Amryt announces Succeeded Bioavailability study for Mycapssa® (octreotide capsules)
Data Supporther planned phase 3 study in treatment of carcinoid symptoms associated with Neuroendocrine tumors (REPORT)
Global REPORT the market opportunity is estimated at approx. $1.9 billion* with Opportunity in the US market estimated at approx. $1.0 billion*
DUBLIN, Ireland and Boston MA, March 8, 2022 Amryt (Nasdaq: AMYT), a global, commercial-stage biopharmaceutical company dedicated to acquiring, developing and commercializing novel treatments for rare diseases, today announces the completion of a successful pharmacokinetic (PK) study for Mycapssa® (oral octreotide). The data supports a planned Phase 3 study of Mycapssa® in the treatment of patients with carcinoid symptoms due to neuroendocrine tumors (NETs).
Mycapssa® – REPORT Opportunity
Amryt’s TPE® platform allows for the oral delivery of the octreotide molecule which is otherwise delivered in injectable form. Mycapssa® (oral octreotide) is FDA-approved for long-term maintenance therapy in patients with acromegaly who have responded to and tolerated injectable treatment with octreotide or lanreotide (i.e. somatostatin analogues (SSA)).
Injectable SSAs are also approved and are the pharmaceutical standard of care in the treatment of carcinoid symptoms in NETs and their use in NETs accounts for approximately $1.9 billion* worldwide and approximately $1.0 billion* in the US. United States. The potential population of addressable patients under SSA in the United States is estimated at 24,000**. Compared to acromegaly, patients with NETs are known to require higher average doses of injectable SSA to achieve adequate symptom control.
package Study Results
The objective of the PK study was to demonstrate that doses of Mycapssa® up to 80 mg provide the desired bioavailability and dose proportionality with an acceptable safety and tolerability profile. The phase 1 study was an open label, six-sequence, 3-period crossover study, recruiting 30 healthy subjects who received single doses (20 mg, 60 mg and 80 mg) of Mycapssa®. Study results showed dose linearity from 20 mg to 80 mg. Safety and tolerability were acceptable and as expected and no serious adverse events were reported.
The study met its objectives and the data supports the planned Phase 3 study in patients with carcinoid symptoms due to NET. The study results are consistent with the previous pharmacokinetic study that was undertaken with these higher doses (20 mg, 60 mg and 80 mg) and compared to injectable ASS.
The U.S. Food and Drug Administration (FDA) has confirmed that a single positive Phase 3 study would be sufficient for approval, under the previously agreed 505(b)(2) regulatory pathway. Amryt is currently finalizing the study protocol with the FDA and expects to initiate the Phase 3 study in Q4 2022.
Dr. Joe Wiley, CEO of Amryt Pharma, said: “Today’s news represents an important important step in our Mycapssa® development plan for patients affected by carcinoid symptoms associated with NET. Jits additional indication for NET, if approved, creates a significant New market opportunity for Amryt estimated at $1.9 billion worldwide. We are very excited at the poprovisional for Mycapssa® and our TPE® technology deliver oral therapy option for REPORT the patients in need”.
About Neuroendocrine Tumors (NETs)
NETs originate from neuroendocrine cells throughout the body, most commonly in the gastrointestinal tract, the lungs, and rarely the pancreas. While well-differentiated neuroendocrine tumors are known for their slow growth, they are often asymptomatic in the early stages, resulting in a significant number of patients being diagnosed when the tumors have already spread regionally or distantly. Capable of secreting hormones and bioactive amines, approximately 19% of patients present with carcinoid syndrome characterized by secretory diarrhea and flushing.
* Based on management estimates
** National Cancer Institute SEER database; Halperin et al. 2017 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6066284/
Amryt is a commercial-stage global biopharmaceutical company focused on acquiring, developing and commercializing innovative treatments to help improve the lives of patients with rare and orphan diseases. Amryt comprises a strong and growing portfolio of commercial and development assets.
