Transaction provides new royalty and potential milestone revenues, opportunity to reduce cost structure, and continuity of patient access to lomitapide
Cambridge, MA, December 5, 2016 - Novelion Therapeutics, Inc. (NASDAQ: NVLN) (TSX: NVLN), today announced that its subsidiary, Aegerion Pharmaceuticals, Inc., has entered into a licensing agreement with Amryt Pharma (“Amryt”) for the exclusive rights to LOJUXTA® (lomitapide) hard capsules in certain European and Middle Eastern territories. LOJUXTA, marketed as JUXTAPID® in the United States, is currently approved by the European Commission as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without low density lipoprotein (LDL) apheresis in adult patients with homozygous familial hypercholesterolemia (HoFH).
Under the terms of the agreement, Amryt will receive an exclusive license to promote and distribute LOJUXTA in the European Economic Area, Switzerland, Turkey, and certain Middle Eastern and North African countries, including Israel. Amryt will pay Aegerion sales-related milestone payments and royalties on product sales in the licensed territories. Amryt will also be responsible for ongoing regulatory and post-marketing obligations and commitments in support of the brand.
“We are very pleased that Aegerion has entered into this agreement with Amryt, which will enable adult HoFH patients in these markets to remain on treatment and continue benefiting from this important therapy,” said Mary Szela, chief executive officer of Novelion. “Our objective as a company is to always put patients at the center of our decisions, and we believe with this outcome we have achieved that goal. In addition to the critical benefit for patients, we believe this is the right decision for our business. Our ability to reduce both corporate expenses and post-approval commitments allows us to reprioritize resources. Additionally, our stakeholders will now benefit from a royalty and potential milestone stream to Aegerion derived from Amryt’s sales of LOJUXTA. Amryt’s unique combination of orphan drug experience, commitment to developing and commercializing transformative rare disease therapies, and management’s familiarity with LOJUXTA, give us confidence in their ability to serve current and future adult HoFH patients. We are pleased to have them as a partner.”
“We believe that LOJUXTA, which was approved in the EU in mid-2013, is an important and needed therapy,” said Joe Wiley, chief executive officer of Amryt. “We believe there are a meaningful number of adult HoFH patients for whom LOJUXTA is a vital therapeutic option, and given our management team’s in-depth knowledge of the drug, we look forward to contributing to their care. This agreement is tremendously exciting and underlines our focus on building our portfolio of medicines to treat rare and orphan diseases where there is large unmet medical need.”
Lomitapide was approved in July 2013 by the European Commission and, until assumed by Amryt under the announced transaction, was marketed by Aegerion Pharmaceuticals under the brand name LOJUXTA. Aegerion is commercializing lomitapide under the brand name JUXTAPID in the U.S., Canada, and certain countries in Latin America, and expects to launch in Japan next month.
About JUXTAPID (lomitapide) capsules
JUXTAPID is a prescription medicine used along with diet and other lipid-lowering treatments, including low-density lipoprotein (LDL) apheresis where available, in adults with homozygous familial hypercholesterolemia (HoFH) to reduce LDL (“bad”) cholesterol, total cholesterol, a protein that carries bad cholesterol in the blood (apolipoprotein B), and non-high-density lipoprotein cholesterol (non-HDL-C).
Studies have not been conducted to tell us whether JUXTAPID can help prevent problems from high cholesterol, such as heart attack, stroke, death, or other health problems. Studies have also not been conducted to tell us whether JUXTAPID is safe for use in people with high cholesterol who do not have HoFH, including those with heterozygous familial hypercholesterolemia (HeFH).
Important Safety Information from U.S. Prescribing Information, including BOXED WARNING:
WARNING: RISK OF HEPATOTOXICITY
JUXTAPID can cause elevations in transaminases. In the JUXTAPID clinical trial, 10 (34%) of the 29 patients treated with JUXTAPID had at least one elevation in alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥3x upper limit of normal (ULN). There were no concomitant clinically meaningful elevations of total bilirubin, international normalized ratio (INR), or alkaline phosphatase.
