OnMay 3, 2016 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 March 31, 2016 (Press release, Corcept Therapeutics, MAY 3, 2016, http://www.corcept.com/news_events/view/pr_1462308579 [SID:1234511885]).

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Corcept reported revenue of $16.1 million and a GAAP net loss of $0.00 per share for the first quarter of 2016, compared to revenue of $10.1 million and a GAAP net loss of $0.05 per share in the first quarter of 2015. The company’s cash and cash equivalents were $40.7 million at year-end, an increase of $300,000 from December 31, 2015.

The company reiterated its 2016 revenue guidance of $76-81 million.

"Corcept’s business model continues to prove itself," said Dr. Joseph K. Belanoff, Corcept’s Chief Executive Officer. "Our Cushing’s syndrome franchise remains on track to generate $76-81 million in revenue this year, which will fully support the planned advancement of our cortisol modulation platform. It is gratifying to see our discovery program, which has identified so many promising compounds, mature into a development program with the potential to produce treatments for a wide range of serious diseases."

"The breadth of the clinical program we’re building is impressive," added Robert S. Fishman, M.D., Corcept’s Chief Medical Officer. "In the coming months, we expect results from our Phase 2A trial of mifepristone in combination with eribulin to treat TNBC. CORT125134, which has already produced promising pre-clinical and Phase 1 results, has entered Phase 1/2 as a treatment for patients with a range of solid-tumor cancers and this quarter we plan for it to start Phase 2 as a treatment for Cushing’s syndrome. Equally exciting, more selective cortisol modulators are advancing towards the clinic. These compounds have shown promise in animal models of a wide range of serious diseases, including oncologic disorders, fatty liver disease, antipsychotic induced weight gain and alcoholism."

Financial Discussion

Corcept’s GAAP net loss in the first quarter of 2016 was $19,000, compared to a GAAP net loss of $4.8 million in the first quarter of 2015. Excluding non-cash expenses related to stock-based compensation and accreted interest on the company’s capped royalty obligation (the "Royalty Financing"), Corcept generated $2.2 million of non-GAAP net income in the first quarter, compared to a non-GAAP net loss of $2.7 million in the first quarter of 2015. A reconciliation of GAAP to non-GAAP net operating results is set forth below

Operating expenses for the first quarter increased to $15.5 million, from $14.1 million in the first quarter of 2015, primarily due to increases in patient support costs, compensation and professional services costs associated with our expanded field sales force, the additional distribution expense resulting from higher sales volumes, and increased spending on the clinical development of CORT125134.

Corcept’s cash and cash equivalents totaled $40.7 million as of March 31, 2016, compared to $40.4 million as of December 31, 2015. These cash balances reflect Corcept’s scheduled payments due under the Royalty Financing. Pursuant to the terms of the agreement, Corcept paid $3.0 million in the first quarter of 2016, compared to $2.8 million in the fourth quarter of 2015. Corcept expects to make its final payment under the Royalty Financing in 2017.

Conference Call

Corcept will hold a conference call on May 3, 2016, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time) to discuss this announcement. To participate, dial 1-888-771-4371 from the United States or 1-847-585-4405 internationally approximately 10 minutes before the start of the call. The passcode is 42398569. A replay will be available through May 17, 2016 at 1-888-843-7419 from the United States and 1-630-652-3042 internationally. The passcode is 42398569.

About Cushing’s Syndrome

Endogenous Cushing’s syndrome is caused by prolonged exposure of the body’s tissues to high levels of the hormone cortisol and is generated by tumors that produce cortisol or ACTH. Cushing’s syndrome is an orphan indication that most commonly affects adults aged 20-50. An estimated 10-15 of every one million people are newly diagnosed with this syndrome each year, resulting in over 3,000 new patients annually in the United States. An estimated 20,000 patients in the United States have Cushing’s syndrome. Symptoms vary, but most people have one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Cushing’s syndrome can affect every organ system in the body and can be lethal if not treated effectively.

About Triple-Negative Breast Cancer (TNBC)

TNBC is a form of the disease in which the three receptors that fuel most breast cancer growth – estrogen, progesterone and the HER-2/neu gene – are not present. Because the tumor cells lack the necessary receptors, treatments that target estrogen, progesterone and HER-2 receptors are ineffective. In 2013, approximately 40,000 women were diagnosed with TNBC. We estimate that more than 75 percent of these women’s tumor cells expressed the GR receptor to which cortisol binds. There is no FDA-approved treatment and neither a targeted treatment nor an approved standard chemotherapy regimen for relapsed TNBC patients exists.

