On November 9, 2017 Novelion Therapeutics Inc. (NASDAQ: NVLN), a biopharmaceutical company dedicated to developing and commercializing innovative new therapies for individuals living with rare diseases ("Novelion" or the "Company"), reported financial results for the third quarter ended September 30, 2017, announced that it has commenced a search for a new chief executive officer ("CEO") to lead the Company in the next stage of growth, and provided an overview of recent business highlights (Press release, QLT, NOV 9, 2017, View Source [SID1234521900]). Chief Executive Officer Mary Szela has resigned for personal reasons, effective immediately.

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To lead the company until a permanent CEO is appointed, the Board announced the creation of an interim Office of the Chief Executive Officer, comprised of Jeffrey Hackman, Novelion’s Chief Operating Officer, Jason Aryeh, Chairman of the Board of Directors and Mark Corrigan, Director. The Board has also formed a search committee, which has commenced efforts to identify candidates who will bring strong strategic and operational direction to the enterprise.

Jason Aryeh said, "Novelion’s strategy and mission will continue to focus on supporting access to our marketed therapies for patients globally and pursuing the development of our product portfolio in order to support long-term and sustainable growth. The board is confident in its ability to identify a successor who will effectively drive improved growth and shareholder value by reinvigorating the Company’s plans in support of our key objectives."

Mr. Aryeh continued, "We recognize the energy that Mary Szela brought to her role as CEO. Much has been accomplished in her tenure, including the merger to create Novelion, and we thank her for her dedication."

Third Quarter 2017 Highlights & Business Update

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JUXTAPID: Novelion reported net revenues of JUXTAPID of $15.2 million in the third quarter of 2017, $10.3 million, or 68%, of which were from prescriptions written in the U.S.

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MYALEPT: Novelion reported net revenues of MYALEPT of $13.5 million in the third quarter of 2017. $11.3 million, or 84%, of these revenues were from prescriptions written in the U.S.

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Novelion reported total net revenues of $28.7 million in the third quarter of 2017.

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The Company is continuing its efforts to stabilize JUXTAPID in the U.S., and continues to make progress with the launch in Japan.

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Novelion ended the third quarter of 2017 with $70.5 million in unrestricted cash, compared with $83.3 million at the end of the second quarter of 2017.

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Novelion’s marketing authorization application for metreleptin was accepted by the European Medicines Agency in January of 2017. In October 2017, the company submitted its responses to the Day 120 Questions, and based on current timelines, continues to anticipate EMA approval in the first half of 2018.

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Novelion appointed Jeffrey Hackman as Executive Vice President and Chief Operating Officer, effective November 1, 2017. Mr. Hackman will lead the company’s commercial efforts, business development, manufacturing and supply chain initiatives. Mr. Hackman was most recently senior vice president, head of the US Internal Medicine and Oncology franchises for Shire. In this role, Mr. Hackman was a member of the US commercial leadership team and was responsible for sales, marketing, business insights and analytics for eight licensed products within Shire’s US rare disease portfolio.

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Novelion appointed Murray Stewart, M.D. as Executive Vice President, Head of R&D, effective November 27, 2017. Dr. Stewart will lead the company’s clinical development activities, provide strategic regulatory guidance for the pipeline and commercial product initiatives, and maintain oversight of global medical affairs, publications and registry activities. Dr. Stewart will join Novelion from GlaxoSmithKline (GSK) where he is currently chief medical officer with global responsibility for patient well-being across the vaccines, pharmaceutical and consumer business units.

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Novelion appointed Suzanne Bruhn, Ph.D. to its board of directors, effective October 1, 2017. Dr. Bruhn is president and chief executive officer of Proclara Biosciences, Inc. Prior to joining Proclara, Dr. Bruhn served as president and chief executive officer of Promedior, Inc. She also served as a member of the board of directors of Raptor Pharmaceuticals from 2011 until it was acquired by Horizon Pharma in 2016. Previously, Dr. Bruhn served as senior vice president, strategic planning and program management at Shire from 1998 until 2012.

