CTI BioPharma 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, CTI BioPharma, 2018, MAR 7, 2018, View Source [SID1234524492]).

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On March 7, 2018 CTI BioPharma Corp. (NASDAQ:CTIC) reported financial results for the fourth quarter and full year ended December 31, 2017 (Press release, CTI BioPharma, MAR 7, 2018, View Source;p=RssLanding&cat=news&id=2336850 [SID1234524503]).

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Clinical / Regulatory

In July 2017, the first patient was enrolled in PAC203, a Phase 2 clinical trial of pacritinib in patients with primary myelofibrosis who have failed prior ruxolitinib therapy and/or have thrombocytopenia, to evaluate the dose response relationship for safety and efficacy. The Company expects to complete an interim analysis of data in the second quarter of 2018, with full top-line data expected in the first quarter of 2019.
In January 2018, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) granted a three-month extension for submitting responses to the Day 120 List of Questions (D120 LoQ) for the MAA for pacritinib. As a result of the extension, the Company anticipates submitting its response to the D120 LoQ in May 2018 and receipt of the CHMP’s opinion on the Marketing Authorization Application (MAA) in the third quarter of 2018.
In August 2017, enrollment was completed in the PIX306 Phase 3 trial of PIXUVRI (pixantrone). Top-line results are event-driven and are expected in the second quarter of 2018.
Financial and Partnerships

In February 2018, received gross proceeds of $69 million from an underwritten public offering of common stock. In June 2017, the Company received gross proceeds of $45 million through an underwritten public offering of preferred stock.
In January 2018, the Company reincorporated in the State of Delaware, triggering an automatic delisting of the Company’s common stock from the Borsa Italiana MTA exchange.
In January 2018 and again in June 2017, the Company announced that it received a $10 million milestone payment from Teva Pharmaceutical Industries Ltd. related to a regulatory approval milestone for TRISENOX.
In April 2017, the Company announced the expansion of the existing license and collaboration agreement with Servier, providing Servier with rights to PIXUVRI (pixantrone) in all markets except in the United States.
Board of Directors and Management

In September 2017, Laurent Fischer, M.D. was appointed Chairman of the Board of Directors. The Company also announced the appointment of David Parkinson, M.D. and Michael A. Metzger to the Board of Directors in 2017. Phillip M. Nudelman, Ph.D. and Jack W. Singer, M.D. resigned as members of the Board.
In March 2017, Adam Craig, M.D., Ph.D., was appointed President and CEO and a Director of the Company, succeeding Richard Love, who continues to serve on the Company’s Board. Additional management changes in 2017 included the promotion of David H. Kirske to Chief Financial Officer and Bruce J. Seeley to Chief Operating Officer.
"CTI BioPharma is now focused on the enrollment of patients in the PAC203 study of our lead compound, pacritinib, for the treatment of myelofibrosis. We are well-financed after recently completing an over-subscribed public offering of our common stock, and we believe we have sufficient cash to carry us through key clinical and regulatory milestones over the next 24 months," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI BioPharma. "We look forward to several key clinical and regulatory milestones in 2018, including an interim data analysis for the PAC203 study of pacritinib and top-line results in the PIX306 Phase 3 trial of PIXUVRI in the second quarter."

Fourth Quarter Financial Results

Total revenues for the fourth quarter and twelve months ended December 31, 2017, were $0.46 million and $25.1 million, respectively, compared to $9.1 million and $57.4 million for the respective periods in 2016. The decrease in total revenues for the twelve months of 2017 is primarily due to recognition of $32 million in milestone revenue related to pacritinib in the first quarter of 2016. Net product sales of PIXUVRI for the fourth quarter and twelve months ended December 31, 2017, were zero and $0.9 million, respectively, compared to $1.0 million and $4.1 million for the respective periods in 2016. The decrease in net product sales for the periods in 2017 compared to 2016, is primarily related to the April 2017 expansion of the PIXUVRI agreement with Servier under which they have rights in all markets except the United States.

GAAP operating loss for the fourth quarter and twelve months ended December 31, 2017, was $13.7 million and $39.5 million, respectively, compared to GAAP operating loss of $5.6 million and $49.2 million for the respective periods in 2016. Non-GAAP operating loss, which excludes non-cash share-based compensation expense, for the fourth quarter and twelve months ended December 31, 2017 was $12.3 million and $33.8 million, respectively, compared to non-GAAP operating loss of $3.5million and $35.8 million for the respective periods in 2016. Non-cash share-based compensation expense for the fourth quarter and twelve months ended December 31, 2017, was $1.4 million and $5.7 million, respectively, compared to $2.1 million and $13.3 million for the respective periods in 2016. The decrease in operating loss for the fourth quarter and twelve months of 2017 was due to a significant decrease in research and development and selling, general and administrative expenses primarily related to a decrease in pacritinib development costs as a result of the completion of the Phase 3 clinical studies in 2017 and a decrease in expenses for the manufacture of pacritinib and personnel costs. For information on CTI BioPharma’s use of non-GAAP operating loss and a reconciliation of such measure to GAAP operating loss, see the section below entitled "Non-GAAP Financial Measures."

