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Sierra Oncology Reports 2017 Year End Results

On February 27, 2018 Sierra Oncology, Inc. (Nasdaq: SRRA), a clinical stage drug development company focused on advancing next generation DNA Damage Response (DDR) therapeutics for the treatment of patients with cancer, today reported its financial and operational results for the year ended December 31, 2017 (Press release, Sierra Oncology, FEB 27, 2018, View Source [SID1234524262]).

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"During 2017, we made substantial progress advancing our portfolio of promising next generation DDR therapeutics. In particular, emerging preclinical and clinical data continue to reinforce the fundamental biological role of Chk1 in regulating cancer-driven replication stress associated with genomic instability, a hallmark of cancer with potentially broad therapeutic relevance. Accordingly, during the year we significantly advanced and expanded the development program for our potential best-in-class Chk1 inhibitor, SRA737, which will now be evaluated across ten cancer indications in two ongoing Phase 1/2 trials, targeting aggregate enrollment of approximately 200 genetically-selected patients. We look forward to reporting preliminary data from these trials, expected in the fourth quarter of 2018," said Dr. Nick Glover, President and CEO of Sierra Oncology. "We also announced, subsequent to the end of the year, the advancement of a promising third development opportunity for SRA737 with the signing of an agreement with Janssen to supply us with ZEJULA (niraparib). This agreement facilitates the advancement of a novel Chk1i/PARPi combination trial in prostate cancer that we plan to initiate in the fourth quarter of 2018, which will be led by Professor Johann de Bono, Regius Professor of Cancer Research at The Institute of Cancer Research and The Royal Marsden NHS Foundation Trust.

2017 Highlights:

In January 2017, we relaunched as Sierra Oncology with a stated focus on developing oncology drugs targeting the DNA Damage Response (DDR) network.
Also in January, we successfully transferred sponsorship of the two ongoing Phase 1/2 clinical trials for our lead DDR drug candidate, SRA737, from the Cancer Research UK Centre for Drug Development, enabling us to submit protocol amendments aimed at enhancing these studies to include cohort expansions of prospectively selected patients with tumors identified to have genetic aberrations hypothesized to confer sensitivity to Chk1 inhibition via synthetic lethality.
In February, we raised net proceeds of US$27.4 million to further advance our programs.
In April, our collaborator, the Institute of Cancer Research (ICR), London, UK, reported preclinical results for SRA737 in a poster at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, supporting our genetically-driven clinical development strategy for SRA737.
In May, we were issued a patent providing SRA737 with protection in the United States to 2033, before any potential patent term extensions.
In June, having received clearance for our protocol amendments, we presented these innovative SRA737 Phase 1 clinical designs in two Trials in Progress posters at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. We concurrently reported encouraging initial progress from the two ongoing trials; SRA737 was well-tolerated and a maximum tolerated dose (MTD) had not yet been reached in the dose escalation phase of the monotherapy trial.
In August, we established a DDR Advisory Committee composed of leading experts in DDR biology, chemistry and medicine. We also held two Key Opinion Leader (KOL) events, in September and October 2017, which featured members from our Advisory Committee who shared their insights on emerging DDR biology and the potential of Chk1 inhibition in oncology, providing further support for our programs.
In October, we presented a poster at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), reporting on preclinical data demonstrating that sub-therapeutic, non-cytotoxic doses of gemcitabine induce replication stress and hence potentiate the anti-tumor activity of SRA737. These data support the design of our ongoing Phase 1/2 clinical trial which is specifically evaluating this combination.
We appointed Dr. Andrew Allen to our Board of Directors, effective October 23, 2017. Dr. Allen is the co-founder of Gritstone Oncology and was the co-founder of Clovis Oncology (Nasdaq: CLVS).

