On Mach 23, 2017 C4 Therapeutics (C4T) and Calico reported a five-year collaboration to discover, develop and commercialize therapies for treating diseases of aging, including cancer (Press release, C4 Therapeutics, MAR 23, 2017, View Source [SID1234518257]).

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Under the terms of the agreement, the parties will leverage C4T’s expertise and capabilities in targeted protein degradation to jointly discover and advance small molecule protein degraders as therapeutic agents to remove certain disease-causing proteins. The partnership will pursue preclinical research and Calico will be responsible for subsequent clinical development and commercialization of resulting products that may emerge from the collaboration.

"We are thrilled to have Calico as partners in pioneering novel therapeutics based on targeted protein degradation," said Andrew Phillips, President and Chief Scientific Officer of C4 Therapeutics. "Calico’s leadership team has a long record of innovation and our combined efforts are aimed at bringing forward new options for patients affected by devastating diseases such as cancer."

"We know from decades of translational research that it can be incredibly challenging to find effective pharmacologic inhibitors of many of the biologically well-validated targets, particularly in cancer," said Hal Barron, President of Research and Development at Calico. "Through the alternative strategy of specifically targeting such proteins for degradation, we believe we have the opportunity to identify promising new therapeutics in cancer and in other diseases as well. We’re looking forward to collaborating with C4T’s scientists and applying their protein degradation technology to the discovery and development of effective new treatments."

On March 23, 2017 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported that it is exploring strategic options for taladegib and has entered into an amended and restated license, development and commercialization agreement with Eli Lilly and Company for the taladegib oncology program (Press release, Ignyta, MAR 23, 2017, View Source [SID1234518256]). Ignyta had previously disclosed it was in discussions with Lilly regarding the optimal path forward for taladegib in the context of its pipeline priorities, which are focused substantially on molecularly targeted therapies, including its lead product candidate, entrectinib.

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The agreement amends and restates the prior license, development and commercialization agreement, dated November 6, 2015, by and between Ignyta and Lilly. Taladegib is a potent, orally bioavailable small molecule hedgehog/smoothened antagonist that has achieved clinical proof-of-concept and a recommended Phase 2 dose based on results from prior clinical studies.

"Ignyta is excited to continue to develop potential combinations of taladegib with other products to address residual disease, while exploring strategic options for single agent taladegib oral and the topical formulation of taladegib by sub-licensing or selling the franchise to dermatology-focused companies who may have an interest in superficial, nodular, and advanced basal cell carcinoma (BCC). We have received inbound interest on these assets in these BCC indications, and will be weighing the merits of these potential partnerships with other opportunities to develop taladegib internally for the benefit of patients with rare cancers outside of BCC. We are pleased to have achieved an amicable resolution with Lilly to provide Ignyta with a clear path forward for pursuing these various strategic options for taladegib," said Jonathan Lim, M.D., Chairman and CEO of Ignyta.

On March 23, 2017 EMD Serono, the biopharmaceutical business of Merck KGaA, Darmstadt, Germany in the US and Canada, and Pfizer Inc. (NYSE: PFE) reported that the US Food and Drug Administration (FDA) has approved BAVENCIO (avelumab) Injection 20 mg/mL, for intravenous use, for the treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (mMCC) (Press release, Pfizer, MAR 23, 2017, View Source [SID1234518255]). This indication is approved under accelerated approval based on tumor response and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.1 BAVENCIO was developed, reviewed and approved through the FDA’s Breakthrough Therapy Designation and Priority Review programs.

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BAVENCIO, a human anti-PD-L1 antibody, is the first FDA-approved therapy for patients with mMCC.2 Metastatic MCC is a rare and aggressive skin cancer, with fewer than half of patients surviving more than one year and fewer than 20% surviving beyond five years.3

"At the heart of this FDA approval is our drive to make a meaningful difference for patients with hard-to-treat cancers like metastatic Merkel cell carcinoma," said Belén Garijo, CEO Healthcare and Member of the Executive Board of Merck KGaA, Darmstadt, Germany. "BAVENCIO’s journey has included years of hard work – from the scientists who discovered this molecule in our labs, to our alliance with Pfizer and to the study participants and investigators worldwide. We are grateful to all who have made it possible for us to bring this important new treatment option to patients."

