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Amryt Pharma plc (AIM: AMYT)  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. Amryt’s commercial business comprises two orphan disease products:

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®). HoFH is a rare genetic disorder which impairs the body’s ability to remove low density lipoprotein (“LDL”) cholesterol.

Myalept® / Myalepta® (metreleptin) is approved in the US as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (GL). It is also approved in the EU for the treatment of leptin deficiency in patients with congenital or acquired GL in adults and children two years of age and above and familial or acquired partial lipodystrophy (PL) in adults and children 12 or over for whom standard treatments have failed to achieve adequate metabolic control. Metreleptin is also approved for lipodystrophy in Japan. Generalised and partial lipodystrophy are rare disorders characterised by loss or lack of adipose tissue resulting in the deficiency of the hormone leptin, produced by fat cells and are associated with severe metabolic abnormalities including severe insulin resistance, diabetes, hypertriglyceridemia and fatty liver disease.


  • Ray Stafford (NE Chair) – 30yrs pharma experience. He was Chairman, Chief Executive Officer and majority shareholder of the Tosara Group who launched Sudocrem
  • Dr Joe Wiley (CEO) – co-founder of Amryt. 20+ years of experience in the pharmaceutical, medical and VC industries
  • Rory Nealon (CFO & COO) – co-founder of Amryt. Prior to that, he was the CFO & COO of Trinity Biotech plc, a  Nasdaq listed diagnostic Co.
  • NED’s – Stephen Wills, Dr Patrick Vink, Donald Stern, Dr Alain Munoz, George Hampton Jr.


Profit & Loss2019
(Cost of sales)(42,001)(6,266)
Gross Profit16,12310,829
(Loss for the year)(64,754)(30,654)
Balance Sheet2019
Casn & cash equivalents67,22911,226
Total assets95,83614,798

Peer Group

  • Abeona Therapeutics (NASDAQ: ABEO)
  • Fibrocell Science (NASDAQ: FCSC)
  • Krystal Biotech (NASDAQ: KRYS)
  • Summit Therapeutics (AIM: SUMM)

Major Shareholders

  • Athyrium Capital Management, LP – 27.2%
  • Novelion Therapeutics Inc. – 8.9%
  • Highbridge Capital Management, LLC – 8.2%
  • Software AG-Stiftung – 6.5%
  • UBS O’Connor LLC – 5.4%
  • AXA Investment Management – 4.1%

Macro Indicators

Clinical OMICS (October 2019)

“…There is a great need for new treatments for rare diseases. Of an estimated 6,500 to 7,000 known such conditions, only a fraction (approximately 5%) have U.S. Food and Drug Administration-approved treatments.”


Media coverage

Shares Magazine (1st Oct 2019)

“Biopharmaceutical business Amryt Pharma (AMYT:AIM) added 1.5p at 132.5p on news its treatment for EB, a rare genetic skin disorder, is being fast-tracked by the US Food and Drug Administration (FDA), a positive development that builds on yesterday’s encouraging interim results.” (26th Sep 2019)

“Amryt Pharma plc raised $60 million in new equity financing to complete its all-stock takeover of Aegerion Pharmaceuticals Inc., creating a global rare diseases company with three marketed products, a U.S. commercial infrastructure and a couple of near-term opportunities to expand its portfolio.”

Broker Coverage

Shore Capital (House Broker – October 2019)

“The transaction creates a profitable, fast growing biotech company with a portfolio of products for rare and orphan diseases. The all-equity acquisition was contingent on a number of factors including the restructuring of Aegerion’s debt and also successful
consummation of a back-stopped $60m equity placing, which should provide ample financing from deal-close for the foreseeable future…The combined company has a global portfolio (ex-Japan) of two marketed rare disease products and a pipeline of assets for Epidermolysis Bullosa (EB), including AP101 which is in Phase 3 (data early FY 2020F).”

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