From Dr. Theodore Friedman: Cushings-Help.com Announces: The Availability Of An Investigational Drug For Severe Cushing’s Syndrome On a Compassionate Use Basis
We would like to make patients aware that mifepristone, an investigational drug that blocks the action of cortisol and is being developed by Corcept Therapeutics Incorporated, is now available on a compassionate use basis for eligible patients in the United States with Cushing’s syndrome who have no other treatment options.
Under this compassionate use program, the FDA allows seriously ill patients who lack satisfactory alternative treatment options to use an investigational new drug that is still under development. Corcept has completed a Phase III trial investigating the safety and efficacy of mifepristone in patients with endogenous Cushing’s syndrome. The information from that study has been submitted to the FDA for review of safety and efficacy.. For information on the trial results see http://www.corcept.com/cushings_clinical_trials. The company has submitted a New Drug Application (NDA) seeking approval for this drug.
Patients interested in using mifepristone should consult with their endocrinologist. Their endocrinologist, in turn, should contact Corcept for information about the compassionate use program. Please note that Corcept will provide information solely to physicians.
Toll Free: 1-877-367-6550
Corcept Therapeutics: Clinical Development and Trials
Hong Kong (HKSAR) - The Department of Health (DH) is today (November 30) investigating a suspected case of Cushing's syndrome involving an eight-year-old girl who patronised Mr Chan Kwok-wing of PCRC Chinese Medicine Clinic in Mongkok for management of allergic conditions.
The case was reported to the DH by the Hospital Authority. The girl, who had history of eczema and allergic rhinitis, consulted Chan for around two months since mid-September 2011 and was supplied with some pills, including pills in orange and black colour respectively, and green capsules and green tablets.
"The girl has stopped taking the pills after noting DH's announcement on November 28 of a previous incident related to Chan involving a seven-year-old patient who developed features compatible with steroid overdose after taking pills supplied by Chan," a spokesman said. However, her mother started to notice that the girl had some features of obesity and moon face.
The girl was admitted to the Prince of Wales Hospital today where the girl was found to have moon face, truncal obesity and recent weight gain.
Her clinical diagnosis was iatrogenic Cushing's syndrome. Cushing's syndrome can be caused by steroid overdose. The patient is now in stable condition.
The spokesman added, "Investigation of the present case, including testing of the pills for adulteration of western medicines, is in progress,"
The spokesman again appealed to members of the public, who patronised Chan for management of allergy and was supplied with a type of green and another type of orange oral tablets, to consult healthcare professionals for advice as soon as possible.
"Chan is suspected of practising Chinese medicines without licence as there is no record to show that he is either a registered medical practitioner or a pharmacist.
DH is assisting with Police investigation," the spokesman said.
Source: HKSAR Government
David Unuane, MD (Doctor), Department of Endocrinology, Universitair Ziekenhuis Brussel, UZ Brussel, Vrije Universiteit Brussel, Brussels, Belgium, Herman Tournaye, MD, PhD (Professor, Doctor), Brigitte Velkeniers, MD, PhD (Professor, Doctor), Kris Poppe, MD, PhD (Professor, Doctor)
Female infertility occurs in about 37% of all infertile couples and ovulatory disorders account for more than half of these. The ovaries are in continuous interaction with the other endocrine organs. The interplay may account for infertility occurring at different levels and may render the diagnosis of infertility a difficult exercise for the involved physician. A hypothalamic cause of female infertility should be considered in an appropriate clinical context, with tests pointing to a hypogonadotropic hypogonadism. It can be functional, physiological or related to organic causes. Hyperprolactinemia has well characterized effects on the normal gonadal function and treatment is well established.
Acromegaly and Cushing’s disease may impair fertility at different levels, mechanisms involved however remain ill defined. Thyroid disorders, both hyperthyroidism and hypothyroidism, can interact with the ovaries, through a direct effect on ovarian function, but autoimmunity may be involved, as well as alterations of the sex hormone binding protein levels. Primary ovarian disorders, such as the polycystic ovary syndrome and primary ovarian insufficiency are frequent diseases, for which novel treatments are currently being developed and discussed.
We will propose an algorithm for the diagnosis and approach of the female patient presenting with infertility on the basis of the available evidence in literature.