Net loss for the fourth quarter of 2017 was $14.3 million, or ($0.33) per share, compared to a net loss of $6.4 million, or ($0.23) per share, for the same period in 2016. Net loss for the twelve months ended December 31, 2017, was $45.0 million, or ($1.24) per share, compared to a net loss of $52.0 million, or ($1.86) per share, for the same period in 2016.

As of December 31, 2017, cash, cash equivalents and restricted cash totaled $43.2 million, compared to $44.0 million at December 31, 2016.

Conference Call Information

CTI BioPharma management will host a conference call to review its fourth quarter and full year 2017 financial results and provide an update on business activities. The event will be held today at 1:30 p.m. PT / 4:30 p.m. ET / 10:30 p.m. CET. Participants can access the call at 1-800-289-0517 (domestic) or + +1 323-994-2084 (international). To access the live audio webcast or the subsequent archived recording, visit www.ctibiopharma.com. Webcast and telephone replays of the conference call will be available approximately two hours after completion of the call. Callers can access the replay by dialing 1-888-203-1112 (domestic) or +1 719-457-0820 (international). The access code for the replay is 2516357. The telephone replay will be available until March 14, 2018.

On March 7, 2018 -Relay Therapeutics, a biotech company dedicated to developing breakthrough medicines by leveraging insights from protein motion, reported that Sanjiv Patel, M.D., President and Chief Executive Officer, will present at Cowen and Company’s 38th Annual Health Care Conference (Press release, Relay Therapeutics, JUL 7, 2018, View Source [SID1234524523]).

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The presentation will take place on Monday, March 12, 2018 at 4:00 p.m. Eastern Time at the Boston Marriott Copley Place in Boston, MA.

On March 7, 2018 Idera Pharmaceuticals, Inc. ("Idera") (NASDAQ:IDRA), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel nucleic acid-based therapeutics for oncology and rare diseases, reported its financial and operational results for the fourth quarter and year ended December 31, 2017 (Press release, Idera Pharmaceuticals, MAR 7, 2018, View Source [SID1234524648]).

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"Our company made tremendous progress throughout 2017 advancing our development programs and we expect the first half of 2018 to be marked by a number of key inflection points for each of these programs," stated Vincent Milano, Idera’s chief executive officer. "In 2018, we are continuing to enroll patients in the ILLUMINATE 204 Phase 2 trial of IMO-2125, with the next planned data update at ASCO (Free ASCO Whitepaper) and completion of enrollment expected by year end. We also recently initiated the Phase 3 ILLUMINATE 301 trial. For IMO-8400, we plan to report top-line data from our Phase 2 trial in dermatomyositis by the end of the 2nd quarter. We also intend to finalize our development plans for our lead nucleic acid chemistry research candidate, IDRA-008 shortly," continued Milano.

"In January, we announced our proposed merger with BioCryst Pharmaceuticals, Inc. that we believe will build greater and more sustainable value for the benefit of stockholders as well as patients with rare diseases beyond what we could achieve alone. The Idera Board determined this combination was compelling from both a strategic and financial perspective following a careful evaluation of a range of strategies to enhance long-term stockholder value. The transaction will create a leading rare disease company with a robust pipeline including two promising Phase 3 rare disease programs and combines synergistic discovery engines that will not only expand the number of rare diseases we can target but create meaningful opportunities for differentiation in the market through joint small molecule and oligo treatments. Importantly, joining with BioCryst will also enable us to achieve cost synergies and increase our financial strength and flexibility," Milano expressed.

Clinical Development Program Updates:
ILLUMINATE (IMO-2125) Clinical Development

ILLUMINATE 204 – Phase 1/2 trial of IMO-2125 in combination with ipilimumab or pembrolizumab in patients with PD-1 refractory metastatic melanoma:

As of March 7, 2018, enrolled 26 patients at 8 mg (RP2D) dose with ipilimumab, enrollment completion expected by year end 2018;
5 of the first 10 evaluable patients at the 8 mg dose of IMO-2125 were responders (50% Overall Response Rate [ORR]);
7 trial sites currently enrolling patients with goal of expansion to 10 sites during first half of 2018;
In pembrolizumab combination arm of the trial, phase 1 dose escalation continues into the last dosing cohort (32 mg); and
Next clinical data update expected at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2018.
ILLUMINATE 101 – Phase 1b trial of intratumoral IMO-2125 monotherapy in patients with refractory solid tumors:

