June 2, 2015
By Alex Keown, BioSpace.com Breaking News Staff
CAMBRIDGE, Mass. -- In only two months at the helm of Sarepta Therapeutics , Edward Kaye, the interim-chief executive officer, is bringing the company’s lead drug to treat Duchenne muscular dystrophy closer to regulatory approval in the United States, and he’s doing well enough to be considered as a candidate for the position permanently, a spokesman told BioSpace Tuesday.
Ian Estepan, a spokesman for Sarepta, told BioSpace that the board of directors has not set a timetable for a new CEO search. He said Kaye will certainly be a candidate for the position. “The focus of the company is to get through the regulatory process,” Estepan said.
Duchenne muscular dystrophy, caused by an absence of dystrophin, a protein that helps keep muscle cells intact, is a genetic disorder characterized by progressive muscle degeneration and weakness. The disease affects approximately one in every 3,500 boys born worldwide. There are about 20,000 new cases diagnosed globally each year. The condition is universally fatal, and death usually occurs before the age of 30. There is currently no approved therapy in the United States for DMD.
Since taking over at Sarepta, Kaye seems to be doing what is necessary to raise the profile of the company’s treatment platform and of Duchenne muscular dystrophy, a disease that often appears in children, particularly young boys, and leads to death of most by the time they reach age 30. As he attempts to guide the company to its first regulatory-approved drug, Kaye is combining both a practical and empathic approach to guiding Sarepta and achieving regulatory approval for eteplirsen, Sarepta’s treatment for Duchenne. A recent interview with Kaye conducted by the Boston Business Journal provides several key insights into how Kaye will, at least in the interim, guide the company.
1. Kaye is reminding Sarepta’s staff of the human element in developing drugs for deadly disorders. As someone who knows what it is like to lose a child, Kaye is taking an empathic approach to working with would-be customers.
2. Cost is also an important factor Kaye understands. Although no price point for the drug has yet to be set, a company spokesman said the drug will likely have a cost of hundreds of dollars. In the interview, Kaye certainly understood that economic factor. “My whole vision is, every person in this company who touches the patient understand the importance of Duchenne muscular dystrophy and what it means. And that means having systems in place to help people get reimbursement. It’s going to be an expensive drug,” he said in the interview.
3. While eteplirsen has encountered some speed bumps along the way to regulatory approval, Kaye has the experience of guiding drugs through the process. