Pfenex Reports Fourth Quarter and Full Year 2016 Results and Provides Business Update

On March 15, 2017 Pfenex Inc. (NYSE MKT: PFNX), a clinical-stage biotechnology company engaged in the development of biosimilar therapeutics, including high value and difficult to manufacture proteins, reported financial results for the fourth quarter and full year ended December 31, 2016 and provided a business update (Press release, Pfenex, MAR 15, 2017, View Source [SID1234518198]).

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Business Review and Update

PF708 is the Pfenex product candidate that is being developed as a therapeutic equivalent to Forteo (teriparatide), marketed by Eli Lilly for the treatment of osteoporosis patients at high risk of fracture. The PF708 bioequivalence study, conducted in 70 healthy subjects, was completed in the second quarter of 2016 and met its primary objectives. We initiated an immunogenicity/pharmacokinetics clinical study in osteoporosis patients in the fourth quarter of 2016. The interim pharmacokinetic data from this study is expected in the second half of 2017 and the immunogenicity data is expected in the first half of 2018. Pfenex believes this study, along with the positive bioequivalence study, is anticipated to satisfy the filing requirements for PF708 through the 505(b)(2) regulatory pathway.

In August 2016, Pfenex regained full rights from Pfizer to PF582, our biosimilar candidate to Lucentis (ranibizumab) for the treatment of retinal diseases, and announced positive results from the phase 1/2 trial, which showed that PF582 was pharmacologically active and with a safety profile that was consistent with that of Lucentis. We are continuing to explore strategic options for PF582.

Px563L, a novel anthrax vaccine candidate, is being developed by Pfenex in response to the United States government’s unmet demand for increased quantity, stability and dose sparing regimens of anthrax vaccine. We initiated a randomized, placebo-controlled Phase 1a trial in healthy subjects in the second half of 2015 to investigate the safety and immunogenicity of Px563L, and we announced the interim analysis results in the second half of 2016. Findings indicated that the vaccine was well-tolerated, with the potential to afford immunogenicity protection against anthrax infection with fewer injections. The development of Px563L is funded by the U.S. Department of Health and Human Services, through the Biomedical Advanced Research and Development Authority, or BARDA, in accordance with a cost plus fixed fee advanced development contract valued at up to approximately $143.5 million. In addition to the base period, BARDA has now exercised additional phases of the development contract effective January 2017, allowing for the continuing development of Px563L. The phase 2 study could initiate in 2018, provided the program continues to successfully advance with the support of BARDA. Pfenex believes the successful completion of the activities under this contract could lead to a procurement contract for supply of Px563L to the Strategic National Stockpile.

Regulatory feedback for PF529, our biosimilar candidate to Neulasta (pegfilgrastim), was received in 2016 and supported the feasibility of development under the 351(k) biosimilar pathway. Pfenex continues to evaluate the potential resource requirements and timeline for development.

In January 2017, Pfenex announced that Dr. Bertrand C. Liang resigned his position as Chief Executive Officer, President, Secretary and as a member of the Board of Directors of the Company following the completion of an independent investigation overseen by the Audit Committee that revealed Dr. Liang had not acted in accordance with the Company’s Board Approval Process Policy and Code of Ethics and Conduct. The misconduct did not impact the operating results for Pfenex. Patrick K. Lucy, the Company’s Chief Business Officer, was appointed to serve as Interim Chief Executive Officer, President, and Secretary. The Board of Directors has formed a search committee consisting of select independent directors to initiate an executive search for a replacement CEO.

Financial Highlights for the Fourth Quarter and Full Year 2016

Total Revenue increased by $50.6 million, or 528%, from $9.6 million in 2015 to $60.2 million in 2016. The increase in revenue was primarily due to the termination of our development and license agreement with Pfizer. As a result of the termination in August 2016, the estimated performance period was accelerated resulting in the recognition of $45.8 million of revenue that had been previously deferred. As a result of the termination, we will not recognize any additional future revenue under the Pfizer development and license agreement. Revenue increased by $2.2 million, or 68%, to $5.5 million in the three month period ended December 31, 2016 compared to $3.3 million in the same period in 2015. The increase in the three month period was due primarily to one-time milestone payments which contributed approximately $2 million to license fee revenue for the quarter ended December 31, 2016.