Keywords: female infertility, pituitary, adrenal, ovarian, thyroid
(Ivanhoe Newswire) – Cushing’s disease is a hormone disorder that causes many symptoms, such as high blood pressure, fat accumulation, osteoporosis and ultimately ending in death. It is caused by a tumor in the anterior pituitary gland that secretes excess amounts of adrenocorticotrophic hormone (ACTH). The only treatment is removal of the tumor, however, researchers have found a new treatment for these reoccurring tumors.
Researchers, led by Shlomo Melmed, at Cedars-Sinai Medical Center, Los Angeles, have now identified a potential new therapeutic target -- the protein EGFR, which is the target of a drug used to treat some patients with non–small cell lung cancer (gefitinib). As discussed by Melmed and colleagues in their paper, as well as Frederic Wondisford, at Johns Hopkins University School of Medicine, Baltimore, in an accompanying commentary, the data generated in human, canine, and mouse models provide strong support to investigate the clinical effects of gefitinib in patients with Cushing disease.
SOURCE: Journal of Clinical Investigation, published online November 21, 2011
Keywords Nasal endoscopy – Pituitary adenoma – Complication – Surgical skill
Source: Johns Hopkins Medicine
FOR IMMEDIATE RELEASE
Newswise — Infertility is common among obese women, but the reasons remain poorly understood and few treatments exist. Now a team of Johns Hopkins Children's Center scientists, conducting experiments in mice, has uncovered what they consider surprising evidence that insulin resistance, long considered a prime suspect, has little to do with infertility in women with type-2 diabetes, polycystic ovary syndrome (PCOS) and metabolic syndrome, all obesity-related conditions in which the body becomes desensitized to insulin and loses the ability to regulate blood sugar.
In a report, published online Nov.10 in the journal Diabetes, the Johns Hopkins scientists say the real culprit appears to be insulin sensitivity in the ovaries and the pituitary.
The Johns Hopkins team said its findings show that these organs escape insulin resistance and, awash with high levels of circulating insulin common in obesity, develop abnormal cell signaling that disrupts ovulation and eventually leads to infertility.
"Our findings suggest that the focus should shift from treating insulin resistance in peripheral tissue to taming insulin sensitivity in the pituitary and ovaries," says lead investigator Sheng Wu, Ph.D., of the Johns Hopkins Children's Center. Scientists traditionally have treated obesity-induced infertility by lowering blood insulin to counter the effects of insulin resistance.
A 2010 study by the same team discovered that the pituitary gland, insensitive to insulin in lean mice, became sensitive to elevated levels of insulin seen in human and rodent obesity. By knocking out the insulin receptors in the pituitary glands of obese mice, the researchers were able to partially restore fertility, thus proving that abnormal insulin signaling in the pituitary was only part of the story.
"In the original study, disrupting insulin signaling in the pituitary restored 50 percent of fertility in obese mice, but the search was on for the accomplice," says senior investigator Andrew Wolfe, Ph.D., an endocrinologist at the Johns Hopkins Children's Center. "Our new findings point to the ovaries."
In the pituitary, faulty insulin signaling stimulates increased secretion of luteinizing hormone, the researchers say. In the ovary, it puts testosterone production into overdrive. Both disrupt ovulation, the researchers explain.
In the latest study, lean mice and mice made obese on a three-month high-fat diet received injections of progressively higher doses of insulin to mimic the effects of high circulating insulin seen in obesity, diabetes and PCOS. In lean mice, the ovaries and pituitaries were insensitive to the hormone at low-dose injections, and responded only when injected with higher doses of insulin. The "trigger" doses corresponded to insulin levels typically seen in obesity. Obese mice with naturally elevated insulin levels exhibited high levels of insulin signaling in their pituitary and ovarian cells. When injected with insulin, the livers and muscles of obese mice showed greatly reduced response to insulin -- or insulin resistance. Their ovaries and pituitary glands, however, responded to insulin injections, confirming that in obese mice, these reproductive organs escape the insulin resistance seen in other organs.
To determine insulin sensitivity, the researchers focused on two signaling proteins, IRS-1 and IRS-2, regulators of cell-insulin communication involved in the development of insulin resistance in liver and muscle tissue. The scientists hypothesized that in the pituitary and ovaries, these messenger proteins would remain dormant under normal insulin levels, but would get activated once exposed to high levels of insulin. Indeed, the researchers found, the pituitary glands of obese mice showed higher IRS-2 signaling activity compared with lean mice, while the ovaries of obese mice had higher signaling activity in both IRS-1 and IRS-2 proteins, compared with lean mice.
