On March 8, 2018 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), today provided a corporate update and reported financial results for the full year ended December 31, 2017 (Press release, Onconova, MAR 8, 2018, View Source [SID1234524544]).

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"The recently completed year was a pivotal period for rigosertib development programs in MDS. We achieved important goals across our full pipeline, highlighted by the recently announced promising interim analysis and the advancement of the INSPIRE pivotal trial for rigosertib, our lead Phase 3 clinical candidate. With no FDA approved therapies available for patients with higher-risk MDS who are refractory to hypomethylating agents, Onconova has taken a leadership position in this indication. Looking ahead, we now expect topline analysis to be concurrent with enrollment completion, which can be achieved in the first half of 2019," said Dr. Ramesh Kumar, President and Chief Executive Officer.

"We also announced three important collaborations in recent months. Regional licensing of our pre-IND stage next generation CDK 4/6 inhibitor for Greater China which we believe advnaces this program on the IND track and towards clinical data in 2019. Our collaboration with the National Cancer Institute for clinical development of rigosertib in children suffering from incurable inherited diseases (RASopathies) could provide Onconova with the opportunity to establish a rare disease development program. Finally, our licensing agreement for rigosertib in Latin America further expands the global commercial footprint of rigosertib, and is in addition to our existing partnership in Japan and Korea. Execution of these transactions we believe indicates the ability to leverage our late stage and pipeline assets to finance multiple programs. Based on the progress achieved in our oral rigosertib-azacitidine combination Phase 2 program in front-line MDS indications, we expect to secure additional collaborations and regional partnerships to help support a pivotal Phase 3 trial for oral rigosertib."

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

Interim Analysis (IA)

On January 17, 2018, Onconova announced that it is moving forward with its Phase 3 INSPIRE pivotal trial following the interim analysis and the Data Monitoring Committee’s (DMC) recommendation, together with unanimous approval by the Executive Committee overseeing this trial. The DMC recommended continuation of the trial with a one-time expansion in enrollment, using a pre-planned sample size re-estimation, consistent with the Statistical Analysis Plan.
The expanded INSPIRE study will increase enrollment by adding 135 patients to the original target to reach a total enrollment of 360 patients.
At the topline analysis of the INSPIRE trial, the primary endpoint of overall survival will be analyzed in both the ITT population and the Very High Risk (VHR) subgroup.
In the INSPIRE trial enrollment so far, the predefined subgroup of VHR patients constitutes greater than 70% of patients enrolled to date.
The Company remains blinded to the interim analysis results.
Trial Progress

The INSPIRE study is open in more than 170 sites in 22 countries across four continents.
More than half of the expanded study is now enrolled.
The Company is planning to add sites in Europe and new territories, including in Latin America, in concert with our new partner Pint Pharma ("Pint").
The INSPIRE trial was designed with 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.
Oral Rigosertib in Combination with Azacitidine for 1st-line HR-MDS

Pivotal Phase 3 Trial Protocol

Phase 2 Expansion Trial is expected be fully enrolled this month with the addition of more than 40 patients.
Onconova plans to present initial data from this study at a scientific conference in 2018, highlighting the results of dose selection and optimization of the combination regimen.
On March 2, 2018, Onconova presented data relating to the mechanism of action of rigosertib in combination with azacitidine at the AACR (Free AACR Whitepaper) Special Conference. The results suggested potential novel clinical strategies to improve outcomes for patients with higher-risk MDS and reversal of resistance to treatment with epigenetic therapies.
Progress in Business Development around Rigosertib and Pipeline Products

Onconova and Pint Pharmaceutical Announce Licensing Agreement for Rigosertib in Latin America

On March 5, 2018, Onconova and Pint announced that they had entered into a Latin American licensing agreement for rigosertib. Pint is a private, European-based pharmaceutical company focused on the development, registration and commercialization of specialty-based treatments for the Latin American market.
Under the terms of the agreement, Pint will make an investment in Onconova totaling up to $2.5 million by purchasing shares at a premium to market. In addition, Pint will make potential additional regulatory, development and sales-based milestone payments to Onconova of up to $42.75 million and pay double digit tiered royalties on net sales in Latin America.
Rigosertib Collaboration for Pediatric RASopathies

On January 4, 2018, Onconova announced that it had entered into a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), part of the National Institutes of Health. Under the terms of the CRADA, the NCI will conduct research, including preclinical laboratory studies and a clinical trial, on rigosertib in pediatric cancer associated RASopathies. The RASopathies are a group of rare diseases which share a well-defined molecular basis in expression or defects involving Ras Effector Pathways.
License and Collaborative Development Agreement with HanX Biopharmaceuticals for ON 123300