Cost of revenue increased by $0.7 million, or 15%, to $5.3 million in 2016 compared to $4.6 million in 2015. The change was due primarily to a net increase of $0.7 million in costs for our proprietary novel vaccine program Px563L, which is funded by a government agency. Cost of revenue decreased by approximately $0.4 million, or 24%, to $1.3 million in the three month period ending December 31, 2016 compared to $1.7 million in the same period in 2015 due primarily to timing of costs related to the development of our Px563L product candidate. Given the nature of the novel vaccine development process, cost of revenue will fluctuate depending on stage of development.

Research and development expenses increased by approximately $14.2 million, or 78%, to $32.4 million in 2016 compared to $18.2 million in 2015. The increase in research and development expenses in 2016 compared to 2015 was due to manufacturing and development activities of PF708, which is being developed as a therapeutically equivalent candidate Forteo, and our other biosimilar product candidates. R&D expenses increased by approximately $4.6 million, or 75%, to $10.7 million in the three months ended December 31, 2016 compared to the same period in 2015. The increase in the three month period was due primarily to manufacturing and development costs associated with our product candidates.

Selling, general and administrative expenses increased by $2.7 million, or 19% to $17.3 million in 2016 compared to $14.6 million in 2015 and increased by $0.7 million, or 18%, to $4.4 million in the three month period ended December 31, 2016 from $3.7 million in the same period in 2015. The increases in selling, general and administrative expenses were primarily due to higher personnel-related expenses.

Cash and cash equivalents as of December 31, 2016 was $81.5 million.

The National Institutes of Biomedical Innovation, Health and Nutrition, Daiichi Sankyo,and Mitsubishi UFJ Capital Announce Open Innovation Research on New Cancer Immunotherapy

On March 16, 2017 National Institutes of Biomedical Innovation, Health and Nutrition (Director General: Yoshihiro Yoneda; Ibaraki-shi, Osaka; hereinafter "NIBIOHN"), Daiichi Sankyo Company, Limited (Representative Director, President and CEO: George Nakayama; head office: Chuo-ku, Tokyo; hereinafter "Daiichi Sankyo"), and Mitsubishi UFJ Capital Co., Ltd. (President: Muneki Handa; head office: Chuo-ku, Tokyo; hereinafter "Mitsubishi UFJ Capital") reported that they will launch open innovation research ("the research") on new cancer immunotherapy (Press release, Daiichi Sankyo, MAR 15, 2017, View Source [SID1234518205]).

To carry out the research, a new company called OiDE Adjubilee, Inc. (head office: Chuo-ku, Tokyo; hereinafter "Adjubilee") was established and wholly funded by the OiDE Fund Investment Limited Partnership ("OiDE Fund"), operated by Mitsubishi UFJ Capital.

If the goals of the two-year joint research are achieved, Daiichi Sankyo will purchase all Adjubilee stock in order to continue research and development of the new cancer immunotherapy on its own, paying sales royalties to NIBIOHN following a successful product launch.

This research into new cancer immunotherapy is the second OiDE Fund investment, and Daiichi Sankyo and Mitsubishi UFJ Capital plan to continue to promote open innovation activities to develop new drug discovery platforms using the OiDE Fund.

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

Aclaris Therapeutics 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, Aclaris Therapeutics, 2017, MAR 15, 2017, View Source [SID1234521533]).

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20-F – Annual and transition report of foreign private issuers [Sections 13 or 15(d)]

(Filing, Annual, AEterna Zentaris, 2016, MAR 15, 2017, View Source [SID1234518135])

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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]).

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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.