On May 03, 2016 Supernus Pharmaceuticals, Inc. (NASDAQ:SUPN), a specialty pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported financial results for first quarter 2016 and associated company developments (Press release, Supernus, MAY 3, 2016, View Source [SID:1234511895]).

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Commercial Update

First quarter 2016 product prescriptions for Trokendi XR and Oxtellar XR, as reported by IMS, totaled 114,773, a 49.7% increase over the first quarter of 2015.

Prescriptions
Q1 2016 Q1 2015 Change %

Trokendi XR 85,987 55,227 55.7 %
Oxtellar XR 28,786 21,463 34.1 %

Total 114,773 76,690 49.7 %

Source Data: Product prescriptions as reported by IMS

Total revenue for the first quarter of 2016 was $43.1 million, a 53.1% increase over $28.1 million in the same period last year. Total revenue for both periods consisted almost exclusively of net product sales.

Net Product Sales ($mil.)
Q1 2016 Q1 2015 Change %

Trokendi XR $ 32.3 $ 20.9 54.5 %
Oxtellar XR $ 10.7 $ 7.2 49.3 %

Total $ 43.0 $ 28.1 53.1 %

Consistent with industry trends and the Company’s experience in the first quarter last year, product prescriptions and net product sales for Trokendi XR and Oxtellar XR were unfavorably impacted by the reset of insurance coverage at the beginning of the year, with high deductible and high co-pay programs. Additionally, changes in wholesaler inventory levels and ordering patterns during the first quarter of 2016 had the effect of reducing net product sales by approximately $3 million in the first quarter of 2016.

"Our business continues to post strong growth compared to last year. Total first quarter IMS prescriptions, and net product sales grew by 50% or more compared to the same period last year. We continue to build on the momentum we finished with last year promoting the benefits of our products to patients with epilepsy," said Jack Khattar, President and CEO of Supernus Pharmaceuticals.

Regarding our supplemental new drug application requesting approval to expand the Trokendi XR label to include treatment of migraine in adults, we are ready to launch the new migraine indication once we receive full approval from the FDA.

Progress of Product Pipeline

Enrollment continues for both Phase III trials for SPN-810, which is currently in development for Impulsive Aggression in patients who have ADHD, and for the Phase IIb trial for SPN-812, currently in development for ADHD. The Company continues to expect Phase III data for SPN-810 to be available by mid 2017, and data from the SPN-812 Phase IIb trial to be available by early 2017.

"In addition to our recent findings on the favorable emerging clinical profile of SPN-812 as it relates to adverse events, we completed the evaluation of the cardiac effects portion of the single ascending and multiple ascending dose study. We are pleased to report that there was no clinically significant change in QT interval and other ECG parameters. We believe these additional safety data in adult healthy volunteers, which show a lack of cardiac effects, are very encouraging and further strengthen the differentiation of SPN-812," said Jack Khattar.

Collaboration Update

Shire recently announced positive results of SHP465 in a safety and efficacy study in children and adolescents with ADHD. The study addresses a key U.S. Food and Drug Administration (FDA) requirement, keeping SHP465 on track for resubmission in the fourth quarter of 2016 and a potential launch in the second half of 2017, if it is approved by the FDA. SHP465 was originally developed by Shire Laboratories, the former division of Shire that subsequently became Supernus Pharmaceuticals. Based on the agreement between Supernus and Shire, Shire will pay to Supernus a single-digit percentage royalty on net sales of the product.

Operating Expenses

Research and development expenses in the first quarter of 2016 were $10.6 million, as compared to $3.7 million in the same quarter the prior year. This increase is primarily due to the ongoing Phase III testing of SPN-810 and Phase IIb testing of SPN-812.

Selling, general and administrative expenses in the first quarter of 2016 were $25.2 million, as compared to $19.4 million in the same quarter the prior year. The increase is primarily due to the continued increase in our sales and marketing efforts for both Trokendi XR and Oxtellar XR and the efforts in preparing for the launch of the migraine indication for Trokendi XR.

Operating Income and Earnings Per Share

Operating income in the first quarter of 2016 was $5.3 million, an increase of 55.0% over operating income of $3.4 million in the same period last year. This improvement in operating income is primarily due to the increase in net product sales.

Diluted earnings per share were $0.08 in the first quarter ended March 31, 2016, compared to $0.02 in the same period last year.

Weighted-average diluted common shares outstanding were approximately 51.2 million in the first quarter of 2016, as compared to approximately 44.9 million in the same period the prior year.

Capital Resources

As of March 31, 2016, the Company had $114.0 million in cash, cash equivalents, marketable securities, and long term marketable securities, as compared to $117.2 million at December 31, 2015. As of March 31, 2016, approximately $6.6 million of the Company’s six year, $90 million notes, bearing interest at 7.5% per annum, remain outstanding.

Financial Guidance

For full year 2016, the Company reiterates its expectation that net product sales will range from $200 million to $210 million, R&D expenses to range from $55 million to $65 million, and operating income to range from $28 million to $35 million.

On May 3, 2016 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) reported financial results for the first quarter ended March 31, 2016 (Press release, Rigel, MAY 3, 2016, View Source;p=RssLanding&cat=news&id=2164508 [SID:1234511894]).

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"We look forward to our upcoming Phase 3 data as we continue with our planning for the potential commercial launch of fostamatinib in the United States," said Raul Rodriguez, president and chief executive officer of Rigel. "Also, we initiated the Phase 2 proof-of-concept study with fostamatinib in autoimmune hemolytic anemia (AIHA). We anticipate that the results of these studies as well as the IgA nephropathy study will be forthcoming later this year," he added.