2017 Financial Guidance

The Company reiterated its previously stated net revenues financial guidance for full year 2017 and expects:

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Total net revenues between $135 million and $145 million;
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JUXTAPID net revenues between $70 million and $75 million; and
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MYALEPT net revenues between $65 million and $70 million.

Third Quarter 2017 Financial Results

On November 29, 2016, the Company completed its acquisition of Aegerion Pharmaceuticals, Inc. ("Aegerion"). The acquisition has been accounted for as a business combination in which Novelion was considered the acquirer of Aegerion. As such, under U.S. Generally Accepted Accounting Principles ("GAAP"), the financial statements of Novelion are treated as the historical financial

statements of the consolidated companies, with the results of Aegerion being included from November 29, 2016. This release also includes pro forma adjusted non-GAAP financial information showing pro forma results of operations of Novelion as if the acquisition had occurred on January 1, 2016. Reconciliation of the financial results on a GAAP versus non-GAAP basis are provided below the financial information that follows.

GAAP total net revenues for the third quarter of 2017 were $28.7 million compared to the prior year’s third quarter net revenues of $0. GAAP net revenues for JUXTAPID in the third quarter of 2017 were $15.2 million compared to $0 in the prior year. GAAP net revenues for MYALEPT in the third quarter of 2017 were $13.5 million compared to $0 for the same period in 2016.

GAAP total operating expenses for the third quarter of 2017 were $38.6 million compared to total operating expenses of $6.0 million for the same period in 2016. GAAP SG&A expenses were $21.4 million in the third quarter of 2017 compared to $3.2 million for the same period in 2016. GAAP R&D expenses were $17.1 million in the third quarter of 2017 compared to $2.9 million for the same period in 2016.

On a pro forma basis, during the third quarter of 2017, SG&A expenses were $20.2 million compared to $28.2 million for the same period in 2016. The decrease in pro forma SG&A expenses in the third quarter of 2017 compared with the same period in 2016 was primarily related to a reduction in headcount and legal and consulting fees.

On a pro forma basis, during the third quarter of 2017, R&D expenses were $16.9 million compared to $12.7 million for the same period in 2016. The decrease in R&D expenses in the third quarter of 2017 compared with the same period in 2016 was primarily related to a reduction in headcount.

GAAP net loss in the third quarter of 2017 was $49.7 million compared to GAAP net loss of $5.9 million during the same period in 2016.

On a pro forma basis, net loss in the third quarter of 2017 was $16.6 million, compared to $14.3 million for the same period in 2016.

As part of the Merger between QLT and Aegerion, the Company acquired inventory, a portion of which is classified as non-current based on its forecasted consumption exceeding one year. An excess and obsolescence analysis is run to determine the need to adjust inventory carrying values. In the third quarter, that analysis led to a write down of inventory of approximately $17.3 million.

First Nine Months of 2017 Financial Results

GAAP total net revenues for the first nine months of 2017 were $99.5 million compared to $0 for the same period of 2016. GAAP net revenues for JUXTAPID for the first nine months of 2017 were $51.9 million compared to $0 in same period in 2016. GAAP net revenues for MYALEPT for the first nine months of 2017 were $47.6 million compared to $0 for the same period in 2016.

GAAP total operating expenses for the first nine months of 2017 were $112.1 million compared to total operating expenses of $22.3 million for the same period in 2016. GAAP SG&A expenses were $72.4 million in the first nine months of 2017 compared to $13.6 million for the same period in 2016.

GAAP R&D expenses were $37.2 million in the first nine months of 2017 compared to $8.8 million for the same period in 2016.

On a pro forma basis, for the first nine months of 2017, SG&A expenses were $68.2 million compared to $111.5 million for the same period in 2016. For the first nine months of 2017, R&D expenses on a pro forma basis were $36.6 million compared to $37.9 million for the same period in 2016.

GAAP net loss for the first nine months of 2017 was $102.1 million compared to GAAP net loss of $33.0 million during the same period in 2016.