Subsequent Events:
On February 27, 2018, we provided an update on the SRA737 and SRA141 development programs:

For the SRA737 Monotherapy Phase 1/2 trial, we reported that the Dose Escalation Phase 1 portion of the study was in the final stages of optimizing the SRA737 dose regimen and the Cohort Expansion Phase 2 portion of the study was ongoing. The Cohort Expansion Phase 2 portion of the study was being expanded to include more patients, including a sixth indication (CCNE1-driven ovarian cancer), and would in aggregate target enrollment of 120 patients across six genetically-defined cohorts. We also reported that we plan to expand the number of sites recruiting patients into the trial from three active sites (as of the third quarter of 2017) to fifteen leading centers across the United Kingdom. The Cohort Expansion Phase 2 portion of the study is expected to report preliminary data in the fourth quarter of 2018.
For the SRA737 Low-Dose Gemcitabine Phase 1/2 Combination trial, we reported that the Standard-Dose Triplet Combo Dose Escalation Phase 1 was complete and that the Low-Dose Gemcitabine Combo Dose Escalation Phase 1 had made significant progress. In addition, the Low-Dose Gemcitabine Combo Cohort Expansion Phase 2 was anticipated to commence in the second quarter of 2018 and would be expanded to target enrollment of 80 genetically-selected patients across four indications. An update on the study is expected to be reported in the fourth quarter of 2018.
We reported signing a supply agreement with Janssen Research & Development, LLC where they will supply TESARO’s ZEJULA (niraparib), an orally administered poly ADP-ribose polymerase (PARP) inhibitor, facilitating the initiation of a combination trial of niraparib with SRA737 in patients with prostate cancer in the fourth quarter of 2018. The trial is to be led by Professor Johann de Bono, Regius Professor of Cancer Research, Head of the Division of Clinical Studies and Professor in Experimental Cancer Medicine at The Institute of Cancer Research and The Royal Marsden NHS Foundation Trust.
We presented emerging evidence of biological synergy between immune checkpoint blockade and Chk1 inhibition. Sierra is currently designing a clinical study for this combination, which potentially could be submitted to regulatory authorities in the fourth quarter of 2018.
We reported supportive preclinical research for SRA141 demonstrating noteworthy anti-cancer activity in two independent oncology models, generated in preparation for an Investigational New Drug (IND) filing expected in the second half of 2018.

2017 Financial Results (all amounts reported in U.S. currency)

Research and development expenses were $30.2 million for the year ended December 31, 2017, compared to $33.9 million for the year ended December 31, 2016. The decrease is primarily due to expenses incurred in 2016, including $9.0 million related to the SRA737 license agreement, a $0.9 million upfront payment for the exclusive license of SRA141, and a $2.3 million restructuring charge related to close-out expenses for PNT2258. These decreases were partially offset by increases of $4.6 million in third-party manufacturing costs, $2.6 million in research and other costs related to SRA737 and SRA141, and $1.0 million in clinical trial costs. Research and development expenses included non-cash stock-based compensation of $4.0 million and $3.6 million for the years ended December 31, 2017 and 2016, respectively.

General and administrative expenses were $12.5 million for the year ended December 31, 2017 compared to $14.2 million for the year ended December 31, 2016. The decrease is primarily due to a $1.1 million decrease in business development expenses and a $0.5 million decrease in restructuring costs as compared to 2016. General and administrative expenses included non-cash stock-based compensation of $1.9 million for both the year ended December 31, 2017 and the year ended December 31, 2016.

For the year ended December 31, 2017, Sierra incurred a net loss of $42.0 million compared to a net loss of $47.9 million for the year ended December 31, 2016.

Cash and cash equivalents totaled $100.3 million as of December 31, 2017, compared to $107.8 million as of September 30, 2017, and $109.0 million as of December 31, 2016. The company believes that its existing cash and cash equivalents will be sufficient to fund current operating plans through approximately mid-2019.

At December 31, 2017, there were 52,395,223 shares of common stock issued and outstanding, and stock options to purchase 7,470,601 shares of common stock issued and outstanding.

Data From SELLAS Life Sciences’ Positive Phase 2 Acute Myeloid Leukemia Study Published in American Society of Hematology’s Journal, Blood Advances

On February 27, 2018 SELLAS Life Sciences Group Inc. (Nasdaq:SLS) (SELLAS), a clinical-stage biopharmaceutical company focused on novel cancer immunotherapies for a broad range of cancer indications, reported that data from the Phase 2 trial of its lead candidate, galinpepimut-S (GPS), in acute myeloid leukemia (AML) have been published in the current issue of Blood Advances (Press release, Sellas Life Sciences, FEB 27, 2018, View Source [SID1234524259]). GPS met its pre-specified primary endpoint of ≥34% actual overall survival (OS) rate at three years with a GPS-induced OS rate of 47.4%. Median disease-free survival (DFS) from first complete response was 16.9 months, while the overall survival (OS) from diagnosis has not yet been reached, but is predicted to be > 67.6 months. GPS targets the antigen, Wilms tumor 1 (WT1) protein, which has been ranked by the National Cancer Institute as the leading target for cancer immunotherapy.

