On November 6, 2017 CTI BioPharma Corp. (NASDAQ and MTA:CTIC) reported financial results for the third quarter ended September 30, 2017 (Press release, CTI BioPharma, NOV 6, 2017, View Source;p=RssLanding&cat=news&id=2314662 [SID1234521603]).

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Recent Highlights

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. PAC203 is designed to evaluate the dose response relationship for safety and efficacy (spleen volume reduction at 12 and 24 weeks) of three dose regimens: 100 mg once-daily, 100 mg twice-daily (BID) and 200 mg BID. The 200 mg BID dose regimen was used in the Phase 3 PERSIST-2 trial of pacritinib in patients with myelofibrosis. The trial is expected to enroll up to approximately 105 patients.
In July 2017, the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for pacritinib for the treatment of patients with myelofibrosis who have thrombocytopenia (platelet counts less than 100,000 per microliter). Validation confirms that the submission is complete and initiates the centralized review process by the EMA’s Committee for Medicinal Products for Human Use (CHMP).
Board of Directors and Management

In September 2017, Laurent Fischer, M.D. was appointed Chairman of the Board of Directors. Dr. Fischer has more than 20 years of experience in developing and commercializing novel medicines in the biopharmaceutical industry and currently serves as liver therapeutic area head at Allergan following its acquisition of Tobira Therapeutics in 2016.
In September 2017, David H. Kirske was promoted to Chief Financial Officer and Bruce J. Seeley to Chief Operating Officer of the company.
"In the third quarter, we solidified our board and senior management leadership and continue to make significant progress in reducing expenses to operate as a leaner organization as we approach important milestones," said Adam R. Craig, M.D., Ph.D., President and Chief Executive Officer of CTI BioPharma. "We believe there remains a significant unmet need for myelofibrosis patients with low platelets and continue activating sites in the PAC203 trial of pacritinib. We also look forward to continuing to work with the EMA over the next nine months during their review of the MAA for pacritinib."

Third Quarter Financial Results

Total revenues for the third quarter and nine months ended September 30, 2017, were $1.7 million and $24.7 million, respectively, compared to $4.4 million and $48.3 million for the respective periods in 2016. The decrease in total revenues for the nine 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 third quarter and nine months ended September 30, 2017, were zero and $0.9 million, respectively, compared to $0.9 million and $3.1 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 PXUVRI agreement with Servier under which they have rights in all markets except the U.S.

GAAP operating loss for the third quarter and nine months ended September 30, 2017, was $11.8 million and $25.8 million, respectively, compared to GAAP operating loss of $28.7 million and $43.6 million for the respective periods in 2016. Non-GAAP operating loss, which excludes non-cash share-based compensation expense, for the third quarter and nine months ended September 30, 2017 was $10.4 million and $21.5 million, respectively, compared to non-GAAP operating loss of $23.6 million and $32.4 million for the respective periods in 2016. Non-cash share-based compensation expense for the third quarter and nine months ended September 30, 2017, was $1.4 million and $4.3 million, respectively, compared to $5.1 million and $11.2 million for the respective periods in 2016. The decrease in operating loss for the third quarter and nine 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 third quarter of 2017 was $12.0 million, or ($0.28) per share, compared to a net loss of $29.2 million, or ($1.04) per share, for the same period in 2016. Net loss for the nine months ended September 30, 2017, was $30.8 million, or ($0.90) per share, compared to a net loss of $45.6 million, or ($1.63) per share, for the same period in 2016.

As of September 30, 2017, cash and cash equivalents totaled $52.8 million, compared to $44.0 million at December 31, 2016.

Conference Call Information

CTI BioPharma management will host a conference call to review its third quarter 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 1-888-461-2021 (domestic) or +1 719-325-2359 (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 1048572. The telephone replay will be available until November 13, 2017.

Gilead Sciences has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Gilead Sciences, 2017, NOV 6, 2017, View Source [SID1234521655]).

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On November 6, 2017 STORM Therapeutics, the drug discovery company focused on the discovery of small molecule therapies modulating RNA epigenetics, reported the appointment of Professor Paul Workman to its Board (Press release, STORM Therapeutics, NOV 6, 2017, View Source [SID1234561049]). This appointment continues STORM’s evolution of its management and advisory networks as it fulfils its ambition to become the leading therapeutics company in RNA epigenetic modulation.

