On November 7, 2017 Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to improve the survival and quality of life of cancer patients, reported financial results for the third quarter ended September 30, 2017 and provided an overview of certain corporate developments and plans (Press release, Verastem, NOV 7, 2017, View Source;p=RssLanding&cat=news&id=2314866 [SID1234521654]).

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"The third quarter was a pivotal time for Verastem with the announcement of positive top-line data from the pivotal Phase 3 DUO study evaluating oral duvelisib monotherapy in patients with relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL)," said Robert Forrester, President and Chief Executive Officer of Verastem. "The DUO study met its primary endpoint, with progression-free survival (PFS) significantly favoring duvelisib monotherapy over ofatumumab, an approved standard of care treatment for patients with CLL/SLL. We recently met with the U.S. Food and Drug Administration (FDA), and based on the meeting and written feedback, we intend to submit, during the first quarter of 2018, a New Drug Application (NDA) requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory CLL/SLL and accelerated approval for the treatment of patients with relapsed or refractory follicular lymphoma (FL). The NDA will be based on a comprehensive clinical data package, including the Phase 3 DUO and Phase 2 DYNAMO studies evaluating duvelisib in patients with advanced hematologic malignancies."

Mr. Forrester concluded, "At the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) 2017 Annual Meeting (ASH 2017), we will be presenting data from multiple studies, including the detailed positive results from the Phase 3 DUO study, which were selected for an oral presentation."

Third Quarter 2017 and Recent Highlights:

Duvelisib

Announced Regulatory Strategy for Duvelisib NDA – Verastem recently announced that a meeting was held with the U.S. Food and Drug Administration (FDA) regarding the regulatory path for duvelisib. Based on the meeting with, and written feedback from the FDA, Verastem intends to submit a New Drug Application (NDA) requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory CLL/SLL and accelerated approval for the treatment of patients with relapsed or refractory FL. Along with the clinical data from the DUO study, the duvelisib NDA submission will also contain the favorable results from the Phase 2 DYNAMO study in double-refractory indolent non-Hodgkin’s lymphoma (iNHL), which also achieved its primary endpoint with an ORR of 46% (p<0.0001). In the subset of patients enrolled in the DYNAMO study with double-refractory FL (n=83), duvelisib demonstrated an ORR of 41%. The Company expects to submit the duvelisib NDA during the first quarter of 2018.
Reported Positive Top-line Data from the Pivotal Phase 3 DUO Study in Relapsed or Refractory CLL/SLL – In September 2017, Verastem reported that the Phase 3 DUO study met its primary endpoint with oral duvelisib monotherapy demonstrating superiority over ofatumumab for PFS in patients with CLL/SLL. In this study, duvelisib achieved a statistically significant improvement in median PFS of 13.3 months, compared to 9.9 months for ofatumumab with a hazard ratio of 0.52 (p<0.0001), representing a 48% reduction in the risk of progression or death. Duvelisib monotherapy had a manageable safety profile, with results from this study consistent with the well-characterized safety profile of duvelisib monotherapy in patients with advanced hematologic malignancies.
Expanded Duvelisib Program to Include Peripheral T-Cell Lymphoma – In September 2017, Verastem announced the expansion of its duvelisib development program to include targeting the treatment of patients with Peripheral T-Cell Lymphoma (PTCL). Duvelisib was granted Fast Track designation by the FDA for the treatment of patients with PTCL who have received at least one prior therapy. Development of duvelisib in PTCL is supported by encouraging Phase 1 clinical data which demonstrated a 50% investigator-assessed overall response rate in 16 heavily pre-treated patients with relapsed or refractory PTCL, including 3 (19%) complete responses and 5 (31%) partial responses. Verastem intends to initiate an open-label, multicenter, Phase 2 clinical trial evaluating the efficacy and safety of duvelisib in patients with relapsed or refractory PTCL by the end of 2017.
Clinical Data from Pivotal Phase 3 DUO Study Selected for Oral Presentation at ASH (Free ASH Whitepaper) 2017 – Verastem recently announced that an abstract highlighting clinical data from the Phase 3 DUO study was selected for oral presentation at ASH (Free ASH Whitepaper) 2017. The presentation, titled "Results from the Phase 3 DUO Trial: A Randomized Comparison of Duvelisib Vs Ofatumumab in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma," will be presented by principal investigator, Ian Flinn, M.D., Ph.D., Director of the Blood Cancer Research Program at Sarah Cannon Research Institute, on Sunday, December 10, 2017 at 4:30pm ET at the Georgia World Congress Center, in Building B, Level 5, Murphy BR 3-4.
Additional Duvelisib Abstracts Selected for Presentation at ASH (Free ASH Whitepaper) 2017 – Along with the Phase 3 DUO results, two additional duvelisib abstracts were selected for presentation at ASH (Free ASH Whitepaper) 2017. The abstract, titled "In Vitro, In Vivo, and Parallel Phase I Evidence Support the Safety and Activity of Duvelisib, a PI3K-δ,γ Inhibitor, in Combination with Romidepsin or Bortezomib in Relapsed/Refractory T-Cell Lymphoma," will be given as an oral presentation by Steven Horowitz, M.D., Memorial Sloan Kettering Cancer Center, on Monday, December 11, 2017 at 5:00pm ET at the Georgia World Congress Center, in Building A, Level 4, Marcus Auditorium. The abstract, titled "The Dual PI3K-δ,γ Inhibitor Duvelisib Stimulates Anti-Tumor Immunity and Enhances Efficacy of Immune Checkpoint and Co-Stimulatory Antibodies in a B Cell Lymphoma Model," will be given as a poster presentation by Jonathan Pachter, Ph.D., Chief Scientific Officer of Verastem, on Saturday, December 9, 2017 from 5:30-7:30pm ET at the Georgia World Congress Center, in Building A, Level 1, Hall A2.
Verastem to Host Key Opinion Leader Event at ASH (Free ASH Whitepaper) 2017 – On Sunday, December 10, 2017, Verastem will host an investor and analyst reception, which will feature a moderated panel discussion/Q&A including Ian Flinn, MD, Director of the Blood Cancer Research Program at Sarah Cannon Cancer Center in Nashville, TN. The event will take place during the ASH (Free ASH Whitepaper) 2017 annual meeting and interested parties can access a live webcast of the event beginning December 10, 2017 at 8pm ET by going to the "News and Press" section of the Verastem website at www.verastem.com.
Defactinib