On a pro forma basis, net loss for the first nine months of 2017 was $26.7 million, compared to $111.7 million for the same period in 2016.

As of September 30, 2017, the Company’s consolidated unrestricted cash balance was $70.5 million, compared to $83.3 million at June 30, 2017 and $108.9 million at December 31, 2016. As of September 30, 2017, there were 18.6 million shares outstanding. At September 30, 2017, total debt was $325 million, reflecting the principal amount of convertible debt issued by Aegerion and consolidated as a result of the acquisition.

On November 9, 2017 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported financial results for the third quarter and nine months ended September 30, 2017 (Press release, Puma Biotechnology, NOV 9, 2017, View Source [SID1234521899]). Unless otherwise stated, all comparisons are for the third quarter and nine months of 2017 compared to the third quarter and nine months of 2016.

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On July 17, 2017, Puma Biotechnology received approval from the U.S. Food and Drug Administration (FDA) for NERLYNX (neratinib) for the treatment of early stage HER2-positive breast cancer following adjuvant trastuzumab-based therapy, and began shipments to wholesalers at the end of July 2017. In the third quarter of 2017, the Company reported net revenue from initial sales of NERLYNX of approximately $6.1 million.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss applicable to common stock of $77.2 million, or $2.07 per share, for the third quarter of 2017, compared to a net loss applicable to common stock of $65.8 million, or $2.02 per share, for the third quarter of 2016. Net loss applicable to common stock for the first nine months of 2017 was $227.9 million, or $6.15 per share, compared to $203.4 million, or $6.26 per share, for the first nine months of 2016.

Non-GAAP adjusted net loss was $50.7 million, or $1.36 per share, for the third quarter of 2017, compared to non-GAAP adjusted net loss of $36.0 million, or $1.11 per share, for the third quarter of 2016. Non-GAAP adjusted net loss for the nine months ended September 30, 2017 was $144.7 million, or $3.90 per share, compared to non-GAAP adjusted net loss of $115.4 million, or $3.55 per share, for the nine months ended September 30, 2016. Non-GAAP adjusted net loss excludes stock-based compensation expense, which represents a significant portion of overall expense and has no impact on the cash position of the Company. For a reconciliation of GAAP net loss to non-GAAP adjusted net loss and GAAP net loss per share to non-GAAP adjusted net loss per share, please see the financial tables at the end of this news release.

Net cash used in operating activities for the third quarter of 2017 was $54.9 million. Net cash used in operating activities for the nine months ended September 30, 2017 was $136.9 million. At September 30, 2017, Puma had cash and cash equivalents of $79.7 million and marketable securities of $26.6 million, compared to cash and cash equivalents of $194.5 million and marketable securities of $35.0 million at December 31, 2016.

Effective October 31, 2017, Puma entered into a credit facility with Silicon Valley Bank and Oxford Finance for a term loan of up to $100 million, subject to funding in two tranches. The Company received gross proceeds of $50 million from the first tranche of the credit facility upon closing on October 31, 2017 and intends to use the funds for general corporate purposes and to further support NERLYNX commercial initiatives. The second tranche of $50 million may be drawn at the Company’s option and is subject to the achievement of certain milestones. The loan will mature on October 31, 2022.

"With the U.S. approval and launch of NERLYNX in the third quarter, we began providing early stage HER2-positive breast cancer patients with an additional option to reduce their risk of disease recurrence," said Puma Chief Executive Officer and President Alan H. Auerbach. "We are pleased with the feedback that we have received from patients, prescribers and payors during the initial launch and we look forward to continuing to execute our commercial activities throughout 2017 and beyond.

"Looking forward, we anticipate the following milestones: (i) reporting additional data from the Phase II CONTROL trial in the fourth quarter of 2017; (ii) receiving a regulatory opinion from the Committee for Medicinal Products for Human Use (CHMP) for neratinib in extended adjuvant HER2-positive early stage breast cancer in the first quarter of 2018; and (iii) reporting Phase III trial results in third-line HER2- positive metastatic breast cancer patients in the first half of 2018."

