 2015 Annual Meeting of SITC

On October 30, 2015 Adaptimmune Therapeutics plc (Nasdaq: ADAP), a clinical stage biopharmaceutical company focused on the use of T-cell therapy to treat cancer, reported that it will present new data from trials of its NY-ESO affinity enhanced T-cell therapy in patients with synovial sarcoma and myeloma, as well as data from preclinical safety assessments of its affinity enhanced T-cell therapy directed at MAGE A-10, at the 2015 Annual Meeting of the Society of Immunotherapy for Cancer (SITC) (Free SITC Whitepaper) (Press release, Adaptimmune, OCT 30, 2015, View Source [SID:1234507856]). SITC (Free SITC Whitepaper) is the world’s leading member-driven organization specifically dedicated to professionals working in the field of cancer immunology and immunotherapy. The meeting will take place at the McCormick Place exhibition center in National Harbor, MD on November 4 through 8, 2015.

Adaptimmune’s poster presentations will take place on November 6, 2015 in the Prince George’s Exhibition Hall from 12:45 to 2:00 pm and are as follows:

Friday November 6, 2015
Poster Presentations
Presentation Time: 12:45-2:00 pm
Location: Prince George’s Exhibition Hall

Track Name: Clinical Trials in Progress

Abstract number: 112286
Title: "Optimizing engineered TCR T-cell therapy for synovial sarcoma"
Sandra D’Angelo, M.D., Assistant Attending, Sarcoma Medical Oncology / Immunotherapeutics Core at Memorial Sloan-Kettering Cancer Center will provide additional data from the Company’s NY-ESO-1 synovial sarcoma study. Updates will include data on the expanded study group, longer follow-up and time-to-event, as well as updated correlative and safety data, and characterization of the product pre and post infusion.

Track Name: Mechanisms and Responses to Immune Therapy

Abstract number: 112346
Title: "Deep phenotypic characterization of NY-ESO TCR engineered T cells and tumor in patients with advanced myeloma"
Presenter: Eduardo Davila, Ph.D., Associate Professor of Microbiology and Immunology at the University of Maryland School of Medicine, Program Leader for Tumor Immunology and Immunotherapy Research Program at the Greenebaum Cancer Center at the University of Maryland will present follow-up data from the recently published Nature Medicine paper, including details on NY-ESO-1 T-cell phenotyping and functional data, as well as clinical and basic correlative data in myeloma patients.

Track Name: Adoptive Immunotherapy

Abstract number: 112244
Title: "Preclinical safety testing of an affinity-optimized MAGE-A10 T cell receptor for adoptive T cell therapy"
Presenter: Andrew Gerry, Ph.D., Director of Preclinical Research, Adaptimmune Therapeutics will provide a summary of the preclinical safety testing of the Company’s next affinity optimized TCR entering clinical studies in 2015, an affinity-enhanced T-cell therapy targeting MAGE-A10 in patients with non-small cell lung cancer.

Adaptimmune’s affinity enhanced T-cell candidates are novel cancer immunotherapies that have been engineered to target and destroy cancer cells by strengthening a patient’s natural T-cell response. T-cells are a type of white blood cell that play a central role in a person’s immune response. Adaptimmune’s goal is to harness the power of the T-cell and, through its multiple therapeutic candidate, significantly impact cancer treatment and clinical outcomes of patients with multiple solid and hematologic cancers.

8-K – Current report

On October 30, 2015 Heat Biologics, Inc. ("Heat") (Nasdaq:HTBX), an immuno-oncology company developing novel therapies to activate a patient’s immune system against cancer, rreported its financial results for the third quarter ended September 30, 2015 (Filing, 8-K, Heat Biologics, OCT 30, 2015, View Source [SID:1234507855]).

"During the third quarter, we continued to advance our programs while demonstrating our ability to quickly shift to accommodate the rapidly evolving immunotherapy space," said Jeff Wolf, Founder and CEO of Heat Biologics. "We treated the first patient in a new trial in non-small cell lung cancer combining our HS-110 with Opdivo, a Bristol Myers-Squibb PD-1 checkpoint inhibitor.i This trial represents the first checkpoint inhibitor/vaccine combination in non-small cell lung cancer, as well as an important paradigm for future checkpoint combinations, since published data indicates our vaccines may be synergistic with checkpoint inhibitors. Additionally, we recently completed enrollment in the randomized portion of our Phase 2 trial with our HS-410 therapy in non-muscle invasive bladder cancer and we expect to announce Phase 1 results this quarter."