"Today is a significant milestone for people fighting metastatic Merkel cell carcinoma, who until now have not had any options beyond chemotherapy," said Albert Bourla, Group President, Pfizer Innovative Health. "This approval demonstrates the power of collaboration to accelerate meaningful new choices for cancer patients."

"Merkel cell carcinoma is rarer than some of the more well-known skin cancers, however, it’s very aggressive and the proportion of people who die from MCC is much higher than that of people with melanoma," said Deborah S. Sarnoff, MD, President of the Skin Cancer Foundation. "With this approval, I believe there is new hope for people and their families touched by this rare form of skin cancer."

The efficacy and safety of BAVENCIO was demonstrated in the JAVELIN Merkel 200 trial, an open-label, single-arm, multi-center study conducted in 88 patients with histologically confirmed metastatic MCC whose disease had progressed on or after chemotherapy administered for distant metastatic disease. Sixty-five percent of patients were reported to have had one prior anti-cancer therapy for metastatic MCC and 35% had two or more prior therapies. The major efficacy outcome measures were confirmed overall response rate (ORR) according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 as assessed by a blinded independent central review committee (IRC) and IRC-assessed duration of response.

The overall response rate (ORR) was 33% (95% confidence interval [CI]: 23.3–43.8%).1 Eleven percent of patients experienced a complete response (95% CI: 6.6-19.9%) and 22% of patients experienced a partial response (95% CI: 13.5-31.7%). Tumor responses were durable, with 86% of responses lasting for at least six months (n=25).1 Forty-five percent of responses lasted at least 12 months (n=13).1 Duration of response ranged from 2.8 to over 23.3 months.

The warnings and precautions for BAVENCIO include immune-mediated adverse reactions (such as pneumonitis, hepatitis, colitis, endocrinopathies, nephritis and renal dysfunction, and other adverse reactions), infusion-related reactions and embryo-fetal toxicity. The most common adverse reactions (reported in at least 20% of patients) included fatigue (50%), musculoskeletal pain (32%), diarrhea (23%), nausea (22%), infusion-related reactions (22%), rash (22%), decreased appetite (20%) and peripheral edema (20%).1 For more information, please see Important Safety Information for BAVENCIO below.

BAVENCIO is designed to potentially engage both the adaptive and innate immune systems. By binding to PD-L1, BAVENCIO is thought to prevent tumor cells from using PD-L1 for protection against white blood cells, such as T-cells, exposing them to anti-tumor responses.1 BAVENCIO has been shown to induce antibody-dependent cell-mediated cytotoxicity (ADCC) in vitro.1

BAVENCIO is available for order now.

The alliance is committed to providing industry-leading patient access and reimbursement support through its CoverOne program. This program provides a spectrum of patient access and reimbursement support services intended to help patients receive appropriate access to BAVENCIO in the United States. CoverOne may be reached by phone at 844-8COVER1 (844-826-8371) or online at www.CoverOne.com (link is external).

About JAVELIN Merkel 200

The efficacy and safety of BAVENCIO was demonstrated in the JAVELIN Merkel 200 trial, an open-label, single-arm, multi-center study conducted in 88 patients with histologically confirmed metastatic MCC whose disease had progressed on or after chemotherapy administered for distant metastatic disease. Sixty-five percent of patients were reported to have had one prior anti-cancer therapy for metastatic MCC and 35% had two or more prior therapies. The major efficacy outcome measures were confirmed overall response rate (ORR) according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 as assessed by a blinded independent central review committee (IRC) and IRC-assessed duration of response.

