On February 23, 2016 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its full-year 2015 financial results and provided an update on its pipeline, including duvelisib, an oral, dual inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma (Press release, Infinity Pharmaceuticals, FEB 23, 2016, View Source;p=RssLanding&cat=news&id=2142341 [SID:1234509160]). Infinity expects to report topline data from DYNAMO, a Phase 2 study of duvelisib in patients with refractory indolent non-Hodgkin lymphoma (iNHL), early in the third quarter of 2016. Infinity also anticipates completing an interim analysis of DUO, a Phase 3 study of duvelisib in patients with relapsed/refractory chronic lymphocytic leukemia (CLL), early in the second half of 2016. The company expects marketing applications, if supported by these data, to be submitted to the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) in the fourth quarter of 2016. Infinity also recently expanded its pipeline with the addition of IPI-549, an oral immuno-oncology development candidate targeting PI3K-gamma. A Phase 1 study of IPI-549 is ongoing.

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"We made important progress across our pipeline in 2015, which included completing patient enrollment in DYNAMO and DUO, our registration-focused studies of duvelisib. These studies represent two initial paths to potential approval, and we are continuing to prepare for regulatory submissions based on data from these studies," stated Adelene Perkins, Infinity’s president and chief executive officer. "We are also advancing additional clinical studies designed to further differentiate duvelisib, with an ultimate goal of providing a potential cure for patients with hematologic malignancies through combination therapy. To implement this strategy, our partner AbbVie has under way a Phase 1b/2 clinical study of duvelisib in combination with venetoclax."

"Infinity is committed to building a pipeline of medicines that can meaningfully impact the standard of care for patients. We recently initiated a Phase 1 clinical study of IPI-549, our oral immuno-oncology development candidate. IPI-549, a selective PI3K-gamma inhibitor, has first-in-class potential and represents an important extension of our oncology portfolio to include development candidates directed at both hematologic malignancies and solid tumors," Ms. Perkins continued.

Recent achievements include the following:

Enrollment completed in registration-focused studies, DYNAMO and DUO: In September, Infinity announced it had reached target enrollment in DYNAMO, a global, Phase 2 open-label, single-arm, monotherapy study of duvelisib in approximately 120 patients with iNHL whose disease is refractory to rituximab and to either chemotherapy or radioimmunotherapy. The primary endpoint of the study is overall response rate.

In November 2015, Infinity announced it reached target enrollment in DUO, a randomized Phase 3 monotherapy study evaluating the safety and efficacy of duvelisib compared to ofatumumab (an anti-CD20 antibody) in approximately 300 patients with relapsed or refractory CLL. The primary endpoint of this study is progression-free survival. Infinity anticipates completing an interim analysis of DUO early in the second half of 2016.

Infinity expects marketing applications, if supported by the DYNAMO and DUO data, to be submitted to the FDA and EMA in the fourth quarter of 2016.

Duvelisib-venetoclax Phase 1b/2 study under way: AbbVie has under way a Phase 1b/2 clinical study of duvelisib in combination with venetoclax, an investigational B-cell lymphoma-2 (BCL-2) selective inhibitor. This study is designed to evaluate the safety and efficacy of duvelisib in combination with venetoclax in approximately 174 patients with relapsed or refractory iNHL, aggressive NHL, small lymphocytic lymphoma or CLL.

BRAVURA study initiated: In December 2015, Infinity initiated BRAVURA, a Phase 3, double-blind, placebo-controlled study in patients with relapsed iNHL. BRAVURA is designed to evaluate the safety and efficacy of duvelisib plus rituximab and bendamustine (RB) compared to placebo plus RB in approximately 600 patients. The primary endpoint is progression-free survival.

FRESCO study initiated: In December 2015, Infinity initiated FRESCO, a Phase 2 study in patients with relapsed/refractory follicular lymphoma (FL). FRESCO is designed to evaluate the safety and efficacy of duvelisib plus rituximab versus rituximab in combination with chemotherapy in approximately 230 patients. The primary endpoint is progression-free survival.