During the first quarter, Rigel announced that patient enrollment was completed for the two studies in the FIT Phase 3 clinical program of fostamatinib in immune thrombocytopenic purpura (ITP). The results from the first study are expected in the middle of 2016, with the results for the second study expected shortly thereafter. Rigel plans to submit a New Drug Application to the Food and Drug Administration in the first quarter of 2017, subject to the results of the program. In addition, Rigel is in the early stages of establishing its sales and marketing infrastructure for the commercial launch of fostamatinib.

For the first quarter of 2016, Rigel reported a net loss of $17.5 million, or $0.19 per share, compared to a net loss of $18.2 million, or $0.21 per share, in the first quarter of 2015.

Contract revenues from collaborations of $5.0 million in the first quarter of 2016, compared to $2.2 million in the first quarter of 2015, were comprised of the amortization of the $30.0 million upfront payment and FTE fees earned pursuant to Rigel’s collaboration and license agreement with Bristol-Myers Squibb.

Rigel reported total costs and expenses of $22.6 million in the first quarter of 2016, compared to $20.4 million in the first quarter of 2015. The increase in costs and expenses was primarily due to the increase in research and development costs related to Rigel’s clinical research programs with fostamatinib in ITP and AIHA.

As of March 31, 2016, Rigel had cash, cash equivalents and short-term investments of $103.6 million, compared to $126.3 million as of December 31, 2015. Rigel expects this amount to be sufficient to fund operations into the third quarter of 2017.

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."

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 3, 2016 Foundation Medicine, Inc. (NASDAQ:FMI) reported the launch of FoundationACT (Assay for Circulating Tumor DNA), an analytically validated and accurate blood-based circulating tumor DNA (ctDNA) assay that provides patients and oncologists with a new option for comprehensive genomic profiling when a tissue biopsy is not feasible or when tissue is not available (Press release, Foundation Medicine, MAY 3, 2016, View Source [SID:1234511842]). By analyzing circulating tumor DNA isolated from a patient’s blood, FoundationACT can identify clinically relevant genomic alterations, and like Foundation Medicine’s tissue-based genomic profiles, FoundationOne and FoundationOne Heme, FoundationACT delivers this comprehensive molecular information in a concise report that matches the findings with potentially relevant targeted therapies and clinical trials.

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"Our goal has always been to deliver a highly accurate and sensitive assay available for blood based samples, and the confirmatory data produced by our clinical collaborators indicates that we have succeeded in achieving that with the release of FoundationACT," said Vincent Miller, M.D., chief medical officer of Foundation Medicine. "Bringing the rigor used to develop and validate FoundationOne to this assay, we believe we have overcome many of the clinical limitations presented by other tests in the liquid biopsy field. Launching FoundationACT strengthens our market leading position as the go-to resource for a complete, end-to-end offering of comprehensive molecular information solutions."

The presence of ctDNA, which is DNA shed from tumors that circulates in blood, is a well-established phenomenon that has led to the development of non-invasive tumor sequencing assays. However, the concentration of ctDNA compared to other DNA fragments derived from other tissue sources can vary significantly depending on tumor type and disease stage. For many cancer patients, this means that the proportion of detectable tumor DNA in the blood is extremely low, making the detection of therapeutically relevant genomic alterations much more difficult and error prone as compared to tissue-based approaches.

FoundationACT, which was launched to Foundation Medicine’s pharmaceutical partners for research use in December 2015, interrogates all clinically relevant alterations across 62 genes and fusions across six genes, and it has been optimized for sensitivity and specificity of all classes of molecular alterations, including base substitutions, insertions and deletion, focal amplifications and gene fusions. The assay was developed for patients who are not candidates for comprehensive genomic profiling with FoundationOne. Factors that may preclude patients from undergoing FoundationOne testing include insufficient or inadequate tissue from a recent biopsy, safety risks associated with biopsy, or medical contraindications to re-biopsy. In July 2015, Foundation Medicine initiated a large-scale, multi-center prospective clinical study to clinically validate FoundationACT across various cancers and stages of the disease. A portion of the patients in the study will include those with earlier-stage disease, allowing the company to investigate how different tumor types shed DNA into the bloodstream at different stages of the disease.

Technical Validation Data Confirms Accuracy, Sensitivity of FoundationACT Genomic Profiling Assay

At the Annual Meeting of the Advances in Genome Biology and Technology (AGBT) in February 2016, Foundation Medicine reported data from its analytic validation study in a presentation titled, "Assessment of the Relative Clinical Utility of ctDNA and Tissue Biopsies for the Detection of Actionable Genomic Alterations in Routine Clinical Oncology Specimens." Study highlights demonstrated that FoundationACT results were 100 percent concordant with FoundationOne and droplet digital PCR (ddPCR) results across 87 base substitutions (43 at < 5% MAF), three indels and five genomic alterations.

At the American Association of Cancer Research Annual Meeting in April 2016, Foundation Medicine further reported analytical validation data in a poster presentation titled, "Rigorous Validation of a Clinical Circulating Tumor DNA Assay for Cancer Molecular Profiling." As part of this study, results compared alterations from patient-matched ctDNA and FFPE biopsies across more than 200 samples from lung, breast and other cancers. This rigorous analytic validation study demonstrated ≥99 percent sensitivity in the detection of alterations present in blood at low frequency with a very low rate of false positives, realizing the potential of ctDNA-based molecular profiling for the management of patients with cancer. In 48 clinical ctDNA samples, 95 alterations of all classes were 100 percent confirmed by orthogonal testing.