About Korlym

Korlym modulates the effect of cortisol at GR, one of the two receptors to which cortisol binds, thereby inhibiting the effects of excess cortisol in patients with Cushing’s syndrome. Since 2012, Corcept has made Korlym available as a once-daily oral treatment of hyperglycemia secondary to endogenous Cushing’s syndrome in adult patients with glucose intolerance or diabetes mellitus type 2 who have failed surgery or are not candidates for surgery. Korlym was the first FDA-approved treatment for that illness and the FDA has designated it as an Orphan Drug for that indication.

About CORT125134

CORT125134 is the lead compound in Corcept’s portfolio of selective cortisol modulators. It is a non-steroidal competitive antagonist of GR that does not bind to the body’s other hormone receptors, including the progesterone receptor. It is the affinity of Korlym for the progesterone receptor that results in termination of pregnancy and can cause endometrial thickening and irregular vaginal bleeding in some women. CORT125134 will not have these effects. The compound is proprietary to Corcept and is protected by composition of matter and method of use patents extending into 2033.

On May 03, 2016 Stemline Therapeutics, Inc. (Nasdaq:STML) reported that it has completed the initial dosing cohort in its SL-801 Phase 1 advanced solid tumor trial (Press release, Stemline Therapeutics, MAY 3, 2016, View Source [SID:1234511819]). The second cohort is currently open. SL-801 is a novel, oral, small molecule that reversibly inhibits the XPO1 (Exportin-1) nuclear transport protein, a clinically validated target active in a wide variety of cancer types.

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This Phase 1 dose escalation trial will enroll up to 50 patients with advanced solid tumors at multiple centers in the U.S. The trial is designed to evaluate safety, identify an optimal dosing regimen, and detect potential signals of efficacy in one or more cancer types.

Ivan Bergstein, M.D., Stemline’s Chief Executive Officer, stated, "We are very pleased to have dosed the first patients in our SL-801 program during the first quarter, in-line with our expectations, and recently cleared the first dosing cohort. We are continuing to enroll patients with a wide range of solid cancer types in this trial in order to identify specific indications with both sensitivity to the agent as well as viable registration pathways. We believe we can leverage the unique properties of this compound along with our knowledge of the XPO1 space to create significant value from the program early on."

On May 3, 2016 Kancera AB reported their interim results for Q1 2016, January 1 – March 31, 2016 (Press release, Kancera, MAY 3, 2016, View Source;releaseID=1143395 [SID:1234511821]).

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The period January to March 2016 in brief

R&D expenses for the period amounted to SEK 4.2m (SEK 4.2m).
Operating income for the period amounted to SEK -5.1m (SEK 4.9m).
Income after financial items for the period amounted to SEK -5.0m (SEK -4.9m).
Earnings per share for the period were SEK -0.05 (SEK -0.05).
Cash flow from operating activities for the period amounted to SEK -6.3m (SEK -5.1m).
Equity as of March 31, 2016 amounted to SEK 17.0m (SEK 22.6m) or SEK 0.16 (SEK 0.22) per share. The equity/assets ratio as of March 31, 2016 was 70 percent (71 percent).
Cash and cash equivalents as of March 31, 2016 amounted to SEK 12.1m (SEK 17.8m).
Significant events during the period

Kancera has from the 1st of January 2016 extended the lease of the company’s laboratories within the Karolinska Science Park for three years through an agreement with Humlegården Fastigheter.
Kancera has provided an update of the small molecule patent portfolio.
– A patent covering small molecule PFKFB3 inhibitors has been approved in the USA.

– A patent application covering new chemical series in the HDAC6 project has been filed.

– An international patent application covering ROR inhibitors has been strengthened by adding examples of additional highly potent ROR inhibitors.

Kancera reported that the company has developed a new series of ROR inhibitors that show improved pharmaceutical properties which will allow preclinical studies of their effect on e.g. solid tumors. These results have prompted Kancera to concentrate the investments in the ROR project to small molecule inhibitors and terminate the product development of a ROR-based vaccine. Furthermore, Kancera reported results from the Fractalkine project showing that KAN0440567 after oral administration to mice effectively blocks the function of the Fractalkine receptor.
Kancera announced that the company according to plan has received another payment of about SEK 2.8 million in January, 2016 from the EU for the A-PARADDISE project, which aims to develop drugs against parasitic diseases.
Significant events after the end of the reporting period

