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Intercept Pharmaceuticals, Inc.

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Mark Pruzanski, M.D., CEO & President, commented on the 90 day extension for the OCA PDUFA data. He stated, "I would like to touch briefly on the announcement we made in December regarding the FDA notification of a 90-day extension on our PDUFA date. Just to reiterate, the extension was the result of FDA's request to us for additional clinical data analysis based on our completed studies with no new clinical studies requested. We maintained a very productive and constructive relationship with the FDA and look forward to continued interactions with the agency over the coming weeks as both the Intercept team and the agency prepare for the planned advisory committee meeting in April."
Source: Q4 2015 earnings conference call, 2/22/16.

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FDA Extends PDUFA Date for Obeticholic Acid for the Treatment of PBC

NEW YORK, Dec. 17, 2015 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT), a clinical stage biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic, underserved liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for its Priority Review of obeticholic acid (OCA) in primary biliary cirrhosis, recently renamed primary biliary cholangitis (PBC). In response to an information request from the FDA, additional clinical data analyses have been submitted. To provide time for a full review of the submission, the original PDUFA date of February 29, 2016 has been extended by three months, resulting in a new PDUFA date of May 29, 2016. The FDA has also notified Intercept of a planned advisory committee meeting date of April 7, 2016.

Intercept is seeking approval of OCA for the treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.
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Source: press release, 12/17/15. http://ir.interceptpharma.com/releasedetail.cfm?ReleaseID=947530

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Mark Pruzanski, M.D., CEO & President, commented on the upcoming PDUFA date for OCA. HE stated, "In August, we announced that the FDA granted priority review for OCA in PBC, with a PDUFA date of February 29, 2016. We are pleased that the FDA has been very engaged with us in the review process and we continue to expect and are preparing for an advisory committee meeting early next year. Keep in mind that OCA, if approved, will be the first new medicine for PBC in 20 years. We are also continuing our regulatory process in Europe and continue to plan for approval and launch in the second half of 2016."
Source: Q3 2015 earnings conference call, 11/09/15.

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FDA Grants Priority Review for Intercept's Obeticholic Acid for the Treatment of Primary Biliary Cirrhosis

NEW YORK, Aug. 31, 2015 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept), a clinical stage biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic underserved liver diseases, today announced the U.S. Food and Drug Administration (FDA) has accepted for review the company's New Drug Application and granted Priority Review for obeticholic acid (OCA) for the treatment of primary biliary cirrhosis (PBC).

OCA is being developed to treat PBC patients with an inadequate therapeutic response to, or who are unable to tolerate, ursodeoxycholic acid (UDCA), the only drug currently approved to treat PBC. The FDA has set a target date of February 29, 2016 to take action under the Prescription Drug User Fee Act (PDUFA).

"Despite current treatment, PBC remains a leading cause of liver transplant among women, so there is a clear ongoing high unmet medical need for new therapies for patients with PBC," said Mark Pruzanski, M.D., President and Chief Executive Officer. "Priority review designation accelerates the FDA review timelines, potentially bringing Intercept closer to its goal of delivering the first new medicine for PBC in close to 20 years. We look forward to working with FDA to bring OCA to PBC patients in need as soon as possible."

The FDA grants Priority Review to medicines that, if approved, would be a significant improvement in the safety or effectiveness of treatment, prevention or diagnosis of serious diseases. A priority review designation means FDA's goal is to take action on the marketing application within six months of acceptance as compared to 10 months under standard review. OCA was previously granted Fast Track designation by FDA. A drug development program with Fast Track designation is afforded greater access to FDA for the purpose of expediting the drug's development, review and potential approval.
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Source: press release, 8/31/15. http://ir.interceptpharma.com/releasedetail.cfm?ReleaseID=929359

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Intercept Pharmaceuticals Submits Applications in the U.S. and Europe for Marketing Approval of Obeticholic Acid for the Treatment of Primary Biliary Cirrhosis

- First Primary Biliary Cirrhosis Medication Submitted for Regulatory Review in Nearly Two Decades -

NEW YORK, June 29, 2015 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept), a clinical stage biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic underserved liver diseases, today announced the achievement of two important regulatory milestones for obeticholic acid (OCA) in primary biliary cirrhosis (PBC): submission of a New Drug Application (NDA) for accelerated approval to the U.S. Food and Drug Administration (FDA) and acceptance of the Marketing Authorization Application (MAA) by the European Medicines Agency (EMA). Intercept is seeking approval of OCA for the treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.