"These data follow on prior positive data in AML and various cancer indications, supporting the development of GPS as an important potential therapy in the treatment of AML and other cancers," said Angelos Stergiou, MD, ScD h.c., President & Chief Executive Officer of SELLAS. "We are encouraged by the collective supporting evidence generated by our Phase 1 and Phase 2 AML studies. We wholeheartedly appreciate the participation of patients and their families in all our clinical studies, as well as the exceptional physicians and study teams. We look forward to advancing GPS into a Phase 3 trial in AML."

For patients in the older cohort (age >60; n=13), median OS post-diagnosis was 35.8 months. Historical controls for comparable patients over sixty years of age who reach first complete remission (CR1) show median OS since initial diagnosis of 9.5-15.8 months.

"We were especially pleased with the findings in AML patients over sixty years of age, which are important considering the poor prognosis particularly for those patients, even with optimal use of current care standards" stated Peter Maslak, M.D., Chief, Immunology Laboratory Service at Memorial Sloan Kettering Cancer Center and Principal Investigator of the study.

The open-label Phase 2 study evaluated GPS in 22 adult patients with AML (median age – 64 years) in CR1. Patients received 6 vaccinations administered over 10 weeks with the potential to receive 6 additional monthly doses if they remained in CR1. Immune responses (IR’s) were evaluated after the sixth and twelfth vaccinations by CD4+ T-cell proliferation, CD8+ T cell interferon-γ secretion (ELISPOT) or the CD8-relevant WT1 peptide MHC tetramer assay (HLA-A*02 patients only).

"Older adults with AML who achieve complete remission (CR) are in critical need of new treatment options to prevent emerging relapses, especially in cases where allogeneic stem cell transplant is infeasible or is not predicted to improve outcome," stated Gert Ossenkoppele, M.D., Ph.D., professor of Hematology at the VU University Medical Center in Amsterdam, The Netherlands, and chair of the AML working party of HOVON (Dutch-Belgian Hematology Trial Group). "Results from this GPS Phase 2 study reinforce the potential of this innovative WT1-targeting immunotherapy in the post-CR maintenance setting. I look forward to co-leading the Phase 3 study in AML patients older than 60 years, currently being planned by SELLAS." Dr. Ossenkoppele did not participate in the GPS Phase 2 study.

In the study, GPS was well tolerated, with the most common side effects being Grade 1/2 injection site reactions (46%), fatigue (32%), and skin induration (32%). Fourteen patients (64%) completed more than six vaccinations, and nine (41%) received all 12 vaccine doses. Nine of 14 tested patients (64%) had an immune response (IR) in more than one of three assays used (one for CD4 or two for CD8).

The article, "Phase 2 trial of a multivalent WT1 peptide vaccine (galinpepimut-S) in acute myeloid leukemia," is available in the current issue of Blood Advances, a peer-reviewed medical journal published by the American Society of Hematology (ASH) (Free ASH Whitepaper). The complete article can be accessed here (View Source).

Rigel Announces Conference Call and Webcast to Report Fourth Quarter and Year End 2017 Financial Results

On February 27, 2018 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) reported that it will report its fourth quarter and year end 2017 financial results after market close on Tuesday, March 6, 2018. Rigel senior management will follow the announcement with a live conference call and webcast at 5:00pm Eastern Time (2:00pm Pacific Time) to discuss the financial results and provide a company update (Press release, Rigel, FEB 27, 2018, View Source;p=RssLanding&cat=news&id=2334848 [SID1234524258]).

Participants can access the live conference call by dialing 855-892-1489 (domestic) or 720-634-2939 (international) and using the Conference ID number 7289803. The conference call will also be webcast live and can be accessed from Rigel’s website at www.rigel.com. The webcast will be archived and available for replay for 30 days after the call via the Rigel Website.