Published Scientific Data Highlighting Potential Role of Focal Adhesion Kinase (FAK) Inhibition in Pancreatic and Breast Cancer – In July 2017, Verastem announced the publication of two papers in the peer-reviewed journals, PLoS One and Oncotarget. The two published articles reported scientific findings from studies evaluating FAK inhibition in preclinical models of pancreatic and breast cancer and continue to validate the underlying thesis for ongoing clinical collaborations evaluating Verastem’s lead FAK inhibitor, defactinib, in combination with chemotherapeutic and leading immunotherapeutic agents in several difficult to treat types of cancer. The PLoS One paper in pancreatic cancer is available here and the Oncotarget paper in breast cancer is available here.
Defactinib Preclinical Abstract Selected for Presentation at ASH (Free ASH Whitepaper) 2017 – An abstract describing preclinical data in combination with BCL-2 was selected for presentation at ASH (Free ASH Whitepaper) 2017. The abstract, titled "Combinatorial Inhibition of Focal Adhesion Kinase and BCL-2 in AML," will be given as a poster presentation by Xiangmeng Wang, Ph.D., on Sunday, December 10, 2017 from 6:00-8:00pm ET at the Georgia World Congress Center, in Building A, Level 1, Hall A2.
Corporate and Financial

Brian Stuglik, R.Ph. Appointed to the Board of Directors – In September 2017, Verastem announced the appointment of Mr. Stuglik to its Board. Mr. Stuglik, former Chief Marketing Officer for Lilly Oncology, brings to Verastem 35 years of experience in pharmaceutical and oncology commercialization in both the U.S. and international markets. He has successfully launched several multi-billion dollar brands over his career, including Gemzar, Alimta and Erbitux.
NgocDiep Le, M.D., Ph.D., Appointed Chief Medical Officer – In October 2017, Verastem announced the appointment of Dr. Le as its Chief Medical Officer. A trained medical oncologist, Dr. Le is board certified in internal medicine and has 15 years of drug development experience across all phases in both solid and hematologic malignancies as well as IND and NDA submissions. Dr. Le joins Verastem from MedImmune (a subsidiary of AstraZeneca) where she served as Vice President, Immuno-Oncology Innovative Medicines and led the product development teams for multiple high-priority immuno-oncology assets. Prior to joining MedImmune, Dr. Le held roles of increasing responsibilities at Novartis and at GlaxoSmithKline where she led the MEK inhibitor, trametinib (MekinistTM), from the first-in-human studies to FDA approval. Dr. Le received a Bachelor in Science degree from the California Institute of Technology, and earned both MD and PhD degrees from Stanford University School of Medicine. Dr. Le will oversee the development strategy and activities for Verastem’s duvelisib and defactinib.
Julie B. Feder Appointed Chief Financial Officer – In July 2017, Verastem announced the appointment of Ms. Feder as its Chief Financial Officer. Ms. Feder is an accomplished financial professional with invaluable leadership experience in the healthcare industry. She joins Verastem from the Clinton Health Access Initiative, Inc. (CHAI), where she served as Chief Financial Officer. Prior to joining CHAI, Ms. Feder held finance roles of increasing responsibility at Genzyme Corporation including leading the internal audit function. Ms. Feder began her career at Deloitte & Touche LLP and she holds a Bachelor of Science in Accounting from Yeshiva University’s Sy Syms School of Business.
Achieved First Development Milestone Related to the Duvelisib License Agreement – In September 2017, upon achievement of positive top-line results from the Phase 3 DUO study, Verastem determined that the pre-specified criteria for the first milestone under the license agreement with Infinity Pharmaceuticals, Inc. (Infinity) had been met and recorded $6.0 million as research and development expense. Subsequently, in October 2017, Verastem made the milestone payment of $6.0 million to Infinity. The milestone was paid using funds drawn from Verastem’s existing loan and security agreement with Hercules Capital, Inc.
Third Quarter 2017 Financial Results

Net loss for the three months ended September 30, 2017 (2017 Quarter) was $23.1 million, or $0.61 per share, as compared to a net loss of $7.9 million, or $0.21 per share, for the three months ended September 30, 2016 (2016 Quarter). Net loss includes non-cash stock-based compensation expense of $1.7 million and $1.3 million for the 2017 Quarter and 2016 Quarter, respectively. Verastem used $11.8 million for operating activities during the 2017 Quarter.

Research and development expense for the 2017 Quarter was $17.7 million compared to $4.2 million for the 2016 Quarter. The $13.5 million increase from the 2016 Quarter to the 2017 Quarter was primarily related to the achievement of a $6.0 million milestone pursuant to Verastem’s license agreement with Infinity, an increase of $4.8 million in contract research organization (CRO) expense for outsourced biology, development and clinical services, which includes Verastem’s clinical trial costs, an increase of approximately $2.0 million in consulting fees, an increase in stock-based compensation of approximately $423,000 and an increase in personnel related costs of approximately $153,000.

