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10-K – Annual report [Section 13 and 15(d), not S-K Item 405]

(Filing, 10-K, Kura Oncology, MAR 14, 2017, View Source [SID1234518147])

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Mirna Therapeutics Reports Financial Results for the Fourth Quarter and Full Year Ended December 31, 2016

On March 14, 2017 Mirna Therapeutics, Inc. (Nasdaq: MIRN), a biopharmaceutical company, reported financial results for the fourth quarter and year ended December 31, 2016 and provided a corporate update (Press release, Mirna Therapeutics, MAR 14, 2017, View Source [SID1234518172]).

Following its November 2016 decision to discontinue all research and development activities including the development of MRX34, Mirna began implementing operating cost reductions and organizational restructuring to reduce overall cash burn, including a reduction in its workforce. The Company engaged a financial advisor and has been pursuing activities to identify and evaluate strategic alternatives, including a possible merger or sale of the Company.

"Our strategic process is active and ongoing and we are pursuing discussions with third parties on a range of potential transactions," said President and CEO Paul Lammers, M.D., M.Sc. "We are committed to serving our shareholders’ best interests through our efforts to identify, evaluate and potentially consummate a transaction that may result from these activities."

2016 FINANCIAL RESULTS

Cash Position and Guidance: Cash, cash equivalents, and marketable securities totaled $60.5 million as of December 31, 2016, compared to $89.7 million as of December 31, 2015. The Company has no debt.

The Company expects its quarterly cash burn rate to remain within the range of $2.1 million and $2.3 million. This quarterly guidance includes contractual commitments and obligations for future minimum lease payments, but excludes any one-time charges related to any strategic transaction should such be consummated and contractual payments for executive severance or change-in-control provisions.
Research and development expenses: Research and development expenses were approximately $2.3 million and $13.9 million for the quarter and year ended December 31, 2016 compared to research and development expenses of $6.4 million and $18.9 million during the comparable periods in 2015. The decrease was primarily due to the closing of the Company’s Phase 1 clinical trial of MRX34 in September 2016 and discontinuing all research and development activities.
General and administrative expenses: General and administrative expenses were approximately $2.0 million and $8.1 million for the quarter and year ended December 31, 2016, compared to general and administrative expenses of $2.5 million and $6.1 million during the comparable periods in 2015. The increase for the year ended December 31, 2016 was primarily attributable to increased employee compensation expense due to a higher headcount and higher outside professional and consulting costs, the majority of which were incurred to comply with public company operating and reporting requirements in the Company’s first full year operating as a public company.
Restructuring charges: Restructuring charges were approximately $4.4 million for the quarter and year ended December 31, 2016 and $0 for the year ended December 31, 2015. In September 2016, Mirna announced its decision to close the ongoing Phase 1 study of MRX34 and voluntarily halted the enrollment and dosing of patients in the study prior to receiving notice from the U.S. Food and Drug Administration ("FDA") that its Investigational New Drug Application for MRX34 had been placed on full clinical hold. Following the Company’s announcement and notification from the FDA, Mirna’s Board of Directors approved a reduction of the total number of full-time employees from 36 to 12. The restructuring charges recognized during the year ended December 31, 2016 included approximately $1.5 million for employee severance and benefits, an accounting charge under U.S. Generally Accepted Accounting Principles ("U.S. GAAP") of $1.5 million for lease facility termination costs, and $1.4 million for non-cash impairment charges of property and equipment. The majority of these employee severance and related benefits are expected to be settled in the first quarter of 2017. The Company expects to incur additional restructuring charges under U.S. GAAP of approximately $0.3 million through the six months ended June 30, 2017.
Net Loss: Net loss was approximately $8.7 million and $26.3 million for the quarter and year ended December 31, 2016, compared to a net loss of $8.8 million and $25.0 million for the comparable periods in 2015. The results included non-cash, stock-based related compensation charges of $1.6 million and $1.0 million for the years ended December 31, 2016 and December 31, 2015, respectively.