Product Revenue

Net revenue consists of sales of NERLYNX, Puma’s first and only commercial product to date. The FDA approved NERLYNX in July 2017 and the Company commenced shipment to wholesalers in late July. For the three and nine months ended September 30, 2017, net revenue was approximately $6.1 million.

Operating Expenses

Operating expenses were $83.5 million for the third quarter of 2017, compared to $66.0 million for the third quarter of 2016. Operating expenses for the nine months ended September 30, 2017 were $234.9 million, compared to $203.7 million for the nine months ended September 30, 2016.

Cost of Sales:

Cost of sales was $1.5 million for the third quarter and nine months ended September 30, 2017. The Company had no product sales prior to the third quarter of 2017.

Selling, General and Administrative Expenses:

Selling, general and administrative (SG&A) expenses were $32.5 million for the third quarter of 2017, compared to $14.0 million for the third quarter of 2016. SG&A expenses for the nine months ended September 30, 2017 were $75.8 million, compared to $37.3 million for the nine months ended September 30, 2016. The $38.5 million increase during the first nine months of 2017, compared to the first nine months of 2016, resulted primarily from increases of approximately $24.2 million for professional fees and expenses, $8.2 million in payroll and related costs, $3.2 million for stock-based compensation, $2.4 million for other expenses such as travel and related costs to support the commercial launch of NERLYNX, and $0.4 million for facility and equipment costs. These increases reflect overall corporate growth.

Research and Development Expenses:

Research and development (R&D) expenses were $49.5 million for the third quarter of 2017, compared to $52.0 million for the third quarter of 2016. R&D expenses for the nine months ended September 30, 2017 were $157.6 million, compared to $166.4 million for the nine months ended September 30, 2016. The approximately $8.8 million decrease during the first nine months of 2017, compared to the first nine months of 2016, resulted primarily from decreases of approximately $5.0 million due to a decrease in regulatory submission activity, decreased preclinical study activities and decreased drug supply manufacturing logistics, and $8.0 million for stock-based compensation, partially offset by increases during the first nine months of 2017, compared to the first nine months of 2016, of approximately $2.2 million for internal clinical development, internal regulatory affairs and quality assurance and internal chemical manufacturing, and $2.0 million for consultants and contractors.

Conference Call

Puma Biotechnology will host a conference call to report its third quarter financial results and provide an update on the company’s business and outlook at 1:30 p.m. PST/4:30 p.m. EST on Thursday, November 9, 2017. The call may be accessed by dialing 1-877-709-8150 (domestic) or 1-201-689-8354 (international) at least 10 minutes prior to the start of the call and referencing the "Puma Biotechnology Conference Call." A live webcast of the conference call and presentation slides may be accessed on the Investors section of the Puma Biotechnology website at View Source A replay of the call will be available approximately one hour after completion of the call and will be archived on the company’s website for 90 days.

On November 9, 2017 Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) ("Prometic") reported that it will report its financial results for the third quarter ended September 30, 2017 on Monday November 13, 2017 after market close (Press release, ProMetic Life Sciences, NOV 9, 2017, View Source [SID1234521898]).

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Prometic will host a conference call at 11:00am (ET) on Tuesday November 14, 2017. The telephone numbers to access the conference call are 1-888-231-8191 and 647-427-7450. A live audio webcast of the conference call will be available via: View Source;s=1&k=99570B881A8129DE3DCA8DE3EBD93544

An audio replay of the call will be available as of Tuesday November 14, 2017 at 2:00pm (ET). The numbers to access the audio replay are 416-849-0833 and 1-855-859-2056 using the following password (3866799).