In a follow-up study now under way, the Hopkins team is trying to determine whether knocking out the insulin receptors in both the ovaries and the pituitary would fully restore fertility in obese mice with high insulin levels.
Other co-investigators on the study included Sara Divall, M.D., and Fred Wondisford, M.D., both of the Johns Hopkins Children's Center.
The research was funded by the Endocrine Fellow Foundation, by The Eunice Kennedy Shriver National Institute of Child Health and Human Development, part of the National Institutes of Health, and by the Baltimore Diabetes Research and Training Center, which is supported by the National Institute for Diabetes and Digestive and Kidney Diseases.
Founded in 1912 as the children's hospital of the Johns Hopkins Medical Institutions, the Johns Hopkins Children's Center offers one of the most comprehensive pediatric medical programs in the country, treating more than 90,000 children each year. Hopkins Children's is consistently ranked among the top children's hospitals in the nation.
Hopkins Children's is Maryland's largest children's hospital and the only state-designated Trauma Service and Burn Unit for pediatric patients. It has recognized Centers of Excellence in dozens of pediatric subspecialties, including allergy, cardiology, cystic fibrosis, gastroenterology, nephrology, neurology, neurosurgery, oncology, pulmonary, and transplant. For more information, please visitwww.hopkinschildrens.org
By Lisa Smyth
A Northern Ireland man has defied medical predictions and overcome the effects of a rare condition that left him in a coma for three months.
Jonathan Fisher is a survivor of an Addisonian crisis which affected him so badly doctors feared he would never recover.
His mother ignored medical advice to switch off his life-support system as doctors believed he was brain dead.
However, convinced he could recover after noticing that he was reacting to her with the occasional slight movement of his little finger she refused to give up on her son.
Now — against all the odds — he has made a remarkable recovery and has regained many of the skills lost as a result of his condition.
Addison’s disease is a rare disorder of the adrenal glands. It affects the production of two hormones — cortisol and aldosterone — which help to regulate blood pressure.
If left untreated, the amount of steroid hormones in the body will gradually fall and the symptoms of Addison’s disease will get progressively worse.
Eventually, this will cause an adrenal crisis — when the symptoms become very severe and blood pressure drops to a dangerously low level. An adrenal crisis can be fatal if it is not treated immediately.
Jonathan can now get around Lisburn in his electric wheelchair and has even managed to complete his first book, August Always — a triumph as he has considerable speech impairment and great difficulty using his hands to operate a keyboard.
The process has been long and laborious but Jonathan said he was determined to share his experience with others.
“There are moments in life that define us, like birth and death,” he said.
“Along the journey there will be crises of passion, of love, of faith and desire, but none so devastating as an Addisonian crisis. I am Jonathan Fisher, a survivor.
“August Always is my memoir. I believe in the incredible. I dream of a better future.”
Addison’s disease is a rare disorder of the adrenal glands which are located on top of the kidneys. The condition affects the production of two hormones. Cortisol, which helps to regulate blood pressure, maintaining blood glucose and heart function. And aldosterone which also helps regulate blood pressure. Addison’s sufferers must get treatment if their blood pressure falls as it can be fatal.
1Department of Endocrinology, University Hospitals Birmingham NHS Foundation Trust, Birmingham, B15 2TH, United Kingdom
2 Experimental and Clinical Endocrinology, MedizinischeKlinik I, RatzeburgerAllee 160, D-23538, Lübeck, Germany
3Department. of Endocrinology, CliniquesUniversitaires Saint-Luc, Avenue Hippocrate, 54.74, 1200 Brussels
4Department of Women's and Children's Health, Uppsala University, Uppsala, Sweden
5Department of Medical Endocrinology, Rigshospitalet, Copenhagen, 2100, Denmark
6KIMS Pfizer Endocrine Care, Specialty Business Unit, Pfizer Health AB, Sollentuna, 190 91, Sweden
7Department of Neurosurgery, University of Erlangen Nuernberg, Erlangen, 91045, Germany
8 Pfizer Endocrine Care Europe, Tadworth, United Kingdom
9Neuroendocrine Unit, Massachusetts General Hospital, Harvard Medical School, 55 Fruit Street, Boston, MA 02114, United States
Objective: To determine whether the ITT, arginine (AST) and glucagon stimulation tests (GST) identify patients who have similar features of GH deficiency using a diagnostic threshold of 3 μg/l.