Kura Oncology Reports Fourth Quarter and Full Year 2016 Operational and Financial Results

On March 14, 2017 Kura Oncology, Inc., (NASDAQ:KURA) a clinical stage biopharmaceutical company focused on the development of precision medicines for oncology, reported fourth quarter and full year 2016 financial results and provided a corporate update (Press release, Kura Oncology, MAR 14, 2017, View Source;p=RssLanding&cat=news&id=2253894 [SID1234518171]).

"Our precision medicine approach continues to deliver results, and we are pleased to have achieved important milestones in each of our programs," said Troy Wilson, Ph.D., J.D., President and CEO of Kura Oncology. "We are encouraged by the durability of responses observed in patients with HRAS mutant squamous cell carcinomas of the head and neck treated with tipifarnib, and believe our data validate HRAS as a driver oncogene in that disease. In our Phase 2 trial in PTCL, we have observed initial signals of clinical activity and identified potential biomarkers, including genes that are expressed and/or altered, which appear to be associated with the activity of tipifarnib. In our Phase 2 trial of tipifarnib in lower-risk MDS, based on anecdotal evidence of hematological improvement observed in several patients, we have amended the study to evaluate further dose regimens in an effort to optimize those initial findings. With KO-947, our ERK inhibitor, we anticipate initiating the Phase 1 study, and through preclinical studies have identified potential development opportunities, including KRAS and BRAF mutant cancers and squamous cell carcinomas. We also selected a development candidate in our menin-MLL inhibitor program, KO-539, which demonstrates potent anti-tumor activity in certain preclinical models of acute leukemia."

Recent Operational Highlights

Selection of KO-539, an orally-available small molecule inhibitor of the menin-MLL interaction, as a development candidate for the treatment of mixed lineage leukemias, a genetically-defined subset of the two most common forms of acute leukemia, acute myeloid leukemia and acute lymphoblastic leukemia
FDA acceptance of an Investigational New Drug (IND) application to begin Phase 1 clinical testing of KO-947, a small molecule inhibitor of extracellular-signal-regulated kinases 1 and 2 (ERK1/2) as a treatment for cancers in which the mitogen activated protein kinase (MAPK) pathway is dysregulated
Appointment of Steven H. Stein, M.D., to Kura’s board of directors. Dr. Stein currently serves as Executive Vice President and Chief Medical Officer of Incyte Corporation and has extensive experience in the discovery, development and commercialization of oncology therapeutics.
First patient dosed in Phase 2 clinical trial of tipifarnib in patients with chronic myelomonocytic leukemia (CMML)
Presentation of preclinical data highlighting the characterization of KO-947, and preclinical data relating to the identification and optimization of potent and selective inhibitors of the menin-MLL interaction. Both presentations took place at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics in Munich, Germany.
Upcoming Potential Milestones and Expectations for Clinical and Preclinical Programs

Initiation of the Phase 1 clinical trial for KO-947 during the first half of 2017
Data from the first and second stages of the Phase 2 trial of tipifarnib in peripheral T-cell lymphomas (PTCL) and associated biomarkers in the first half of 2017
Preclinical data for KO-947 and KO-539 in the first half of 2017
Additional data from the Phase 2 trial of tipifarnib in HRAS mutant squamous cell carcinomas of the head and neck (SCCHN) during the second half of 2017
Additional preclinical and clinical data for tipifarnib in PTCL in the second half of 2017
Data from the Phase 2 tipifarnib trials in lower risk myelodysplastic syndromes (MDS) and in CMML during the first half of 2018
Financial Results for the Fourth Quarter and the Full Year 2016