On November 9, 2017 Pfenex Inc. (NYSE American: PFNX) a clinical-stage biotechnology company developing complex therapeutic proteins, using the patented Pfenex Expression Technology platform, reported that it has created an advanced pipeline of therapeutic equivalents, vaccines, biologics and biosimilars (Press release, Pfenex, NOV 9, 2017, View Source2017-11-09-Pfenex-Reports-Third-Quarter-2017-Results-Interim-PK-data-from-PF708-Study-Completion-of-Process-Development-Milestone-on-Jazz-Program-and-Provides-Business-Update" target="_blank" title="View Source2017-11-09-Pfenex-Reports-Third-Quarter-2017-Results-Interim-PK-data-from-PF708-Study-Completion-of-Process-Development-Milestone-on-Jazz-Program-and-Provides-Business-Update" rel="nofollow">View Source [SID1234521895]). The company reported financial results for the third quarter ended September 30, 2017 and provided a business update.

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Business Review and Update

Today, Pfenex is pleased to announce the interim pharmacokinetic (PK) results from ongoing Study PF708-301, which compares the effects of PF708 and Forteo in osteoporosis patients. PF708 is a teriparatide product candidate that is being developed through the 505(b)(2) regulatory pathway in the US and references Forteo, marketed by Eli Lilly for the treatment of osteoporosis patients at a high risk of fracture, as the Reference Listed Drug. Study PF708-301 completed enrollment in the third quarter of 2017 with a total of 181 patients – 90 randomized to PF708 and 91 to Forteo. The primary study endpoint is anti-drug antibody formation after 24 weeks of drug treatment. The secondary study endpoints are changes in bone mineral density and bone turnover markers after 24 weeks of drug treatment, as well as PK parameters for up to 4 hours after the first dose.

The interim PK data from the study are shown in Figure 1. The PF708 and Forteo PK profiles are comparable, and there are no statistically significant differences in key PK parameters. The study is on-track for completion and top-line immunogenicity data readout in the first half of 2018. Pfenex believes that results from Study PF708-301, along with the previously-announced bioequivalence findings from Study PF708-101 in healthy subjects, should satisfy the clinical filing requirements for PF708 and expects to submit the new drug application (NDA) to the FDA in the third quarter of 2018. Pfenex believes it has sufficient cash resources to fund all necessary activities leading up to and including the submission of the NDA to the FDA.

Pfenex has completed a strategic review of PF582 and PF529, biosimilar product candidates to Lucentis and Neulasta, respectively. The strategic review considered timeline for development and cost. While the market opportunities remain attractive, Pfenex has decided to pause development activities on PF582 and PF529 and focus development efforts elsewhere within the product portfolio until strategic partnerships for these candidates are forged. The company continues to engage with potential strategic partners to monetize the assets.

Px563L and RPA563, novel anthrax vaccine candidates, are 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. The development of our anthrax candidates 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. Recently, Pfenex completed a meeting with the FDA, in which the Agency provided clear guidance for the proposed clinical development and licensure plans for post-exposure prophylaxis indication. Ahead of the phase 2 study initiation, Pfenex expects to continue to demonstrate stability of the vaccine candidates and complete manufacturing of the clinical supply. Pfenex expects to initiate the phase 2 study by year-end 2018.

Pfenex is pleased to announce that during the third quarter, it completed a process development milestone associated with its collaboration with Jazz Pharmaceuticals. In July 2016, Pfenex and Jazz announced an agreement under which Pfenex granted Jazz worldwide rights to develop and commercialize multiple early stage hematology product candidates. Under the agreement, Pfenex received upfront and option payments totaling $15 million and may be eligible to receive additional payments of up to $166 million based on the achievement of certain development-, regulatory-, and sales-related milestones.

Figure 1. Study PF708-301: Interim PK Results

Financial Highlights for the Third Quarter 2017

Total Revenue decreased to $5.0 million in the three-month period ended September 30, 2017 compared to $48.8 million in same period in 2016. This decrease in revenue was due to the termination of our development and license agreement with Pfizer in the third quarter of 2016 which resulted in accelerated recognition of $45.8 million of revenue. This decrease was partially offset by a $2.0 million increase in revenue comprised of revenue recognized in connection with meeting a process development milestone under our collaboration agreement with Jazz and increased government contract revenue for our Px563L/RPA563 product candidate, as greater activity occurred from two options exercised in January 2017 under our existing contract with the U.S. government allowing for continued development. Given the nature of the development process, revenue will fluctuate depending on stage of development.