Phase 1 clinical study of IPI-549 initiated: In January 2016, Infinity initiated a Phase 1 study of IPI-549, an oral immuno-oncology development candidate that selectively inhibits PI3K-gamma. The study is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with an anti-PD-1 antibody, a checkpoint inhibitor, in approximately 150 patients with advanced solid tumors, including non-small cell lung cancer and melanoma. IPI-549 is the only investigational PI3K-gamma inhibitor in clinical development.
2016 Duvelisib Goals

Infinity expects to achieve the following duvelisib milestones in 2016:

Report topline DYNAMO data in 3Q16

Report topline DUO data in 2H16*

Submit a New Drug Application (NDA) for iNHL and CLL in 4Q16*

AbbVie submission of Marketing Authorization Application (MAA) for FL and CLL in 4Q16*

Report initial data from CONTEMPO, a Phase1b/2 study in treatment-naïve patients with FL, in 2H16

Advance Phase 1b/2 study of duvelisib in combination with venetoclax

*Topline DUO data report, CLL NDA filing and FL/CLL MAA filing predicated on DUO interim analysis.

Full-Year 2015 Financial Results

At December 31, 2015, Infinity had total cash, cash equivalents and available-for-sale securities of $245.2 million, compared to $333.2 million at December 31, 2014.

Revenue during 2015 was $109.1 million, which included a $75.2 million license fee associated with the $130 million milestone payment from AbbVie for the completion of patient enrollment in DYNAMO and $33.9 million in research and development (R&D) services. R&D services revenue for 2015 was composed of $12.8 million associated with the $130 million milestone payment and $21.1 million associated with the $275 million upfront payment from AbbVie received in September 2014. Revenue during 2014 was $165.0 million, which was composed of a $159.1 million license fee and $5.9 million in R&D services, both of which related to the $275 million upfront milestone from the strategic collaboration with AbbVie. Infinity will recognize the remainder of the $130 million milestone payment and $275 million upfront payment over the period in which R&D services will be provided.

R&D expense for full-year 2015 was $199.1 million, compared to $143.6 million for 2014. R&D expense for 2015 included a $52.5 million payment related to the exercise of an option to buy out the company’s royalty obligations to Takeda Pharmaceutical Company Limited for duvelisib worldwide oncology sales. R&D expense for 2014 included a $10.0 million milestone payment to Takeda for the initiation of the first duvelisib Phase 3 study and a $5.0 million payment to Takeda related to the royalty buy-out option. Excluding these payments to Takeda, the increase in R&D expense in 2015 compared to 2014 was primarily due to higher clinical development expenses for duvelisib.

General and administrative expense was $37.1 million for the full-year 2015 compared to $29.3 million for 2014. The increase in general and administrative expense in 2015 compared to 2014 was primarily due to additional personnel as well as commercial expenses in preparation for the potential 2017 duvelisib launch.

Net loss for the full-year 2015 was $128.4 million, or a basic and diluted loss per common share of $2.62, compared to $17.4 million, or a basic and diluted loss per common share of $0.36 for 2014.
2016 Financial Guidance
Infinity’s 2016 financial outlook remains as follows:

Revenue: Infinity expects revenue for 2016 to range from $225 million to $245 million, assuming the achievement of $200 million in anticipated regulatory milestones under the company’s collaboration with AbbVie: $125 million associated with the acceptance of the first NDA submission and $75 million associated with the acceptance of the first MAA submission. Infinity expects to record the $200 million in milestone revenue in the fourth quarter of 2016 and expects payment by AbbVie in the first quarter of 2017.

Net Income: Infinity expects net income for 2016 to range from $15 million to $35 million.

Cash and Investments: Infinity expects to end 2016 with a year-end cash and investments balance ranging from $45 million to $65 million. This year-end cash and investments balance excludes the $200 million in anticipated milestones, which Infinity expects to be paid by AbbVie in the first quarter of 2017.

On February 23, 2016 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported financial results for the full year and the fourth quarter of 2015 and provided financial guidance for 2016 (Filing, 8-K, Jazz Pharmaceuticals, FEB 23, 2016, View Source [SID:1234509162]).

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"In 2015, we delivered solid growth on the top- and bottom-line while increasing investment in new growth opportunities for our current products and our promising R&D pipeline," said Bruce C. Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals plc. "We look forward to 2016 as we focus on delivering growth of our key commercial products, preparing for our planned launch of defibrotide in the U.S., reaching important clinical milestones, including completing enrollment and determining preliminary results of our JZP-110 Phase 3 safety and efficacy studies, and potentially expanding our commercial and development portfolio through corporate development activities."

Adjusted net income attributable to Jazz Pharmaceuticals plc for 2015 was $600.1 million, or $9.52 per diluted share, compared to $520.5 million, or $8.31 per diluted share, for 2014. Adjusted net income attributable to Jazz Pharmaceuticals plc for the fourth quarter of 2015 was $163.5 million, or $2.60 per diluted share, compared to $151.1 million, or $2.40 per diluted share, for the fourth quarter of 2014.

