1. Sarepta Therapeutics

(Sarepta)

1. Sarepta Therapeutics
2018 median employee pay: $329,229
2018 number of employees: 499
CEO: Douglas Ingram
2018 CEO pay: $1.43 million
CEO-to-employee pay ratio: 4.4:1

At first glance, the ratio of Sarepta Therapeutics CEO Doug Ingram’s 2018 pay to that of its employees, at 4.4 to 1, is among the lowest in the industry. But that doesn’t mean Sarepta is extra harsh on Ingram or overly generous with its staffers.

In 2018, Ingram didn’t receive any equity awards, which make up the biggest chunk of many executives’ compensation. That’s because the performance-based option award he got in 2017 as part of his welcome-aboard package covered all equity awards for the first five years of his employment. Ingram’s total stock awards in 2017 amounted to $56.1 million. Divided evenly across five years, omitting fluctuations in Sarepta’s stock price, his 2018 pay would be around $12.6 million.

Nevertheless, Sarepta employees saw their median pay jump nearly 30% in 2018, to $329,229, putting them at No.1 on our employee pay ranking. It came as Ingram declined a base salary increase even though the board had planned one considering his “exceptional performance,” the company said in a proxy filing.

As of the end of 2018, Sarepta had 499 employees, including 255 working in R&D and 48 in sales. The company’s total headcount is now tracking to about 900 by the end of 2019, Ingram told investors during the firm’s second-quarter earnings call in August. These staffers are spread across its offices in Massachusetts, Switzerland, Germany, France, Italy, Ireland, Sweden, the U.K., Brazil and Japan.

Last year, The Boston Globe named Sarepta as a top place to work in Massachusetts among 36 organizations with between 250 and 999 employees. The award was based on employee surveys on the companies’ management, pay and benefits, etc. In its annual securities filing, Sarepta describes its culture as “based on bias to action, a self-starter mentality, smart and appropriate risk-taking and high ethics.”

Product-wise, Sarepta is still a one-drug company. Last year, its Duchenne muscular dystrophy therapy Exondys 51 sold nearly $301 million, almost double its tally the previous year. It was a good rally considering the controversies around its price and efficacy.

RELATED: BIO: Lightning round with Sarepta's Doug Ingram

However, the EMA gave the exon-skipping drug a thumbs-down last year. The European reviewers were skeptical about the small size of the study Sarepta used in its application, and about how the drug’s dystrophin production improvement could translate into lasting clinical benefit: Exondys 51 didn’t help phase 2 trial patients do better in the six-minute walk test. In fact, the same concerns were raised within the FDA in the agency’s much-debated approval back in 2016.

Then earlier this year, increasingly influential drug cost watchdog the Institute for Clinical and Economic Review put out an analysis that ruled Exondys 51 too pricey “even with assuming extremely favorable treatment effects that are not credible given clinical experience.” In response, Sarepta called ICER’s methodology “fatally flawed” because it doesn’t apply to treatment for serious rare diseases such as DMD.

Exondys 51 could see some competition, though several of its potential rivals have their own problems. Pfizer, for example, recently stopped dosing of its DMD gene therapy PF-06939926 after one of the first six people to receive the treatment in a phase 1b study was hospitalized with acute renal injury.

RELATED: FDA surprises Sarepta by spurning its second Duchenne drug

Looking to expand its RNA offerings, Sarepta recently suffered a setback with an FDA complete response letter for golodirsen, which is designed for DMD patients who have genetic mutations subject to skipping exon 53. It’s also preparing to file an application for casimersen soon, with eyes on a 2020 approval.

Also building out its own gene therapy arsenal, Sarepta recently bought out former partner Myonexus along with its five gene therapies for limb-girdle muscular dystrophy. Several gene therapies are also under development either in house or via licensing for DMD, Pompe disease and others.

One of those candidates just triggered an alarm after a mistakenly filed entry in the FDA’s adverse events reporting system revealed that a clinical trial participant was hospitalized with rhabdomyolysis. Because the trial is blinded, there was no telling whether the patient had received the experimental gene therapy or placebo, Sarepta has said.

1. Sarepta Therapeutics

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