Amryt Pharma (AMYT ) has received “encouraging” data from a study of lomitapide in Familial Chylomicronaemia Syndrome (“FCS”) which was conducted by expert lipidologists in Italy.

Lomitapide is approved as an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with Homozygous Familial Hypercholesterolaemia ("HoFH") a rare and genetic disease which causes excessive levels of ‘bad cholesterol’ to accumulate in the body.

Lomitapide is approved in the US, Canada, Columbia, Argentina and Japan (under the trade name Juxtapid®) as well as in the EU, Israel and Brazil (under the trade name Lojuxta®). 

Today, Amryt told investors that it has received efficacy and safety data from the “Lochnes” investigator sponsored study of lomitapide in Familial Chylomicronaemia Syndrome (“FCS”), a group of rare genetic disorders that cause very high levels of triglycerides in the blood.

The study enrolled 18 adult patients with a genetic diagnosis of FCS and a history of pancreatitis. The median baseline fasting triglyceride level of patients was 1884 mg/dL.

Patients were administered open-label treatment with escalating doses of lomitapide for 26 weeks. The median fasting triglyceride level at 26 weeks was 672 mg/dL representing a 70.5% median reduction versus baseline. The study showed that treating patients with lomitapide was generally ‘well tolerated’ with patients completing 26 weeks of treatment.

By the end of the study, 14 patients had achieved a reduction in triglycerides to less than 1000 mg/dL and 13 of these achieved triglycerides of 750 mg/dL or below at 26 weeks.  

Amryt said adverse events were ‘mild to moderate’ and mostly related to gastrointestinal tolerability and liver enzyme elevations.  Liver MRI imaging also revealed an increase in hepatic fat content which was between 30-50% at week 26 in 3 patients, it noted.

“Interestingly, at the end of the study, 14 out of 18 patients achieved triglyceride levels below 1000 mg/dL , which is considered the pancreatitis risk threshold,” said Professor Maurizio Averna, Lead Investigator with Professor Angelo B. Cefalù - Study Co-Investigator.

Maurizio Averna and Angelo B. Cefalù concluded that, “The results of the study support the hypothesis that lomitapide may be a potential treatment for reducing triglycerides in FCS.”

Amryt told investors that it will now evaluate and analyse the full data set from the study. It said it plans to discuss the potential development path forward with the FDA and the EMA.  

Commenting on the data set, Dr Mark Sumeray, Chief Medical Officer of Amryt, stated, “We are encouraged by the data from the study and look forward to our discussions with the respective health authorities to progress lomitapide as a potential treatment for FCS.”

Today’s news follows a recent string of successes as Amryt expands its portfolio’s reach into both existing and new territories. 

The group will now evaluate the data which supports the hypothesis that lomitapide may be a potential treatment for reducing triglycerides in FCS.

The data received from the study gives the group further confidence ahead of its plans to discuss lomitapide’s potential development path forward with the FDA and the EMA. 

Shares in Amryt Pharma have increased by over 8% in value since the beginning of the year. The stock was trading 2.18% lower this morning at 201.05p 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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