Cash, cash equivalents and short-term investments totaled $67.8 million as of December 31, 2016, compared with $74.6 million as of September 30, 2016 and $85.7 million as of December 31, 2015. Management expects that current cash, cash equivalents and short-term investments will be sufficient to fund current operations into the second half of 2018.
Research and development expenses for the fourth quarter of 2016 were $5.5 million, compared to $5.1 million for the fourth quarter of 2015. Research and development expenses for the full year 2016 were $20.4 million, compared to $17.8 million for the prior year.
General and administrative expenses for the fourth quarter of 2016 were $2.0 million, compared to $1.7 million for the fourth quarter of 2015. General and administrative expenses for the full year 2016 were $8.0 million, compared to $6.1 million for the prior year.
Net loss for the fourth quarter of 2016 was $7.3 million, compared to a net loss of $6.5 million for the fourth quarter of 2015. Net loss for the full year 2016 was $27.6 million, compared to a net loss of $22.6 million for the prior year.

Infinity Provides Company Update and Reports Fourth Quarter 2016 Financial Results

On March 14, 2017 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its full-year 2016 financial results and provided an update on the company, including its progress with IPI-549, a potentially first-in-class immuno-oncology product candidate that selectively inhibits PI3K-gamma (Press release, Infinity Pharmaceuticals, MAR 14, 2017, View Source;p=RssLanding&cat=news&id=2253890 [SID1234518169]). Infinity is evaluating IPI-549 as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor, in a Phase 1 study in patients with advanced solid tumors, and updated data from the study will be presented in April at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2017 in Washington, D.C. IPI-549 is believed to be the only selective PI3K-gamma inhibitor in clinical development.

Infinity anticipates completing the monotherapy dose-escalation phase of the study in the first half of 2017 and initiating a monotherapy expansion cohort in the second half of the year. The company also expects to complete the dose-escalation phase evaluating IPI-549 in combination with Opdivo in the second half of 2017, and subsequently initiate multiple combination expansion cohorts. The combination expansion cohorts will include patients with non-small cell lung cancer (NSCLC), melanoma, and squamous cell carcinoma of the head and neck (SCCHN) whose tumors show initial resistance or subsequently develop resistance to immune checkpoint therapy. There is a great need for additional treatment options for the growing number of patients living with these cancers, which account for more than 17 percent of all new cancer cases in the U.S.[1],[2]

"We are very pleased with the progress we have made in our Phase 1 clinical study of IPI-549. Both the monotherapy and combination dose-escalation phases of the study have been rapidly enrolling, and we are on track to initiate multiple expansion cohorts in the second half of the year," stated Adelene Perkins, Infinity’s chair and chief executive officer. "While new immunotherapies, such as checkpoint inhibitors, are showing great promise in the treatment of various cancers, additional treatment options are needed for patients who relapse or do not respond to currently available therapies. Our preclinical data recently published in Nature demonstrate that IPI-549 reprograms macrophages from a pro-tumor to an anti-tumor phenotype and is able to help overcome resistance to checkpoint inhibition, providing a compelling rationale for our ongoing clinical study."

Recent developments include the following:

IPI-549

Updated Phase 1 data to be presented at AACR (Free AACR Whitepaper): In March, Infinity announced that updated data from the ongoing Phase 1 study of IPI-549 will be presented at the AACR (Free AACR Whitepaper) Annual Meeting 2017 in Washington, D.C. during two sessions. Jeffery Kutok, M.D., Ph.D., Infinity’s chief scientific officer, will review the IPI-549 program during an educational session. The presentation, "Reprogramming Tumor-Associated Macrophages by Targeting PI3K-gamma Through a Small Molecule Approach," will take place during an educational session on Saturday, April 1, 2017, from 3:15 – 3:45 p.m. ET.

Additionally, a poster presentation, "IPI-549-01 – A Phase 1/1b, First-in-Human Study of IPI-549, a PI3K-Gamma Inhibitor, as Monotherapy and in Combination with Nivolumab in Patients with Advanced Solid Tumors" (Abstract CT089), will take place during a clinical trial poster session on Tuesday, April 4, 2017, from 8:00 a.m. – 12:00 p.m. ET. Jedd Wolchok, M.D., Ph.D., Chief of the Melanoma and Immunotherapeutics Service at Memorial Sloan Kettering Cancer Center (MSK), as well as Associate Director of the Ludwig Center for Cancer Immunotherapy and Director of the Parker Institute for Cancer Immunotherapy, both at MSK, is the lead author for the poster. He also serves as the lead investigator for the Phase 1 clinical study of IPI-549.