JUXTAPID also increases hepatic fat, with or without concomitant increases in transaminases. The median absolute increase in hepatic fat was 6% after both 26 and 78 weeks of treatment, from 1% at baseline, measured by magnetic resonance spectroscopy. Hepatic steatosis associated with JUXTAPID treatment may be a risk factor for progressive liver disease, including steatohepatitis and cirrhosis.
Measure ALT, AST, alkaline phosphatase, and total bilirubin before initiating treatment and then ALT and AST regularly as recommended. During treatment, adjust the dose of JUXTAPID if the ALT or AST are ≥3x ULN. Discontinue JUXTAPID for clinically significant liver toxicity.
Because of the risk of hepatotoxicity, JUXTAPID is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the JUXTAPID REMS PROGRAM. Prescribe JUXTAPID only to patients with a clinical or laboratory diagnosis consistent with HoFH. The safety and effectiveness of JUXTAPID have not been established in patients with hypercholesterolemia who do not have HoFH.
• Concomitant administration of moderate or strong CYP3A4 inhibitors
• Moderate or severe hepatic impairment or active liver disease including unexplained persistent elevations of serum transaminases
WARNINGS AND PRECAUTIONS
JUXTAPID can cause elevations in transaminases and hepatic steatosis. Although cases of hepatic failure have not been reported, there is concern that JUXTAPID could induce steatohepatitis, which can progress to cirrhosis over several years. Modify the dose of JUXTAPID if elevations of transaminases are observed and discontinue JUXTAPID for persistent or clinically significant elevations. If transaminase elevations are accompanied by clinical symptoms of liver injury, such as nausea, vomiting, abdominal pain, fever, jaundice, lethargy, flu-like-symptoms, increases in bilirubin ≥2x ULN, or active liver disease, discontinue treatment with JUXTAPID and identify the probable cause. Use JUXTAPID with caution when co-administered with agents known to be hepatotoxic. Alcohol may increase levels of hepatic fat and induce or exacerbate liver injury.
Measure ALT, AST, alkaline phosphatase, and total bilirubin before initiating treatment. During the first year, measure liver-related tests (ALT and AST, at a minimum) prior to each increase in dose or monthly, whichever occurs first. After the first year, do these tests at least every three months and before any increase in dose.
Females of reproductive potential should have a negative pregnancy test before starting Juxtapid and should use effective contraception during therapy with Juxtapid. The recommended maximum dosage of Juxtapid is 40 mg daily when used concomitantly with oral contraceptives.
Given its mechanism of action in the small intestine, JUXTAPID may reduce the absorption of fat-soluble nutrients. Patients treated with JUXTAPID should take daily supplements that contain 400 international units vitamin E and at least 200 mg linoleic acid, 210 mg alpha-linolenic acid (ALA), 110 mg eicosapentaenoic acid (EPA), and 80 mg docosahexaenoic acid (DHA).
Gastrointestinal adverse reactions are common and may lead to treatment discontinuation. To reduce the risk of gastrointestinal adverse reactions, patients should adhere to a low-fat diet supplying less than 20% of energy from fat and the dosage of JUXTAPID should be increased gradually.
Weak CYP3A4 inhibitors can increase the exposure of lomitapide approximately two-fold; therefore, when JUXTAPID is administered with weak CYP3A4 inhibitors, the dose of JUXTAPID should be decreased by half and the recommended maximum dosage of JUXTAPID is 30 mg daily. The recommended maximum dosage is 40 mg daily when used concomitantly with oral contraceptives. Strong and moderate CYP3A4 inhibitors should not be used with Juxtapid. Patients taking JUXTAPID 5 mg daily may continue with the same dosage.
Due to risk of myopathy associated with simvastatin or lovastatin, doses of these agents should be limited when co-administered with JUXTAPID.
JUXTAPID increases the plasma concentrations of warfarin. Increases or decreases in the dose of JUXTAPID may lead to supra- or subtherapeutic anticoagulation, respectively. Patients taking warfarin should undergo regular monitoring of the INR, especially after any changes in JUXTAPID dosage.