The trial excluded patients with autoimmune disease; medical conditions requiring systemic immunosuppression; prior organ or allogenic stem cell transplantation; prior treatment with anti-PD-1, anti-PD-L1 or anti-CTLA-4 antibodies; CNS metastases; infection with HIV, hepatitis B or hepatitis C; or ECOG performance score greater than or equal to two. Patients received BAVENCIO 10 mg/kg as an intravenous infusion over 60 minutes every two weeks until disease progression or unacceptable toxicity.

The international clinical development program for avelumab, known as JAVELIN, involves at least 30 clinical programs, including nine Phase III trials, and more than 4,000 patients across more than 15 tumor types. In October 2016, the alliance announced the European Medicines Agency accepted the Marketing Authorisation Application for avelumab for the proposed indication of metastatic MCC.

For full prescribing information and medication guide for BAVENCIO, please see www.BAVENCIO.com (link is external) or the FDA website (link is external).

IMPORTANT SAFETY INFORMATION and INDICATION

BAVENCIO can cause immune-mediated pneumonitis, including fatal cases. Monitor patients for signs and symptoms of pneumonitis and evaluate suspected cases with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold BAVENCIO for moderate (Grade 2) and permanently discontinue for severe (Grade 3), life-threatening (Grade 4), or recurrent moderate (Grade 2) pneumonitis. Pneumonitis occurred in 1.2% (21/1738) of patients, including one (0.1%) patient with Grade 5, one (0.1%) with Grade 4, and five (0.3%) with Grade 3.

BAVENCIO can cause immune-mediated hepatitis, including fatal cases. Monitor patients for abnormal liver tests prior to and periodically during treatment. Administer corticosteroids for Grade 2 or greater hepatitis. Withhold BAVENCIO for moderate (Grade 2) immune-mediated hepatitis until resolution and permanently discontinue for severe (Grade 3) or life-threatening (Grade 4) immune-mediated hepatitis. Immune-mediated hepatitis was reported in 0.9% (16/1738) of patients, including two (0.1%) patients with Grade 5 and 11 (0.6 %) with Grade 3.

BAVENCIO can cause immune-mediated colitis. Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold BAVENCIO until resolution for moderate or severe (Grade 2 or 3) colitis and permanently discontinue for life-threatening (Grade 4) or recurrent (Grade 3) colitis upon re-initiation of BAVENCIO. Immune-mediated colitis occurred in 1.5% (26/1738) of patients, including seven (0.4%) with Grade 3.

BAVENCIO can cause immune-mediated endocrinopathies, including adrenal insufficiency, thyroid disorders, and type 1 diabetes mellitus.

Monitor patients for signs and symptoms of adrenal insufficiency during and after treatment and administer corticosteroids as appropriate. Withhold BAVENCIO for severe (Grade 3) or life-threatening (Grade 4) adrenal insufficiency. Adrenal insufficiency was reported in 0.5% (8/1738) of patients, including one (0.1%) with Grade 3.



Thyroid disorders can occur at any time during treatment. Monitor patients for changes in thyroid function at the start of treatment, periodically during treatment, and as indicated based on clinical evaluation. Manage hypothyroidism with hormone replacement therapy and hyperthyroidism with medical management. Withhold BAVENCIO for severe (Grade 3) or life threatening (Grade 4) thyroid disorders. Thyroid disorders including hypothyroidism, hyperthyroidism, and thyroiditis were reported in 6% (98/1738) of patients, including three (0.2%) with Grade 3.



Type 1 diabetes mellitus, including diabetic ketoacidosis: Monitor patients for hyperglycemia or other signs and symptoms of diabetes. Withhold BAVENCIO and administer anti-hyperglycemics or insulin in patients with severe or life-threatening (Grade 3 or greater) hyperglycemia and resume treatment when metabolic control is achieved. Type 1 diabetes mellitus without an alternative etiology occurred in 0.1% (2/1738) of patients, including two cases of Grade 3 hyperglycemia.