GAAP net income attributable to Jazz Pharmaceuticals plc for 2015 was $329.5 million, or $5.23 per diluted share, compared to $58.4 million, or $0.93 per diluted share, for 2014. GAAP net income attributable to Jazz Pharmaceuticals plc for the fourth quarter of 2015 was $82.8 million, or $1.32 per diluted share, compared to $81.6 million, or $1.30 per diluted share, for the fourth quarter of 2014. Reconciliations of applicable GAAP reported to non-GAAP adjusted information are included in this press release.

2015 Revenues and Product Sales
Total revenues for the year ended December 31, 2015 were $1,324.8 million, an increase of 13% over total revenues of $1,172.9 million for the year ended December 31, 2014. Total revenues for the fourth quarter of 2015 were $340.9 million, an increase of 4% over total revenues of $328.1 million for the fourth quarter of 2014. The increase in total revenues was driven by higher net product sales of Xyrem (sodium oxybate) oral solution. Total revenues include net product sales, royalties and contract revenues.

Net product sales for 2015 and the fourth quarter of 2015 were as follows:
•
Xyrem: 2015 Xyrem net sales increased by 23% to $955.2 million compared to $778.6 million during the prior year. Xyrem net sales increased by 13% to $251.8 million in the fourth quarter of 2015 compared to $222.5 million in the fourth quarter of 2014.

• Erwinaze/Erwinase (asparaginase Erwinia chrysanthemi): 2015 Erwinaze/Erwinase net sales were $203.3 million compared to $199.7 million during the prior year. Erwinaze/Erwinase net sales were $50.4 million in the fourth quarter of 2015 compared to $52.8 million in the fourth quarter of 2014. Product sales volume for the full year and fourth quarter of 2015 increased

compared to the same periods in 2014; however, net sales were negatively impacted by higher chargebacks and rebates and unfavorable foreign currency exchange rates. In the fourth quarter of 2015, the company experienced supply challenges that disrupted the ability to fully supply certain markets.

• Defitelio (defibrotide): 2015 Defitelio/defibrotide net sales were $70.7 million compared to 2014 full year pro forma Defitelio/defibrotide net sales of $73.4 million. Defitelio/defibrotide net sales from the period beginning from the closing of its acquisition of Gentium S.r.l. on January 23, 2014 to December 31, 2014 were $70.5 million. Defitelio/defibrotide net sales were $18.5 million in the fourth quarter of 2015 compared to $19.2 million in the fourth quarter of 2014. Product sales volume for the full year and fourth quarter of 2015 was consistent with the company’s expectations and higher than the same periods in 2014, but net sales were impacted by unfavorable foreign currency exchange rates.

• Prialt (ziconotide) intrathecal infusion: Prialt net sales were $26.4 million in both 2015 and 2014. Prialt net sales were $6.5 million in the fourth quarter of 2015 compared to $10.0 million in the fourth quarter of 2014. Net sales in the fourth quarter of 2014 included shipments to Eisai Co., the European distributor of Prialt.

• Psychiatry products: 2015 net sales of the company’s psychiatry products were $37.1 million compared to $40.9 million in the prior year. Net sales of the company’s psychiatry products were $8.8 million in the fourth quarter of 2015 compared to $8.4 million in the fourth quarter of 2014.

• Other: 2015 net sales of other products were $24.1 million compared to 2014 full year pro forma net sales of other products of $47.0 million. Net sales of other products in the fourth quarter of 2015 were $3.0 million compared to $11.3 million in the fourth quarter of 2014. In March 2015, the company completed the sale of certain products and the related business that the company acquired as part of the acquisition of EUSA Pharma Inc. in 2012.

Tables showing actual net product sales for the three months and year ended December 31, 2015 and 2014 and pro forma net product sales for the year ended December 31, 2014 are included in this press release.

Operating Expenses and Other
Operating expenses for 2015 were $816.6 million compared to $977.3 million for 2014. Operating expenses for 2014 included acquired in-process research and development expenses of $202.6 million. Operating expenses for the fourth quarter of 2015 were $234.3 million compared to $198.2 million for the fourth quarter of 2014. Operating expenses changed over the prior year periods primarily due to the following:

• Cost of product sales for 2015 was $102.5 million compared to $117.4 million for 2014. Cost of product sales for the fourth quarter of 2015 was $24.0 million compared to $28.8 million for the same period in 2014. Gross margin for 2015 was 92.2% compared to 89.9% for 2014. Gross margin for the fourth quarter of 2015 was 92.9% compared to 91.1% for the same period in 2014.