IPI-549 featured at TAT 2017: In March, data from 12 patients enrolled in the monotherapy dose-escalation portion of the Phase 1 study of IPI-549 were presented at the 15th International Congress on Targeted Anticancer Therapies (TAT 2017). The presentation, "A Phase 1/1b, First-in-Human Study of IPI-549, a PI3K-Gamma Inhibitor and Myeloid Targeting Agent, as Monotherapy and in Combination with Nivolumab in Patients with Advanced Solid Tumors," was given by Anthony Tolcher, M.D., FRCP(C), clinical director at South Texas Accelerated Research Therapeutics and an investigator for the Phase 1 study. Due to AACR (Free AACR Whitepaper) embargo policies, the data presented at TAT will be made available during the AACR (Free AACR Whitepaper) Annual Meeting 2017. IPI-549 was also discussed during the TAT 2017 Honorary Award Invited Lecture, "Immunologic Checkpoint Blockade: Exploring Combinations and Mechanisms," which was given by Dr. Wolchok.

Preclinical and Phase 1 clinical data on IPI-549 presented at PI3K Keystone Symposia Conference: In January, during a plenary session at the Keystone Symposia Conference, "PI3K Pathways in Immunology, Growth Disorders and Cancer," Infinity presented preclinical data for IPI-549 which demonstrated that IPI-549 is able to help overcome resistance to checkpoint inhibition by reprograming macrophages from a pro-tumor to an anti-tumor phenotype. Initial Phase 1 monotherapy data from nine patients with advanced solid tumors were also summarized and showed that the safety, pharmacokinetics and pharmacodynamics of IPI-549 monotherapy treatment appeared favorable.
Corporate

Jeffery Kutok, M.D., Ph.D., promoted to chief scientific officer: In February, Infinity announced the promotion of Dr. Kutok to the role of chief scientific officer. In this role, he will be responsible for overseeing biology and translational science efforts, including preclinical collaborations, to support the ongoing development of IPI-549. Dr. Kutok joined Infinity in 2010 and was promoted to vice president, biology and translational science in 2013.
Additional organizational changes: In January, Infinity announced the promotion of Lawrence Bloch, M.D., J.D., to president. In his new role, Dr. Bloch retained responsibility for the finance, business development, corporate communications and investor relations functions at Infinity and assumed responsibility for pharmaceutical development as well as facilities operations. Also in January, the company promoted Claudio Dansky Ullmann, M.D., to senior vice president, clinical development; Joseph Pearlberg, M.D., Ph.D., to vice president, clinical development; and Melissa Hackel to vice president, finance. Additionally, Infinity announced the retirement of Julian Adams, Ph.D., who had led the company’s research and development (R&D) teams since joining Infinity in 2003.
2017 Program Goals for IPI-549

In January 2017, Infinity outlined its anticipated program milestones for the year:

Present preclinical and clinical data from Phase 1 study at the PI3K Keystone Symposia Conference in January 2017
Report Phase 1 data from the monotherapy dose-escalation phase as well as the IPI-549 in combination with Opdivo dose-escalation phase in 2017
Complete the dose-escalation phase evaluating IPI-549 monotherapy in the first half of 2017
Begin enrolling patients with advanced solid tumors in the monotherapy expansion cohort during the second half of 2017
Complete the dose-escalation phase evaluating IPI-549 in combination with Opdivo in the second half of 2017
Begin enrolling patients with NSCLC, melanoma and SCCHN in expansion cohorts evaluating IPI-549 in combination with Opdivo in the second half of 2017
Full-Year 2016 Financial Results