BAVENCIO can cause immune-mediated nephritis and renal dysfunction. Monitor patients for elevated serum creatinine prior to and periodically during treatment. Administer corticosteroids for Grade 2 or greater nephritis. Withhold BAVENCIO for moderate (Grade 2) or severe (Grade 3) nephritis until resolution to Grade 1 or lower. Permanently discontinue BAVENCIO for life-threatening (Grade 4) nephritis. Immune-mediated nephritis occurred in 0.1% (1/1738) of patients.

BAVENCIO can result in other severe and fatal immune-mediated adverse reactions involving any organ system during treatment or after treatment discontinuation. For suspected immune-mediated adverse reactions evaluate to confirm or rule out an immune-mediated adverse reaction and to exclude other causes. Depending on the severity of the adverse reaction, withhold or permanently discontinue BAVENCIO, administer high-dose corticosteroids, and initiate hormone replacement therapy if appropriate. Resume BAVENCIO when the immune-mediated adverse reaction remains at Grade 1 or lower following a corticosteroid taper. Permanently discontinue BAVENCIO for any severe (Grade 3) immune-mediated adverse reaction that recurs and for any life-threatening (Grade 4) immune-mediated adverse reaction. The following clinically significant immune-mediated adverse reactions occurred in less than 1% of 1738 patients treated with BAVENCIO: myocarditis with fatal cases, myositis, psoriasis, arthritis, exfoliative dermatitis, erythema multiforme, pemphigoid, hypopituitarism, uveitis, Guillain-Barré syndrome, and systemic inflammatory response.

BAVENCIO can cause severe (Grade 3) or life-threatening (Grade 4) infusion-related reactions. Patients should be premedicated with an antihistamine and acetaminophen prior to the first 4 infusions and for subsequent doses based upon clinical judgment and presence/severity of prior infusion reactions. Monitor patients for signs and symptoms of infusion-related reactions, including pyrexia, chills, flushing, hypotension, dyspnea, wheezing, back pain, abdominal pain, and urticaria. Interrupt or slow the rate of infusion for mild (Grade 1) or moderate (Grade 2) infusion-related reactions. Permanently discontinue BAVENCIO for severe (Grade 3) or life-threatening (Grade 4) infusion-related reactions. Infusion-related reactions occurred in 25% (439/1738) of patients, including three (0.2%) patients with Grade 4 and nine (0.5%) with Grade 3.

BAVENCIO can cause fetal harm when administered to a pregnant woman. Advise patients of the potential risk to a fetus including the risk of fetal death. Advise females of childbearing potential to use effective contraception during treatment with BAVENCIO and for at least one month after the last dose of BAVENCIO. It is not known whether BAVENCIO is excreted in human milk. Advise a lactating woman not to breastfeed during treatment and for at least one month after the last dose of BAVENCIO due to the potential for serious adverse reactions in breastfed infants.

The most common adverse reactions (all grades, greater than or equal to 20%) in patients with metastatic MCC were fatigue (50%), musculoskeletal pain (32%), diarrhea (23%), nausea (22%), infusion-related reactions (22%), rash (22%), decreased appetite (20%), and peripheral edema (20%). The most common adverse reaction requiring dose interruption was anemia.

Selected treatment-emergent laboratory abnormalities (all grades, greater than or equal to 20%) in patients with metastatic MCC were lymphopenia (49%), anemia (35%), increased aspartate aminotransferase (34%), thrombocytopenia (27%). and increased alanine aminotransferase (20%). Selected treatment-emergent Grade 3-4 laboratory abnormalities (greater than or equal to 2%) were lymphopenia (19%), anemia (9%), hyperglycemia (7%), increased alanine aminotransferase (5%), and increased lipase (4%).

INDICATION
BAVENCIO is indicated for the treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Please see full Prescribing Information (link is external) and Medication Guide (link is external).

AboutBAVENCIO (avelumab)
BAVENCIO is a human programmed death ligand-1 (PD-L1) blocking antibody indicated in the US for the treatment of adults and pediatric patients 12 years of age and older with metastatic Merkel cell carcinoma.1 This indication is approved under accelerated approval based on tumor response and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

BAVENCIO is not approved in any market outside the US.

