Sarepta files $250M fundraising, says it's considering more bolt-on deals

Company says developing its pipeline is "urgent."

Sarepta Therapeutics' brand-new CEO Doug Ingram is hoping to raise $250 million in a public stock offering as the company looks beyond its first product, the Duchenne muscular dystrophy (DMD) therapy Exondys 51.

The company says that it plans to use the cash injection to fund trials of its pipeline therapies, rolling out Exondys 51 (eteplirsen) in international markets following its FDA approval last September and—potentially—licensing in new drug candidates.

Ex-Allergan president and Chase Pharma CEO Ingram, who took over the helm of Sarepta on last month, has said he intends to buy around $2 million worth of the offered shares.

The filing comes as Sarepta is in a buoyant phase, despite some lingering controversy over the approval of Exondys 51 and continued speculation about how well the company will do convincing payers to stump up for its $300,000-a-year price tag without placing serious restrictions on its use.

Right now the portents seem good. Sales of Exondys 51 are ramping up nicely in the U.S., reaching $35 million in the second quarter, which the biotech said was ahead of consensus estimates. In Europe the EMA formally kicked off its review at the end of 2016, with the company predicting a possible approval in the first half of 2018.

Ingram told investors on the company's second-quarter results call last week that the company is launching a managed access program designed to expand the availability of Exondys 51 to eligible patients outside the U.S. on a named-patient basis. The company is expecting sales to reach $125 million to $130 million this year, higher than its initial prediction of around $95 million.

The company has also just settled a potentially disruptive patent dispute with Biomarin, with Sarepta taking a license to some patents held by the Dutch biotech on exon-skipping therapies for DMD, and sold a priority review voucher it claimed from the FDA for the Exondys 51 approval to Gilead for $125 million.

"We intend to extend our global leadership in DMD and we intend to become a substantial global leader in rare neuromuscular disorders," said Ingram. "The development of our pipeline with urgency is critical, because … there are many patients still waiting for treatment options," he added.

Adding another $250 million or so to the coffers will give Sarepta the financial muscle it needs to continue to build its portfolio. The company has already signed two pipeline-expanding deals this year, joining forces with Genethon of France and Nationwide Children's Hospital on gene therapy candidates for DMD.  

DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. The disease primarily affects young boys, and occurs in about one out of every 3,600 male infants worldwide.

Sarepta's current FDA-approved drug can only treat certain patients, namely those with the mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13% of the population with DMD. Along with gene therapies to try to correct the underlying genetic defect in DMD, the company also has exon-skipping candidates targeting different mutations.