• Selling, general and administrative (SG&A) expenses for 2015 on a GAAP basis were $449.1 million compared to $406.1 million for 2014. SG&A expenses for the fourth quarter of 2015 on a GAAP basis were $125.6 million compared to $105.7 million for the same period in 2014. Adjusted SG&A expenses for 2015 were $355.4 million, or 27% of total revenues, compared to $321.5 million, or 27% of total revenues, for 2014. Adjusted SG&A expenses for the fourth quarter of 2015 were $87.4 million, or 26% of total revenues, compared to $83.5 million, or 25% of total revenues, for the same period in 2014. The increases were primarily due to higher headcount and other expenses resulting from the expansion of the company’s business, except that the increase in GAAP SG&A expenses for the fourth quarter of 2015 compared to the same period in 2014 was primarily due to a one-time charge for settlement of a contract claim originally

asserted against Azur Pharma Public Limited Company (Azur Pharma) prior to the 2012 merger between Azur Pharma and Jazz Pharmaceuticals, Inc.

• Research and development (R&D) expenses for 2015 on a GAAP basis were $135.3 million compared to $85.2 million for 2014. R&D expenses for the fourth quarter of 2015 on a GAAP basis were $29.5 million compared to $24.6 million for the same period in 2014. Adjusted R&D expenses for 2015 were $96.7 million, or 7% of total revenues, compared to $71.8 million, or 6% of total revenues, for 2014. Adjusted R&D expenses for the fourth quarter of 2015 were $26.0 million, or 8% of total revenues, compared to $21.2 million, or 6% of total revenues, for the same period in 2014. The increases were primarily due to higher costs for clinical studies and outside services for the development of JZP-110 and line extensions for the company’s existing products, except that the increase in GAAP R&D expenses for full year 2015 compared to the same period in 2014 was primarily due to a $25.0 million milestone payment that was triggered by the acceptance for filing by the U.S. Food and Drug Administration (FDA) of the first new drug application (NDA) for defibrotide.

• Acquired in-process research and development expenses of $202.6 million in 2014 primarily related to upfront and milestone payments of $127.0 million made in connection with the acquisition of rights to JZP-110 and an upfront payment of $75.0 million made in connection with the acquisition of rights to defibrotide in the Americas.

• Impairment charges of $31.5 million in 2015 resulted from the termination of the suspended JZP-416 study. Impairment charges of $39.4 million in 2014 related to certain products we sold in March 2015 that we acquired as part of the EUSA acquisition.
Net interest expense in 2015 was $56.9 million compared to $52.7 million for 2014. Net interest expense for the fourth quarter of 2015 was $12.2 million compared to $16.7 million for the fourth quarter of 2014. In June 2015, the company refinanced its existing term loans and revolving credit facility and obtained more favorable interest rates, which reduced interest expense in the second half of 2015.

As of December 31, 2015, cash and cash equivalents were $988.8 million, and the outstanding principal balance of the company’s long-term debt was $1.3 billion. Cash and cash equivalents increased during 2015 primarily due to cash generated by the business.
During 2015, the company repurchased 0.4 million ordinary shares for $61.6 million at an average cost of $150.24 per ordinary share.

On February 22, 2016 PDL BioPharma, Inc. (PDL) (NASDAQ: PDLI) reported financial results for the fourth quarter and twelve months ended December 31, 2015 (Filing, 8-K, PDL BioPharma, FEB 22, 2016, View Source [SID:1234509130]).

Total revenues in 2015 increased two percent to $590.4 million from $581.2 million in 2014. Revenues for the year ended December 31, 2015 included $485.2 million in royalties from PDL’s licensees to the Queen et al. patents, $68.4 million in net royalty payments from acquired royalty rights and a change in fair value of the royalty rights assets, which included approximately $43.4 million in net cash royalty payments, $36.2 million in interest revenue from notes receivable debt financings to late-stage healthcare companies, and $0.7 million in realized gains from the sale of PDL’s investment in AxoGen Inc. common stock. During the years ended December 31, 2015 and 2014, our Queen et al. royalty revenues consisted of royalties and maintenance fees earned on sales of products under license agreements associated with our Queen et al. patents. During the years ended December 31, 2015 and 2014, royalty rights – change in fair value consisted of revenues associated with the change in estimated fair value of our royalty right assets, primarily Depomed, Inc., The Regents of the University of Michigan, Viscogliosi Brothers, LLC, ARIAD Pharmaceuticals Inc. and AcelRx Pharmaceuticals, Inc. The full year 2015 revenue growth over the full year 2014 is driven by increased sales of Perjeta, Xolair, and Kadcyla by PDL’s licensees, an increase in the estimated fair value of the acquired royalty rights from the Company’s purchase of Depomed’s diabetes-related royalties, as well as a foreign exchange gain and lower rebate paid to Novartis AG for Lucentis, partially offset by decreased interest revenues due to the early payoff of the AxoGen and Durata Therapeutics, Inc. notes receivables.