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CEO of STORM Therapeutics, Keith Blundy commented on the appointment: "I am pleased to welcome Paul asIndependent Director to STORM’s board. Paul brings a wealth of experience in cancer biology and drug discovery, in addition to the translational expertise of moving emerging biology to therapeutic applications through his experience of leading a world class cancer institute and being a founder of companies such as Piramed. We look forward to Paul’s input on development and positioning of STORM’s emerging RNA epigenetics platform and programmes."

Professor Paul Workman, Chief Executive and President of The Institute of Cancer Research, London, said: "I am delighted to be joining the board of STORM and working with the company in this exploding area of biology. RNA epigenetic modulation is an exciting area of science with real potential to deliver new targeted cancer therapies. I look forward to working with the board to help guide STORM’s innovative research programmes, adding my experience from drug discovery in academia, pharma and biotech."

On November 6, 2017 Portola Pharmaceuticals Inc. (Nasdaq:PTLA) will report financial results and provide a corporate update for the quarter ended September 30, 2017 (Press release, Portola Pharmaceuticals, NOV 6, 2017, View Source [SID1234521609]).

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“Following FDA approval of our first product, Bevyxxa, our team has been working diligently to bring this important new medicine to patients in need, and we are pleased to report good progress with the FDA on achieving product release,” said Bill Lis, chief executive officer of Portola. “We are also continuing to advance AndexXa, the first-ever Factor Xa inhibitor antidote. This treatment is highly anticipated by the medical community and we are continuing discussions with U.S. and European regulatory authorities on our filings for this novel therapy.”

Recent Achievements, Upcoming Events and Milestones

Betrixaban -– an oral, once-daily Factor Xa inhibitor

Bevyxxa (betrixaban) received U.S. Food and Drug Administration (FDA) approval for hospital and extended duration prophylaxis (35 to 42 days) of venous thromboembolism (VTE) in adult patients hospitalized for an acute medical illness who are at risk for thromboembolic complications due to moderate or severe restricted mobility and other risk factors for VTE
Committee for Medicinal Products for Human Use (CHMP) opinion anticipated by the end of 2017
Multiple abstracts accepted at the upcoming American Heart Association (AHA) and American Society of Hematology (ASH) (Free ASH Whitepaper) annual meetings
AndexXa (andexanet alfa) – a Factor Xa inhibitor antidote in development for patients treated with a Factor Xa inhibitor when reversal of anticoagulation is needed due to life-threatening bleeding or when urgent surgery is required; designated a Breakthrough Therapy and an Orphan Drug by the FDA

The FDA has set an action date of February 3, 2018 to respond to our Biologics License Application (BLA)
CHMP opinion anticipated in Q1 2018
Multiple abstracts accepted at the upcoming AHA and ASH (Free ASH Whitepaper) annual meetings
Corporate

Public offering successfully completed in September 2017, the net proceeds of which, including exercise of the underwriters’ over-allotment option, were $380.6 million.
Industry veteran John “Jack” Lawrence, M.D., appointed senior vice president and chief medical officer. Dr. Lawrence previously served as vice president and cardiovascular therapeutic area head for Bristol-Myers Squibb, including global responsibilities for apixaban.
Third Quarter 2017 Financial Results
Collaboration and license revenue earned under Portola’s collaboration and license agreements with Bristol-Myers Squibb Company, Pfizer, Bayer Pharma, Janssen Pharmaceuticals and Daiichi Sankyo was $3.8 million for the third quarter of 2017, compared with $9.3 million for the third quarter of 2016.

Total operating expenses for the third quarter of 2017 were $84.3 million, compared with $100.8 million for the same period in 2016. Total operating expenses for the third quarter of 2017 included $10.1 million in stock-based compensation expense, compared with $7.8 million for the same period in 2016.

Research and development expenses were $55.3 million for the third quarter of 2017, compared with $87.2 million for the third quarter of 2016. The decrease in R&D expenses was largely attributable to a $27.3 million one-time charge taken in the third quarter of 2016 resulting from the Company’s decision to suspend Line C manufacturing at CMC Biologics.

Selling, general and administrative expenses for the third quarter of 2017 were $28.9 million, compared with $13.6 million for the same period in 2016.