Cost of revenue increased to $1.8 million in the three-month period ended September 30, 2017 compared to $1.3 million in same period in 2016. This increase in cost of revenue was mainly due to an increase in costs for our Px563L/RPA563 product candidate under our government contracts related to the two options exercised in January 2017. Given the nature of the development process, these costs will fluctuate depending on stage of development.

Research and development expenses decreased to $8.1 million in the three-month period ended September 30, 2017 compared to $8.7 million in same period in 2016. This decrease in research and development expenses was primarily due to the stage of development of our pipeline programs. Expenses decreased for materials and subcontractors for PF529 and PF530, which were partially offset due to clinical trial expenses for PF708.

Selling, general and administration expenses decreased to $4.0 million in the three-month period ended September 30, 2017 compared to $4.4 million in same period in 2016.

Cash and cash equivalents as of September 30, 2017 was $48.4 million.

Cautionary Note Regarding Forward-Looking Statement

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements generally relate to future events or Pfenex’s future financial or operating performance. In some cases, you can identify forward-looking statements because they contain words such as "may," "will," "should," "expects," "plans," "anticipates," "could," "intends," "target," "projects," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these words or other similar terms or expressions that concern Pfenex’s expectations, strategy, plans or intentions. Forward-looking statements in this press release include, but are not limited to, statements regarding the future potential of Pfenex’s product candidates, including future plans to advance, develop, manufacture and commercialize its product candidates; Pfenex’s expectation to receive data from the PF708 clinical program in the first half of 2018 and its expectation to submit an NDA in the third quarter of 2018; Pfenex’s expectations with respect to the sufficiency of its cash resources; Pfenex’s expectations regarding the timing of the release of additional clinical trial data for its product candidates; Pfenex’s expectations regarding the timing and advancement of clinical trials and the types of future clinical trials for its product candidates, including PF708 and Px563L/RPA563; Pfenex’s expectations regarding the expected regulatory pathways for its product candidates, including the development of PF708 pursuant to the 505(b)(2) regulatory pathway; Pfenex’s expectations regarding the sufficiency of its clinical trials to satisfy regulatory requirements; Pfenex’s expectation for potential partnership opportunities for its product candidates; Pfenex’s expectation that it will continue to demonstrate stability of Px563L/RPA563 and complete manufacturing of the clinical supply; Pfenex’s expectation that it will initiate a phase 2 study for Px563L/RPA563 by year-end 2018; and Pfenex’s expectations with respect to the timing of future regulatory filings for its product candidates. Pfenex’s expectations and beliefs regarding these matters may not materialize, and actual results in future periods are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Actual results may differ materially from those indicated by these forward-looking statements as a result of the uncertainties inherent in the clinical drug development process, including, without limitation, Pfenex’s ability to successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for its product candidates, which may not support further development of product candidates or may require Pfenex to conduct additional clinical trials or modify ongoing clinical trials or regulatory pathways; challenges related to commencement, patient enrollment, completion, and analysis of clinical trials; difficulties in achieving and demonstrating biosimilarity in formulations; Pfenex’s ability to manage operating expenses; Pfenex’s ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives; Pfenex’s dependence on third parties for development, manufacture, marketing, sales and distribution of products; unexpected expenditures; and difficulties in obtaining and maintaining intellectual property protection for its product candidates. Information on these and additional risks, uncertainties, and other information affecting Pfenex’s business and operating results is contained in Pfenex’s Annual Report on Form 10-K for the period ended December 31, 2016 and in its other filings with the Securities and Exchange Commission. Additional information will also be set forth in Pfenex’s Quarterly Report on Form 10-Q for the period ended September 30, 2017 to be filed with the Securities and Exchange Commission. The forward-looking statements in this press release are based on information available to Pfenex as of the date hereof, and Pfenex disclaims any obligation to update any forward-looking statements, except as required by law.

