On November 5, 2015 BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) reported financial results for the third quarter ended September 30, 2015 (Filing, 8-K, BioCryst Pharmaceuticals, NOV 5, 2015, View Source [SID:1234508001]).

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"The completion of enrollment of our OPuS-2 trial of avoralstat and the successful outcome of the Phase 1 healthy volunteer study of BCX7353 have positioned both programs to reach important value creating events in 2016. We expect to initiate the APeX-1 (Angioedema ProphylaXis) proof of concept trial of BCX7353 in HAE patients and to report OPuS-2 results by early 2016," said Jon P. Stonehouse, President & Chief Executive Officer of BioCryst. "These oral kallikrein inhibitors have the potential to revolutionize HAE treatment, providing patients the ability to lead normal lives."

Third Quarter Financial Results

For the three months ended September 30, 2015, revenues increased to $11.0 million from $3.2 million in the third quarter of 2014. The increase was the result of higher levels of product and collaboration revenue associated with the sale of RAPIVAB (peramivir injection) and the Seqirus, formerly bioCSL, out-licensing transaction, as well as increased collaboration revenue associated with BCX4430 development as a medical counter measure for Ebola virus and other filovirus diseases.

Research and development (R&D) expenses for the quarter increased to $20.1 million from $13.0 million in the third quarter of 2014. The increase in 2015 R&D expenses was primarily due to the advancement of the Company’s hereditary angioedema (HAE) programs, and to a lesser extent, higher expenses associated with two discovery-stage rare disease projects.

Selling, general and administrative (SG&A) expenses for the third quarter 2015 increased to $2.7 million from $1.8 million in 2014. This increase was due primarily to the initiation of activities in preparation for commercialization of the Company’s HAE product candidates, and to a lesser extent, commercial expenses associated with RAPIVAB.

In the third quarter of both 2015 and 2014, interest expense was $1.2 million and related to non-recourse notes payable. In addition, a mark-to-market loss on our foreign currency hedge of $460,000 was recognized in the third quarter of 2015, compared to a gain of $4.1 million in the third quarter of 2014. These gains and losses resulted from periodic changes in the U.S. dollar/Japanese yen exchange rate and the related mark-to-market valuation of our underlying hedge arrangement. During the third quarter of 2015, we also realized a currency hedge gain of $108,000 from the exercise of a U.S. Dollar/Japanese yen currency option.

The net loss for the third quarter of 2015 was $14.6 million, or $0.20 per share, as compared to a net loss of $8.7 million, or $0.12 per share, for the third quarter of 2014.

Cash, cash equivalents and investments totaled $119.7 million at September 30, 2015, compared to $114.0 million at December 31, 2014. Net operating cash use for the third quarter of 2015 was $12.3 million, as compared to $8.0 million in the third quarter of 2014. Net operating cash use for the first nine months of 2015 was $28.1 million, as compared to $19.8 million for the 2014 period.

Year to Date Financial Results

For the nine months ended September 30, 2015, total revenues increased to $43.7 million from $8.2 million in the same period of 2014. The increase in 2015 was primarily due to revenue recognition of $21.7 million associated with the upfront payment from the Seqirus out-licensing transaction, RAPIVAB product revenue, and increased collaboration revenue associated with BCX4430 development.

R&D expenses increased to $53.7 million for the first nine months of 2015 from $33.3 million in the same period of 2014. The increase in 2015 expenses was primarily due to increased spending associated with the Company’s HAE development programs, and to a lesser extent, higher BCX4430 development costs and discovery activity on two rare disease projects.

SG&A expenses increased to $10.3 million for the nine months ended September 30, 2015 from $5.4 million for the nine months ended September 30, 2014. The increase was primarily associated with the initiation of a commercial organization in preparation for commercialization of the Company’s HAE drug candidates, unrestricted grants awarded to the U.S. and international HAE patient advocacy groups, as well as deal-related expenses associated with the Seqirus out-licensing transaction.

In the first nine months of 2015 and 2014, interest expense was $3.9 million and $3.7 million, respectively, and related to the non-recourse notes payable. In addition, a mark-to-market loss on our foreign currency hedge of $793,000 was recognized in the first nine months of 2015, compared to a gain of $732,000 in the same period of 2014. These gains and losses result from periodic changes in the U.S. dollar/Japanese yen exchange rate and the related mark-to-market valuation of our underlying hedge arrangement. We also realized a currency hedge gain of $1.7 million from the exercise of a U.S. Dollar/Japanese yen currency option.

