On March 12, 2018 Altimmune, Inc. (Nasdaq:ALT), a clinical-stage immunotherapeutics company, reported data from a pre-clinical study comparing SparVax-L and BioThrax against anthrax infection (Press release, Altimmune, MAR 12, 2018, View Source [SID1234524667]). Data from the study showed a 67% survival rate in animals challenged with a lethal dose of anthrax after a single dose of SparVax-L, an anthrax vaccine currently being developed through funding from the National Institute of Allergy and Infectious Diseases (NIAID) and previously through funding from the Biomedical Advanced Research and Development Authority (BARDA).

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"We are very encouraged by these data and look forward to continuing to develop this important vaccine," said William J. Enright, president and chief executive officer of Altimmune. "Nearly 700 volunteers have been dosed with a liquid formulation of this vaccine in previous Phase 1 and Phase 2 human studies. These data using a new, highly stable, lyophilized (freeze dried) formulation, continue to validate this approach and strongly support further development of SparVax-L."

The study compared SparVax-L to BioThrax, the currently approved vaccine against anthrax infection. SparVax-L uses a well-accepted adjuvant formulation with a clear regulatory pathway for safety and efficacy. The results of this study showed a 67% survival rate after a single dose of SparVax-L, and a 100% survival rate after two doses at day 0 and day 14 when challenged at Day 28, which was comparable to BioThrax’s 96% survival rate after two doses. The dose of SparVax-L used in this study was low compared to what is supported by existing safety and toxicology studies which indicates that better single dose protection may be achieved with a higher dose. Additionally, SparVax-L showed significantly more toxin neutralizing antibodies (TNA) after the two doses when compared to BioThrax. TNA is the key surrogate for protection in clinical studies of anthrax vaccines.

SparVax-L is being developed as a second generation, highly purified recombinant anthrax vaccine requiring only two vaccinations for protection. SparVax-L is further differentiated by its ability to be stored at room temperature, making it well-suited for stockpiling in the Strategic National Stockpile.

This project has been funded in part with Federal funds from the National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health, Department of Health and Human Services, under Contract No. HHSN272201400040C and the Biomedical Advanced Research and Development Authority (BARDA) under contract No. HSS01002009000103C. The Company is seeking additional government funding to further advance the stable lyophilized formulation in human clinical studies.

NantKwest has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, NantKwest, 2018, MAR 12, 2018, View Source [SID1234524658]).

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On March 12, 2018 Kura Oncology, Inc. (Nasdaq:KURA), a clinical-stage biopharmaceutical company focused on the development of precision medicines for oncology, today provided a regulatory update for its lead product candidate, tipifarnib, and reported fourth quarter and full year 2017 financial results (Press release, Kura Oncology, MAR 12, 2018, View Source [SID1234524678]).

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"Following a successful end of Phase 2 meeting with the FDA, we plan to initiate a single-arm, registration-directed trial of tipifarnib in at least 59 recurrent or metastatic patients with HRAS mutant squamous cell head and neck cancer (HNSCC) with objective response rate (ORR) as the primary endpoint," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "We expect to initiate this trial, which we are calling the AIM-HN trial, in the second half of 2018. We are encouraged by the feedback we received from the FDA regarding the development path for tipifarnib in HRAS mutant HNSCC, and we look forward to providing more specific information regarding the design and execution of the trial in the months ahead."

Recent Operational Highlights

Feedback from end of Phase 2 clinical meeting with the FDA – Based on feedback from the FDA, Kura is planning for the initiation of its AIM-HN trial of tipifarnib in HRAS mutant HNSCC patients, pending completion of site feasibility activities and submission of the final protocol to the FDA. The AIM-HN trial will be a global, multi-center, single-arm, study of at least 59 recurrent or metastatic patients with measurable disease as determined by RECIST version 1.1 criteria. The primary endpoint will be ORR, as determined by independent radiological review. The FDA indicated in the minutes from the meeting that the AIM-HN trial, as currently designed, may be adequate to support an NDA seeking accelerated approval.

Update on Phase 2 trial of tipifarnib in HRAS mutant HNSCC – In February 2018, Kura reported updated preliminary results from its Phase 2 trial of tipifarnib in patients with HRAS mutant HNSCC at the 2018 Multidisciplinary Head and Neck Cancers Symposium. The update showed that five of the six evaluable patients achieved a confirmed, partial response. Two of these patients achieved durable responses beyond a year and a half. The one evaluable patient who did not achieve a response, based on standard RECIST criteria, experienced prolonged disease stabilization for more than six months. Tipifarnib has been generally well-tolerated with adverse events observed consistent with its known safety profile.