Kancera announced that the Extraordinary General Meeting of Kancera AB approved the Board’s decision of 6 April 2016 to issue new shares and warrants in the form of units. The decision includes a preferential rights issue of shares and warrants (units) ("New Share Issue"), which upon full subscription brings Kancera about SEK 52 million before issue expenses and with a full exercise of the warrants brings Kancera an additional SEK 52-62 million. The issue assets will be used for Kancera’s drug development, clinical studies and the further development of the Company’s capacity to commercialize products. The majority of Kancera’s resources are now concentrated on taking at least one of Kancera’s drug candidates in the ROR and Fractalkine projects to clinical trial for chronic lymphocytic leukemia and pancreatic cancer, respectively. In parallel, the Company intends to validate a broader use of the drug candidates from these projects in order to demonstrate their full commercial potential.
Kancera reported that ROR inhibitors have been tested against human triple negative breast cancer transferred to zebra fish. The experiments showed that Kancera’s small molecule ROR inhibitors are able to both reduce tumor size and metastases (spread) of this aggressive tumor form. Further, Kancera reported that the company´s PFKFB3 inhibitors are active in the same model of triple negative breast cancer and that a patent application has been filed covering the discovery that PFKFB3 inhibitors enhance the effect of radiation treatment
Kancera reported that the Company due to positive efficacy data in disease models of cancer and pain has decided to exercise the exclusive option to acquire the Fractalkine project. The acquisition will be carried out in connection with the completion of the ongoing transfer of results and know-how from Acturum and AstraZeneca to Kancera. Payment for the project to Acturum Life Science AB will be made into three steps by a total of 6 million shares, of which the first payment is due at the submission of the application for authorization of a clinical trial after an approval by Kancera´s shareholders. In parallel, the company intends to validate a broader use of the drug candidate (KAN0440567) in order to demonstrate its full commercial potential.
Statement from the CEO

In 2016, we have reported progress in the development of a new generation of ROR inhibitors with properties that we expect will enable effect against both lymphoma and solid tumors. A first study where human triple negative breast cancer was transplanted into zebra fish, shows that ROR inhibitors are able to both inhibit tumor growth and prevent spread. Further, we have been able to validate that Kancera PFKFB3 inhibitor is able to inhibit tumor growth in the same model.

On the patent side, we have completed an international patent application in the ROR project with the addition of approximately 100 selected potent compounds. Also, a patent application covering PFKFB3 substances has been approved in the United States and a new application covering the discovery that Kancera’s PFKFB3 inhibitor counteract cancer by enhancing the effect of radiation treatment has been submitted. An additional patent application has been filed in the HDAC6 project covering a new class of active substances which together with the first patent application in the project, broadens the protection around our unique selective and effective compounds against myeloma.

Furthermore, due to positive efficacy data in disease models of cancer and pain, the Kancera Board has decided to exercise the exclusive option to acquire the Fractalkine project.

The goal for the next 18-24 months is to take at least one of Kancera’s drug candidates from the Fractalkine and ROR projects to clinical trials and thus first clinical use (chronic lymphocytic leukemia/ pancreas cancer). In parallel, we also complete the evaluation of a broader use of the drug candidates from these projects in order to reduce the risk in the product development and to identify full commercial potential of the projects. The operational objectives also include delivering drug candidates from the HDAC6 and PFKFB3 projects.

Given these ambitious goals, Kancera’s Board supported by the decision of the Extraordinary General Meeting on April 22, 2016, decided to launch a new share issue. The decision includes a preferential rights issue of shares and warrants (units) which upon full subscription brings Kancera about SEK 52 million before issue costs.

With eight patent families, which together make up the intellectual property protection for four small-molecule cancer projects, we expect that Kancera has good opportunities to translate scientific results into commercially attractive new drugs against cancer. However, the development of new drugs is biologically and technically risky and challenged by international competition.

This share issue aims to push Kancera’s cancer project from a promising development phase to completion in order to be tested in cancer patients. I am convinced that we will succeed in this work.

On May 4, 2016 Innate Pharma SA (the "Company" – Euronext Paris: FR0010331421 – IPH) reported its revenues and cash position for the first three months of 2016 (Press release, Innate Pharma, MAY 3, 2016, View Source [SID:1234511888]).

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Cash, cash equivalents and financial instruments of the Company amounted to €255.8 million as of March 31, 2016, including short term investments (€51.2m) and non-current financial instruments (€37.5m). At the same date, financial liabilities amounted to €3.6 million.

Revenues for the first three months of 2016 amounted to €5.7 million (€0.4 million for the same period in 2015). This revenue results from Innate Pharma’s collaboration and licensing agreement with Bristol-Myers Squibb and co-development and commercialization agreement with AstraZeneca:

€5.5 million resulting from the agreement with AstraZeneca, corresponding to the recognition over the period of the initial payment received in June 2015;
€0.2 million relating to the agreement with Bristol-Myers Squibb resulting from the recognition over the period of the upfront payment received in July 2011.
In 2015, revenue for the first three months resulted mainly from the recognition of the upfront payment from the agreement with Bristol-Myers Squibb.

Hervé Brailly, Chief Executive Officer and co-founder of Innate Pharma, commented: "During the first quarter of 2016, our pipeline of innovative immuno-oncology candidates continued to progress and expand. At the AACR (Free AACR Whitepaper) 2016 Annual Meeting, we presented data supporting the development of our clinical and preclinical programs, including two new programs targeting the tumor microenvironment. We are proud of these programs which further demonstrate our unique positioning in immuno-oncology.