The net loss for the nine months ended September 30, 2015 decreased to $24.9 million, or $0.34 per share, compared to a net loss of $33.5 million, or $0.52 per share, for the same period in 2014.

Clinical Development Update & Outlook

BioCryst recently announced that the OPuS-2 (Oral ProphylaxiS-2) clinical trial of avoralstat completed enrollment of approximately 100 HAE patients, and the Company expects to report results in early Q1 2016.

BioCryst also announced that the randomized, placebo-controlled, Phase 1 clinical trial of orally-administered BCX7353 in healthy volunteers successfully met all of its objectives. Oral BCX7353 was generally safe and well tolerated at all doses up to 500 mg once-daily for 7 days and 350 mg once-daily for 14 days in healthy volunteers, and no dose-limiting toxicity was identified. There were no serious adverse events (AEs) and most AEs were mild. The safety, tolerability, drug exposure and on-target plasma kallikrein inhibition results strongly support advancing the development program into the (APeX-1) Phase 2 proof of concept clinical trial of BCX7353 in HAE patients.

The APeX-1 (Angioedema ProphylaXis) Phase 2, four week dose ranging trial to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of BCX7353 as a preventative treatment to reduce or eliminate attacks in HAE patients is expected to begin by early 2016, with results expected mid-2016.

On October 27, The Japanese Ministry of Health Labor & Welfare (MHLW) announced that BioCryst’s BCX7353 was one of six products designated under MLHW’s new "Sakigake" fast track review system. The Sakigake Designation System promotes R&D in Japan, aiming at early market availability for innovative pharmaceutical products. This designation provides for additional interactions with the regulatory agency in Japan (PMDA) from early development through filing, prioritized development and review, and introduction of the product as soon as possible to address a serious unmet medical need.

Financial Outlook for 2015

Based upon current trends, assumptions, and development plans, BioCryst expects its 2015 net operating cash use to be in the range of $8 to $18 million, upon adjusting the Company’s previously predicted range for the first nine months of operations and including the favorable impact of the Seqirus transaction. In addition, BioCryst continues to expect its operating expenses to be within the range of $75 to $95 million. Our operating expense range excludes equity-based compensation expense due to the difficulty in accurately projecting this expense, as it is significantly impacted by the volatility and price of the Company’s stock, as well as vesting of the Company’s outstanding performance-based stock options.

On November 5, 2015 Rexahn Pharmaceuticals, Inc. (NYSE MKT: RNN), a clinical stage biopharmaceutical company developing next generation therapeutics for the treatment of cancer, reported that interim clinical data from an ongoing Phase IIa study of its novel anti-cancer drug candidate, Archexin, will be presented on Friday, November 6, 2015 at the 14th International Kidney Cancer Symposium in Miami, Florida (Filing, 8-K, Rexahn, NOV 5, 2015, View Source [SID:1234508020]).

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"We are excited to present interim data from the ongoing Phase IIa clinical trial showing that Archexin, in combination with everolimus (Afinitor), appears to be safe and well tolerated at the doses tested to date. We have also noted early evidence of clinical activity at low doses in patients with metastatic kidney cancer," commented Peter D. Suzdak, Chief Executive Officer. "We look forward to completing the randomized, open-label, 2-arm dose expansion study of Archexin in combination with everolimus versus everolimus alone in order to further evaluate Archexin in metastatic renal cell carcinoma."

Archexin Clinical Data

Interim data from the Phase IIa Archexin clinical trial will be presented on Friday, November 6, 2015 by study investigators, Drs. S. Tagawa, G. Chatta and N. Agarwal in a poster presentation entitled "RX-0201, An Anti-Sense Targeting AKT-1 to Treat Metastatic Renal Cancer – Preliminary Phase IIa Data."

The interim results show that at the dose levels tested to date, Archexin appeared to be safe and well tolerated. The most commonly reported adverse events in the patients taking both Archexin and everolimus included: thrombocytopenia, mouth ulcerations, decreased weight, facial edema, and hyponatremia. To date, none of these adverse events has been dose limiting.

Early evidence of the potential clinical activity of Archexin in combination with everolimus has been observed. Among the patients enrolled in the study, two patients experienced stable disease, which has persisted for 170 and 334 days (as of October 28, 2015). In addition, at the lowest dose tested one patient experienced a 15% reduction in tumor size, as compared to a baseline CT scan taken prior to treatment with Archexin and everolimus.