Patients and Methods: 5453 tests were available from 4,867 patients registered in the KIMS database (49.9% females, ITT = 3111, AST = 1390, GST = 952). Comparisons were made for GH peak, BMI, lipids, waist circumference, waist:hip ratio and quality of life (QoL-AGHDA questionnaire).
Results. There were significant (p<0.0001) intra-individual correlations between the GH peaks for the ITT vs AST (r = 0.655), ITT vs GST (r = 0.445) and AST vs GST (r = 0.632). GH peaks in response to all tests were negatively correlated to the number of additional pituitary hormone deficiencies, and positively correlated to IGF-I SDS. BMI had a negative influence on all three tests.
Comparing GHD patients according to the diagnostic test used, most clinical variables did not differ between the groups. The only exceptions showing any difference were BMI being slightly higher in the AST and GST groups, triglyceride levels increased in the GST group, and IGF-I SDS was lower in the ITT and AST than in the GST group. Waist circumference was larger and quality of life was worse in the GST group than in the other groups.
Conclusions. This study demonstrates that the ITT, AST and GST produce similar GH peaks, are influenced by similar clinical factors and identify patients with similar features of GH deficiency at a diagnostic threshold of 3 μg/L.
Diabetes mellitus is a frequent complication of Cushing syndrome (CS) which is caused by chronic exposure to glucocorticoid excess, either endogenous or exogenous, and that is characterized by several clinical symptoms such as central obesity, purple striae, proximal muscle weakness, acne, hirsutism and neuropsychological disturbances.
Diabetes occurs as a consequence of an insulin-resistant state together with impaired insulin secretion which are induced by glucocorticoid excess. The management of patients with CS and diabetes mellitus includes the treatment of hyperglycemia and, when possible, the correction of glucocorticoid excess.
This review focuses on the disorders of glucose metabolism in patients exposed to glucocorticoid excess, addressing both the pathophysiological aspects and the clinical and therapeutic implications.
Read the entire article at http://www.cell.com/trends/endocrinology-metabolism/abstract/S1043-2760(11)00138-X
Something new of interest to Cushies most every day. Please note that there is a current backlog of about three weeks for submitted bios to be added to the website.
November 10, 2011:
November 5, 2011:
November 2, 2011:
HELSINGBORG, Sweden and EXTON, Pa., Nov. 7, 2011 /PRNewswire/ -- The Swedish specialty pharma company, DuoCort Pharma, announced today that the European Commission has granted a European Marketing Authorisation for Plenadren®(hydrocortisone, modified release tablet), an orphan drug for treatment of adrenal insufficiency in adults, bringing these patients their first pharmaceutical innovation in over 50 years.
Developed by DuoCort Pharma, Plenadren® is a dual release hydrocortisone replacement therapy designed to better mimic the normal physiological cortisol profile in order to improve outcomes for patients suffering from adrenal insufficiency. Plenadren® is given as an oral tablet once daily. It has an outer layer releasing hydrocortisone immediately and an inner core releasing the rest of the drug more slowly during the day.
Although glucocorticoid hormone replacement therapy for adrenal insufficiency has been available for decades, studies have recorded complications and comorbidities including premature death, impaired quality of life, increased risk of cardiovascular diseases, and decreased bone mineral density in treated patients, most likely because it is difficult to match the natural secretion pattern of cortisol.
Maria Forss, CEO of DuoCort Pharma, said: "The marketing authorization for Plenadren® in Europe is an important step towards addressing the unmet needs of these patients."
The approval of Plenadren® follows the positive opinion adopted by the Committee for Medicinal Products for Human Use (CHMP) in July 2011. Plenadren® is now approved for marketing in all countries of the European Union (EU) as well as in the European Economic Area (EEA), namely Iceland, Norway and Lichtenstein.
Professor Gudmundur Johannsson of the Department of Endocrinology, Sahlgrenska University Hospital, Gothenburg, Sweden, and Chief Medical Officer of DuoCort Pharma, said: "Plenadren® offers a welcome new treatment option to help patients suffering from adrenal insufficiency. Plenadren® can improve therapy for many of the almost 200,000 patients in Europe who suffer from this disease and who need life-long cortisol replacement therapy for their survival."