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Total revenues for the fourth quarter of 2015 increased 52 percent, to $178.1 million from $117.1 million in the fourth quarter of 2014. Revenues for the fourth quarter of 2015 included $121.2 million in royalty payments from PDL’s licensees to the Queen et al. patents, $49.1 million in net royalty payments from acquired royalty rights and a change in fair value of the royalty rights assets, which included approximately $34.4 million in net cash royalty payments, $7.6 million in interest revenue from notes receivable debt financings to late-stage healthcare companies, and $0.1 million in realized gains from the sale of PDL’s investment in AxoGen common stock. The fourth quarter of 2015 revenue growth over the fourth quarter of 2014 is driven by the change in estimated fair value of our royalty right assets, primarily Depomed, Inc.

Operating expenses in 2015 were $40.1 million, compared with $34.9 million in 2014. Operating expenses in the fourth quarter of 2014 were $16.5 million, compared with $17.7 million in 2014. The increase in operating expenses for the year ended December 31, 2015, when compared to the year ended December 31, 2014, was a result of total restructuring costs of $7.9 million in connection with the LENSAR notes receivable extinguishment, which is comprised of a loss on extinguishment of notes receivable of $4.0 million primarily related to a lower estimated fair value of the ALPHAEON Class A common stock, and additional general and administrative expenses of $3.9 million for closing and legal fees related to the LENSAR notes receivable restructuring, and other legal expenses mostly related to $1.2 million in funding the ongoing operation management of Wellstat Diagnostics, partially offset by a decrease in professional services from asset acquisition expenses. The decrease in operating expenses for the quarter ended December 31, 2015, when compared to the quarter ended December 31, 2014, was a result of a decrease in professional services from asset acquisition expenses and a decrease in compensation related expenses,

partially offset by the LENSAR restructuring loss and other closing fees, and an increase for legal expenses mostly related to Wellstat ongoing operation management.

Net income in 2015 was $332.8 million, or $2.03 per diluted share as compared with net income in 2014 of $322.2 million, or $1.86 per diluted share. Net income for the fourth quarter of 2015 was $100.6 million, or $0.61 per diluted share, as compared with net income of $55.1 million in the same period of 2014, or $0.32 per diluted share.

Net cash provided by operating activities in 2015 was $301.5 million, compared with $292.3 million in the same period in 2014. PDL had cash, cash equivalents and short-term investments of $220.4 million and $293.7 million at December 31, 2015 and 2014, respectively. The decrease was primarily attributable to the extinguishment of convertible notes of $220.4 million, purchase of royalty rights at fair value of $115.0 million, payment of dividends of $98.3 million, repayment of a portion of the March 2015 Term Loan of $75.0 million, purchase of notes receivable of $35.2 million, and payment of debt issuance costs related to the February 2018 Note issuance of $0.6 million, partially offset by proceeds from the March 2015 Term Loan of $100.0 million, proceeds from royalty rights of $43.4 million, repayment of notes receivables of $25.2 million, sale of investments of $1.9 million, and cash generated by operating activities of $301.5 million.

Recent Developments

In December 2015, Lion Buyer, a wholly owned subsidiary of ALPHAEON assumed $42.0 million in loans as part of the borrowings under PDL’s prior credit agreement with LENSAR and changed its name to LENSAR, LLC in connection with ALPHAEON’s acquisition of substantially all of the assets of LENSAR. In addition, ALPHAEON issued 1.7 million shares of its Class A common stock to PDL for an estimated fair value of $3.84 per share.

In December 2015 and January 2016, PDL and Direct Flow Medical modified the existing credit agreement. PDL funded an additional $5.0 million to Direct Flow Medical in the form of a short-term secured promissory note that we expect will be converted into a loan under the credit agreement with substantially the same interest and payment terms as the existing loans.

PDL’s $100.0 million term loan entered into on March 20, 2015 with the Royalty Bank of Canada was repaid with the final principal payment of $25.0 million plus accrued interest paid on February 12, 2016.