UDCA is currently the only approved medication for the treatment of PBC and is the standard of care for all PBC patients. However, a majority of patients continue to experience persistent elevations above the upper limit of normal in the serum marker alkaline phosphatase (ALP), corresponding with increased risk of liver failure, need for liver transplant and death. Thus, there continues to be a critical need for new treatments for patients with PBC.

The NDA and MAA submissions include a total of 1,507 subjects exposed to at least a single dose of OCA. Of these subjects, 432 were patients with PBC, with 290 treated for at least six months and 232 treated for at least one year. The key efficacy and safety data are derived from three randomized double-blind, placebo-controlled clinical trials in patients with PBC evaluating the effect of OCA on ALP and bilirubin. All three trials met their primary endpoints with high statistical significance and improvements were seen in secondary endpoints including markers of liver injury, immunity, inflammation and apoptosis. OCA treatment was generally well-tolerated, with primarily mild or moderate pruritus being the most common adverse event.

The regulatory submissions are also supported by two clinical databases that include more than 10,000 patients from the Global PBC Study Group and UK-PBC Group, both independently confirming that achieving lower ALP and/or bilirubin levels is significantly correlated with increased transplant-free survival.

"Over the past several years, the medical community has gained a deeper appreciation of the extent of the unmet medical need in PBC," said Mark Pruzanski, M.D., President and Chief Executive Officer. "In each of our PBC clinical trials, OCA has demonstrated the ability to rapidly and sustainably lower ALP and improve bilirubin levels, both when added to UDCA and as monotherapy. If approved, we believe OCA will become an important new treatment option that will help patients with PBC."

OCA has received orphan drug designation for PBC in both the United States and Europe and fast track designation for PBC in the United States. In December 2014, Intercept initiated its rolling NDA submission for accelerated approval of OCA in PBC and began a Phase 3b confirmatory clinical outcomes trial in PBC, in accordance with FDA accelerated approval regulations. This trial is anticipated to enroll approximately 350 patients with advanced PBC at 150 centers in more than 20 countries, and is expected to be completed on a post-marketing basis.
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Source: press release, 6/29/15. http://ir.interceptpharma.com/releasedetail.cfm?ReleaseID=919926

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Mark Pruzanski, M.D., CEO & President, commented on the progress being made for the regulatory filings for Obeticholic acid (OCA) in PBC . He stated, "First item, I'd like to cover today is our program in primary Biliary Cirrhosis or PBC. Our supporting studies are proceeding on track and we continue to anticipate completing our NDA and MAA filings in the first half of 2015. We will continue to work closely with FDA and MAA and are preparing for pre-NDA and pre-MAA meetings that we anticipate will take place in the fourth quarter 2014. We believe that the successful outcome of these meetings will give us an increased confidence in our regulatory strategy for OCA and PBC and facilitates for filing our NDA and MAA."
Source: Q2 2014 earnings conference call, 8/11/14.

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Intercept Announces that FDA Grants Fast Track Designation to Obeticholic Acid for the Treatment of Patients with Primary Biliary Cirrhosis

NEW YORK, May 28, 2014 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept), a clinical stage biopharmaceutical company, today announced that the United States Food and Drug Administration (FDA) has granted Fast Track designation to obeticholic acid (OCA) for the treatment of patients with primary biliary cirrhosis (PBC). OCA is being developed to treat PBC patients with an inadequate therapeutic response to, or who are unable to tolerate, ursodiol, the only drug currently approved to treat the disease. Intercept intends to complete its New Drug Application (NDA) of OCA for PBC in the first half of 2015. The NDA will include data from the Phase 3 POISE trial and two randomized Phase 2 trials, all of which met their primary endpoints with high statistical significance.

Established under the FDA Modernization Act of 1997, the Fast Track program is designed to facilitate the development and review of drugs intended to treat serious conditions and fill an unmet medical need. A drug development program with Fast Track designation is afforded greater access to FDA for the purpose of expediting the drug's development, review and potential approval.