Avoid use of JUXTAPID in patients with rare hereditary disorders of galactose intolerance.
About Amryt Pharma
Amryt Pharma is a specialty pharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare or ‘orphan’ diseases. The Company is building a diversified portfolio of commercially attractive, best-in-class, proprietary new drugs to help address some of these rare and debilitating illnesses for which there are currently no available therapies.
Amryt’s lead product, Episalvan, received marketing approval for the treatment of partial-thickness wounds from the European Commission in January 2016. Amryt intends to further develop Episalvan as a new treatment for Epidermolysis Bullosa (“EB”), a rare and distressing genetic skin disorder affecting young children for which there is currently no treatment. Amryt is currently planning a phase III study of Episalvan in EB, which has been granted US and EU orphan drug designation. The market opportunity for EB is estimated to be circa US $1.5 billion.
Amryt’s earlier stage products are focused on developing novel, next generation somatostatin analogue (“SSA”) peptide medicines for patients with rare neuroendocrine diseases, where there is a high unmet medical need. These include acromegaly and Crushing’s disease.
The Company joined AIM and Dublin’s ESM in April 2016 following the reverse takeover of Fastnet Equity PLC.
About Novelion Therapeutics, Inc.
Novelion Therapeutics is a biopharmaceutical company dedicated to developing new standards of care for individuals living with rare diseases. The company seeks to advance its portfolio of rare disease therapies by investing in science and clinical development. Novelion has a diversified commercial portfolio through its indirect subsidiary, Aegerion Pharmaceuticals, Inc., which includes MYALEPT® and JUXTAPID®, and is also developing zuretinol acetate for the treatment of inherited retinal disease caused by underlying mutations in RPE65 or LRAT genes.
Forward Looking Statements
This press release contains forward-looking statements, including statements regarding the anticipated benefits of the licensing arrangement with Amryt; the ability of Amryt to promote and distribute LOJUXTA effectively and to pay the sales-related milestones and royalties; and the planned launch by Aegerion of metreleptin in the EU and JUXTAPID in Japan. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond our control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include the possibility that the anticipated benefits of the licensing arrangement with Amryt may not be realized; the ability of Amryt to promote and distribute LOJUXTA effectively and to pay sales-related milestones and royalties, which may be impeded by factors beyond its control; the ability of Amryt to comply with regulatory and post-marketing obligations and commitments; the risk that the prevalence of the diseases LOJUXTA treats may be lower than we estimate, and that it may be more difficult to identify patients than we expect; the risk that the side effect profile or other results for LOJUXTA are inconsistent, in scope or severity, with the side effect profile and other results observed in clinical trials or of competitive products; the risk that the negative impact of the launch of PCSK9 inhibitors on LOJUXTA sales will be greater than we expect or that other competitive products will negatively impact sales; the risk that private or government payers may refuse to reimburse LOJUXTA or may impose onerous restrictions that hinder reimbursement or significantly limit or cap the price or the number of reimbursed patients who receive LOJUXTA; the risk that exchange rates will negatively impact revenues realized from the licensing arrangement with Amryt; risks related to intellectual property and data exclusivity; and the risk of unexpected manufacturing issues affecting future supply. The risks related to the planned launch of metreleptin in the EU and JUXTAPID in Japan include that the regulatory authorities do not approve the planned filing of metreleptin; that pricing and reimbursement authorities do not provide requisite approvals of meterleptin in the EU, and the other regulatory, development and commercialization risks related to developing and commercializing rare disease pharmaceutical products generally and in the EU and in Japan.
For additional disclosure regarding these and other risks we face, see the disclosure contained in the "Risk Factors" section of Aegerion's Quarterly Report on Form 10-Q filed on November 4, 2016, Novelion’s Annual Report on Form 10-K filed on February 25, 2016 (and amended on April 29, 2016) and Quarterly Report on Form 10-Q filed on November 1, 2016 and each company’s other public filings with the SEC, available on the SEC's website at www.sec.gov. Except as required by law, we undertake no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.
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Amanda Murphy, Associate Director, Investor & Public Relations