General and administrative expense for the 2017 Quarter was $5.4 million compared to $3.8 million for the 2016 Quarter. The increase of $1.6 million from the 2016 Quarter to the 2017 Quarter primarily resulted from increases in consulting and professional fees of $1.3 million and personnel costs of approximately $330,000.

As of September 30, 2017, Verastem had cash, cash equivalents and investments of $60.3 million compared to $80.9 million as of December 31, 2016.

The number of outstanding common shares as of September 30, 2017, was 39,945,028.

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 the second half of 2018.

About Duvelisib

Duvelisib is an investigational, dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells and T-cells. PI3K signaling may lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 Duvelisib is currently being evaluated in late- and mid-stage clinical trials, including DUO, a randomized, Phase 3 monotherapy study in patients with relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL),4 and DYNAMO, a single-arm, Phase 2 monotherapy study in patients with refractory indolent non-Hodgkin lymphoma (iNHL).5 Both DUO and DYNAMO achieved their primary endpoints and Verastem is preparing to submit a New Drug Application to the U.S. Food and Drug Administration for the treatment of patients with relapsed or refractory CLL/SLL and patients with follicular lymphoma (FL) whose disease has progressed and are refractory to rituximab and to either chemotherapy or radioimmunotherapy. Duvelisib is also being developed by Verastem for the treatment of peripheral T-cell lymphoma (PTCL), and is being investigated in combination with other agents through investigator-sponsored studies.6 Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

About Defactinib

Defactinib is an investigational inhibitor of FAK, a non-receptor tyrosine kinase that mediates oncogenic signaling in response to cellular adhesion and growth factors.7 Based on the multi-faceted roles of FAK, defactinib is used to treat cancer through modulation of the tumor microenvironment, enhancement of anti-tumor immunity, and reduction of cancer stem cells.8,9 Defactinib is currently being evaluated in three separate clinical collaborations in combination with immunotherapeutic agents for the treatment of several different cancer types including pancreatic, ovarian, non-small cell lung cancer, and mesothelioma. These studies are combination clinical trials with pembrolizumab and avelumab from Merck & Co. and Pfizer/Merck KGaA, respectively.10,11,12 Information about these and additional clinical trials evaluating the safety and efficacy of defactinib can be found on www.clinicaltrials.gov.

On November 7, 2017 Nektar Therapeutics (Nasdaq: NKTR) reported that new clinical data will be presented for NKTR-214, Nektar’s lead immuno-oncology candidate, a CD122-biased agonist, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting at the Gaylord National Hotel & Convention Center in National Harbor, Maryland (Press release, Nektar Therapeutics, NOV 7, 2017, View Source [SID1234521653]). Data from the ongoing PIVOT Phase 1/2 study, which is evaluating NKTR-214 in combination with the checkpoint inhibitor nivolumab, will be presented in an oral presentation on Saturday, November 11th. Six additional presentations for immuno-oncology (I-O) candidates in Nektar’s pipeline will also be presented at the meeting; specifically, one additional clinical and three preclinical presentations on NKTR-214; one preclinical presentation on NKTR-255, an IL-15 memory T-cell stimulating cytokine; and one preclinical presentation on NKTR-262, a novel toll-like receptor (TLR) agonist. The abstracts published in advance of the SITC (Free SITC Whitepaper) Annual Meeting were made available at 8 a.m. Eastern Time today on the Journal for ImmunoTherapy of Cancer’s website at View Source

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"The initial abstract data for these first set of 16 patients from the PIVOT study evaluating NKTR-214 in combination with nivolumab is very exciting," said Mary Tagliaferri, M.D., Senior Vice President of Clinical Development at Nektar Therapeutics. "We look forward to presenting additional new data from all of the 38 patients enrolled in the dose escalation cohort of the study in Dr. Diab’s oral presentation at SITC (Free SITC Whitepaper) on Saturday afternoon, including the first efficacy data for patients with PD-L1 negative non-small cell lung cancer who had progressed on prior chemotherapy. We believe there is tremendous need for more efficacious and better tolerated I-O combination treatment regimens, particularly treatments that can be used for the majority of cancer patients whose tumors don’t express PD-L1 as these patients can’t currently benefit from treatment with checkpoint inhibitors."

NKTR-214 is an investigational immuno-stimulatory therapy designed to expand specific cancer-fighting CD8+ effector T cells and natural killer (NK) cells directly in the tumor micro-environment and increase expression of PD-1 on these immune cells.

Details of the SITC (Free SITC Whitepaper) presentations for Nektar’s I-O portfolio are below.

Oral Presentation

Date: Saturday, November 11, 2017, Presentation Time: 5:00 p.m. Eastern Time
Session Title: Clinical Trials: Novel Combinations
Presentation Title: PIVOT-02: Preliminary safety, efficacy and biomarker results from the Phase 1/2 study of CD-122-biased agonist NKTR-214 plus nivolumab in patients with locally advanced/metastatic solid tumors
Presenter: Dr. Adi Diab, MD Anderson Cancer Center
Location: Gaylord National Hotel & Convention Center, Maryland Ballroom A

The online abstract for the oral clinical presentation includes preliminary data as of July 25, 2017 for 16 patients with Stage IV melanoma and renal cell carcinoma enrolled in the dose-escalation phase of the PIVOT study of NKTR-214 in combination with nivolumab (NCT02983045). New and updated interim data for a total of 38 patients with Stage IV melanoma, renal cell carcinoma and non-small cell lung cancer which were enrolled in the dose-escalation phase of the PIVOT study, will be presented at the SITC (Free SITC Whitepaper) Annual Meeting. A copy of the slides from Dr. Diab’s presentation will be made available on Nektar’s corporate website on Saturday, November 11th at 6:00 p.m. Eastern Time, in accordance with the embargo policies set forth by SITC (Free SITC Whitepaper).