At December 31, 2016, Infinity had total cash, cash equivalents and available-for-sale securities of $92.1 million, compared to $245.2 million at December 31, 2015.
Revenue during 2016 was $18.7 million related to Infinity’s previous collaboration agreement with AbbVie Inc. Revenue during 2015 was $109.1 million, all of which related to the AbbVie collaboration.
R&D expense for the full-year 2016 was $119.6 million, compared to $199.1 million for 2015. The decrease in R&D expense in 2016 compared to 2015 was primarily related to a 2015 option exercise payment to Takeda Pharmaceutical Company Limited and a reduction in clinical development expenses for duvelisib in 2016.
General and administrative expense was $42.2 million for the full-year 2016 compared to $37.1 million for 2015. The increase in general and administrative expense in 2016 compared to 2015 was primarily due to restructuring activities.
In 2016, Infinity recorded a non-recurring gain on AbbVie’s opt-out of the duvelisib collaboration of $112.2 million. This non-recurring gain represents the remaining deferred revenue of $112.2 million as of June 24, 2016, from the previous collaboration with AbbVie for duvelisib. The AbbVie opt-out is irrevocable, and Infinity has no obligation to continue to provide AbbVie any services. Infinity did not record any gains during 2015.
Net loss for the full-year 2016 was $30.1 million, or a basic and diluted loss per common share of $0.61, compared to a net loss of $128.4 million, or a basic and diluted loss per common share of $2.62 for the full-year 2015.
Cash and Investments Outlook

Infinity’s 2017 financial outlook remains as follows:

Net loss: Infinity expects net loss for 2017 to range from $40 million to $50 million.
Cash and Investments: Infinity expects to end 2017 with a year-end cash, cash equivalents and available-for-sale securities balance ranging from $40 million to $50 million.
Based on its current operational plans, Infinity expects that its existing cash, cash equivalents and available-for-sale securities at December 31, 2016, will be adequate to satisfy the company’s capital needs into the first quarter of 2019. The company’s financial outlook excludes additional funding or business development activities.

Ignyta Announces Full Year 2016 Company Highlights and Financial Results

On March 14, 2017 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported company highlights and financial results for the full year ended December 31, 2016 (Press release, Ignyta, MAR 14, 2017, View Source [SID1234518168]). The company is issuing this press release in lieu of conducting a conference call.

"In 2016, we continued to advance our pipeline of molecularly targeted therapies for the benefit of patients with cancer, including the STARTRK-2 registration-enabling Phase 2 clinical trial of our lead product candidate, entrectinib, our CNS-active tyrosine kinase inhibitor targeting tumors that harbor TRK, ROS1 or ALK fusions," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "We demonstrated robust clinical proof-of-concept data for entrectinib, as well as RXDX-105—our investigational, VEGFR-sparing, potent RET inhibitor—and made significant preclinical progress on RXDX-106, which represents a novel class of immunomodulatory agents that appears to restore innate immunity in preclinical models via potent inhibition of the TYRO3, AXL and MER (or TAM) family of receptors. Furthermore, in 2016, our in-house Trailblaze Pharos diagnostic assay achieved multiple regulatory milestones, we bolstered our balance sheet, and strengthened our leadership team."

Company Highlights

Entrectinib Clinical Progress

In February 2017, the company announced that updated results from two Phase 1 trials of entrectinib were published in the peer-reviewed journal, Cancer Discovery. Highlights of that publication included that entrectinib has the largest published safety experience of any TRK inhibitor in clinical development and continues to be well tolerated. As of the September 2016 data cutoff, RECIST responses were noted in three of three patients with TRK-positive extracranial solid tumors, with the longest ongoing TRK responder on therapy for 17 months; and RECIST responses in 12 of 14 patients with ROS1-positive solid tumors, with the longest ongoing ROS1 responder on therapy for 32.2 months. Within the subset of 13 patients with ROS1-positive non-small cell lung cancer (NSCLC), the response rate was 85% with a median duration of response of 17.3 months. Additionally, RECIST responses were noted in 63% of patients (five out of eight) with primary or metastatic disease involving the brain.