At the same time, our most advanced programs moved forward in their clinical development. A Phase I clinical trial was started by AstraZeneca that is testing our first-in-class NKG2A checkpoint inhibitor, monalizumab, in combination with durvalumab (AstraZeneca/ MedImmune’s PD-L1 inhibitor). This fifth trial completes the roll-out of the initial clinical plan, due to start reading out in 2017. With regards to lirilumab, our first-in-class anti-KIR checkpoint inhibitor, the DSMB completed its sixth assessment of the Phase II EffiKIR study in March and recommended continuation of the trial without modification. Data on the primary endpoint are expected in the second half of 2016."

On May 3, 2016 Cerus Corporation (NASDAQ:CERS) reported financial results for the first quarter ended March 31, 2016 (Press release, Cerus, MAY 3, 2016, View Source [SID:1234511827]).

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Recent company highlights include:
Entered into long-term INTERCEPT platelet and plasma supply agreement with the American Red Cross, the largest U.S. supplier of blood components.

Entered into framework agreement with Blood Centers of America (BCA) as its pathogen reduction technology supply partner. Fifteen BCA members are now under contract for use of the INTERCEPT Blood System.

Received U.S. Food and Drug Administration (FDA) approval for use of the INTERCEPT Blood System for platelets suspended in 100% plasma, expanding the potential market for INTERCEPT in the U.S.

Pathogen reduction obviates the need for both primary and secondary bacterial screening under FDA’s revised draft guidance for controlling the risk of bacterial contamination of platelets.

Signed INTERCEPT supply agreement with Banco de Sangre de Servicios Mutuos to help sustain local platelet and plasma collections during the Zika epidemic.

FDA Zika guidance document recognizes pathogen reduction as a method to reduce transfusion risk and maintain local platelet and plasma collections in areas of active Zika transmission.

"Concerns about both the Zika virus and bacterial contamination are stimulating U.S. interest in INTERCEPT from both industry and regulators," said William ‘Obi’ Greenman, Cerus’ president and chief executive officer. "We now have a significant portion of the U.S. market under contract for INTERCEPT and will be working diligently to support these customers as they begin their validation processes. Once customers have completed their validations over the next few quarters, we expect the revenue contribution from the U.S. to begin in earnest."

Revenue
Revenue for the first quarter of 2016 was $7.6 million and relatively flat compared to the prior year. Because revenue for the three months ended March 31, 2016 and 2015, was predominantly driven by Euro denominated markets, reported revenue was negatively affected by an approximate 2% weakening of the Euro compared to the U.S. dollar, the Company’s reporting currency. The Company continues to expect 2016 global revenue in the range of $37 million to $40 million.

Gross Margins
Gross margins for the first quarter of 2016 were 44%, compared to 39% for the first quarter of 2015. Margins for the first quarter of 2016 were positively impacted by the decline in the value of the Euro relative to the Company’s reporting currency, the U.S. dollar, lifting gross margins when comparing the first quarter of 2016 to the comparable period in 2015. In addition, product mix helped improve reported gross margins with higher margin platelet kits contributing proportionately more than in the prior year.

Operating Expenses
Total operating expenses for the first quarter of 2016 were $18.7 million, compared to $17.3 million for the first quarter of 2015. Selling, general and administrative expenses were relatively flat, driven by increased 2016 U.S. commercialization costs which were offset by lower accounting and other administrative fees. Research and development expenses increased as a result of activities to support our platelet label claim extension efforts, required post marketing platelet studies in the U.S. and preparation of the anticipated 2016 CE Mark submission for the red blood cell system.

Operating and Net Loss
Operating losses during the first quarter of 2016 were $15.3 million, compared to $14.4 million for the first quarter of 2015.
Net loss for the first quarter of 2016 was $16.9 million, or $0.17 per diluted share, compared to a net loss of $9.5 million, or $0.17 per diluted share, for the first quarter of 2015.

Net losses for the first quarter of 2016 were favorably impacted by approximately $1.0 million of lower foreign exchange losses during the first quarter of 2016, when compared to the corresponding prior period. Net losses in the prior year period were positively impacted by the mark-to-market adjustments of the Company’s previously outstanding warrants to fair value, which resulted in non-cash gains of $6.3 million during the first quarter of 2015. The Company has no remaining outstanding warrants and as such, does not expect mark-to-market adjustments going forward.

Cash, Cash Equivalents and Investments
At March 31, 2016, the Company had cash, cash equivalents and short-term investments of $96.4 million compared to $107.9 million at December 31, 2015. The Company’s short-term investments include a marketable equity security which was valued at $5.1 million at March 31, 2016 and $11.2 million at December 31, 2015.

At March 31, 2016, the Company had approximately $20 million in outstanding debt under its loan agreement with Oxford Finance.