On October 26, 2011, ViroPharma Incorporated (NASDAQ: VPHM) signed a definitive agreement to acquire DuoCort Pharma AB. The companies expect to complete the acquisition in November 2011. On closing, ViroPharma will pay an upfront closing cost of 220 million Swedish kroner (SEK) or $33 million in US dollars (USD). Additionally, there are contingent milestone payments of up to 860 million SEK or $130 million USD associated with manufacturing, sales thresholds and territory expansion.
About Adrenal insufficiency
Adrenal insufficiency (cortisol deficiency) is a rare, life-threatening disease that affects patients in their active years. To survive, patients suffering from this disease need lifelong replacement therapy with hydrocortisone. Treatment of adrenal insufficiency involves replacing, or substituting, the hormones that the patient's own adrenal glands are not producing. Cortisol is replaced using hydrocortisone, the synthetic form of cortisol.
About Plenadren® (hydrocortisone, modified release tablet)
Plenadren is the first true innovation in over 50 years in the treatment of adrenal insufficiency.
Hypersensitivity to the active substance of Plenadren or to any of the excipients may occur. During acute adrenal insufficiency, parenteral administration of hydrocortisone in high doses, together with physiological sodium chloride solution for injection, must be given. Use of Plenadren with potent CYP 3A4 inducers and inhibitors may merit an adjustment of hydrocortisone dosage. High (supra-physiological) dosages of cortisone can cause elevation of blood pressure, salt and water retention, and increased excretion of potassium. Long-term treatment with higher than physiological hydrocortisone doses can lead to clinical features resembling Cushing's syndrome with increased adiposity, abdominal obesity, hypertension and diabetes, and thus result in an increased risk of cardiovascular morbidity and mortality. All glucocorticoids increase calcium excretion and reduce the bone remodeling rate. Patients with adrenal insufficiency on long term glucocorticoid replacement therapy have been found to have reduced bone mineral density. Psychiatric adverse events may occur with systemic glucocorticoids.
The most common adverse reactions observed in clinical studies have been fatigue, gastroenteritis, upper respiratory tract infection, sedation, vertigo and dry eyes.
About ViroPharma Incorporated
ViroPharma Incorporated is an international biopharmaceutical company committed to developing and commercializing novel solutions for physician specialists to address unmet medical needs of patients living with diseases that have few if any clinical therapeutic options, including C1 esterase inhibitor deficiency, treatment of seizures in children and adolescents, and C. difficile infection (CDI). Our goal is to provide rewarding careers to employees, to create new standards of care in the way serious diseases are treated, and to build international partnerships with the patients, advocates, and health care professionals we serve. ViroPharma's commercial products address diseases including hereditary angioedema (HAE), seizures in children and adolescents, and CDI; for full U.S. prescribing information on our products, please download the package inserts athttp://www.viropharma.com/Products.aspx; the prescribing information for other countries can be found atwww.viropharma.com.
ViroPharma routinely posts information, including press releases, which may be important to investors in the investor relations and media sections of our company's web site, www.viropharma.com. The company encourages investors to consult these sections for more information on ViroPharma and our business.
About DuoCort Pharma
DuoCort Pharma is a drug development company focused on improving glucocorticoid therapy. The company has its origins among researchers at the Sahlgrenska Academy at Gothenburg University and at Uppsala University in Sweden. DuoCort Pharma has developed Plenadren®, an improved glucocorticoid replacement therapy for patients with adrenal insufficiency, which is a rare disease. DuoCort Pharma has orphan drug designations in EU, Switzerland and the USA for Plenadren®. Plenadren® is a once daily, dual-release hydrocortisone oral tablet. It has an outer layer that releases the drug immediately and an inner core that releases the drug over the day. The tablets come in both 5 mg and 20 mg strengths. For more information please visit www.duocort.com.
DuoCort Pharma is a project company of the life science incubator PULS. For more information visit www.pulsinvest.se.