Potential biomarkers identified for tipifarnib in hematologic malignancies – In December 2017, Kura presented new findings at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting that identified activation of the CXCL12 pathway and bone marrow homing of myeloid cells as potential biomarkers of tipifarnib’s activity in certain hematologic malignancies, including peripheral T-cell lymphoma (PTCL), myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML). Based on these observations, the company is now prospectively investigating these potential biomarkers in its ongoing Phase 2 trials in various hematologic malignancies.
Upcoming Potential Milestones and Expectations for Clinical Programs

Preclinical data for tipifarnib in HRAS mutant squamous non-small cell lung tumor models at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2018

Preclinical biomarker data from KO-947 in squamous cell carcinomas at AACR (Free AACR Whitepaper) in April 2018

Initiation of the tipifarnib SEQ-HN trial, a screening and outcomes study in HRAS mutant HNSCC in the first half of 2018

Initiation of the tipifarnib AIM-HN trial in HRAS mutant HNSCC in the second half of 2018

Data from Phase 1 dose-escalation trial of KO-947 in the second half of 2018

Additional updates from the ongoing Phase 2 study of tipifarnib in HRAS mutant HNSCC in the second half of 2018

Initiation of a proof-of-concept study of tipifarnib in HRAS mutant squamous non-small cell lung cancer through the Spanish Lung Cancer Group in 2018

Additional clinical data from tipifarnib in hematologic malignancies in the second half of 2018

Submission of an investigational new drug (IND) application for KO-539 in late 2018 or early 2019
Financial Results for the Fourth Quarter and the Full Year 2017

Research and development expenses for the fourth quarter of 2017 were $8.1 million, compared to $5.5 million for the fourth quarter of 2016. Research and development expenses for the full year 2017 were $26.4 million, compared to $20.4 million for the prior year.

General and administrative expenses for the fourth quarter of 2017 were $2.9 million, compared to $2.0 million for the fourth quarter of 2016. General and administrative expenses for the full year 2017 were $9.7 million, compared to $8.0 million for the prior year.

Net loss for the fourth quarter of 2017 was $10.7 million, compared to a net loss of $7.3 million for the fourth quarter of 2016. Net loss for the full year 2017 was $35.4 million, compared to a net loss of $27.6 million for the prior year.

Cash, cash equivalents and short-term investments totaled $93.1 million as of December 31, 2017, compared with $100.8 million as of September 30, 2017 and $67.8 million as of December 31, 2016.

Subsequently, in January 2018, Kura sold an aggregate of approximately 3.1 million shares of its common stock under an ATM facility for net proceeds of $57.4 million.

Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund its current operations into the first half of 2020.
Conference Call and Webcast

Kura’s management will host a webcast and conference call today at 4:30 p.m. ET / 1:30 p.m. PT today, March 12, 2018, to discuss the regulatory update and financial results. The live call may be accessed by dialing (877) 516-3514 for domestic callers and (281) 973-6129 for international callers and using conference ID # 5196505. A live webcast of the call will be available from the Investors and Media section of the company website at www.kuraoncology.com, and will be archived there for 30 days.

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On March 12, 2018 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK), a clinical-stage oncology company focused on biomarker-defined cancers, reported its fourth quarter and full year 2017 financial results for the period ended December 31, 2017 (Press release, Merrimack, MAR 12, 2018, View Source [SID1234524679]).

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"2017 was a transformative year for Merrimack, in which we reset the company’s foundation to focus on our ten wholly owned clinical and preclinical programs, all targeting biomarker-defined cancers. We are very pleased with the advancements we have made across our pipeline, including today’s announcement to expand enrollment in the SHERLOC study, a randomized Phase 2 trial evaluating MM-121 in non-small cell lung cancer, and our recent dosing of the first patient in the SHERBOC study, a randomized Phase 2 trial evaluating MM-121 in post-menopausal metastatic breast cancer," said Richard Peters, M.D., Ph.D., President and Chief Executive Officer. "We are well-positioned to carry this momentum forward, with three clinical readouts expected in 2018, including randomized Phase 2 data from MM-141 and MM-121 and Phase 1 data from MM-310."