Amryt’s commercial activity includes three orphan disease products: metreleptin (Myalept®/ Myalepta®); oral octreotide (Mycapssa®); and lomitapide (Juxtapid®/Lojuxta®).
Myalept®/Myalepta® (metreleptin) is approved in the United States (under the trade name Myalept®) as an adjunct to diet as replacement therapy to treat complications of leptin deficiency in patients with generalized lipodystrophy ( GL) congenital or acquired and in the EU (under the trade name Myalepta®) as an adjunct to diet for the treatment of leptin deficiency in patients with congenital or acquired GL in adults and children two years of age and older and familial or acquired partial lipodystrophy (PL) in adults and children 12 years and older for whom standard treatments have failed to achieve adequate metabolic control. For more information, please follow this link.
Mycapssa® (octreotide capsules) is approved in the United States for long-term maintenance therapy in patients with acromegaly who have responded to and tolerated treatment with octreotide or lanreotide. Mycapssa® is the first and only FDA-approved oral somatostatin analog. Mycapssa® has also been submitted to the EMA and is not yet approved in Europe. For more information, please follow this link.
Juxtapid®/Lojuxta® (lomitapide) is approved as an adjunct to a low-fat diet and other lipid-lowering medications for adults with the rare cholesterol disorder, homozygous familial hypercholesterolemia (“HoFH”) in the United States , in Canada, Colombia, Argentina and Japan (under the trade name Juxtapid®) and in the EU, Israel, Saudi Arabia and Brazil (under the trade name Lojuxta®). For more information, please follow this link.
Amryt’s lead development candidate, Oleogel-S10, is a potential treatment for the cutaneous manifestations of junctional and dystrophic epidermolysis bullosa (EB), a rare and distressing genetic skin disease affecting young children and adults for which there is currently no approved treatment. Filsuvez® was chosen as the brand name for Oleogel-S10. The product does not currently have regulatory approval to treat EB.
Amryt’s preclinical gene therapy candidate, AP103, offers a potential treatment for patients with dystrophic EB, and the polymer-based delivery platform has the potential to be developed for the treatment of other genetic disorders.
Amryt also intends to develop oral drugs that are currently only available as injectable therapies through its Transient Permeability Enhancer (TPE®) technology platform. For more information about Amryt, including products, please visit www.amrytpharma.com.
This announcement may contain forward-looking statements and the words “expect”, “anticipate”, “intend”, “plan”, “estimate”, “aim”, “expect”, “project” and similar expressions (or their negative) identify some of these forward-looking statements. The forward-looking statements contained in this announcement are based on numerous assumptions and about Amryt’s current and future business strategies and the environment in which Amryt expects to operate in the future. Forward-looking statements involve inherent known and unknown risks, uncertainties and contingencies because they relate to events and depend on circumstances that may or may not occur in the future and may cause actual results, performance or achievements are materially different from those expressed or expressed. implied by such forward-looking statements. These statements are not guarantees of future performance or the ability to identify and make investments. Many of these risks and uncertainties relate to factors that are beyond Amryt’s ability to control or accurately estimate, such as future market conditions, developments in the COVID-19 pandemic, fluctuations currencies, the behavior of other market participants, the results of clinical trials, the actions of regulators and other factors such as Amryt’s ability to obtain financing, changes in the political, social and regulatory framework in which Amryt operates in either economic, technological or consumer trends or conditions. Past performance should not be taken as an indication or guarantee of future results, and no representations or warranties, express or implied, are made regarding future performance. No one is under any obligation to update or keep current the information contained in this announcement or to provide the recipient with access to any additional relevant information that may arise in connection with it. These forward-looking statements reflect the Company’s current beliefs and assumptions and are based on information currently available to management.
Joe Wiley, CEO / Rory Nealon, CFO/Managing Director, +353 (1) 518 0200, [email protected]
Tim McCarthy, LifeSci Advisors, LLC, +1 (212) 915 2564, [email protected]