On December 19, 2017, Onconova announced the signing of a license and collaboration agreement with HanX Biopharmaceuticals, Inc., a company focused on development of novel oncology products, for the further development, registration and commercialization of ON 123300 in China. ON 123300 is a first-in-class dual inhibitor of CDK4/6 + ARK5, which is currently in advanced pre-clinical development. This compound has the potential to overcome the limitations of current generation CDK 4/6 inhibitors.
Under the terms of the agreement, Onconova will receive an upfront payment, and is eligible to receive potential regulatory and commercial milestone payments, as well as royalties on Chinese sales. HanX will provide all funding required for Chinese IND enabling studies performed for Chinese Food and Drug Administration IND approval. The Companies also intend for these studies to comply with US Food and Drug Administration (FDA) standards. Accordingly, such studies may be used by Onconova for an IND filing with the FDA. Onconova will maintain global rights outside of China.
Pre-clinical Stage CDK4/6 + ARK5 Inhibitor Program

Following signing of the collaboration agreement with HanX, Onconova initiated a pre-IND process with the U.S. Food and Drug Administration (FDA).

Presentations of Data

Rigosertib in MDS at the ASH (Free ASH Whitepaper) 2017 Meeting

Onconova delivered two poster presentations highlighting drug activity and the mechanism of action of rigosertib in MDS during the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Atlanta in December, 2017.
Among the highlights of the presentation were: Oral rigosertib as a single agent demonstrated activity in a Phase 2 trial for lower-risk MDS; 32% of 62 evaluable patients, and 44% of patients receiving optimal dosing, achieved transfusion independence; and new data on the molecular basis of the combination therapy with rigosertib and azacitidine in epigenetic studies in patient derived stem cells.
Full Year 2017 Financial Results:

Cash and cash equivalents as of December 31, 2017, totaled $4.0 million, compared to $21.4 million as of December 31, 2016. Subsequently, on February 12, 2018, Onconova announced the closing of a $10 million underwritten public offering of 9,947,500 shares of common stock or common stock equivalents and warrants to purchase an aggregate of 994,750 shares of Onconova’s Series A convertible preferred stock, including the exercise in full of the underwriter’s option to purchase additional securities, at the public offering price of $1.01 per share and accompanying Preferred Stock Warrant. Onconova also issued to the underwriter a preferred stock warrant to purchase 49,737.5 shares of Series A convertible preferred stock. Based on the Company’s cash burn for 2017 and its current projections, Onconova expects that cash and cash equivalents will be sufficient to fund ongoing trials and operations into the third quarter of 2018.
Net loss was $24.1 million for the year ended December 31, 2017, compared to $19.7 million for the year ended December 31, 2016, primarily due to the lack of collaboration cost sharing revenue in the 2017 period and a smaller change in fair value of warrant liability in the 2017 period.
Research and development expenses were $19.1 million for the year ended December 31, 2017, and $20.1 million for the comparable period in 2016.
General and administrative expenses were $7.4 million for the year ended December 31, 2017, and $9.2 million for comparable period in 2016.
The Company will host a conference call on March 8th at 9:00 a.m. Eastern Time to provide a corporate update and discuss fourth quarter and full-year 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: 2947108.

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

A replay will be available at this link until June 29, 2018

On March 8, 2018 PDL BioPharma, Inc. (PDL or the Company) (NASDAQ: PDLI) reported financial results for the fourth quarter and year ended December 31, 2017 including (Press release, PDL BioPharma, MAR 8, 2018, View Source [SID1234524568]):

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Total revenues of $68.0 million and $320.1 million for the three and twelve months ended December 31, 2017, respectively.
GAAP diluted EPS of $0.15 and $0.71 for the three and twelve months ended December 31, 2017, respectively.
GAAP net income attributable to PDL’s shareholders of $22.3 million and $110.7 million for the three and twelve months ended December 31, 2017, respectively.
Non-GAAP net income attributable to PDL’s shareholders of $24.8 million and $100.7 million for the three and twelve months ended December 31, 2017. A full reconciliation of all components of the GAAP to non-GAAP financial results can be found in Table 3 at the end of the release.
"2017 was a great year for us and one where we experienced a 31 percent increase in revenue from the previous year," stated John P. McLaughlin, chief executive officer of PDL. "Since 2012, we have built a rich portfolio of income generating assets and products to replace revenues from our expired Queen et al patents. We expect the revenues from these assets, whose net book value is $5.54 per share, to fuel the building of our specialty pharma business. It’s important to note that $264 million, or 83 percent of our 2017 revenues, came from sources other than the Queen et al patents. In 2018, we need to continue to execute successfully on our business model as well as close the gap between our share price and our book value per share."