Poster Presentations

Biomarkers and Immune Monitoring

Clinical Data Abstract #P77: The Novel IL-2 Cytokine Immune Agonist NKTR-214 Harnesses the Adaptive and Innate Immune System for the Treatment of Solid Cancers
Presenter: Salah Eddine Bentebibel, University of Texas MD Anderson Cancer Center
Date and Time: Friday, November 10, 2017, 12:30-2:00 p.m. Eastern Time

Cancer Vaccines

Preclinical Data Abstract #P140: NKTR-214 enhances anti-tumor T-cell immune responses induced by checkpoint blockade or vaccination
Presenter: Meenu Sharma, University of Texas MD Anderson Cancer Center
Date and Time: Saturday, November 11, 2017, 12:30-2:00 p.m. Eastern Time

Combination Therapy

Preclinical Data Abstract #P274: Combination of NKTR-214 and radiotherapy (RT) to reverse anergy and expand tumor-specific CD8 T Cells
Presenter: Joshua Walker, Oregon Health & Science University
Date and Time: Saturday, November 11, 2017,12:30-2:00 p.m. Eastern Time

Preclinical Data Abstract #P275: Harnessing the innate and adaptive immune system to eradicate treated and distant untreated solid tumors
Presenter: Saul Kivimae, Nektar Therapeutics
Date and Time: Friday, November 10, 2017, 12:30-2:00 p.m. Eastern Time

Immune Modulation, Cytokines, and Antibodies

Preclinical Data Abstract #P332: Pre-clinical efficacy and tolerability of NKTR-255, a polymer-conjugated IL-15 for immuno-oncology
Presenter: Peiwen Kuo, Nektar Therapeutics
Date and Time: Saturday, November 11, 2017, 12:30-2:00 p.m. Eastern Time

Personalized Vaccines and Technologies/Personalized Medicines

Preclinical Data Abstract #P434: Great Apes Adenoviral vaccine encoding neoantigens synergizes with immunomodulators to cure established tumors in mice
Presenter: Anna Morena D’Alise, Nouscom srl
Date and Time: Saturday, November 11, 2017, 12:30-2:00 p.m. Eastern Time

Analyst and Investor Event
Nektar Therapeutics will webcast an analyst and investor event with clinical investigators during the SITC (Free SITC Whitepaper) Annual Meeting beginning on Saturday, November 11, 2017 at 6:15 p.m. Eastern Time. Featured speakers include Dr. Patrick Hwu of MD Anderson Cancer Center, Dr. Adi Diab of MD Anderson Cancer Center, Dr. Michael E. Hurwitz of Yale Cancer Center, Dr. Antoni Ribas of UCLA Medical Center and Dr. Nizar M. Tannir of MD Anderson Cancer Center. The webcast is accessible from the investor relations page of Nektar’s website at www.nektar.com.

About NKTR-214
NKTR-214 is a clinical-stage, immuno-stimulatory therapy designed to expand specific cancer-fighting CD8+ effector T cells and natural killer (NK) cells directly in the tumor micro-environment and increase expression of PD-1 on these immune cells. NKTR-214 is a CD122-biased IL-2 pathway agonist that targets cancer-fighting immune cells to stimulate their proliferation and activation. In clinical and preclinical studies, treatment with NKTR-214 resulted in expansion of these cells and their mobilization into the tumor.1,2 NKTR-214 has an antibody-like dosing regimen similar to the existing checkpoint inhibitor class of approved medicines.

About NKTR-255
NKTR-255 is an immune-stimulating cytokine designed to engage all three IL-15 receptors and signal through all of these to activate the IL-15 pathway. NKTR-255 stimulates proliferation and survival of Natural Killer (NK) Cells, CD8+ T Cells and enhances long-term immunological memory which may lead to sustained anti-tumor immune response.

About NKTR-262
NKTR-262 is a first-in-class investigational small molecule intended to target toll-like receptors (TLRs) on innate immune cells in the body. NKTR-262 is designed to overcome the body’s dysfunction of antigen-presenting cells (APC), and is being developed as a single intra-tumoral injection to be administered at the start of therapy with NKTR-214 to induce an abscopal response and achieve the goal of complete tumor regression in cancer patients treated with both therapies.

On November 7, 2017 Acceleron Pharma Inc. (NASDAQ:XLRN), a leading biopharmaceutical company in the discovery and development of TGF-beta therapeutics to treat serious and rare diseases, reported a corporate update and financial results for the third quarter ended September 30, 2017 (Press release, Acceleron Pharma, NOV 7, 2017, View Source [SID1234521623]).

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"As we think about and plan to execute on our long-term vision and strategy, there were several significant corporate events in the third quarter. At our September R&D day, we outlined our core research and development focus in three disease areas of high unmet medical need: hematology, neuromuscular, and pulmonary disease. We announced that we gained rights to sotatercept, an internally discovered Phase 2 asset, for the development in pulmonary arterial hypertension. We also completed a successful equity offering that will provide sufficient funding through key inflection points in each of our clinical programs," said Habib Dable, President and Chief Executive Officer of Acceleron. "We and our partner Celgene continue to invest heavily in our luspatercept development plan with seven clinical trials expected to be ongoing in 2018. In neuromuscular diseases, ACE-083 continues to advance in the Phase 2 trials in FSHD and CMT, and we remain on track to launch a Phase 2 trial with sotatercept in the first half of 2018 as we work to grow our pulmonary franchise, and ultimately deliver transformative treatment options to patients in need."