Third Quarter 2015 Corporate Highlights & Recent Developments

· In October, Heat completed enrollment of the 75 patients in the company’s blinded, randomized, placebo-controlled arms of its Phase 2 clinical trial of HS-410 (vesigenurtacel-L) for the treatment of high-risk, non-muscle invasive bladder cancer (NMIBC). In these three arms, Heat is evaluating the ability of HS-410 in combination with standard of care, Bacillus Calmette-Guérin (BCG), to prevent cancer recurrence. Heat continues to enroll an additional 25 patients to evaluate HS-410 as a monotherapy in an unblinded, open-label arm. The company expects to report topline efficacy, immune-response and safety data in the fourth quarter of 2016.

· In September, Heat reported further findings from its preclinical research conducted with ComPACT in a poster at the CRI-CIMT-EATI-AACR Inaugural International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper). Heat’s next generation combination immunotherapy platform, ComPACT, combines a pan-antigen T cell priming vaccine and T cell co-stimulator in a single product, producing the first potential dual-acting immunotherapy. Heat expects to announce its selection of the first product candidate based on the ComPACT platform in the first quarter of 2016.

· In September, Heat announced the dosing of its first patient in a Phase 1b clinical trial investigating the combination of HS-110 (viagenpumatucel-L) and a PD-1 checkpoint inhibitor in patients with non-small cell lung cancer (NSCLC). Primary and second endpoints include safety and tolerability, immune response, overall response rate and progression-free survival. Topline data is expected in the fourth quarter of 2016.

Third Quarter 2015 Financial Highlights

· Research and development expenses totaled $0.7 million for the third quarter of 2015 compared to $1.0 million for the third quarter of 2014, a decrease of $0.3 million. The decrease is attributable to a reduction in pre-manufacturing costs associated with preparing to produce HS-410 and HS-110 for use in our clinical trials, offset by increases in compensation costs, lab supplies and other fees.

· Clinical and regulatory expenses totaled $3.7 million for the third quarter of 2015 compared to $1.3 million for the third quarter of 2014, an increase of $2.4 million. The increase is attributable to increases in clinical trial execution costs, investigator payments and expenses related to the production of HS-410 and HS-110 for our clinical trials.

· General and administrative expenses totaled $0.9 million for the third quarter of 2015 compared to $0.8 million for the third quarter of 2014, an increase of $0.1 million. The increase is attributable to increased employee and professional service fees.

· Net loss was $5.4 million for the third quarter of 2015, compared to $3.1 million for the third quarter of 2014.

· Cash, cash equivalents and short-term investments totaled approximately $15.0 million at September 30, 2015, compared to $14.4 million at December 31, 2014.

Year to Date 2015 Financial Highlights

· Research and development expenses totaled $1.8 million for the nine months ended September 30, 2015 compared to $2.4 million for the nine months ended September 30, 2014, a decrease of $0.6 million. The decrease is attributable to a reduction in pre-manufacturing costs associated with preparing to produce HS-410 and HS-110 for use in our clinical trials, offset by increases in compensation costs, lab supplies and other fees.

· Clinical and regulatory expenses totaled $9.3 million for the nine months ended September 30, 2015 compared to $3.2 million for the nine months ended September 30, 2014, an increase of $6.1 million. The increase is attributable to increases in clinical trial execution costs, investigator payments and expenses related to the production of HS-410 and HS-110 for our clinical trials.

· General and administrative expenses totaled $3.2 million for the nine months ended September 30, 2015 compared to $2.8 million for the nine months ended September 30, 2014, an increase of $0.4 million. The increase is attributable to increased employee and professional service fees.

· Net loss was $14.4 million for the nine months ended September 30, 2015, compared to $8.5 million for the nine months ended September 30, 2014.