On February 18, 2016, PDL was advised that Sanofi and kaléo will terminate their license and development agreement later this year. At that time, all U.S. and Canadian commercial and manufacturing rights to Auvi-Q will be returned to kaléo, and they intend to evaluate the timing and options for bringing Auvi-Q back to the market. PDL entered into a secured note purchase agreement with Accel 300, a wholly-owned subsidiary of kaléo, which as of December 31, 2015, had a principal balance of $144.8 million due to PDL. An interest reserve account previously set up as part of the note agreement will substantially cover interest payments due to PDL through the end of the second quarter of 2016, and kaléo has indicated that it intends to make payments due to PDL under the note agreement until Auvi-Q is returned to the market.

2016 Dividends
On January 26, 2016, our board of directors declared a quarterly dividend to be paid to our stockholders in the first quarter of 2016 of $0.05 per share of common stock, payable on March 11, 2016 to stockholders of record on March 4, 2016, the record date of the dividend payment. At the same time our board of directors elected to announce its future dividend plans on a quarter by quarter basis, rather than for the full year as was the previous practice, to allow greater flexibility and focus on long term growth. Our board of directors evaluates the financial condition of the Company and considers the economic outlook, profitability, corporate cash flow, the Company’s liquidity needs and the health and stability of credit markets when determining the dividend.

On February 22, 2016 Cleveland BioLabs, Inc. (NASDAQ: CBLI) reported financial results and development progress for the fourth quarter and year ended December 31, 2015.

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Cleveland BioLabs reported a net loss, excluding minority interests, of $(1.4) million for the fourth quarter of 2015, or $(0.13) per share, compared to net income of $11.3 million, or $3.95 per share, for the fourth quarter of 2014. Net loss, excluding minority interests, for full year 2015 was $(12.6) million, or $(1.79) per share, compared to a net income of $1.6 million, or $0.60 per share, for full year 2014. The 2014 periods reported net income due to a $14.2 million gain on the deconsolidation of the Company’s joint venture, Incuron LLC. Excluding the gain on the deconsolidation of Incuron, net loss per share for the fourth quarter of 2014 was $(1.02) and net loss per share for full year 2014 was $(4.66).

As of December 31, 2015, the Company had $19.6 million in cash, cash equivalents and short-term investments, which, based on the Company’s current operational plan, is expected to fund the Company’s operating requirements beyond one year.

Yakov Kogan, Ph.D., MBA, Chief Executive Officer, stated, "The past year was one of significant momentum and accomplishment for CBLI. We strengthened our financial resources through the addition of a $25 million strategic investor and the award of $15.8 million in funding from the Department of Defense Congressionally Directed Medical Research Programs for continued development of entolimod’s biodefense indication. We streamlined our corporate structure with the sale of Incuron, while retaining a royalty on Incuron’s future success. We achieved several major milestones with our development programs, including the submission of a pre-Emergency Use Authorization (pre-EUA) dossier for entolimod as a radiation countermeasure and presentation of clinical oncology data for entolimod at the 2015 annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper). And, we commenced or continued clinical studies designed to further substantiate the potential of our Toll-like receptor agonists, entolimod, CBLB612 and Mobilan."

"The pursuit of commercialization for entolimod as a radiation countermeasure remains our top priority," continued Dr. Kogan. "We continue to work with the U.S. Food and Drug Administration to facilitate the review of our pre-EUA dossier. Products with pre-EUA status may be purchased by certain US government stakeholders for stockpiling in the event of a disaster and we believe achievement of this status may also increase interest from foreign governments. We recently initiated a regulatory process with the European Medicines Agency, which has granted entolimod orphan drug designation for the treatment of acute radiation syndrome, and we continue to evaluate other foreign markets."

Other Recent Operational Highlights

Studies elucidating immunotherapeutic mechanisms through which entolimod suppresses metastasis were published in Proceedings of the National Academy of Sciences of the United States of America (PNAS). The studies presented in the PNAS publication decipher the cascade of cell-signaling events that are triggered by entolimod activation of the TLR5 pathway in the liver. (View Source )
Dosing commenced in a Phase 2 clinical study of the safety and tolerability of entolimod as a neo-adjuvant therapy in treatment-naïve patients with primary colorectal cancer who are recommended for surgery. This study is being conducted in the Russian Federation.
A Phase 2 clinical study of CBLB612 as myelosuppressive prophylaxis in patients with breast cancer receiving doxorubicin-cyclophosphamide chemotherapy started dosing in the Russian Federation.
Panacela Labs continued dosing in a Phase 1 study with Mobilan evaluating single injections administered directly into the prostate of patients with prostate cancer. This study is being conducted in the Russian Federation.
All of the studies being conducted in the Russian Federation are supported by development contracts with the Russian Federation Ministry of Industry and Trade, or MPT.