For the third quarter of 2017, Portola reported a net loss of $82.9 million, or $1.41 net loss per share, compared with a net loss of $92.9 million, or $1.64 net loss per share, for the same period in 2016.

Cash, cash equivalents and investments at September 30, 2017 totaled $597.8 million, compared with cash, cash equivalents and investments of $318.8 million as of December 31, 2016. The increase is due to the net proceeds of $380.6 million from the Company’s September 12, 2017 public offering.

If the FDA approves andexanet in Q1 2018, the Company will receive an additional $100 million from its royalty-based financing with Health Care Royalty Partners.

Conference Call Details
Portola will host a conference call today, Monday, November 6, 2017, at 4:30 p.m. Eastern Time, during which management will provide updates on the U.S. launch of Bevyxxa, third quarter 2017 financial results and other matters. The live call can be accessed by phone by calling (844) 452-6828 from the United States and Canada or (765) 507-2588 internationally and using the passcode 7269839. The webcast can be accessed live on the Investor Relations section of the Company’s website at View Source It will be archived for 30 days following the call.

On November 7, 2017 MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY), a leader in the field of therapeutic antibodies, reported results for the third quarter of 2017 (Press release, MorphoSys, NOV 7, 2017, View Source [SID1234521632]).

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"Our proprietary development program MOR208 for aggressive lymphoma was recently awarded Breakthrough Therapy designation by the FDA based on encouraging clinical data from our L-MIND trial in relapsed/refractory DLBCL. We are now focused on working closely with the regulatory authorities to bring MOR208 to market as fast as possible. The launch in the United States of Janssen’s TremfyaTM (guselkumab) for moderate to severe plaque psoriasis was a major highlight of this quarter. Approval in a second major territory may be imminent based on the positive recommendation for guselkumab by the European regulatory authority. We expect guselkumab to become an important pillar of our revenue in the years ahead", commented Dr. Simon Moroney, Chief Executive Officer of MorphoSys AG.

"We will continue to drive our proprietary portfolio forward. The approval of TremfyaTM (guselkumab) marks our transition to a company whose revenues will be increasingly based on recurring income from product sales in the years to come. This will contribute to the funding of our proprietary development activities," stated Jens Holstein, Chief Financial Officer of MorphoSys AG.

Financial Review for the third quarter of 2017 (IFRS; all figures rounded)

MorphoSys continues to focus on applying its proprietary technology and expertise to the research and development of innovative drug candidates. In the third quarter of 2017, group revenues amounted to EUR 15.0 million, up 20% compared to Q3 2016 (EUR 12.5 million). The revenue increase was mainly driven by licence income as well as milestone payments from Janssen. Q3 2017 revenues do not include royalties on TremfyaTM (guselkumab) sales as the first royalty reporting from Janssen has not yet been received. Thus, royalties on TremfyaTM (guselkumab) sales for Q3 2017 since launch in late July 2017 will be part of Q4 2017 results.

In the Proprietary Development segment, MorphoSys focuses on research and clinical development of its own drug candidates in cancer and inflammation. In Q3 2017, this segment recorded revenues of EUR 0.2 million (Q3 2016: EUR 0.1 million).

In the Partnered Discovery segment, MorphoSys applies its proprietary technology to discover new antibodies for pharmaceutical companies, receiving R&D funding and licensing fees from its partners and benefiting from the partners’ development progress through success-based milestone payments and royalties. In Q3 2017, revenues from this segment reached EUR 14.8 million (Q3 2016: EUR 12.3 million). The increase was particularly based on license income as well as milestone payments from Janssen.

Earnings before interest and taxes (EBIT) in Q3 2017 amounted to EUR -23.5 million (Q3 2016: EUR -13.1 million). As anticipated, the Proprietary Development segment reported an EBIT of EUR -29.8 million in Q3 2017 (Q3 2016: EUR -17.7), while the Partnered Discovery segment achieved an EBIT of EUR 10.4 million (Q3 2016: EUR 7.7 million).

In Q3 2017, the consolidated net loss amounted to EUR 24.0 million (Q3 2016: consolidated net loss of EUR 12.8 million). The consolidated net loss per share for Q3 2017 was EUR 0.83 (Q3 2016: consolidated net loss per share of EUR 0.49).