"We believe that the award of Fast Track designation represents important recognition by FDA of OCA's potential to address a significant unmet need in the treatment of PBC patients," stated Mark Pruzanski, M.D., Intercept's Chief Executive Officer. "We will continue to work closely with the FDA with the goal of bringing OCA to PBC patients as quickly as possible," he added.
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Source: press release, 5/28/14. http://ir.interceptpharma.com/releasedetail.cfm?ReleaseID=851012

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Compound/DeviceSpecialtyIndicationCompound ClassTarget
Obeticholic acid (OCA) PBCGastroenterologyPrimary Biliary Cirrhosis (PBC)Farnesoid X Receptor (FXR) AgonistFarnesoid X Receptor (FXR)

Mechanism of action: Obeticholic acid (OCA) is a bile acid analog and first-in-class Farnesoid X Receptor (FXR) agonist derived from the primary human bile acid chenodeoxycholic acid, or CDCA.

Phase of Development: Filed

Event Type: Regulatory FDA: PDUFA DATE

Dates: 2016-05-29

Results:

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FDA Grants Accelerated Approval to Ocalivaâ„¢ (Obeticholic Acid) for the Treatment of Patients with PBC

First new medicine for PBC in nearly 20 years

Investor conference call Tuesday, May 31 at 8:30 a.m. ET

NEW YORK, May 27, 2016 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat non-viral, progressive liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to Ocaliva (obeticholic acid) for the treatment of primary biliary cholangitis, previously known as primary biliary cirrhosis (PBC), in combination with ursodeoxycholic acid(UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA. Ocaliva is an agonist of the farnesoid X receptor (FXR), a nuclear receptor expressed in the liver and intestine and a key regulator of bile acid, inflammatory, fibrotic and metabolic pathways.

"Intercept was founded on the belief that targeting FXR would benefit patients with liver diseases for which there are limited or no treatment options, and Ocaliva's approval marks the culmination of more than a decade of work," said Mark Pruzanski, M.D., Chief Executive Officer and President of Intercept. "We are very pleased that the FDA has approved Ocaliva for PBC and would like to thank all the patients and investigators around the world who participated in our clinical trials to make this possible."

This indication is approved under accelerated approval based on a reduction in alkaline phosphatase (ALP). An improvement in survival or disease-related symptoms has not been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

"Ocaliva fills an important unmet need for the many patients with PBC who have an inadequate response to or are intolerant of UDCA, which until now has been the only approved treatment," said John Vierling, M.D., F.A.C.P., F.A.A.S.L.D., Professor of Medicine and Surgery at Baylor College of Medicine and Past President of the American Association for the Study of Liver Diseases (AASLD). "Ocaliva has demonstrated a clinically meaningful improvement in lowering ALP, a liver enzyme and biomarker that is used to track disease progression in patients with PBC. Importantly, Ocaliva maintained durable ALP reductions, which is critical for treatment of a chronic disease like PBC."

In Intercept's Phase 3 POISE trial, Ocaliva administration in combination with UDCA (or as monotherapy in UDCA-intolerant patients) met the primary composite endpoint in 46% of patients in the titration group, as compared to 10% of those receiving placebo added to UDCA (p<0.0001). Pruritus (itching), a common symptom of PBC that is unrelated to disease stage or outcomes, was the most common side effect observed in Ocaliva-treated patients. However, pruritus associated with Ocaliva treatment was generally less in patients who were on the dose titration regimen (5 mg once-daily increasing to 10 mg once-daily); one patient (1%) in the titration group discontinued from the study due to pruritus. Additional side effects observed during the trial included fatigue, abdominal pain and discomfort, rash, oropharyngeal pain, dizziness, constipation, arthralgia, thyroid function abnormality and eczema.

"PBC affects people in the prime of their lives and, for some, the potential need for a liver transplant is a constant concern during these important years," said Linie Moore, a PBC patient and President of the PBCers Organization, the leading PBC patient support group in the U.S. "After nearly two decades with only one approved treatment, we are thrilled to welcome this important new medicine for people living with PBC."

Ocaliva is expected to be available to PBC patients in the U.S. within 7-10 days and will be distributed through a specialty pharmacy network. Intercept is dedicated to helping ensure that people with PBC can access Ocaliva and has launched Interconnectâ„¢, a comprehensive and personalized patient support services program. Through Interconnect, dedicated Care Coordinators will guide patients through disease education, treatment support and, for eligible patients, financial assistance options, which may include reimbursement support, co-pay assistance or access to Ocaliva at no cost. For more information about Interconnect Support Services and U.S. Distribution, call 1-844-622-4278 or visit www.Interconnectsupport.com.
MoreSource: press release, 5/27/16. http://ir.interceptpharma.com/releasedetail.cfm?ReleaseID=973265

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