Pfenex Inc. to Present at Barclay’s 2018 Global Healthcare Conference

On February 27, 2018 Pfenex Inc. (NYSE AMERICAN: PFNX), reported that the company will present at Barclay’s 2018 Global Healthcare Conference in Miami Beach, Florida (Press release, Pfenex, FEB 27, 2018, View Source2018-02-27-Pfenex-Inc-to-Present-at-Barclays-2018-Global-Healthcare-Conference" target="_blank" title="View Source2018-02-27-Pfenex-Inc-to-Present-at-Barclays-2018-Global-Healthcare-Conference" rel="nofollow">View Source [SID1234524255]).

President and Chief Executive Officer, Eef Shimmelpennink, is scheduled to present on Tuesday, March 13th at 3:50 pm ET.

Interested parties can access the live audio webcast for this presentation from the Investors Section of Pfenex’s website at www.pfenex.com. The webcast replay will be available after the conclusion of the live presentation for approximately 60 days.

Pfenex investors and others should note that we announce material information to the public about the Company through a variety of means, including our website (View Source), our investor relations website (View Source), press releases, SEC filings, public conference calls, corporate Twitter account (View Source), Facebook page (View Source), and LinkedIn page (View Source) in order to achieve broad, non-exclusionary distribution of information to the public and to comply with our disclosure obligations under Regulation FD. We encourage our investors and others to monitor and review the information we make public in these locations as such information could be deemed to be material information. Please note that this list may be updated from time to time.

Ophthotech Reports Fourth Quarter and Full Year 2017 Financial and Operating Results

On February 27, 2018 Ophthotech Corporation (Nasdaq:OPHT) reported financial and operating results for the fourth quarter and full year ended December 31, 2017 and provided a business update (Press release, Ophthotech, FEB 27, 2018, View Source [SID1234524253]).

The Company also announced today that it has initiated an innovative gene therapy program focused on applying novel gene therapy technology to discover and develop new therapies for ocular diseases. The Company intends to investigate promising gene therapy product candidates and other technologies through collaborations with leading companies and academic institutions in the United States and internationally. For its first gene therapy collaboration, the Company has entered into a series of sponsored research agreements with the University of Massachusetts Medical School (UMMS) and its Horae Gene Therapy Center to utilize their "minigene" therapy approach and other novel gene delivery technologies to target retinal diseases. As a condition of each research agreement, UMMS has granted the Company an option to obtain an exclusive license to any patent or patent applications that result from this research. This announcement will be discussed during today’s conference call/webcast (see press release issued earlier today and call in details below).

"In 2017, our team began to execute on a strategic plan to restructure the Company, broaden our Zimura portfolio to include development programs in both orphan diseases and larger indications in the back of the eye, including dry and wet forms of age-related macular degeneration, and to initiate an aggressive business development outreach," stated Glenn P. Sblendorio, Chief Executive Officer and President of Ophthotech. "We start 2018 with a strong balance sheet, multiple clinical trials in our Zimura program, and have now entered into the gene therapy arena with a collaboration with the Horae Gene Therapy Center at University of Massachusetts Medical School. Looking ahead we will continue to seek collaborations, in-licensing and partnering opportunities that offer novel and differentiating approaches, including gene therapy approaches, for treating diseases of the eye."

Zimura Complement Factor C5 Inhibitor Program

During the second half of 2017, the Company modified its on-going Zimura (avacincaptad pegol) clinical trial for the treatment of geographic atrophy (GA) secondary to dry age related macular degeneration (AMD). The modification of the trial design accelerates the anticipated timeline for obtaining top-line data. Initial top-line data is expected to be available during the second half of 2019. The scientific details of the GA secondary to dry AMD clinical trial were presented at the 41st Annual Macula Society Meeting in Beverly Hills, California, February 21-24, 2018.
During the third quarter of 2017, the Company initiated a dose-ranging, open-label Phase 2a clinical trial of Zimura in combination with the anti-vascular endothelial growth factor (anti-VEGF) agent Lucentis (ranibizumab) in patients with wet AMD who have not been previously treated with any anti-VEGF agents. Based on the current enrollment rate, the Company expects initial top-line data from this trial to be available by the end of 2018.
In January 2018, the first patient was enrolled in the Company’s Phase 2b randomized, double-masked, sham-controlled clinical trial assessing the efficacy and safety of Zimura in patients with autosomal recessive Stargardt disease (STGD1). Initial top-line data is expected to be available in 2020. The scientific details of the Stargardt clinical trial will be presented at the 2018 Annual Meeting of the Association for Research in Vision and Ophthalmology in Honolulu, Hawaii, April 29 – May 3, 2018 and at The International Symposium on Ocular Pharmacology and Therapeutics in Tel-Aviv, Israel, March 1-3, 2018.
During the fourth quarter of 2017, the Company initiated an open-label Phase 2a clinical trial evaluating Zimura in combination with the anti-VEGF agent Eylea (aflibercept) for the treatment of idiopathic polypoidal choroidal vasculopathy in treatment experienced patients. Initial top-line data is expected to be available during the second half of 2019.