Development Program Highlights

Hematology

Luspatercept:

Myelodysplastic Syndromes (MDS), Beta-Thalassemia, and Myelofibrosis

Luspatercept is designed to treat chronic anemia and reduce red blood cell (RBC) transfusion burden in adults with rare blood disorders. Luspatercept is being developed as part of the global collaboration between Acceleron and Celgene.

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In addition to the ongoing MEDALIST and BELIEVE Phase 3 trials, Acceleron and Celgene continue to prepare for clinical trial expansion in new patient populations, including the COMMANDS Phase 3 trial in first-line, lower-risk MDS, regardless of ring sideroblast status, the BEYOND Phase 2 trial in non-transfusion-dependent beta-thalassemia, and the Phase 2 trial in myelofibrosis.

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Results from the Phase 2 trial of luspatercept for the treatment of anemia in patients with lower-risk MDS were recently published in The Lancet Oncology.

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Data from two clinical abstracts on luspatercept and sotatercept will be presented at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Atlanta, GA on December 9-12, 2017.

Neuromuscular Disease

ACE-083:

Facioscapulohumeral muscular dystrophy (FSHD) and Charcot-Marie-Tooth (CMT) disease

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ACE-083 is a locally-acting therapeutic designed to have a concentrated effect on muscle mass and strength in target muscles for diseases that cause debilitating focal muscle loss by utilizing the "Myostatin+" approach to inhibit multiple TGF-beta ligands.

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Enrollment and treatment are ongoing in Part 1 of the Phase 2 trial in patients with FSHD, one of the most prevalent forms of muscular dystrophy in adults.

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Enrollment and treatment are ongoing in Part 1 of the Phase 2 trial in patients with CMT disease, one of the most common inherited neurological diseases leading to focal muscle weakness.

ACE-2494:

ACE-2494 is a protein therapeutic designed to have a systemic effect on muscle mass and strength throughout the body by utilizing the "Myostatin+" approach to inhibit multiple TGF-beta ligands.

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The Company plans to initiate a Phase 1 healthy volunteer clinical trial this year and is actively evaluating a potential first indication.

Pulmonary Disease

Sotatercept:

Sotatercept is an activin receptor type IIA fusion protein that acts as a ligand trap for members in the TGF-beta protein superfamily involved in remodeling and regeneration of a variety of different tissues, including the vasculature and fibrotic tissue.

•
Acceleron gained development and commercialization rights for pulmonary arterial hypertension (PAH).

•
Preclinical results presented at R&D day show potential for sotatercept to be a first-in-class disease-modifying therapy that addresses fundamental molecular causes of disease in PAH.

•
Preclinical results of sotatercept in PAH will be highlighted in an oral presentation at the American Heart Association Scientific Sessions 2017 in Anaheim, CA on November 14, 2017.

Key Corporate Priorities

Luspatercept
•
Report top-line results from MEDALIST and BELIEVE Phase 3 trials in mid-2018
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Initiate the COMMANDS Phase 3 trial in first-line, lower-risk MDS in 1H 2018
•
Enroll the first myelofibrosis patient in Phase 2 by YE 2017
•
Initiate the BEYOND Phase 2 trial in non-transfusion-dependent beta-thalassemia by YE 2017

ACE-083
•
Report FSHD Phase 2 results for cohort 1 in Part 1 in January 2018
•
Report FSHD Phase 2 results for all dose-escalation cohorts in Part 1 in 2018
•
Report CMT Phase 2 results from all dose-escalation cohorts in Part 1 by YE 2018

ACE-2494
•
Initiate Phase 1 healthy volunteer trial in 2017

Sotatercept
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Initiate Phase 2 trial in PAH first half of 2018

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Financial Results
•
Cash position – Cash, cash equivalents and investments as of September 30, 2017 were $366.6 million. As of December 31, 2016 the Company had cash, cash equivalents and investments of $234.4 million. Cash, cash equivalents and investments include $187.6 million of net proceeds from a follow-on public offering of common stock in September 2017. In October 2017, the underwriters exercised the over-allotment option in the offering which resulted in additional net proceeds of $28.2 million. We believe that existing cash, cash equivalents and investments, including the net proceeds from the offering and the exercise of the underwriters’ over-allotment option, will be sufficient to fund projected operating requirements into 2021.
•
Revenue – Collaboration revenue for the third quarter was $3.0 million. The revenue is all from our Celgene partnership and is primarily due to cost sharing revenue of $2.9 million related to expenses incurred by the Company in support of our partnered programs.
•
Costs and expenses – Total costs and expenses for the third quarter were $28.6 million. This includes R&D expenses of $21.1 million and G&A expenses of $7.5 million.
•
Net loss – The Company’s net loss for the third quarter ended September 30, 2017 was $25.5 million.

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Conference Call and Webcast
The Company will host a webcast and conference call to discuss its third quarter 2017 financial results and provide an update on recent clinical development and corporate activities on November 7, 2017, at 8:00 a.m. EST.
The webcast will be accessible under "Events & Presentations" in the Investors/Media page of the Company’s website at www.acceleronpharma.com. Individuals can participate in the conference call by dialing 877-312-5848 (domestic) or 253-237-1155 (international) and refer to the "Acceleron Third Quarter Earnings Call".
The archived webcast will be available for replay on the Acceleron website approximately two hours after the event.

On November 7, 2017 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a dermatologist-led biopharmaceutical company focused on identifying, developing, and commercializing innovative and differentiated therapies to address significant unmet needs in medical and aesthetic dermatology, reported financial results for the third quarter of 2017 and provided an update on its clinical development programs (Press release, Aclaris Therapeutics, NOV 7, 2017, View Source [SID1234521624]).