Certain statements in this press release contain forward-looking statements that involve a number of risks and uncertainties. Forward-looking statements provide our current expectations or forecasts of future events, including statements about the benefits of the business combination transaction involving ViroPharma and DuoCort Pharma, including, among others, future financial and operating results, enhanced revenues, ViroPharma's plans, objectives, expectations and intentions and other statements that are not historical facts. The following factors, among others, could cause actual results to differ from those set forth in the forward-looking statements: the ability to achieve the other conditions to closing on the proposed schedule; the risk that the business will not be integrated successfully; the risk that revenues following the acquisition will be lower than expected, including the successful commercialization of Plenadren; potential for disruption from the transaction making it more difficult to maintain relationships with manufacturers, employees or other suppliers; competition and its effect on pricing, spending, third-party relationships and revenues; our ability to achieve favorable pricing for Plenadren from European regulatory authorities; the risk that the safety and/or efficacy results of existing clinical trials for Plenadren will not be consistent with the results of additional clinical studies, including the required registry study, or with commercial usage; market acceptance of Plenadren; and our inability to maintain the orphan drug status associated with Plenadren. These factors, and other factors, including, but not limited to those described in our annual report on Form 10-K for the year ended December 31, 2010 and quarterly reports on Form 10-Q filed with the Securities and Exchange Commission, could cause future results to differ materially from the expectations expressed in this press release. The forward-looking statements contained in this press release are made as of the date hereof and may become outdated over time. ViroPharma does not assume any responsibility for updating any forward-looking statements. These forward looking statements should not be relied upon as representing our assessments as of any date subsequent to the date of this press release.
SOURCE DuoCort Pharma
BACKGROUND: Bilateral adrenalectomy (BLA) is a treatment option to alleviate symptoms in patients with ectopic Cushing's syndrome (ECS) for whom surgical treatment of the responsible nonpituitary tumor is not possible. ECS patients have an increased risk for complications, because of high cortisol levels, poor clinical condition, and metabolic disturbances. This study aims to evaluate the safety and long-term efficacy of endoscopic BLA for ECS.
METHODS: From 1990 to present, 38 patients were diagnosed and treated for ECS in the Erasmus University Medical Center, a tertiary referral center. Twenty-four patients were treated with BLA (21 endoscopic, 3 open), 9 patients were treated medically, and 5 patients could be cured by complete resection of the adrenocorticotropic hormone (ACTH)-producing tumor. The medical records were retrospectively reviewed and entered into a database. For evaluation of the efficacy of BLA, preoperative biochemical and physical symptoms were assessed and compared with postoperative data.
RESULTS: Endoscopic BLA was successfully completed in 20 of the 21 patients; one required conversion to open BLA. Intraoperative complications occurred in two (10%) patients, and postoperative complications occurred in three (14%) patients. Median hospitalization was 9 (2-95) days, and median operating time was 246 (205-347) min. Hypercortisolism was resolved in all patients. Improvements of hypertension, body weight, Cushingoid appearance, impaired muscle strength, and ankle edema were achieved in 87, 90, 65, 61, and 78% of the patients, respectively. Resolution of diabetes, hypokalemia, and metabolic alkalosis was achieved in 33, 89, and 80%, respectively.
CONCLUSION: Endoscopic BLA is a safe and effective treatment for patients with ectopic Cushing's syndrome.
MENLO PARK, CA, Nov 07, 2011 (MARKETWIRE via COMTEX) -- Corcept Therapeutics Incorporated , a pharmaceutical company engaged in the discovery, development and commercialization of drugs for the treatment of severe metabolic and psychiatric disorders, today reported financial results for the quarter ended September 30, 2011, and updated its corporate progress.
"Following the acceptance by the U.S. Food and Drug Administration (FDA) of our New Drug Application (NDA) for the use of our lead product candidate, Korlym(TM), in Cushing's Syndrome," said Joseph Belanoff, M.D., Chief Executive Officer of Corcept, "we continue to focus our efforts on building our commercial capabilities to support the launch of Korlym, if Korlym is approved by the FDA, in order to allow us to provide an important treatment option to patients suffering from Cushing's Syndrome."
Corporate and Development Highlights
-- Received notification in October 2011 that the FDA had accepted our proposed brand name, Korlym (formerly referred to as CORLUX(R)), for our lead product candidate in the treatment of endogenous Cushing's Syndrome. -- Advanced our commercial launch preparations related to Korlym for the treatment of Cushing's Syndrome, including developing our internal infrastructure and engaging third-party vendors to provide market analytics and to support distribution and other logistical needs in the event Korlym is approved by the FDA. -- Received notification in October 2011 that the European Commission had granted Korlym Orphan Designation for the treatment of endogenous Cushing's Syndrome (hypercortisolism) in the European Union (EU). Benefits of Orphan Drug Designation in the EU are similar to those in the U.S., but include ten years of marketing exclusivity in all 27 member states, free scientific advice during drug development, access to a centralized review process and a reduction or complete waiver of fees levied by the European Medicines Agency. -- Enrolled additional patients in our double-blind placebo controlled Phase 3 trial of Korlym for the treatment of the psychotic features of psychotic depression. -- Continued the clinical portion of our Phase 1b/2a multi-dose safety and proof of concept studies of CORT 108297, one of our selective GR-II antagonists. -- Identified additional compounds from among our proprietary series of selective GR-II antagonists to advance toward an Investigational New Drug submission.
