On January 28, 2016 AbbVie (NYSE: ABBV), in cooperation with Neurocrine Biosciences, Inc. (NASDAQ: NBIX), reported the initiation of the first of two planned Phase 3 clinical studies evaluating the safety and efficacy of Elagolix alone or in combination with add-back therapy compared to placebo (Press release, Neurocrine Biosciences, JAN 28, 2016, View Source;p=RssLanding&cat=news&id=2137247 [SID:1234509041]). These studies are designed to assess the change in menstrual blood loss utilizing the alkaline hematin method, comparing baseline to month six of treatment. Additional secondary efficacy endpoints are being evaluated; including assessing changes in fibroid volume, monthly blood loss and hemoglobin levels. Bone mineral density will also be assessed.

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"There are limited, non-surgical treatment options for women suffering from heavy menstrual bleeding associated with uterine fibroids. AbbVie is eager to further explore Elagolix’s potential to address this unmet need," said Michael Severino, M.D., executive vice president, research and development and chief scientific officer, AbbVie.

The Elagolix Phase 3 uterine fibroid clinical development program is part of AbbVie’s pipeline and includes two replicate, randomized, parallel, double-blind, placebo-controlled clinical trials. Each trial is expected to enroll approximately 400 subjects for an initial six-month placebo-controlled dosing period, after which, subjects who are eligible will have an option to continue for an additional six-month dosing period in a safety and efficacy extension study. AbbVie will make a $15MM milestone payment to Neurocrine Biosciences upon enrollment of the first patient.

Uterine fibroids (also called leiomyomas or myomas) are noncancerous muscle tissue tumors of the uterus.1 Fibroids are most common in women aged 30-40 years but can occur at any age.1 They can range in size from nearly undetectable to bulky masses that can distort the uterus.2 Fibroids can be asymptomatic but in some women cause symptoms such as: longer, more frequent, or heavy menstrual bleeding; menstrual pain; vaginal bleeding at time other than menstruation; pain in the abdomen or lower back; pain during sex; difficulty urinating; frequent urination; constipation or rectal pain.1

About Elagolix
Elagolix is an orally administered gonadotropin-releasing hormone (GnRH) antagonist that is currently being investigated in diseases that are mediated by sex hormones, such as uterine fibroids and endometriosis. To date, Elagolix has been studied in over 40 clinical trials totaling more than 3,000 subjects. Phase 3 trials of Elagolix for the management of endometriosis-associated pain are also ongoing.

On January 28, 2016 Sophiris Bio Inc. (NASDAQ: SPHS) (the "Company" or "Sophiris"), a biopharmaceutical company developing PRX302 (topsalysin) for the treatment of urological diseases, reported the biopsy data at 6 months for the first seven patients to complete the Phase 2a proof-of-concept study in localized prostate cancer (Filing, 8-K, Sophiris Bio, JAN 28, 2016, View Source [SID:1234508931]).

A review of the biopsy data from the first seven men to complete the study showed that four patients experienced a response to treatment: One patient experienced complete ablation of the tumor where no evidence of the treated tumor remained on a targeted biopsy at 6 months; three patients experienced either a reduction in the maximum cancer core length or a reduction in Gleason pattern; three patients had no response to treatment.

"This new trial is very exciting — we have promising data showing that topsalysin can ablate cancer cells and we look forward to reviewing the results from the remaining 11 patients as they complete the study. We could be on the cusp of a new class of therapeutics for the focal treatment of localized prostate cancer," stated Professor Mark Emberton, Dean, Faculty of Medical Sciences, University College London and Honorary Consultant Urologist University College London Hospital NHS Foundation Trust.

Dr. Hashim Ahmed, Principal Investigator for the study, Division of Surgery and Interventional Sciences, University College London, said, "Topsalysin could offer a tissue-sparing cancer treatment that carries little in the way of side effects. This treatment has the potential to help men avoid radical treatments such as radiation therapy or complete removal of the prostate."

This one-time administration of topsalysin directly into a pre-identified clinically significant tumor appears to be well tolerated with no serious adverse events and no new safety signals being reported. This is consistent with safety observed in the 365 patients that have been treated with topsalysin in the Company’s BPH program to date.

"The biological activity that we have observed further validates the mechanism of action of topsalysin. We are gaining valuable experience on how we might best optimize both the delivery and dose of topsalysin based on lesion size, and the remaining patients to complete the study will help in that assessment," said Dr. Ahmed.

The ongoing Phase 2a proof of concept study is a single-center, open-label study at University College London, which is well known for the focal treatment of prostate cancer in the UK. In this study, previously obtained multiparametric magnetic resonance images (mpMRIs) of each patient’s prostate tumor lesions are mapped to real-time three-dimensional transrectal ultrasound. These images are used to guide the injection of topsalysin to treat a single, histologically-proven, clinically significant prostate cancer lesion. The primary objective of the study is safety and tolerability, and the key efficacy variable is the change in the treated lesion on targeted biopsy after 6 months. The study is designed to assess whether topsalysin has the potential to provide patients with clinically significant, localized, low to intermediate risk prostate cancer a tissue-sparing cancer treatment that carries little in the way of side effects. A total of 18 patients were enrolled and treated in this study. Sophiris expects to have final data on all patients by the end of the second quarter of 2016.