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"The third quarter of 2017 has been a productive one for Aclaris. In August, we closed the acquisition of Confluence Life Sciences, Inc. ("Confluence"), and we continue to prepare for our December 24th PDUFA date for A-101 40% Topical Solution for the treatment of seborrheic keratosis," said Dr. Neal Walker, President and Chief Executive Officer of Aclaris.

Clinical Pipeline Update
·
A-101 40% Topical Solution
o
In September, published results from a Phase 2 clinical trial evaluating two concentrations (40% and 32.5%) of its drug candidate A-101 for the treatment of facial seborrheic keratosis (SK) lesions in the journal Dermatologic Surgery. In the trial, A-101 achieved statistically significant improvement in clearing SK lesions on the face in a dose-related fashion. A-101 was well tolerated at both concentrations studied.
o
The FDA’s Prescription Drug User Fee Act (PDUFA) action date for the New Drug Application (NDA) is December 24, 2017. If approved, A-101 40% Topical Solution would be the first FDA-approved medication for SK.

·
A-101 45% Topical Solution
o
In June, initiated two Phase 2 clinical trials of A-101 45% Topical Solution (A-101 45%) for the treatment of common warts. The Phase 2 clinical trials are designed to evaluate the safety, tolerability and dose frequency of A-101 45% compared with placebo in adult and pediatric patients.

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Enrollment in both phase 2 trials has been completed and 316 patients have been enrolled in the two double-blinded trials which are being conducted at 34 investigational centers within the United States. Aclaris expects to report data from these two trials in the first half of 2018.

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JAK Inhibitors
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In October, initiated a Phase 2 clinical trial of ATI-50002, a topical Janus Kinase (JAK) 1/3 inhibitor (ATI-50002 Topical) for the treatment of alopecia areata (AA). This trial will evaluate the pharmacokinetics, pharmacodynamics and safety of ATI-50002 Topical compared with placebo in 12 patients with AA. This randomized, double-blind clinical trial is being conducted at two investigational centers within the United States, and data is expected to be available in the first half of 2018.
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In November, initiated a Phase 2 open-label clinical trial of ATI-50002 Topical for the treatment of AA. This trial will evaluate the effect of ATI-50002 Topical on the regrowth of eyebrows in up to 24 patients with AA. This trial is being conducted at two investigational centers in Sydney and Melbourne, Australia, and data is also expected to be available in the first half of 2018.
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Continue plans to initiate a Phase 2 dose ranging trial of ATI-50002 Topical for the treatment of AA next week. This study is a randomized, double-blinded, parallel-group, vehicle controlled trial and plan to enroll approximately 120 patients at 20 investigational centers within the United States. Data is expected to be available by year end 2018.
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Continue plans to initiate a Phase 2 open-label clinical trial of ATI-50002 Topical for the treatment of vitiligo by the end of 2017.
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Now plan to initiate a Phase 2 dose ranging trial of ATI-50001, an oral JAK inhibitor, for the treatment of AA in the first half of 2018.
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Continue to develop another series of topical JAK inhibitors for the treatment of androgenetic alopecia (AGA).

Business Highlights and Recent Developments

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In August, completed the acquisition of Confluence, a privately held biotechnology company focused on the discovery and development of kinase inhibitors to treat inflammatory and immunological disorders and cancer.
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In August, raised net proceeds of $80.9 million from a follow-on offering of our common stock.
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In September, the United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 9,737,469 and U.S. Patent No. 9,730,877, and the Japan Patent Office issued Japanese Letters Patent No. 6212107. These patents are directed to methods related to the use and administration of certain JAK inhibitors for treating hair loss disorders. These newly issued patents are owned by The Trustees of Columbia University in the City of New York and exclusively licensed to Aclaris.
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U.S. Patent No. 9,730,877 covers the use of various JAK inhibitors, including tofacitinib, baricitinib, ruxolitinib and decernotinib, to treat AGA, also known as male/female pattern hair loss. The ‘877 Patent contains 22 claims and expires in November 2031.
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U.S. Patent No. 9,737,469 covers the use of baricitinib for inducing hair growth and for treating hair loss disorders such as AA and AGA. Additional issued claims pertain to methods of using baricitinib to treat particular phenotypes of AA, as well as to treat other hair loss disorders. The ‘469 Patent contains 10 claims and expires in November 2031.
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Japanese Letters Patent No. 6212107 covers the use of tofacitinib in a topical composition or as the sole active therapeutic agent in a pharmaceutical composition for inducing hair growth and for treating hair loss disorders, such as AA and AGA. The ‘107 patent issued with 25 claims and expires in March 2033.
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In October, Columbia University received a Notice of Allowance from the USPTO for a patent application covering methods of treating a hair-loss disorder, such as AA and AGA, or inducing hair growth, by administering tofacitinib topically or by administering tofacitinib as the sole active ingredient, and treating AGA or inducing hair growth in a subject having AGA by administering tofacitinib. The application was allowed with 66 claims.
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In October, hosted inaugural R&D and Investor Day event.

Financial Highlights

Liquidity and Capital Resources
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As of September 30, 2017, Aclaris had aggregate cash, cash equivalents and marketable securities of $227.8 million compared to $174.1 million as of December 31, 2016. The $53.7 million increase during the nine months ended September 30, 2017 included:

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Aggregate net proceeds of $100.2 million from the public offering of common stock in August 2017 and the sale of common stock under an at-the-market facility with Cowen in May 2017.

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$9.6 million of cash used to acquire Confluence, net of cash acquired.