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.

On November 9, 2017 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3 stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS), reported a corporate update and financial results for the third quarter ended September 30, 2017 (Press release, Onconova, NOV 9, 2017, View Source [SID1234521892]).

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"We are getting closer to two key milestones for the INSPIRE pivotal trial for IV rigosertib in patients with higher-risk MDS. A pre-planned interim analysis is anticipated in the coming months, and encouraging recent enrollment progress for this global trial suggests full enrollment may be achieved in the first half of 2018," said Dr. Ramesh Kumar, President and Chief Executive Officer.

"We have also made good progress on our oral rigosertib-azacitidine combination program and plan to initiate a Special Protocol Assessment process with the Food and Drug Administration for a pivotal Phase 3 trial early next year. In addition, we are advancing our collaborative pre-clinical RASopathies drug program and look forward to two poster presentations at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) conference."

INSPIRE Trial of IV Rigosertib in 2nd Line Higher-risk (HR) MDS

Interim Analysis (IA)

· In the quarter, the Company received guidance from the Food and Drug Administration (FDA) and European Medicines Agency (EMA) concerning the statistical analysis plan (SAP) for both the interim and final (top-line) analysis in the INSPIRE trial. Based on guidance received, Onconova finalized the SAP in preparation for the IA.
· The IA will be triggered with the 88th death event in this trial of 225 patients. Based on internal modeling, this could occur in the fourth quarter of 2017 or early 2018. The date of the IA is tied to reaching a pre-identified number of death events. Accordingly, the precise time of completing the IA, which will take place approximately a couple of weeks after reaching the number of events, cannot be accurately forecast.
· This adaptive trial design permits several options after the IA, including a futility analysis, trial expansion using pre-planned statistical criteria, or choosing one of two endpoints (survival analysis of the Intent-to-Treat population or the pre-defined Very High Risk subpopulation).

Trial Progress

· As of October 31, 2017, the INSPIRE study is active at approximately 170 sites in 22 countries across four continents. A final five sites will be opened, which is expected to occur in November.
· The INSPIRE trial has stringent selection criteria so as to identify a more homogenous MDS patient population. Accordingly, extensive eligibility verification and trial site education are integral to the Company’s plan.
· Due to efforts undertaken to increase participation, including the addition and replacement of CROs and opening sites in additional countries, the enrollment rate for the trial increased

recently. Consequently, Onconova expects full enrollment to be achieved in the first half of 2018, followed by top-line analysis after 176 death events in the second-half of 2018.

Oral Rigosertib in Combination with Azacitidine for 1st-line HR-MDS

Pivotal Phase 3 Trial Protocol

· Following input received from the FDA in an end-of-phase 2 meeting and from the EMA as part of the scientific advice process, Onconova has designed a Phase 3 protocol. The Company is awaiting the results of the ongoing Phase 1/2 Expansion Trial before engaging in further protocol development. Once the Expansion Trial is complete, which is expected to be in the fourth quarter of 2017, Onconova plans to submit the Pivotal Phase 3 protocol to the FDA in the first half of 2018 to initiate the SPA process with the FDA.
· Initiation of the Phase 3 trial, which is planned to be conducted globally, requires additional financing and/or business development transactions.
· This Expansion Trial is designed to enroll up to approximately 40 patients. More than half of the trial has been accrued in multiple sites in the USA. Based on this progress, the Company has decided to limit the trial to US sites.
· Onconova plans to present initial data from this study at a scientific conference in early 2018, highlighting the results of dose selection and optimization of the combination regimen.

