GCIAmryt Pharma sees ‘clear path’ for global clinical study of metreleptin
has received positive feedback from the US FDA in regard to its global clinical study for Myalept® (metreleptin) in patients with partial lipodystrophy (PL).
Myalepta® (metreleptin), is a leptin replacement therapy used with a doctor-recommended diet to treat problems caused by not having enough leptin in the body (leptin deficiency).
Myalept® / Myalepta® (metreleptin) is already approved in the US, under the trade name Myalept®, as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (GL).
Myalept® / Myalepta® (metreleptin) is also approved in the EU under the trade name Myalepta® to treat leptin deficiency in patients with congenital or acquired GL in adults and children of two and above and familial or acquired PL in adults and children of 12 and above for whom standard treatments have failed to achieve adequate metabolic control.
Generalised and partial lipodystrophy are rare disorders which result in loss or lack of adipose tissue resulting in the deficiency of the hormone leptin. They are associated with severe metabolic abnormalities including severe insulin resistance and hypertriglyceridemia.
The company said the prevalence of PL patients severely affected by their disease is thought to be similar to GL in the US. Therefore, it noted, if approved for treatment of this patient sub-group, this represents approximately double the current US market opportunity.
Amryt said it had recently received additional feedback via a Type C written response on the proposed development plan and study design to support an indication for patients with PL.
The FDA confirmed that they are willing to consider an efficacy supplement based on 6-months efficacy and safety data from a randomized, placebo-controlled trial in PL patients.
As a result, the group noted that safety data will continue to be collected up to the completion of the 12-month treatment period and the overall assessment of the benefit-risk ratio for PL patients will take into consideration anti-leptin neutralizing activity.
The 12-month randomized, placebo-controlled Phase 3 trial to evaluate the safety and efficacy of daily subcutaneous metreleptin treatment in patients with PL will enroll around 80 patients globally. It is anticipated that this study will be initiated by the end of 2021.
The study will enroll patients with severe metabolic consequences of their disease as reflected by blood glucose control and/or triglyceride levels on optimal background treatment. Amryt said it has already completed assessments to identify eligible PL patients.
ComDr Joe Wiley, CEO of Amryt Pharma, said, “Today’s news represents further progress as we grow the reach of our commercial products across both territories and indications.
We now have a very clear path forward for our global clinical study of metreleptin in PL which, if successful, could offer the potential to address a broader population of patients in need.”
Today’s news follows a recent string of successes as Amryt continues to expand the reach of its portfolio, particularly in regard to Myalepta®, into both existing and new territories.
Feedback from the FDA has paved ‘a clear path’ for Amryt to initiate its global clinical study of metreleptin in PL which represents around double the current US market opportunity.
Shares in Amryt Pharma have increased by over 15% in value since the beginning of the year. The stock was trading 1.35% higher this morning at 217.9p following the announcement.
Reasons to Follow AMYT
Amryt is a biopharmaceutical company focused on developing and delivering innovative new 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.
Leading Portfolio
AMYT’s portfolio includes Amryt's lead development candidate, FILSUVEZ®, a potential treatment for the cutaneous manifestations of EB, a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment.
Amryt continues to grow its existing commercial products while it is currently preparing for the launch of its FILSUVEZ® skin product in the US in 4Q21 and in Europe in 1Q22.
The anticipated launch of FILSUVEZ® follows recent positive results from Amryt’s Phase 3 EASE trial, the largest ever global Phase 3 trial conducted in patients with genetic skin disorder Epidermolysis Bullosa (EB), which was performed across 58 sites in 28 countries.
Amryt now intends to complete the submission of its rolling New Drug Application (“NDA”) to the US Food and Drug Administration (“FDA”) and request priority review for FILSUVEZ®.
In a recent report, CEO,Wiley, highlighted the group’s products metreleptin and lomitapide as delivering growth across revenue, EBITDA, cash generation and market expansion.
Amyrt’s Myalept® / Myalepta® injection is a leptin replacement therapy used with a doctor-recommended diet to treat problems caused by not having enough leptin in the body. It is approved in the US under the trade name Myalept® and in the EU under Myalepta®.
Juxtapid®/ Lojuxta® (lomitapide) is approved as an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the rare cholesterol disorder, Homozygous Familial Hypercholesterolaemia ("HoFH") in the US, Canada, Columbia, Argentina and Japan under the trade name Juxtapid® and in the EU under the trade name Lojuxta®.
Expanding Distribution Capacity
Amyrt recently signed a distribution agreement with Swixx BioPharma AG (“Swixx”), a Swiss pharmaceutical agent operating in Central and Eastern Europe (“CEE”) for Lojuxta®.
The agreement represents new territories for the Group to further accelerate its revenue growth, augmenting sales already being generated through its existing salesforce in the EU.
In recent weeks, Amryt received approval from the National Institute for Health and Care Excellence (“NICE”) for its leptin replacement therapy Myalepta® (metreleptin) in England and Wales, to treat partial and generalised lipodystrophy in adults and children 12+ years of age.
Positive Outlook
Last week, the Company released ‘record’ results for FY20 which it said were demonstrative of ‘the very positive performance and growth’ that its commercial products are delivering.
Amryt is well positioned in the novel therapeutics market and strong momentum achieved in the year to date has prompted management to upgrade its guidance for FY20. “Given the strong performance of our business in 2020, we are now issuing revenue guidance for FY21 of $200-$205m which demonstrates our confidence in our prospects,” Wiley told investors.
The Group said both metreleptin and lomitapide continue to deliver growth ‘across a host of metrics’ including revenue and EBITDA growth, cash generation and market expansion.
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