At the end of Q3 2017, the Company had a cash position of EUR 319.5 million compared to EUR 359.5 million on December 31, 2016. On the balance sheet, this cash position is reported under the following items: cash and cash equivalents; available-for-sale financial assets; bonds, available-for-sale; and current and non-current financial assets classified as loans & receivables. The number of shares issued totaled 29,345,748 at the end of Q3 2017 (year-end 2016: 29,159,770).

Results for the first nine months 2017

During the first nine months of 2017, group revenues amounted to EUR 38.6 million, 5% higher than the previous year (Q1-Q3 2016: EUR 36.7 million). For the first nine months of 2017, revenues do not include royalties on TremfyaTM (guselkumab) sales as the first royalty reporting from Janssen has not yet been received. Thus, Q3 2017 royalties on TremfyaTM (guselkumab) sales since launch in late July 2017, will be reflected in the Company’s Q4 2017 revenues.

As planned, R&D expenses for proprietary drug development and technology development increased considerably to EUR 67.9 million in the first nine months of 2017 (Q1-Q3 2016: EUR 46.2 million). This increase is due to intensified activities in the clinical development of proprietary drug candidates that are transitioning into advanced development stages, requiring larger clinical studies. Consequently, the EBIT in the first nine months of 2017 amounted to EUR -53.8 million (Q1-Q3 2016: EUR -32.3 million).

Financial guidance confirmed

For the financial year 2017, MorphoSys continues to expect Group revenues in the range of EUR 46 to 51 million. R&D expenses for proprietary drug development and technology development are confirmed to be in a corridor of EUR 85 to 95 million. Guidance for earnings before interest and taxes (EBIT) continues to be in the range of EUR -75 to -85 million. This guidance does not include any additional revenue from potential future collaborations and/or licensing partnerships, nor effects from potential in-licensing or co-development deals for new development candidates. As first royalty reporting from Janssen has not been received yet, royalties on net sales for TremfyaTM (guselkumab) cannot be accurately projected at this point in time. Hence the guidance for the financial year 2017 does not include any assumptions on royalty income for sales on TremfyaTM (guselkumab).

Transition in the CSO position

Dr. Markus Enzelberger, who has been serving as Interim CSO since April 15, 2017, was appointed Chief Scientific Officer (CSO) effective November 1, 2017. He succeeds Dr. Marlies Sproll who resigned from her CSO position effective end of October 31, 2017 due to ongoing family reasons. Dr. Sproll has taken on a new part-time role at MorphoSys as Special Adviser to the CEO as of November 1, 2017.

Operational outlook for 2017

In the Proprietary Development segment, MorphoSys expects the following events in 2017:

– MOR208: Presentation of further data from more patients in the ongoing phase 2 trial of MOR208 in combination with lenalidomide in DLBCL (L-MIND study) at the ASH (Free ASH Whitepaper) 2017 conference in December.

– MOR202: Continuation of phase 1/2a dose-escalation trial in multiple myeloma, including MOR202 in the highest dosing cohorts in combinations with pomalidomide and with lenalidomide. The Company is currently pursuing opportunities for a deal with MOR202 with an external partner in order to facilitate its further clinical development.

– MOR103/GSK3196165: According to clinicaltrials.gov, several studies conducted by GSK may reach primary completion including a phase 2b and a phase 2a study in rheumatoid arthritis as well as a phase 2a study in hand osteoarthritis. MorphoSys does not control its partners’ mode of communication. This HuCAL antibody originated in MorphoSys’s Proprietary Development segment, and has been fully out-licensed to GSK.

In its Partnered Discovery segment, the following events are expected:

– TremfyaTM (guselkumab): after the positive opinion of European Medical Agency’s CHMP in September 2017 recommending EU approval for treatment of patients with moderate-to-severe plaque psoriasis, the Company expects a decision by the European Commission soon.

– Novartis collaboration: As previously communicated and as reflected in the Company’s 2017 guidance, the collaboration with Novartis will conclude at the end of November 2017 in accordance with the contract.

– For the remainder of the year, up to 19 different clinical studies in various phases conducted by partners with antibodies made using MorphoSys technology may reach primary completion according to clinicaltrials.gov. MorphoSys does not control its partners’ mode of communication.

As always, MorphoSys is in discussions with other companies in the pharmaceutical industry about technology and/or product-based collaborations, with the goal of strengthening its participation in drug programs aimed at unmet medical needs.