Other Programs for Future Development or Partnership and Presentations

Rigosertib for Pediatric RASopathies

· On October 11, 2017, Onconova hosted a Key Opinion Leader meeting to discuss novel approaches to RASopathies. The meeting featured presentations by Bruce D. Gelb, M.D. (Mount Sinai, New York), and Elliot Stieglitz, M.D. (University of California San Francisco), who discussed new developments for pediatric patients with RASopathies, which are related genetic syndromes usually caused by mutations that alter the Ras subfamily and mitogen activated protein (MAP) kinases that control signal transduction. Onconova’s Chief Medical Officer, Steven Fruchtman, M.D., provided an update on rigosertib, which is initially planned to be studied in pediatric patients with RASopathies complicated by the development of associated cancers.
· The Company has completed and expects to sign a Cooperative Research and Development Agreement with the US National Institutes of Health (NIH) to advance rigosertib in pediatric clinical trials at the National Cancer Institute. This trial is expected to start next year and will be funded by the NIH.

Onconova Enters into Strategic Collaboration with Cellectar Biosciences

· On September 21, 2017, the Company announced it had entered into a strategic collaboration with Cellectar Biosciences to develop new phospholipid drug conjugates combining select proprietary compounds or payloads from Onconova’s early stage product pipeline with Cellectar’ s patented phospholipid ether delivery platform.
· Under the terms of the collaboration, Onconova will provide Cellectar with several compounds, including some from the family of molecules that contains Briciclib, which is an EIF4E targeting small molecule with early Phase 1 data. Cellectar will link the molecules to its phospholipid ether to create new, more precisely targeted antitumor agents.

Upcoming Presentations

Two abstracts relating to the Company’s lead product candidate, rigosertib, were accepted for poster presentation at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Atlanta, Georgia, which takes place December 9-12, 2017. Details of the presentations are listed below.

Long-term follow up of patients in a Phase 2 clinical trial of single agent oral rigosertib in lower-risk transfusion dependent MDS

Abstract Number: 1689

Title: Rigosertib Oral in Transfusion Dependent Lower Risk Myelodysplastic Syndromes (LR-MDS): Optimization of Dose and Rate of Transfusion Independence (TI) or Transfusion Reduction (TR) in a Single-Arm Phase 2 Study

Session Name: 637. Myelodysplastic Syndromes – Clinical Studies: Poster I

Date: Saturday, December 9, 2017; 5:30 – 7:30 PM EST

Studies on the mechanism of action of rigosertib azacitidine combination therapy for MDS

Abstract Number: 4235

Title: Effects of Rigosertib (RIGO) Alone or in Combination with Azacitidine or Vorinostat on Epigenetic Reprogramming of CD34+ Cells in the Myelodysplastic Syndrome

Session Name: 636. Myelodysplastic Syndromes – Basic and Translational Studies: Poster III

Date: Monday, December 11, 2017; 6:00 – 8:00 PM EST

Third-Quarter Financial Results:

· Cash, cash equivalents, and marketable securities as of September 30, 2017 totaled $7.6 million, compared to $21.4 million as of December 31, 2016. Based on our cash burn for the first three quarters of 2017 and our current projections, we expect that our cash and cash equivalents will be sufficient to fund our ongoing trials and operations through the end of 2017.

· Total net revenue was $0.1 million for the third quarter of 2017 and $0.6 million for the nine months ended September 30, 2017, compared to $1.7 million and $5.4 million, respectively, for the comparable periods in 2016.

· Research and development expenses were $5.1 million for the third quarter of 2017 and $14.6 million for the nine months ended September 30, 2017, compared to $4.0 million and $15.4 million, respectively, for the comparable periods in 2016.

· General and administrative expenses were $1.7 million for the third quarter of 2017 and $5.6 million for the nine months ended September 30, 2017, compared to $2.0 million and $7.2 million, respectively, for the comparable periods in 2016.

The Company will host a conference call on November 9th at 9:00 a.m. Eastern Time to provide a corporate update and discuss third quarter financial results. Interested parties may access the call by dialing toll-free (855) 428-5741 from the US, or (210) 229-8823 internationally and using conference ID: 3588306.

The call will also be webcast live. Please click here to access the webcast.

A replay will be available at this link until February 23, 2018.