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Duchenne muscular dystrophy drugs at the crossroads, as newer agents advance

NATURE BIOTECHNOLOGY
VOLUME 34 NUMBER 7 JULY 2016 p675

The first generation of exon-skipping drugs for Duchenne muscular dystrophy (DMD) has reached a critical juncture, as an FDA decision on whether to grant accelerated approval to Sarepta Therapeutics’ controversial drug Exondys (eteplirsen) looms. A rival effort at BioMarin Pharmaceutical, of San Rafael, California, is now over. Biomarin withdrew a European application for Kyndrisa (drisapersen) on May 31, after unsuccessful talks with European Medicines Agency officials, and the US Food and Drug Administration (FDA) refused to approve the same drug in January.

The refusals were unequivocal—drisapersen was neither safe nor effective. In the case of Sarepta’s eteplirsen, however, regulatory uncertainty abounds. The Cambridge, Massachusetts–based biotech, is seeking approval with evidence from just 12 patients. “In any other therapeutic area, the agency would be within its rights to
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“The normal balance of evidence hasn’t been achieved for this drug yet.”

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New data underscore benefits of idebenone in DMD as Biomarin pulls drisapersen MAA

BIOWORLD TODAY

By Cormac Sheridan Staff Writer

Thursday, June 2, 2016

DUBLIN – Even as Biomarin Pharmaceutical Inc.’s dwindling hopes for its exon-skipping pipeline in Duchenne muscular dystrophy (DMD) turned to ash this week, with the withdrawal of its marketing authorization application (MAA) for drisapersen from the EMA, there was also some positive news for DMD patients and their families. Santhera Pharmaceuticals AG released additional clinical data from its Delos phase III study of Raxone (idebenone) in Duchenne muscular dystrophy (DMD), which showed that the drug’s ability to slow the rate of decline of respiratory function over one year converted into a range of clinical benefits.
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The new analysis forms part of its recently completed regulatory filing in Europe and its planned filing in the U.S. The Liestal, Switzerland-based company filed for approval in DMD in Europe Tuesday, as a type II variation to its existing approval for treating visual impairment in Leber’s hereditary optic neuropathy. If the FDA is agreeable, it could complete a rolling new drug application during the third quarter of 2016 – although that depends on the outcome of a meeting in late July.

The primary endpoint of the Delos trial, which recruited 64 DMD patients who were not on concomitant glucocorticoid therapy, was the alteration in lung function over the one-year trial, as measured by the percentage change from baseline to week 52 in peak expiratory flow (PEF) for each patient. PEF for those in the drug treatment group declined by an average 2.57 percent, whereas it declined by an average 8.84 percent for those in the control group. (See BioWorld Today, May 14, 2014.)

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Santhera holds talks with potential Eastern European distribution partners for Raxone in LHON – CEO

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Reimbursement discussions ongoing in multiple countries

DMD regulatory filings expected before YE15

Santhera Pharmaceuticals (SWX:SANN) is in ongoing discussions with potential distributors in Eastern Europe for Raxone (idebenone) in Leber’s hereditary optic neuropathy (LHON), said CEO Thomas Meier. There is a reasonable chance the company will have selected a partner by the end of this year, he said.

The company received EMA marketing authorisation for Raxone in LHON on 9 September, according to a company press release.

The company has built an infrastructure for Western European countries; however, in Eastern European territories it needs logistical help, Meier said.

In terms of deal structure it is likely Santhera would pay a fee to the distribution partner and it may receive a percentage of the sales within those territories, Meier said, adding the exact arrangement is still under evaluation. However, Santhera will retain the license for Raxone in these regions, he noted. The company’s preferred scenario is to find one partner for the entire bloc of Eastern European countries rather than multiple partners for several territories, he explained.

Santhera is hiring a team of in­house experts to help commercialise Raxone within Western European territories and is hoping to launch the product in an undisclosed European country shortly, Meier said. Payer discussions are ongoing in multiple countries, he added. When asked about potential pricing, Meier declined to provide a figure however pointed to a recent analyst report which may provide an estimate. The analyst report noted an estimated net price of USD 7,373 per treatment month in 2016 and USD 7,005 for 2017 and beyond.

LHON is a heritable genetic disease causing blindness. The disease typically presents in young, otherwise healthy adults, mostly men, as rapid, painless loss of central vision in one eye, followed by visual loss in the other eye within a few months of the onset of symptoms, leading to blindness, according to a company press release.

The company will file for FDA approval for Raxone in LHON at a later date.

Additional indications
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The company’s cash position at the end of August was CHF 37m, which will cover the full European launch of Raxone, Meier said. Additional financing may be required to continue development of Raxone in other indications, he noted, declining to comment on further financing details.

The company may consider similar LHON distribution agreements for Duchenne Muscular Dystrophy (DMD) in specific territories necessary, he said.

Santhera is preparing regulatory filings for Raxone in the EU and the US for DMD before the year’s end, Meier noted. On the back of the EMA approval in LHON, the company believes it may be able to apply for a line extension for Raxone in DMD which is under discussion internally. The company will initiate discussions with the Committee for Medicinal Products for Human Use (CHMP) shortly. According to the aforementioned analyst report, the LHON approval may allow for a six­month review of Raxone in DMD within Europe. The company anticipates filing its NDA to the FDA as well.

Meier declined to comment on any speculation with regards to a sale of the company and noted the management’s current preference is to stand alone and build value through obtaining additional approvals and pipeline development. Santhera is also considering additional indications for Raxone and is in discussions internally and with key opinion leaders as it believes that the drug may be beneficial for additional mitochondrial diseases, Meier said. However, he declined to name any specific indications under consideration. Within the next 12 months the company will have a clearly defined strategy for other indications, he added.

The company will not out­license Raxone in either LHON or DMD and will market alone in both the US and EU, Meier said. However, Santhera has set aside certain capital resources for in­licensing activities.

Raxone was the first approval for a mitochondrial disease and the company will consider licensing additional molecules for mitochondrial disease, neuromuscular diseases or paediatric diseases, he noted. The company could take part in codevelopment arrangements or license from a US company looking for a European partner.

Santhera also has a fully recruited Phase II trial ongoing in primary progressive multiple sclerosis, he added.

Santhera’s market cap is CHF 697m.

by Hamish McDougall in London

Email the journalist team at editorialfeedback@biopharminsight.com