Third Quarter Financial Results
For the third quarter of 2011, Corcept reported a net loss of $6.4 million, or $0.08 per share, compared to a net loss of $7.1 million, or $0.10 per share, for the third quarter of 2010.
In the third quarter of 2011, research and development expenses decreased to $3.2 million from $5.2 million in the third quarter of 2010. This decrease in research and development expenses was due primarily to decreases in clinical trial costs related to drug-drug interaction and other NDA-supportive studies with Korlym, which were substantially completed in late 2010, and decreases in the clinical trial costs related to the Phase 1b/2a studies with CORT 108297. These decreases were partially offset by increased costs associated with the prosecution of our NDA for Korlym for the treatment of Cushing's Syndrome. General and administrative expenses increased to $3.2 million for the third quarter of 2011 from $1.9 million for the same period in 2010 due primarily to additional expenditures on commercialization activities for the potential launch of Korlym for Cushing's Syndrome.
Our cash balance as of September 30, 2011 was $45.9 million, up from $24.6 million at December 31, 2010. "We anticipate that our current cash balance is sufficient to fund the company through the end of 2012," said Charles Robb, the company's Chief Financial Officer.
Anticipated Activities for the Remainder of 2011
We continue to concentrate our efforts on advancing Korlym toward approval and commercialization for the treatment of Cushing's Syndrome. We also continue our efforts to be prepared to respond in a timely fashion to any questions posed by the FDA during the course of their review of our NDA.
"We are focused intently on developing the commercial and logistical capabilities we will need to make Korlym available to patients suffering from Cushing's Syndrome, should the FDA approve our drug for this indication," added Dr. Belanoff. "Korlym is the first step in unlocking the value of our scientific platform. The regulation of cortisol is a critical biological function; its dysregulation is equally critical in many important disease states. Our own research and research from increasing numbers of academic investigators point to the potential importance of cortisol antagonism in a wide variety of diseases. We believe our expanding library of selective cortisol antagonists may help address these unmet medical needs."
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 which most commonly affects adults aged 20 to 50. An estimated 10 to 15 of every one million people are newly diagnosed with this syndrome each year, resulting in over 3,000 new patients 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 Psychotic Depression
Psychotic depression is a serious psychiatric disorder that affects approximately three million people annually in the United States. It is more prevalent than either schizophrenia or bipolar I disorder. The disorder is characterized by severe depression accompanied by delusions, hallucinations or both. People with psychotic depression are approximately 70 times more likely to commit suicide than the general population and often require lengthy and expensive hospital stays. There is no FDA-approved treatment for psychotic depression.
About Weight Gain Caused by Antipsychotic Medications
The group of medications known as second-generation antipsychotics, including olanzapine (Zyprexa), risperidone (Risperdal), quetiapine (Seroquel) and clozapine (Clozaril), are widely used to treat schizophrenia and bipolar disorder. All medications in this group are associated with treatment emergent weight gain of varying degrees and also carry warning labels relating to treatment emergent hyperglycemia and diabetes mellitus. There is no FDA-approved treatment for the weight gain associated with the use of antipsychotic medications.
Corcept's first-generation compound, Korlym, also known as mifepristone, directly blocks the cortisol (GR-II) receptor and the progesterone (PR) receptor. Intellectual property protection is in place to protect important methods of use for Korlym. Corcept retains worldwide rights to its intellectual property related to Korlym.
About CORT 108297
CORT 108297 is a potent, selective antagonist of the cortisol (GR-II) receptor that we have discovered and for which Corcept owns worldwide intellectual property rights. In in vitro binding affinity and functional assays this compound has no affinity for the progesterone (PR), estrogen (ER), androgen (AR) or mineralocorticoid (GR-I) receptors.