On March 23, 2016 Tessa Therapeutics, an immunotherapy company dedicated to revolutionizing the treatment of cancer, reported the full acquisition of Euchloe Bio, a biotechnology company specializing in the development and commercialization of antibodies to treat cancer (Press release, Tessa Therapeutics, MAR 23, 2017, View Source [SID1234518253]). Euchloe’s portfolio includes a number of highly potent checkpoint receptor antagonists and immune system agonists.

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Since Euchloe Bio’s spin-out from A*STAR’s Singapore Immunology Network (SIgN), the company has advanced several antibody drug candidates for clinical trials including its suite of checkpoint inhibitors (PD1, PDL1, CTLA4, Tim3 and Lag3 antibodies). Euchloe has also developed a number of these projects in combination with Tessa’s Virus Specific T cell (VST) platform technology. Euchloe’s technologies show strong potential to increase the effectiveness of Tessa’s VST therapies and expand Tessa’s pipeline to a broader range of cancers. Euchloe is collaborating with Tessa on the development of next-generation chimeric antigen receptor (CAR) technologies that are expected to show high specificity and efficacy, for a superior safety profile.

Euchloe Bio has made significant progress in its antibody development program since Tessa’s initial investment in the company last year. The combination of the technologies and research & development capabilities of the two companies is expected to realize strong synergies. Euchloe’s focus will remain on the development and commercialization of antibodies as stand-alone and combination therapies, while research efforts will continue to be led by its founder, Dr Cheng-I Wang.

Andrew Khoo, co-founder and CEO of Tessa Therapeutics, commented on the acquisition "Tessa and Euchloe have worked closely together since Tessa’s initial investment last year. We are very excited by the progress of the Euchloe team, thus the full acquisition of Euchloe Bio was a natural next step for us. Euchloe’s capabilities will help us deepen our understanding and ability to modulate the complex immunological environment around solid tumors and further strengthen our clinical trial pipeline."

Associate Professor John E. Connolly, Chief Scientific Officer of Tessa Therapeutics, said "I have known Dr Wang for many years and he is a leader in his field. Euchloe Bio’s strong expertise and extensive range of proprietary antibodies is an excellent complement to Tessa’s existing portfolio. The strong pre-clinical data we are seeing suggests that the combination of our technologies has strong potential to deliver further breakthroughs in cancer immunotherapy."
Dr Cheng-I Wang, founder of Euchloe Bio, said "We are delighted to complete our integration with Tessa Therapeutics, with whom we have been collaborators for some time. Tessa’s VST platform has proven its ability to extend immunotherapy treatment to solid tumors and I am excited by the promise of combining our different approaches. I look forward to working as a part of the Tessa team to deliver a real change in the lives of cancer patients."

On March 23, 2017 Verastem, Inc. (NASDAQ: VSTM), focused on discovering and developing drugs to treat cancer, reported financial results for the year ended December 31, 2016, and also provided an overview of certain corporate developments (Press release, Verastem, MAR 23, 2017, View Source [SID1234518251]).

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"2016 was a year of significant achievement for Verastem with the in-licensing of duvelisib, a late-stage, clinical product candidate with broad potential across B-cell and T-cell lymphoid malignancies, and the advancement of defactinib into clinical development in combination with immuno-oncology agents," said Robert Forrester, President and Chief Executive Officer of Verastem. "As we enter 2017, we are laser-focused on several important milestones, beginning with reporting top-line duvelisib data from the Phase 3 DUO study in chronic lymphocytic leukemia (CLL) expected mid-year 2017. There remains an unmet medical need for patients with relapsed CLL. We believe duvelisib has potential as a convenient, oral monotherapy with an expected and manageable safety profile for patients with relapsed CLL. For defactinib, we look forward to advancing our ongoing combination trials into important expansion cohorts across several high unmet need indications."