Completed enrollment in first two cohorts (11 patients treated with 8 mg dose of IMO-2125, 8 patients treated with 16 mg dose of IMO-2125);
Two subjects in cohort 1 (8 mg) continue IMO-2125 monotherapy on the 101 study as of March 7, 2018; and
Enrollment of third cohort has commenced (8 patients to be treated with 23 mg dose of IMO-2125).
ILLUMINATE 301 – Randomized phase 3 trial of IMO-2125 in combination with ipilimumab versus ipilimumab alone in patients with PD-1 refractory metastatic melanoma:

Trial initiated in Q1 2018;
Approximately 80 sites planned for trial participation across 12 countries;
Planned enrollment of approximately 300 patients with Overall Response Rate (ORR) and Overall Survival (OS) as primary endpoints; and
U.S. Food and Drug Administration granted Fast Track Designation for IMO-2125 in combination with ipilimumab for treatment of PD-1 refractory metastatic melanoma in fourth quarter of 2017.
Pioneer (IMO-8400) Development Activities

PIONEER – Phase 2 trial of IMO-8400 in adult patients with dermatomyositis:

Enrollment concluded during Q3 2017 (30 patients); and
Topline phase 2 trial data expected in Q2 2018.
Nucleic Acid Chemistry Research Group

IDRA-008 Development Activities:

Selected apolipoprotein C-III (APOC-III) as first gene target for development for treatment of familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy (FPL);
Completion of pre-clinical toxicology and IND-enabling studies in Q1 2018;
Pre-clinical pharmacology study in cyno-model comparing IDRA-008 to competitive clinical development asset, volanesorsen expected to readout towards the latter part of Q1 2018;
Development decision for IDRA-008 expected during Q2 2018; and
The Company is continuing to evaluate rare-disease opportunities for application of its core nucleic acid chemistry research capability and expertise to yield innovative oligonucleotide therapeutic concepts that address significant unmet medical needs.
Financial Results
Fourth Quarter Results
Net loss applicable to common stockholders for the three months ended December 31, 2017 was $14.9 million, or $0.08 per basic and diluted share, compared to net income applicable to common stockholders of $0.8 million, or $0.01 per basic and diluted share, for the same period in 2016. Revenue in the fourth quarter of 2017 was nominal and primarily related to our collaboration with GSK. Revenue in the fourth quarter of 2016 was $15.3 million, primarily related to the agreement we entered into with Vivelix in November 2016 in which we received an upfront, non-refundable fee of $15 million. Research and development expenses for the three months ended December 31, 2017 totaled $10.4 million compared to $11.0 million for the same period in 2016. General and administrative expense for the three months ended December 31, 2017 totaled $4.8 million compared to $3.5 million for the same period in 2016.

Full Year Results
Net loss applicable to common stockholders for the year ended December 31, 2017 was $66.0 million or $0.42 per basic and diluted share, compared to net loss applicable to common stockholders of $38.4 million, or $0.30 per basic and diluted share, for the same period in 2016. Revenue for the year ended December 31, 2017 was $0.9 million compared to revenue of $16.2 million for the same period in 2016. Revenue in the 2017 period primarily related to our collaboration with GSK. Revenue in the 2016 period primarily related to collaborations with both GSK and Vivelix, including an upfront, non-refundable fee of $15 million received in connection with the Vivelix Agreement. Research and development expenses for the year ended December 31, 2017 totaled $50.7 million compared to $39.8 million for the same period in 2016. General and administrative expenses for the year ended December 31, 2017 totaled $16.7 million compared to $15.1 million for the same period in 2016.

As of December 31, 2017, our cash, cash equivalents and investments totaled $112.6 million compared to $109.0 million as of December 31, 2016. We currently anticipate that, based on our current operating plan and without taking into account the transaction with BioCryst, our existing cash, cash equivalents and investments will fund our operations into the second quarter of 2019.

Corporate Updates:

On January 22, 2018, BioCryst Pharmaceuticals, Inc. ("BioCryst") and Idera jointly announced the signing of a definitive merger agreement to create a company focused on the development and commercialization of medicines to serve patients suffering from rare diseases. The combined company will be renamed upon closing, and will be led by Vincent Milano, the current chief executive officer of Idera. Jon Stonehouse, the current chief executive officer of BioCryst, will serve as a member of the Board of Directors. The transaction is subject to approval by the stockholders of both companies, as well as the satisfaction of customary closing conditions. The transaction is expected to be completed by the end of the second quarter of 2018.

(Filing, Annual, Sanofi, 2017, MAR 7, 2018, View Source [SID1234524493])

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