Further Financial Results

Revenue for the fourth quarter of 2015 was $1.3 million compared to $1.4 million for the fourth quarter of 2014. Revenue for full year 2015 was $2.7 million compared to $3.7 million for full year 2014. These decreases are primarily due to the completion of an Incuron research contract with the Skolkovo Foundation in 2014. Other offsetting variances include new revenue from recently awarded Department of Defense contracts, offset by reduced revenue from MPT contracts due largely to variations in the foreign currency exchange rate.

Research and development costs for the fourth quarter of 2015 were $1.9 million compared to $2.8 million for the fourth quarter of 2014. Research and development costs for full year 2015 decreased to $7.1 million compared to $9.7 million for full year 2014. These decreases primarily resulted from the deconsolidation of Incuron and nonrecurring drug production costs for Mobilan, both occurring in 2014. Somewhat offsetting were reductions in entolimod’s biodefense program due to the completion and subsequent submission of the pre-EUA dossier in the second quarter of 2015, offset by increases in entolimod’s oncology development largely due to the clinical activities commenced in the Russian Federation.

General and administrative costs for the fourth quarter of 2015 were $1.2 million compared to $2.0 million for the fourth quarter of 2014. General and administrative costs for full year 2015 decreased to $6.4 million compared to $8.5 million for full year 2014. Approximately 40% of this net decrease was attributable to the deconsolidation of Incuron, with the remainder primarily attributable to reductions in personnel and outside professional costs.

At December 31, 2015 the Company had approximately 11 million shares of common stock outstanding. In addition, the Company has 343,643 shares of common stock reserved for issuance pursuant to outstanding stock options with a weighted average exercise price of $46.60 and 2.2 million shares of common stock reserved for issuance pursuant to outstanding warrants exercisable at a weighted average price of $13.98.

On February 22, 2016 Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, reported financial results for the fourth quarter and full-year ended December 31, 2015 and provided a summary of recent corporate highlights (Press release, Regulus, FEB 22, 2016, View Source [SID:1234509134]).

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"2015 was a milestone year for the company with the filing of three investigational new drug applications in the United States, and two clinical trial applications in the European Union," said Paul Grint, M.D., President and CEO of Regulus. "Our priorities for 2016, based on the data seen to date, include acceleration of our clinical programs, advancing our pipeline and defining the regulatory path to approval for our lead programs."

Fourth Quarter and Year-End 2015 Financial Results & Highlights

Regulus reported a net loss of $7.2 million and $55.7 million for the quarter and year ended December 31, 2015, respectively, compared to a net loss of $22.2 million and $56.7 million for the same periods in 2014. Basic and diluted net loss per share was $0.14 for the quarter ended December 31, 2015, compared to $0.47 for the same period in 2014. Basic and diluted net loss per share was $1.08 for the year ended December 31, 2015, compared to $1.29 for the same period in 2014.

Regulus recognized revenue of $10.9 million and $20.8 million for the quarter and year ended December 31, 2015, respectively, compared to $4.2 million and $7.7 million for the same periods in 2014. Revenue for the quarter and year ended December 31, 2015 included milestones earned under Regulus’ strategic alliances and collaboration agreements of $10.0 million and $13.2 million, respectively, which included a $10.0 million clinical milestone payment upon AstraZeneca’s first patient dosing in a first-in-human Phase I clinical study of RG-125(AZD4076). Revenue from research services performed under Regulus’ strategic alliances and collaborations was $0.4 million and $4.5 million for the quarter and year ended December 31, 2015, respectively. Other revenue during these periods consisted of amortization of up-front payments from Regulus’ strategic alliances and collaborations, which is recognized over the estimated period of performance.

Research and development expenses were $12.8 million and $56.4 million for the quarter and year ended December 31, 2015, respectively, compared to $10.5 million and $41.0 million for the same periods in 2014. This increase was primarily driven by clinical trial costs for RG-101, pre-clinical study costs for RG-125 and an increase in salaries and related employee costs, including non-cash stock-based compensation.

General and administrative expenses were $5.4 million and $19.1 million for the quarter and year ended December 31, 2015, respectively, compared to $3.3 million and $11.5 million for the same periods in 2014. This increase was primarily driven by an increase in salaries and related employee costs, including non-cash stock-based compensation.