Mr. Forrester continued, "On the financial front, we ended 2016 with $80.9 million in cash, cash equivalents and investments, which we believe is sufficient to support our research and development programs and operations into 2018. In March 2017, we entered into a loan facility with Hercules Capital, Inc. for up to $25.0 million, subject to certain conditions including positive DUO data, which would provide us with additional financial flexibility to advance duvelisib."

Fourth Quarter 2016 and Recent Highlights:
Duvelisib
In-licensed Late-stage, Complementary Oncology Product Candidate Duvelisib – Verastem and Infinity Pharmaceuticals, Inc. (Infinity) announced the signing of an agreement under which Verastem licensed exclusive worldwide rights to develop and commercialize duvelisib, an investigational product candidate currently in development for hematologic malignancies. Duvelisib is well aligned with Verastem’s strategic focus of developing novel anti-cancer therapeutics that modulate the tumor microenvironment. The transaction provides a new oncology product candidate with demonstrated activity in lymphoid malignancies.
Ongoing Phase 3 DUO Study in Relapsed or Refractory CLL – The safety and efficacy of duvelisib is currently being evaluated in the randomized Phase 3 DUO study in patients with relapsed or refractory CLL. In the DUO study, approximately 300 patients were randomized 1:1 to receive duvelisib (25mg BID) or ofatumumab (8 weekly infusions, starting with an initial intravenous dose of 300mg on day 1 followed by 7 weekly doses of 2,000mg, then 2,000mg monthly for 4 cycles). The primary endpoint of this study is progression free survival (PFS). Key secondary endpoints include overall response rate (ORR), overall survival, duration of response (DOR) and safety. Verastem expects to report top-line data from the DUO study in mid-year 2017.
Positive Phase 2 DYNAMO Data Reported at ASH (Free ASH Whitepaper) 2016 – Positive Phase 2 clinical data from the DYNAMO study demonstrating the clinical activity of duvelisib in patients with relapsed refractory indolent non-Hodgkin lymphoma (iNHL) were presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2016. In an oral presentation, titled "A phase 2 study demonstrating the clinical activity of duvelisib in patients with relapsed refractory indolent non-Hodgkin lymphoma," (Publication ID: 1218) Ian Flinn, M.D., Ph.D. (Director, Hematologic Malignancies Program, Sarah Cannon Research Institute), described results from 129 patients with double refractory iNHL (median 3 prior anti-cancer regimens, range 1-18). The study met its primary endpoint, achieving an ORR of 46% as determined by an independent review committee (IRC) (p=0.0001; 95% CI 0.37-0.55). Among disease subgroups, the ORR was 41% in follicular lymphoma (n=83), 68% in small lymphocytic lymphoma (n=28), and 33% in marginal zone lymphoma (n=18). The median DOR among all patients was 9.9 months. Notably, 83% of patients had reductions in the size of their target lymph nodes per the IRC. Duvelisib was generally well tolerated, with an expected and manageable safety profile with appropriate risk mitigation. The DYNAMO study showed that duvelisib monotherapy has a favorable benefit-risk profile in refractory iNHL patients and may represent an important treatment option in this population.
Defactinib (VS-6063)
Dosed the First Patient in Combination Trial of Defactinib and Avelumab in Patients with Ovarian Cancer – As announced in January 2017, the first patient was dosed in a new clinical trial evaluating avelumab, an investigational fully human anti-PD-L1 IgG1 monoclonal antibody, in combination with Verastem’s defactinib, an investigational focal adhesion kinase (FAK) inhibitor, in patients with advanced ovarian cancer. This multicenter, open-label, dose-escalation and dose-expansion Phase 1/2 clinical trial is designed to assess the safety, pharmacokinetics, pharmacodynamics, and initial observations of clinical activity of the avelumab/defactinib combination in patients with recurrent or refractory stage III-IV ovarian cancer. The study is being conducted in collaboration with the alliance between Merck KGaA, Darmstadt, Germany, which in the U.S. and Canada operates as EMD Serono, and Pfizer, and is expected to enroll approximately 100 patients at up to 15 sites across the U.S.