Scott Tagawa, MD, MS, Medical Director, Genitourinary Oncology Research Program, Associate Professor of Clinical Medicine and Urology, Division of Hematology & Medical Oncology, Weill Cornell Medical College, commented, "The treatment of patients with metastatic RCC remains a significant unmet medical need and the early evidence supporting the potential clinical benefit of Archexin is therefore very promising. With a unique mechanism of action targeting a well validated cancer pathway (Akt-1 suppression), it is possible that Archexin in combination with everolimus could have a two-fold effect in the treatment of RCC, both by inhibiting the growth and proliferation of RCC, but also potentially by overcoming resistance to mTOR inhibitors. I look forward to further evaluation of this promising approach."

The ongoing Phase IIa clinical study is designed to evaluate the efficacy of Archexin in combination with everolimus (Afinitor) to treat metastatic RCC patients and is being conducted in two stages. Stage 1 is an open-label, dose-escalation study designed to identify a safe and tolerable dose of Archexin when given in combination with everolimus. Stage 2 is a randomized, open-label, 2-arm dose expansion study of Archexin in combination with everolimus versus everolimus alone to determine safety and efficacy of the combination.

In Stage 1, escalating doses of Archexin of 125, 200 and 250 mg/m2/day are administered by continuous IV infusion for 14 days followed by 1 week of rest. Based on previous clinical data, the target dose of Archexin is anticipated to be no more than 250 mg/m2 per day. Patient assessments include safety, pharmacokinetics, laboratory and physical exams. Once the maximum tolerated dose of Archexin in combination with everolimus has been determined, thirty RCC patients will be randomized to receive either Archexin in combination with everolimus, or everolimus alone, in a ratio of 2:1.

The primary endpoint of Stage 2 is the percentage of progression free patients following eight cycles of therapy. Patients are scanned (CT or MRI) for the assessment of tumor progression after every 2 cycles of therapy. Secondary endpoints include pharmacokinetic profile, incidence of adverse events, changes in clinical laboratory tests and vital signs over time, tumor response, duration of response, time to response, and response rate. Exploratory endpoints include blood levels of AKT pathway biomarkers, tumor apoptosis biomarkers, or other relevant biomarkers.

In preclinical studies, Archexin has been shown to inhibit the growth of human renal cell carcinoma (RCC) cells in tissue culture. Archexin has also been shown to exhibit an additive anti-tumor effect when combined with other cancer drugs in inhibiting the growth of human RCC cells in tissue culture.

About Archexin

Archexin is a unique anti-sense drug candidate that specifically inhibits the cancer cell signaling protein Akt-1. Archexin is the only specific inhibitor of Akt-1 in clinical development. The activated form of Akt-1, which is involved in cancer cell growth, survival, angiogenesis, and drug resistance, has been shown to be present or elevated in more than 12 different human cancer cell lines, including pancreatic and renal cell carcinoma. By inhibiting Akt-1, Archexin has been shown to both inhibit the growth of renal cell carcinoma cell lines and exhibit a longer survival benefit in the human renal cell carcinoma animal xenograft model. Thus, while Akt-1 is a very specific anti-cancer target, it may have broad therapeutic potential across multiple types of cancer.

Archexin has completed a Phase I clinical trial in cancer patients with solid tumors and was shown to be safe and well tolerated. The dose-limiting toxicity was Grade 3 fatigue. In a small Phase IIa trial in advanced pancreatic cancer patients, Archexin in combination with gemcitabine was shown to be safe and well tolerated and showed a preliminary efficacy signal with a median survival of 9.1 months in evaluable patients.

Metastatic RCC represents an attractive market opportunity with an estimated annual incidence of 90,000 patients worldwide. Metastatic RCC patients receiving standard of care treatment have a poor prognosis with an overall survival of less than 2 years. Rexahn has received U.S. Food and Drug Administration (FDA) Orphan Drug Designation for Archexin for metastatic RCC as well as four other cancers.

On November 5, 2015 Corcept Therapeutics Incorporated (NASDAQ: CORT), a pharmaceutical company engaged in the discovery, development and commercialization of drugs that treat severe metabolic, oncologic and psychiatric disorders by modulating the effects of cortisol, reported its financial results for the quarter ended September 30, 2015 and provided a corporate update (Press release, Corcept Therapeutics, NOV 5, 2015, http://www.corcept.com/news_events/view/pr_1446758532 [SID:1234508043]).

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Corcept recorded net revenue of $13.3 million in the third quarter of 2015, compared to $7.3 million for the same period in 2014, an increase of 82 percent. The company reiterated its 2015 revenue guidance of $49 – $53 million.