RXDX-105 Clinical Progress

In November 2016, Ignyta announced interim results from the ongoing Phase 1/1b clinical study of RXDX-105, which were presented at the 2016 EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) (ENA) Symposium on Molecular Targets and Cancer Therapeutics in Munich, Germany.

Those results highlighted RXDX-105’s clinical activity in patients harboring RET molecular alterations, with five out of nine patients with RET fusion-positive cancers who were RET inhibitor-naïve achieving a RECIST response (one complete response, three partial responses, and one unconfirmed partial response), for a preliminary objective response rate (ORR) of 56%. Within the subset of seven patients with RET fusion-positive NSCLC, four responded, for a preliminary ORR of 57%.

RXDX-106 Preclinical Progress

In November 2016, preclinical data on RXDX-106 were presented at the 2016 ENA Symposium highlighting immuno-oncological and direct tumor-inhibiting activity of this novel agent via potent inhibition of its targets. Those data showed that RXDX-106 is a potent inhibitor of TAM and c-MET targets, and inhibited TAM-mediated and c-MET-mediated tumor growth in vivo. Also, via in vitro and in vivo studies, RXDX-106, as a single agent, appears to release the molecular brakes on immune activation in macrophages, NK cells, and T cells, resulting in the repolarization of the immune response to elicit an anti-tumor effect; and emerging in vivo combination data suggest that RXDX-106 potentiates the activity of anti-PD-1 and anti-CTLA-4 agents.

Diagnostics Laboratory

In November 2016, the company announced that it has received Expedited Access Pathway (EAP) designation from the U.S. Food and Drug Administration (FDA) and also "European Conformity" (CE) marking for its Trailblaze Pharos companion diagnostic test service. The EAP designation enables Ignyta and FDA to have more interactive discussions of the premarket approval application (PMA) for Trailblaze Pharos, which is the proposed companion diagnostic for entrectinib. In August 2016, the FDA approved an investigational device exemption (IDE) for Trailblaze Pharos.

Financing Transactions

In June 2016, the company secured a $42 million term loan facility from Silicon Valley Bank and Oxford Finance. Under the loan facility, the company received initial funding of $32 million, substantially all of which was used to repay the company’s prior loan with Silicon Valley Bank, and has a conditional option to receive an additional $10 million.

In May 2016, the company issued an aggregate of 9.2 million shares of its common stock in an underwritten public offering at a purchase price of $6.25 per share, which resulted in aggregate gross proceeds of $57.5 million.

Financial Results

For the 2016 fiscal year, net loss was $103.6 million, or $2.69 per share, compared with $92.5 million, or $3.44 per share, for the 2015 fiscal year.

Ignyta had no revenues during 2016 or 2015.

Research and development expenses for 2016 were $76.9 million, compared with $73.5 million for 2015. During 2015, the company incurred total costs of $28.2 million in connection with its acquisition of rights to development programs from Teva and Lilly, consisting of the combined $25.3 million net value of our common stock issued and combined upfront payments totaling $2.9 million made to Teva and Lilly in connection with these transactions. Excluding these costs, R&D costs increased by $31.6 million, or 70%, during 2016 as compared to the same period in 2015. This increase was due to the $20.7 million increase in the chemistry, manufacturing, and control and external clinical development costs associated with entrectinib and the company’s other product candidates, and increased facilities costs due to an expansion of its leased facilities space. Additionally, the company incurred increased personnel costs, including additional stock compensation costs of $0.9 million, due to an increase in R&D personnel.

General and administrative expenses were $23.8 million for 2016, compared with $17.1 million for 2015. The increase was primarily attributable to increases in personnel costs, including additional stock compensation costs of $1.5 million.

At December 31, 2016, the company had cash, cash equivalents, and investment securities totaling $133.0 million and current and long-term debt of $32.0 million. At December 31, 2015, the company had cash, cash equivalents, and investment securities totaling $172.1 million and current and long-term debt of $31.0 million.