In January 2018, the Company announced the election of Jane PritchettHenderson, Chief Financial Officer and Senior Vice President of Corporate Development at Voyager Therapeutics, to its Board of Directors. Ms. Henderson has also been elected the Chair of the Ophthotech Audit Committee.

2018 Operational Update

As of December 31, 2017, the Company had $167 million in cash and cash equivalents. Based on the Company’s current 2018 business plan, including continuation of its development programs for Zimura and initiation of its collaborative gene therapy research programs, the Company expects the cash required to fund its operations and capital expenditures for 2018 will range between $50 million and $55 million. This estimate does not reflect any additional expenditures resulting from the potential in-licensing or acquisition of additional product candidates or technologies or associated development that the Company may pursue.

Fourth Quarter 2017 Financial Highlights

Revenues: Collaboration revenue was $0 for the quarter ended December 31, 2017, compared to $5.3 million for the same period in 2016. For the year ended December 31, 2017, collaboration revenue was $210.0 million, compared to $50.9 million for 2016. Collaboration revenue variances for both the quarter and the year are the result of the completion of deliverables under the Fovista licensing and commercialization agreement with Novartis Pharma AG and the recognition of all associated deferred revenue during the third quarter of 2017. The recognition of this revenue did not impact the Company’s cash balance.
R&D Expenses: Research and development expenses were $7.9 million for the quarter ended December 31, 2017, compared to $59.4 million for the same period in 2016. For the quarter ended December 31, 2017, research and development expenses included approximately $0.7 million in costs related to the Company’s previously announced reduction in personnel. For the year ended December 31, 2017, research and development expenses were $66.3 million, compared to $196.3 million for 2016. For the year ended December 31, 2017, research and development expenses included approximately $7.5 million in costs related to the Company’s previously announced reduction in personnel. Research and development expenses decreased in both the quarter and year ended December 31, 2017 primarily due to its termination of the Fovista development programs in wet AMD.

G&A Expenses: General and administrative expenses were $6.9 million for the quarter ended December 31, 2017, compared to $13.0 million for the same period in 2016. For the quarter ended December 31, 2017, general and administrative expenses included approximately $0.5 million in costs related to the Company’s previously announced reduction in personnel. For the year ended December 31, 2017, general and administrative expenses were $35.7 million, compared to $50.2 million in 2016. For the year ended December 31, 2017, general and administrative expenses included approximately $5.6 million in costs related to the Company’s previously announced reduction in personnel and its termination of facilities leases. General and administrative expenses decreased in both the quarter and year ended December 31, 2017 primarily due to a decrease in personnel and infrastructure costs to support the Company’s operations.
Net Income: The Company reported a net loss for the quarter ended December 31, 2017 of $9.5 million, or ($0.26) per diluted share, compared to a net loss of $66.3 million, or ($1.86) per diluted share, for the same period in 2016. For the year ended December 31, 2017, the Company reported net income of $114.2 million, or $3.17 per diluted share, compared to a net loss of $193.4 million, or ($5.45) per diluted share, for 2016.

Conference Call/Web Cast Information

Ophthotech will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for February 27, 2018 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 800-239-9838 (USA) or 323-794-2551 (International), passcode 9171013. A live, listen-only audio webcast of the conference call can be accessed on the Investor Relations section of the Ophthotech website at: www.ophthotech.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA Toll Free), passcode 9171013.