Corcept reported a net loss on a GAAP basis of $0.6 million for the third quarter of 2015, compared to a GAAP net loss of $6.0 million for the same period in 2014. Excluding non-cash expenses, Corcept generated non-GAAP net income in the third quarter of $1.6 million, compared to a non-GAAP net loss of $3.9 million in the third quarter of 2014. A reconciliation of GAAP to non-GAAP net operating results is set forth below.

At September 30, 2015, Corcept held cash and cash equivalents of $36.5 million, compared to $37.0 million at the end of the prior quarter. This change in cash reflects the repayment of $1.8 million in principal due under the company’s capped royalty financing agreement (Royalty Financing). The company entered into the Royalty Financing in 2012 to fund the commercialization of Korlym and expects to make its final payment in 2017.

Based on its current plans, the company expects to reach cash flow breakeven, including payments of principal due under the Royalty Financing, without needing to raise additional funds.

"Our Cushing’s syndrome franchise continues to grow as more physicians appreciate that using Korlym to modulate the effect of excess cortisol can greatly improve their patients’ health," said Joseph K. Belanoff, M.D., Corcept’s Chief Executive Officer. "As we have said before, our efficient cost structure and the revenue growth in our Cushing’s syndrome business allows us to build our clinical infrastructure, further develop Korlym and advance our next-generation compounds."

Korlym for the Treatment of Triple-Negative Breast Cancer (TNBC)

In December 2015, at the San Antonio Breast Cancer Symposium, Corcept will present preliminary efficacy results of its Phase 1/2 open-label trial of Korlym in combination with eribulin (Halaven) to treat patients with metastatic TNBC.

CORT125134 Phase 2 Trials

CORT125134 is the lead compound in Corcept’s portfolio of proprietary next-generation cortisol modulators. It was well-tolerated in its Phase 1 trial and showed that it shares Korlym’s ability to potently modulate activity at the glucocorticoid receptor (GR), the essential quality in treating Cushing’s syndrome. In addition, when administered with a chemotherapeutic agent, CORT125134 slows tumor growth significantly in mouse models of TNBC and castration-resistant prostate cancer. In vitro, it similarly slows the growth of ovarian cancer tumor cells; in vivo testing in mouse models of ovarian cancer is in progress.

In the first quarter of 2016, the company plans to begin two Phase 2 clinical trials with CORT125134. One trial will be for the treatment of patients with Cushing’s syndrome. The other will administer CORT125134 with a companion chemotherapeutic or hormonal agent to treat patients with a solid tumor cancer.

"It’s an exciting time to join Corcept," said Robert S. Fishman, M.D., Corcept’s Chief Medical Officer. "We are exploring extending Korlym’s label to cover oncologic indications, our lead next-generation cortisol modulator, CORT125134, is about to enter multiple Phase 2 trials and we’re advancing additional selective cortisol modulating compounds toward the clinic."

Financial Discussion

Corcept recorded a GAAP net loss of $0.6 million in the third quarter of 2015, compared to $6.0 million in the third quarter of 2014, including non-cash stock-based compensation and accreted interest expense generated from the Royalty Financing of $2.2 million and $2.1 million in the third quarter of 2015 and 2014, respectively. Excluding these non-cash items, Corcept generated net income on a non-GAAP basis of $1.6 million in the third quarter of 2015, compared to a non-GAAP net loss of $3.9 million in the third quarter of 2014.

Operating expenses for the third quarter were $13.2 million, compared to $12.4 million for the third quarter of 2014. The increase was primarily due to increased staffing costs and additional spending on the Phase 1/2 trial of Korlym for the treatment of TNBC and the development of next-generation cortisol modulators.

In the third quarter of 2015, Corcept made a payment of $2.5 million under the Royalty Financing, of which $0.7 million represented accreted interest and $1.8 million reduced outstanding principal. In the same period of 2014, Corcept paid $1.3 million, of which $0.9 million represented accreted interest and $0.4 million reduced outstanding principal. Corcept expects to make its final payment under the Royalty Financing in 2017.

Corcept’s cash balance at September 30, 2015 was $36.5 million, compared to $37.0 million at June 30, 2015 and $24.2 million at December 31, 2014.

(Filing, 10-Q, bluebird bio, NOV 5, 2015, View Source [SID:1234508031])

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(Filing, 10-Q, Array BioPharma, NOV 5, 2015, View Source [SID:1234508065])

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