About Corcept Therapeutics Incorporated
Corcept is a pharmaceutical company engaged in the discovery, development and commercialization of drugs for the treatment of severe metabolic and psychiatric disorders. The company has completed its Phase 3 study of Korlym for the treatment of Cushing's Syndrome, and has an ongoing Phase 3 study of Korlym for the treatment of the psychotic features of psychotic depression. Corcept also has a Phase 2 program for CORT 108297, a selective GR-II antagonist that blocks the effects of cortisol but not progesterone. Corcept has developed an extensive intellectual property portfolio that covers the use of GR-II antagonists in the treatment of a wide variety of psychiatric and metabolic disorders, including the prevention of weight gain caused by the use of antipsychotic medication, as well as composition of matter patents for our selective GR-II antagonists.
Statements made in this news release, other than statements of historical fact, are forward-looking statements, including, for example, statements relating to the potential benefit of Korlym for patients diagnosed with Cushing's Syndrome, Corcept's clinical development and research programs, the outcome of the FDA's review of our NDA filing, our estimates for our capital requirements and needs for additional financing, the introduction of Korlym and future product candidates, including CORT 108297, the ability to create value from Korlym or other future product candidates or our scientific platform and our commercialization plans. Forward-looking statements are subject to a number of known and unknown risks and uncertainties that might cause actual results to differ materially from those expressed or implied by such statements. For example, there can be no assurances with respect to the cost, rate of spending, completion or success of clinical trials; financial projections may not be accurate; there can be no assurances that Corcept will pursue further activities with respect to the development of Korlym, CORT 108297, or any of its other selective GR-II antagonists. These and other risk factors are set forth in the Company's SEC filings, all of which are available from our website ( www.corcept.com ) or from the SEC's website ( www.sec.gov ). We disclaim any intention or duty to update any forward-looking statement made in this news release.
CORCEPT THERAPEUTICS INCORPORATED CONDENSED BALANCE SHEETS (in thousands) September 30, December 31, 2011 2010 -------------- -------------- (Unaudited) (Note) ASSETS: Current assets: Cash and cash equivalents $ 45,909 $ 24,578 Other current assets 427 418 -------------- -------------- Total current assets 46,336 24,996 Other assets 43 108 -------------- -------------- Total assets $ 46,379 $ 25,104 ============== ============== LIABILITIES AND STOCKHOLDERS' EQUITY: Current liabilities: Accounts payable $ 1,066 $ 817 Other current liabilities 1,647 3,043 -------------- -------------- Total current liabilities 2,713 3,860 Total stockholders' equity 43,666 21,244 -------------- -------------- Total liabilities and stockholders' equity $ 46,379 $ 25,104 ============== ============== Note: Derived from audited financial statements at that date. CORCEPT THERAPEUTICS INCORPORATED CONDENSED STATEMENTS OF OPERATIONS (in thousands, except per share amounts) (Unaudited) For the Three Months Ended For the Nine Months Ended September 30, September 30, -------------------------- -------------------------- 2011 2010 2011 2010 ------------ ------------ ------------ ------------ Operating expenses: Research and development* $ 3,228 $ 5,224 $ 14,355 $ 14,286 General and administrative* 3,209 1,881 8,049 5,327 ------------ ------------ ------------ ------------ Total operating expenses 6,437 7,105 22,404 19,613 ------------ ------------ ------------ ------------ Loss from operations (6,437) (7,105) (22,404) (19,613) Interest and other income, net 3 4 3 758 Other expense (1) (3) (17) (18) ------------ ------------ ------------ ------------ Net loss $ (6,435) $ (7,104) $ (22,418) $ (18,873) ============ ============ ============ ============ Basic and diluted net loss per share $ (0.08) $ (0.10) $ (0.27) $ (0.28) ============ ============ ============ ============ Shares used in computing basic and diluted net loss per share 84,188 72,045 83,000 66,982 ============ ============ ============ ============ *Includes non-cash stock-based compensation of the following: Research and development $ 110 $ 45 $ 432 $ 170 General and administrative 844 500 1,971 1,361 ------------ ------------ ------------ ------------ Total non-cash stock-based compensation $ 954 $ 545 $ 2,403 $ 1,531 ============ ============ ============ ============
CONTACT: Charles Robb Chief Financial Officer Corcept Therapeutics 650-688-8783 Email Contact www.corcept.com
SOURCE: Corcept Therapeutics