Corporate and Financial
Hagop Youssoufian, MSc, M.D., Named Head of Hematology and Oncology Development – In January 2017, Dr. Youssoufian assumed this leadership role at Verastem to oversee the clinical and regulatory development of Verastem’s pipeline, including duvelisib, and provide overall strategic and tactical leadership to our hematology-oncology clinical programs. Dr. Youssoufian brings over 25 years of product development and commercialization experience to Verastem, having served in senior leadership roles at several oncology-focused companies, including BIND Therapeutics, Progenics Pharmaceuticals, Ziopharm Oncology, Imclone Systems, Sanofi Aventis and Bristol-Myers Squibb where he was involved in the development of Sprycel, Taxotere and Erbitux.
Additional Key Personnel Appointments – Recently, Michael Ferraresso joined Verastem as Vice President, Commercial Operations, and Verastem also appointed several highly experienced individuals to the Clinical and Scientific Advisory Board including:
Lori Kunkel, M.D., Former Chief Medical Officer, Pharmacyclics
Edmund J. Pezalla, M.D., MPH, Former VP, Pharmaceutical Policy and Strategy at Aetna
Greg Berk, M.D., Former Chief Medical Officer, Verastem
Cheryl Cohen, Former Chief Commercial Officer, Medivation
Brian Stuglik, PharmD., Former VP and Chief Marketing Officer, Oncology Global Marketing, Eli Lilly
Secured $25 Million Loan Facility – In March 2017, Verastem entered into a Loan and Security Agreement with Hercules Capital, Inc. for up to $25.0 million in financing. Verastem received the first $2.5 million of financing under the Loan and Security Agreement when the transaction closed. The proceeds will be used for Verastem’s ongoing research and development programs and for general corporate purposes. Additional tranches of up to $22.5 million in aggregate will be available subject to certain conditions, including positive data from the Phase 3 DUO clinical trial evaluating duvelisib in patients with relapsed or refractory CLL.
Full Year 2016 Financial Results
Net loss for the year ended December 31, 2016 (2016 Period) was $36.4 million, or $0.99 per share, as compared to a net loss of $57.9 million, or $1.61 per share, for the year ended December 31, 2015 (2015 Period). Net loss includes non-cash stock-based compensation expense of $6.2 million and $9.7 million for the 2016 Period and 2015 Period, respectively.
Research and development expense for the 2016 Period was $19.8 million compared to $40.6 million for the 2015 Period. The $20.8 million decrease from the 2015 Period to the 2016 Period was primarily related to a decrease of $15.6 million in external contract research organization expense for outsourced biology, chemistry, development and clinical services, which includes our clinical trial costs, a $3.4 million decrease in personnel related costs, primarily due to the reduction in workforce in October 2015, a decrease of $1.3 million in stock-based compensation expense and a decrease of $1.5 million in lab supplies, travel and other research and development expense. These decreases were partially offset by an increase of approximately $947,000 in consulting and professional fees.
General and administrative expense for the 2016 Period was $17.2 million compared to $17.6 million for the 2015 Period. The approximate $411,000 decrease from the 2015 Period to the 2016 Period primarily resulted from a decrease of $2.1 million in stock-based compensation expense. This decrease was partially offset by increases of $1.1 million in consulting and professional fees, approximately $280,000 in personnel costs, and a net increase of approximately $306,000 of other general and administrative costs.
As of December 31, 2016, Verastem had cash, cash equivalents and investments of $80.9 million compared to $110.3 million as of December 31, 2015. Verastem used $29.5 million for operating activities during the 2016 Period.
The number of outstanding common shares as of December 31, 2016, was 36,992,418.
Financial Guidance
Based on our current operating plans, we expect to have sufficient cash, cash equivalents and investments to fund our research and development programs and operations into 2018.
Conference Call Information