Revenue Highlights

Total revenues of $68.0 million for the three months ended December 31, 2017 included:
Royalties from PDL’s licensees to the Queen et al. patents of $4.5 million, which consisted of royalties earned on sales of Tysabri;
Net royalty payments from acquired royalty rights and a change in fair value of the royalty rights assets of $30.1 million, which consisted of the change in estimated fair value of our royalty right assets, primarily related to Depomed, Inc. (Depomed) royalty asset;
Interest revenue from note receivable investment to CareView Communications of $0.8 million; and
Product revenues of $32.6 million, which consisted of $25.1 million from sales of Tekturna and Tekturna HCT in the United States, Rasilez and Rasilez HCT in the rest of the world (collectively, the Noden Products) and $7.5 million for product sales of the LENSAR Laser System.
Total revenues increased by 2 percent for the three months ended December 31, 2017, when compared to the same period in 2016.
Royalties from PDL’s licensees to the Queen et al. patents were lower due to reduced sales of Tysabri that was manufactured prior to the patent expiry date;
PDL received $32.8 million in net cash royalties from its royalty rights in the fourth quarter of 2017, compared to $25.3 million for the same period of 2016. The increase in cash royalties is mainly due to a one-time settlement payment from Valeant related to the royalty audit of Glumetza and the launch of the authorized generic for Glumetza sold by Valeant Pharmaceuticals International, Inc. PDL received royalties on the authorized generic equivalents under the same terms as the branded Glumetza;
The decrease in interest revenues was primarily due to the sale of the kaléo, Inc. note receivable in September 2017; and
The increase in product revenues were derived from the sale of the LENSAR Laser System, which PDL did not begin to recognize until May 2017.
Total revenues increased by 31 percent for the year ended December 31, 2017, when compared to the year ended December 31, 2016.
The decrease in royalties from PDL’s licensees to the Queen et al. patents is due to the expiration of the patent license agreement with Genentech, Inc. and reduced royalties on Tysabri.
The increase in royalty rights – change in fair value was primarily due to the year-to-date increase in fair value of the Depomed royalty asset by $134.1 million.
PDL received $107.3 million in net cash royalties, including a one-time settlement payment from Valeant related to the royalty audit of Glumetza, from its royalty rights in the year ended December 31, 2017, compared to $72.6 million for the same period of 2016.
The decrease in interest revenues was primarily due to the early repayment of the Paradigm Spine, LLC note receivable and the sale of the kaléo, Inc. note receivable.
Product revenue increased due to sales of the Noden Products, which PDL did not begin to recognize until the third quarter of 2016, and sales of the LENSAR Laser System, which PDL did not begin to recognize until May 2017.
License and other revenue increased by $19.6 million primarily due to a one-time $19.5 million payment from Merck as part of the previously announced settlement agreement to resolve the patent infringement lawsuit related to Keytruda.
Operating Expense Highlights

Operating expenses were $38.2 million for the three months ended December 31, 2017, compared to $74.2 million for the same period of 2016. The decrease in operating expenses for the three months ended December 31, 2017, as compared to the same period in 2016, was primarily a result of the prior year period loss on extinguishment of Direct Flow Medical notes receivable, partially offset by the increase in operating expenses related to the acquisitions and operations of Noden and LENSAR, contributing an additional $13.8 million of cost of product revenue and $6.0 million in sales and marketing expenses due to an increase in Noden’s sales force.
Operating expenses were $126.3 million for the year ended December 31, 2017, compared to $114.9 million for the year ended December 31, 2016. The increase in operating expenses in 2017 was a result of the acquisitions and operations of Noden and LENSAR, contributing an additional $26.5 million of cost of product revenue, $12.7 million of intangible asset amortizations, $17.1 million in sales and marketing expenses, and $3.6 million in research and development costs for the completion of a pediatric trial for Tekturna. General administrative expenses increased by $5.9 million of which $7.5 million was related to Noden and $3.2 million was related to LENSAR, partially offset by a decrease of $51.1 million from the loss on extinguishment for the Direct Flow Medical notes receivable in 2016.
Recent Developments

On February 1, 2018, PDL completed the retirement of the remaining $126.4 million of aggregate principal of its 4.0% Convertible Senior Notes due 2018 at their stated maturity by making a payment to the noteholders of $126.4 million, plus $2.6 million of accrued interest.
In February 2018, we entered into a modification agreement with CareView whereby we agreed, effective as of December 28, 2017, to modify the credit agreement before remedies could otherwise have become available to us under the credit agreement in relation to certain obligations of CareView that would potentially not be met, including the requirement to make principal payments. Under the modification agreement we agreed that (i) a lower liquidity covenant would be applicable and (ii) principal repayment would be delayed for a period of up to December 31, 2018. In exchange for agreeing to these modifications, among other things, the exercise price of our warrants to purchase 4.4 million shares of common stock of CareView was reduced and, subject to the occurrence of certain events, CareView agreed to grant us additional equity interests.
Other Financial Highlights

PDL had cash, cash equivalents, short-term investments and other investments of $532.1 million at December 31, 2017, compared to $242.1 million at December 31, 2016.
Conference Call and Webcast Details

PDL will hold a conference call to discuss financial results at 4:30 p.m. Eastern Time today, March 8, 2018.