Fourth Quarter and Recent Highlights

Key events from the fourth quarter and more recently include:

As announced separately today, expansion of the enrollment target from 80 to 100 patients in the SHERLOC study, a randomized Phase 2 clinical trial evaluating MM-121 added to standard of care in patients with heregulin-positive non-small cell lung cancer. This augmentation of patient enrollment is driven by the faster than projected enrollment rate seen to date and will result in a strengthened statistical design of the study. Merrimack still expects to report top-line data from this trial in the second half of 2018;

First patient dosed in the SHERBOC study, a randomized, double-blind, placebo-controlled Phase 2 clinical trial evaluating MM-121 added to standard of care in patients with heregulin-positive, hormone-receptor-positive and HER2-negative post-menopausal metastatic breast cancer;

Appointment of George Demetri, M.D., to Merrimack’s Board of Directors. Dr. Demetri is currently a Senior Vice President for Experimental Therapeutics and Director of the Center for Sarcoma and Bone Oncology at Dana-Farber Cancer Institute, as well as a Professor of Medicine at Harvard Medical School, where he is also Co-Director of the Ludwig Center. He is a world-renowned expert in the clinical translation of innovative treatment strategies for cancer, and replaces John Mendelsohn, M.D., who had served on Merrimack’s Board since 2012;
Formation of a new Scientific Advisory Board (SAB) with extensive expertise in precision oncology, bioengineering, drug discovery and clinical development. Members include: Peter Blume-Jensen, M.D., Ph.D.; George Demetri, M.D.; Douglas Lauffenburger, Ph.D.; Peter Sorger, Ph.D.; and Josep Tabernero, M.D., Ph.D. The SAB will work closely with Merrimack’s senior management team to advance the company’s pipeline of targeted cancer therapies; and

Financially, closure of 2017 with $93.4 million in cash and cash equivalents and extinguishment of $60.8 million of convertible debt in the fourth quarter of 2017, resulting in a debt-free balance sheet.
Upcoming Milestones:

Merrimack anticipates the following upcoming clinical milestones:

Top-line results in the first half of 2018 from the CARRIE study, a randomized Phase 2 clinical trial evaluating MM-141, a bispecific antibody targeting the IGF-1 and HER3 receptors, added to standard of care in patients with front-line metastatic pancreatic cancer who have high serum levels of free IGF-1;

Top-line results in the second half of 2018 from the SHERLOC study, a randomized Phase 2 clinical trial evaluating MM-121, a fully human monoclonal antibody targeting the HER3 receptor, added to standard of care in patients with heregulin positive non-small cell lung cancer; and

Safety data and maximum tolerated dose in the second half of 2018 from the Phase 1 clinical study of MM-310, an antibody-directed nanotherapeutic (ADN) targeting the EphA2 receptor, in patients with solid tumors.
Fourth Quarter and Full Year 2017 Financial Results

The following summarizes Merrimack’s financial results for the three months and year ended December 31, 2017:

Research and development expenses from continuing operations for the three months ended December 31, 2017 were $12.4 million, compared to $25.6 million for the three months ended December 31, 2016. Research and development expenses from continuing operations for the year ended December 31, 2017 were $67.3 million, compared to $109.6 million for the year ended December 31, 2016. Research and development spending for the three months and year ended December 31, 2017 was less than expenditures over comparable periods in 2016, primarily due to Merrimack’s refocused clinical and preclinical pipeline;

General and administrative expenses for the three months ended December 31, 2017 from continuing operations were $4.7 million, compared to $11.0 million for the three months ended December 31, 2016. General and administrative expenses for the year ended December 31, 2017 from continuing operations were $28.5 million, compared to $32.1 million for the year ended December 31, 2016. General and administrative spending for the three months and year ended December 31, 2017 was less than expenditures over comparable periods in 2016, primarily due to a decrease in corporate expenses related to reduced headcount levels and stock-based compensation following the asset sale to Ipsen S.A.;

Net loss attributable to Merrimack’s continuing operations for the three months ended December 31, 2017 was $11.8 million, or $0.89 per share, compared to a net loss attributable to Merrimack’s continuing operations of $40.1 million, or $2.93 per share, for the three months

ended December 31, 2016. Net loss attributable to Merrimack’s continuing operations for the year ended December 31, 2017 was $74.8 million, or $5.66 per share, compared to a net loss attributable to Merrimack’s continuing operations of $154.5 million, or $12.33 per share, for the year ended December 31, 2016; and

As of December 31, 2017, Merrimack had 13.3 million shares of common stock, $0.01 par value per share, outstanding.
Financial Outlook

Merrimack continues to believe that its cash and cash equivalents of $93.4 million as of December 31, 2017 and potential net milestone payments anticipated from Shire will be sufficient to fund its planned operations into the second half of 2019.

Conference Call and Webcast

Merrimack will host a live conference call and webcast today, Monday, March 12, 2018 at 8:30 am ET, to provide an update on its operational progress and a summary of these financial results.

Investors and the general public are invited to listen to the call by dialing (877) 564-1301 (domestic) or (224) 357-2394 (international) five minutes prior to the start of the call and providing the passcode 8074029. A listen-only webcast of the call can be accessed in the Investors section of Merrimack’s website, investors.merrimack.com, and a replay of the call will be archived there for six weeks following the call.