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Net loss of $45.6 million and $1.2 million of net cash used in working capital, partially offset by $10.4 million of non-cash stock-based compensation expense, depreciation and amortization.

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Aclaris anticipates that its cash, cash equivalents and marketable securities as of September 30, 2017 will be sufficient to fund its operations into the second half of 2019, without giving effect to any potential new business development transactions or financing activities.

Third Quarter 2017 Financial Results
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Net loss was $18.2 million for the third quarter of 2017, compared to $10.7 million for the third quarter of 2016.
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Revenue of $0.7 million and cost of revenue of $0.5 million for the third quarter of 2017 related to Confluence’s contract research organization (CRO) business acquired in August 2017.
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Total operating expenses for the third quarter of 2017 were $19.0 million, compared to $10.8 million for the third quarter of 2016.
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Research and development expenses were $10.9 million for the third quarter of 2017, compared to $7.2 million for the third quarter of 2016. The increase of $3.7 million was primarily attributable to a $1.2 million increase in expenses related to the Phase 2 clinical trials of A-101 45%, a $1.3 million increase in personnel-related expenses, including stock-based compensation, due to increased

headcount, a $0.9 million increase in preclinical and clinical trial development expenses related to the JAK inhibitor technology and a $0.9 million increase in medical affairs expenses and other costs, including early stage drug discovery, offset in part by a $1.2 million decrease in clinical costs for A-101 40% Topical Solution resulting from the completion of Phase 3 clinical trials in November 2016.

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General and administrative expenses were $8.1 million for the third quarter of 2017, compared to $3.7 million for the third quarter of 2016. The increase of $4.4 million was primarily attributable to $1.9 million in higher personnel-related expenses, including stock-based compensation, due to increased headcount, and $0.4 million in expenses related to the acquisition of Confluence. Additionally, Aclaris had a $1.6 million increase in market research and sales operations expenses related to pre-commercial activities for A-101 40% Topical Solution.

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As of September 30, 2017, Aclaris had approximately 30.8 million shares of common stock outstanding.

2017 Financial Outlook
Aclaris is updating its estimates for the following financial metrics for the full year 2017, which estimates now incorporate the acquisition of Confluence:

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Cash burn for 2017, excluding this year’s financing activities and cash paid in the Confluence acquisition, is now estimated to be in the range of $56 million to $59 million compared to previous guidance of $65 million to $70 million.

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Total operating expenses for 2017 are now estimated to be in the range of $72 million to $75 million, or $57 million to $60 million when excluding estimated stock-based compensation expense of $15 million. Prior guidance for operating expenses was $84 million to $92 million, or $70 million to $75 million when excluding stock-based compensation of $14 to $17 million.

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Research and development expenses for 2017 are now estimated to be in the range of $39 million to $42 million, or $33 million to $36 million when excluding estimated stock-based compensation expense of $6 million. Prior guidance for research and development expenses was $51 million to $58 million, or $46 million to $52 million when excluding stock-based compensation of $5 million to $6 million.

Company to Host Conference Call
Management will conduct a conference call at 8:00 AM ET today to discuss Aclaris’ financial results and provide a general business update. The conference will be webcast live over the Internet and can be accessed by logging on to the "Investors" page of the Aclaris Therapeutics website, www.aclaristx.com, prior to the event. A replay of the webcast will be archived on the Aclaris Therapeutics website for 30 days following the call.
To participate on the live call, please dial (844) 776-7782 (domestic) or (661) 378-9535 (international), and reference conference ID 99295180 prior to the start of the call.

On November 7, 2017 Affimed N.V. (Nasdaq: AFMD), a clinical stage biopharmaceutical company focused on discovering and developing highly targeted cancer immunotherapies, reported financial results for the quarter ended September 30, 2017 (Press release, Affimed, NOV 7, 2017, View Source [SID1234521625]).

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"We continue to make great strides in validating our NK cell engager programs, in particular through encouraging data from studies of our lead candidate AFM13, which is progressing through clinical development as a mono- and combination therapy," said Dr. Adi Hoess, CEO of Affimed. "Leveraging our unique NK cell-based platform for high-affinity CD16A-targeting, we are advancing our tetravalent bispecific antibodies with the potential to tailor immune-engaging therapy to different indications and settings."

Third Quarter Updates

Corporate Update

· In September 2017, Dr. Wolfgang Fischer, former Global Head of Program and Project Management of Sandoz Biopharmaceuticals (Novartis Group) joined Affimed as Chief Operating Officer. Dr. Fischer has over 20 years of R&D experience with a focus on oncology, immunology and pharmacology. With his proven track record in drug development, he will support the Company in advancing its unique immune cell engagers to address the existing medical need in hematologic and solid tumor indications. In addition to his role as COO, Dr. Fischer will assume responsibility as interim CMO, working closely with the Company’s clinical team.

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NK cell engager programs

· In Affimed’s Phase 1b combination study of its lead product candidate, the CD30/CD16A-targeting NK cell engager AFM13, with Merck’s Keytruda (pembrolizumab) in Hodgkin lymphoma (HL), the dose expansion cohort is open and recruiting. Data analysis of three-month response rates is ongoing for the escalation phase of the trial with analysis of 9 out of 12 patients completed to date. Of the 3 patients enrolled into cohort 1, two experienced partial metabolic responses, while one patient progressed. Of the 3 patients enrolled into cohort 2, one patient experienced a complete metabolic response, one patient experienced a partial metabolic response and one patient progressed. Out of the six patients in dose cohort 3, three were analyzed to date, all of which experienced partial metabolic responses at the first tumor response assessment. The Company intends to present detailed dose escalation data including response data of the three remaining patients in cohort 3 at the upcoming ASH (Free ASH Whitepaper) 2017 Annual Meeting in December.