To access the live conference call via phone, please dial (800) 668-4132 from the United States and Canada or (224) 357-2196 internationally. The conference ID is 9384627. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available beginning approximately one hour after the call through one week following the call, and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 9384627.

To access the live and subsequently archived webcast of the conference call, go to the Company’s website at View Source and go to the Investor Relations section and select "Events & Presentations." Please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

On March 8, 2018 VBL Therapeutics (Nasdaq:VBLT), reported top-line results from its pivotal Phase 3 GLOBE study in patients with recurrent glioblastoma (rGBM) which was designed to evaluate VB-111 in combination with bevacizumab (Avastin), compared to the bevacizumab control arm (Press release, VBL Therapeutics, MAR 8, 2018, View Source [SID1234525483]). The study did not meet its pre-specified primary endpoint of overall survival (OS).

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Dror Harats, M.D., Chief Executive Officer of VBL Therapeutics, said "We are disappointed that our encouraging Phase 2 data were not replicated in the GLOBE Phase 3 study, and once we receive the full and final data we will be analyzing them carefully to better understand the outcome of the study. We are grateful to the trial investigators, site personnel, patients and caregivers who participated in GLOBE. We believe that VB-111 may still hold promise for other indications we currently or may study in the future."

Conference Call
Thursday, March 8 at 8:30am Eastern Time
US Investors: 800-239-9838
International Investors: 323-794-2551
Conference ID: 3511679
Webcast: View Source

About the GLOBE study
The GLOBE pivotal Phase 3 trial is a randomized, controlled, double-arm, open-label study of VB-111 dosed every two months in combination with bevacizumab dosed every two weeks, compared to bevacizumab monotherapy. Key inclusion criteria include first or second progression of glioblastoma following standard of care treatment with temozolomide and radiation, a histologically confirmed diagnosis of glioblastoma and measurable disease by RANO criteria at progression.

The study is conducted under a Special Protocol Assessment (SPA) granted by the FDA, with full endorsement by the Canadian Brain Tumor Consortium (CBTC). VB-111 has received orphan drug designation in the United States and Europe and was granted Fast Track designation by the FDA for promising and meaningful long-term survival in patients with glioblastoma that has recurred following treatment with standard chemotherapy and radiation.

About Glioblastoma (GBM)
GBM is the most common and most aggressive form of primary brain tumors. In 2017, it is estimated there were approximately 12,000-13,000 new cases diagnosed in the United States. Median OS from diagnosis averages 12 to 15 months with patients treated usually with surgery, chemotherapy and radiation. Progression occurs within approximately 6 months in virtually all patients, and upon progression median OS is about 6-8 months. Although significant research and clinical efforts have focused on improving treatments for recurrent GBM, no systemic therapy has shown an OS benefit, resulting in a significant unmet medical need.

About VB-111 (ofranergene obadenovec)
VB-111, a potential first-in-class anticancer therapeutic candidate, is the Company’s lead oncology product currently being studied in a Phase 3 trial for ovarian cancer. VB-111 has demonstrated statistically significant OS and PFS in a Phase 2 trial in patients with rGBM, versus current standard of care. VB-111 has received orphan drug designation in both the US and Europe, and fast track designation in the US for prolongation of survival in patients with rGBM. In addition, VB-111 successfully demonstrated proof-of-concept and survival benefit in Phase 2 clinical trials in radioiodine-refractory thyroid cancer and recurrent platinum resistant ovarian cancer. VB-111 has received an Orphan Designation for the treatment of ovarian cancer by the European Medicines Agency (EMA).

Dynavax Technologies 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, Dynavax Technologies, 2018, MAR 8, 2018, View Source [SID1234524551]).

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On March 8, 2018 Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel therapeutics for difficult to treat cancers, reported that Ivan Bergstein, M.D., Stemline’s CEO, will present at two upcoming investor conferences (Press release, Stemline Therapeutics, MAR 8, 2018, View Source [SID1234524545]):

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The 30th Annual ROTH Conference on Monday, March 12, at 4:00 PM PT (7:00 PM ET), which is being held at The Ritz-Carlton, Orange County in Dana Point, California.

The Cowen and Company 38th Annual Health Care Conference 2018 on Wednesday, March 14, at 8:00 AM ET, which is being held at the Boston Marriott Copley Place in Boston, MA.
A live webcast of each presentation can be viewed on the company’s website at www.stemline.com.