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On January 29, 2016 Kyowa Hakko Kirin Co., Ltd. (Tokyo 4151; President and CEO: Nobuo Hanai, "Kyowa Hakko Kirin") reported that it will change the name of all its western pharmaceutical subsidiaries, using "Kyowa Kirin" as a common company brand name (Press release, Kyowa Hakko Kirin, JAN 28, 2016, View Source [SID:1234508911]). Every subsidiary will begin officially operating under this new company trade name at various points during 2016.

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Kyowa Hakko Kirin is expanding its business globally and, as shown in its business vision, "Kyowa Hakko Kirin will be a Japan-based Global Specialty Pharmaceutical Company contributing to human health and well-being worldwide through innovative drug discovery and global commercialization, driven by state-of-the art antibody technologies mainly in the core therapeutic areas of oncology, nephrology and immunology". The company plans to launch late stage development products in the US and Europe in this mid-term business plan. Kyowa Hakko Kirin’s decision to unify all of its western pharmaceutical subsidiaries under one name, "Kyowa Kirin", is in pursuit of its aim to become a Global Specialty Pharmaceutical Company.

"I am convinced that a unified brand name will strengthen interaction and integration in our group." said Nobuo Hanai, Ph.D., President and CEO of Kyowa Hakko Kirin. "This will assist us in leaping forward as a Global Specialty Pharmaceutical Company, creating innovations across the group’s various business bases."

The Kyowa Hakko Kirin Group companies strive to contribute to the health and well-being of people around the world by creating new value through the pursuit of advances in life sciences and technologies.

On January 28, 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 preliminary financial results for the quarter and year ended December 31, 2015 (Press release, Corcept Therapeutics, JAN 28, 2016, http://www.corcept.com/news_events/view/pr_1454017543 [SID:1234508902]). The company also provided an update

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Preliminary 2015 Financial Results; 2016 Revenue Guidance

Corcept reported preliminary revenue of $15.0 million for the fourth quarter of 2015 and $50.3 million for the full year. Preliminary GAAP net income for the fourth quarter of 2015 was $0.01 per share, compared to a net loss of $0.04 per share in the fourth quarter of 2014. For the full year, the company reported a preliminary GAAP net loss of $0.06 per share for 2015, compared to a net loss of $0.31 per share in 2014. The company’s cash and cash equivalents were $40.4 million at year-end, an increase of $4.0 million from September 30, 2015.

The company estimates 2016 revenue will be $76-81 million.

"Our Korlym revenue grew 89 percent last year. We expect significant growth in 2016 and beyond, as there are still many patients who could benefit from the medication and have not received it," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer. "The continued growth in our Cushing’s syndrome business, in conjunction with our lean business model, allowed us to generate our first GAAP profit last quarter and will be sufficient to fund our planned development activities."

2016 Clinical and Pre-Clinical Development

"Our clinical and pre-clinical pipeline will expand significantly in 2016," said Robert S. Fishman, MD, Corcept’s Chief Medical Officer. "Our Phase 1/2 trial of mifepristone with eribulin to treat triple-negative breast cancer will generate efficacy and safety results around mid-year."

"We also plan to begin two Phase 2 studies of our next-generation, selective cortisol modulator, CORT125134, by the end of the first quarter," Dr. Fishman said. "And we are advancing additional selective cortisol modulators towards Phase 1, including CORT118335, a compound that has shown promise in animal models of non-alcoholic fatty liver disease."

Dr. Fishman also noted the importance to Corcept’s development program of its collaborations with independent researchers. "We have more than thirty pre-clinical and clinical studies underway with academic investigators around the world. Their work has deepened our understanding of cortisol modulation’s therapeutic potential and is invaluable as we select new development targets. Our oncology program, for example, is based on pioneering work by University of Chicago researchers in the study of cortisol modulators, both mifepristone and our next-generation compounds, as potential treatments for TNBC, ovarian and prostate cancer."

Mifepristone for the Treatment of Triple-Negative Breast Cancer

Corcept is investigating whether mifepristone, the active ingredient in Korlym, will enhance the effect of eribulin in patients with TNBC. At the San Antonio Breast Cancer Symposium in December 2015, Corcept presented preliminary data from its Phase 1/2 trial’s efficacy phase. Enrollment is ongoing.

Two Phase 2 Trials of CORT125134

CORT125134 is a next-generation, selective cortisol modulator. It was well-tolerated in its Phase 1 trial, which showed that the compound shares Korlym’s ability to modulate activity at the glucocorticoid receptor (the essential quality in treating Cushing’s syndrome). Unlike Korlym, CORT125134 is not active at the progesterone receptor and so does not terminate pregnancy or cause other side effects associated with progesterone receptor antagonism. 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. Corcept has submitted INDs to the FDA covering CORT125134’s use in two Phase 2 trials – one to treat patients with Cushing’s syndrome and another for the treatment of patients with a range of solid tumors.

Conference Call

Corcept will hold a conference call on January 28, 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 will be 4156 8920.

A replay will be available through February 12, 2016 at 1-888-843-7419 from the United States and 1-630-652-3042 internationally. The passcode will be 4156 8920.

About Korlym

Korlym modulates the effect of cortisol at the glucocorticoid receptor (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 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

Triple-negative breast cancer 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. It is estimated 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.