As of December 31, 2015, Regulus had $115.3 million in cash, cash equivalents and short-term investments, including restricted cash of $1.3 million, and 52,669,266 shares of common stock outstanding.

Recent Highlights

RG-101 (GalNAc-conjugated anti-miR122 for the treatment of Hepatitis C Virus)

Interim Results from Phase II Combination Study. Regulus recently announced interim results from one of the company’s ongoing Phase II studies of RG-101 for the treatment of Hepatitis C Virus infection (HCV). The study was designed to evaluate a shortened, four-week treatment regimen containing a subcutaneous administration of 2 mg/kg of RG-101 at Day 1 and Day 29, in combination with 4 weeks of once/daily approved anti-viral agents Harvoni, Olysio, or Daklinza. The study enrolled 79 treatment naïve genotype 1 and 4 HCV patients (Harvoni arm, n=27, Olysio arm, n=27, Daklinza arm, n=25). Thirty-eight patients have been evaluated through 8 weeks of follow up. Ninety-seven percent of those patients (37/38) had HCV RNA viral load measurements below the limit of quantification. To date, RG-101 has been generally well tolerated with the majority of adverse events considered mild or moderate, and with no study discontinuations. For those patients through 12 weeks of follow-up, 100% remained below the limit of quantification (14/14). The primary endpoint analysis (12 week follow up) for all 79 patients in the study are anticipated to be reported in late Q2 2016.

Entered into Clinical Collaboration with GSK with Potential for Single Visit HCV Therapy. In accordance with the GSK clinical collaboration, Regulus has initiated its Phase II clinical trial evaluating the combination of RG-101 and GSK2878175, a non-nucleoside NS5B polymerase inhibitor, in HCV patients. In parallel, GSK is working to develop a long-acting parenteral "LAP" formulation of GSK2878175 as a single intra-muscular injection, providing the potential for a single-visit therapeutic treatment for HCV. Regulus anticipates reporting interim safety and efficacy data from this study by the end of 2016.

Enrollment Nearly Complete in US Phase I Study. Enrollment is nearly complete in a multi-center, open label, non-randomized Phase I study to compare the safety, tolerability, pharmacokinetics, and pharmacodynamics of RG-101 in subjects with severe renal insufficiency or end-stage renal disease ("ESRD") to healthy control subjects, and further explore RG-101 in hepatitis C infected subjects with severe renal insufficiency or ESRD. Regulus anticipates reporting safety and efficacy data from the HCV/severe renal impairment or ESRD arm in the second half of 2016.

RG-012 (anti-miR21 for the treatment of Alport syndrome)

Completed Phase I Study. Regulus completed a single-ascending dose, first-in-human, Phase I study evaluating the safety, tolerability and pharmacokinetics of subcutaneous dosing of RG-012 in healthy volunteers. RG-012 was well-tolerated with no serious adverse events or discontinuations reported. Regulus is scheduled to meet with the FDA towards the end of the first quarter of 2016 to discuss the Phase II program.

Advanced ATHENA Natural History Study. Regulus continues to advance the ATHENA natural history study in patients with Alport syndrome and anticipates reporting initial observations in the second quarter of 2016 at a medical meeting. Longitudinal data obtained in the ATHENA natural history study will help inform the design of the Phase II study.

RG-125 (GalNAc-conjugated anti-miR103/107 for the treatment of NASH)

Entered Phase I Development; Received $10.0M Milestone Payment from AstraZeneca. Dosing commenced in a first-in-human Phase I study of RG-125(AZD4076) by Regulus’ collaboration partner AstraZeneca. Regulus received a $10.0 million milestone payment from AstraZeneca, who will assume all ongoing development of RG-125(AZD4076).

Additional Highlights

Attracted Key Talent. Regulus appointed Joseph "Jay" Hagan as Chief Operating Officer, principal financial officer and principal accounting officer. Mr. Hagan is responsible for leading the company’s operations and corporate development and serves as a key member of the executive leadership team.

Received Key Patents for Lead Programs. The U.S. Patent and Trademark Office granted patents related to Regulus’ most advanced microRNA therapeutics, RG-101 and RG-012, furthering the company’s ability to protect its microRNA candidates.

Achieved All Milestones in Biomarkers Collaboration with Biogen; Earned $3.7M. Regulus realized the full potential of its collaboration with Biogen to identify microRNAs as biomarkers for multiple sclerosis and earned $3.7 million in payments. The scope of the research under the current collaboration agreement has concluded.