· Affimed is supporting a translational Phase 1b/2a study of AFM13 in patients with relapsed or refractory CD30-positive lymphoma with cutaneous manifestation led by Columbia University. The study is designed to allow for serial biopsies, thereby enabling assessment of NK cell biology and tumor cell killing within the tumor microenvironment. The first cohort has been fully enrolled and recruitment into further cohorts is ongoing. The first patient, suffering from anaplastic large-cell lymphoma (ALCL) with cutaneous manifestation, experienced a complete response of cutaneous lesions after the first treatment cycle. Systemic evaluation is ongoing. This provides first evidence that NK cell engagers are able to induce tumor regression in this indication.

· The Company’s investigator-sponsored Phase 2a monotherapy study of AFM13 in HL led by the German Hodgkin Study Group (GHSG), is open to recruit under the new design, which includes patients pre-treated with both brentuximab vedotin (B.V.) and anti-PD1.

· In Affimed’s collaboration with The University of Texas MD Anderson Cancer Center (MDACC) evaluating the Company’s NK cell engager AFM13 in combination with MDACC’s NK cell product, preclinical research activities are progressing.

· The Company has developed multiple tetravalent, bispecific antibody formats in addition to its TandAbs. These molecules confer distinct biophysical properties aimed at tailoring PK profiles. Based on its platform, Affimed is advancing AFM24, an EGFR/CD16A-specific NK cell engager and AFM26, a BCMA/CD16A-specific NK cell engager, through IND-enabling studies. Final candidates have been selected for AFM24 and AFM26, respectively.

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· Candidates for AFM24, developed to treat solid tumors, are based on both TandAb format and on novel proprietary antibody formats. AFM24 molecules offer a differentiated mode of action as compared to cetuximab. Furthermore, binding to NK cells is largely unaffected by IgG competition, resulting in higher efficacy and elimination of cells with low target expression. Affimed is currently evaluating its novel format-based AFM24 molecules, which have significantly longer half-lives, in comparison to the Company’s TandAbs, which have already shown evidence of a beneficial safety profile.

· AFM26 is designed to eliminate malignant cells in multiple myeloma (MM) independent of BCMA expression levels. AFM26 offers a differentiated mode of action, targeting cells expressing very low levels of BCMA, conferring NK cell cytotoxicity and eliciting lower cytokine release compared to a BiTE molecule. Furthermore, binding to NK cells is largely unaffected by IgG competition. These unique features could position AFM26 in patients receiving autologous stem cell transplant (ASCT)-eligible at or shortly after transplant, a period in which no treatment is currently available.

T cell engager programs

· Affimed is conducting two clinical Phase 1 dose-escalation trials with AFM11, a CD3/CD19-targeting tetravalent bispecific T cell engager in patients with relapsed and refractory (r/r) acute lymphocytic leukemia (ALL) and with r/r non-Hodgkin lymphoma (NHL), respectively. Both studies are ongoing and recruiting.

· Recruitment is ongoing into a first-in-human Phase 1 dose escalation trial of AMV564 conducted by Amphivena Therapeutics, Inc. in patients with r/r acute myeloid leukemia (AML). AMV564 is a CD33/CD3-specific antibody based on Affimed’s technology platform. Affimed owns ~18.5% of Amphivena (fully diluted).

Financial Highlights
(Figures for the third quarter and first nine months of 2017 and 2016 represent unaudited figures)

Cash and cash equivalents and financial assets totaled €41.8 million as of September 30, 2017 compared to €44.9 million as of December 31, 2016. Affimed’s operational expenses for the nine months ended September 30, 2017 were primarily offset by net proceeds of €16.4 million from a public offering of common shares in the first quarter and of €2.5 million from the drawdown of the second tranche of the loan from Silicon Valley Bank.

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Net cash used in operating activities was €20.7 million for the nine months ended September 30, 2017 compared to €25.5 million for the nine months ended September 30, 2016. The decrease was primarily related to lower cash expenditure for research and development (R&D) in connection with Affimed’s development and collaboration programs and to the expiration of the Amphivena collaboration.

Revenue for the third quarter of 2017 was €0.5 million compared to €0.9 million for the third quarter of 2016. Revenue in the 2017 period was derived from AbCheck services while revenue in the 2016 period related predominantly to Affimed’s collaboration with Amphivena.

R&D expenses for the third quarter of 2017 were €6.0 million compared to €8.8 million for the third quarter of 2016. The decrease was primarily related to lower expenses for AFM13 and our discovery/early stage development activities.

G&A expenses for the third quarter of 2017 were €1.9 million compared to €2.2 million for the third quarter of 2016.

Net loss for the third quarter of 2017 was €8.1 million, or €0.18 per common share, compared to a net loss of €10.3 million, or €0.31 per common share, for the third quarter of 2016. The decrease of operating expenses was partially offset by lower revenue and higher finance costs.

Note on IFRS Reporting Standards
Affimed prepares and reports the consolidated financial statements and financial information in accordance with International Financial Reporting Standards (IFRS) as issued by the International Accounting Standards Board (IASB). None of the financial statements were prepared in accordance with Generally Accepted Accounting Principles (GAAP) in the United States. Affimed maintains its books and records in Euro.

Conference Call and Webcast Information
Affimed’s management will host a conference call to discuss the company’s financial results and recent corporate developments today at 8:30 a.m. ET. A webcast of the conference call can be accessed in the "Events" section on the "Investors & Media" page of the Affimed website at View Source A replay of the webcast will be available on Affimed’s website shortly after the conclusion of the call and will be archived on the Affimed website for 30 days following the call.