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Orchard plans IPO as gene therapy pipeline advances

UK biotech seeks funds for expanded rare disease portfolio

UK gene therapy specialist Orchard Therapeutics has filed registration documents for an initial public offering, just weeks after its latest $150m private round.

The filing shows the company wants to raise $172.5m to roll out its Strimvelis gene therapy for adenosine deaminase-severe combined immunodeficiency (ADA-SCID) – acquired from GlaxoSmithKline (GSK) earlier this year – in Europe.

It will also help bring forward its rapidly-expanding pipeline of rare disease candidates including ADA-SCID follow-up OTL-101, OTL-200 for metachromatic leukodystrophy (MLD), and OTL-300 for beta thalassaemia – which has just bagged a Priority Medicines (PRIME) designation from the EMA.

Add in capital projects such as a planned US manufacturing facility and the biotech will have plenty of demands on its cashflow in the next couple of years.

Under the terms of its license agreement with GSK, which remain a major shareholder, Orchard has to make efforts to broaden the availability of Strimvelis across Europe “until such time as an alternative gene therapy, such as our OTL-101 product candidate, is commercially available.”

It acknowledges in the prospectus that Strimvelis doesn’t have the market potential to make it profitable, even if it can extend its use beyond the single Italian facility currently licensed to deliver the therapy. Adding the IPO proceeds to its $200m-plus cash reserves will give it the resources it needs to bring its in-house projects forward.

OTL-101 and OTL-200 are the two most advanced products in Orchard’s clinical pipeline, with the company hoping to get US approval for the former in 2020, with an EU filing coming a little later. OTL-200 is also scheduled for a 2020 filing – initially in Europe with the US to follow.

Next up are OTL-103. which is in a registration trial in Wiskott-Aldrich syndrome (WAS), and OTL-102 for X-linked chronic granulomatous disease (X-CGD) which is in a proof of concept trial. Like ADA-SCID, both WAS and X-CGD are forms primary immunodeficiency. The company thinks it is in with a shout of having four therapies approved by the end of 2021, including Strimvelis.

Meanwhile, OTL-300 – another project acquired from GSK – is the first therapy in Orchard’s pipeline to target a haemoglobinopathy. It has been tested so far in nine patients with transfusion-dependent beta-thalassemia, the most severe form of the disease.

After at least 12 months of follow-up as, significant reductions in transfusion frequency and volume requirements have been observed in five of seven evaluable patients, with three of four children transfusion-free a month after dosing.

Orchard is playing catch-up in thalassaemia with Bluebird Bio, which is planning to file its LentiGlobin candidate in Europe before the end of this year, but getting PRIME status in Europe could help accelerate the development of OTL-300 and narrow the gap.

Mark Rothera

Mark Rothera

Mark Rothera, president and chief executive of Orchard said the company was pleased to receive the fast-track status  for OTL-300. He commented: "PRIME designation allows us the opportunity to engage more closely with the EMA and potentially accelerate our ability to provide an innovative new treatment option for patients and families affected by TDBT."

Article by
Phil Taylor

9th October 2018

From: Sales



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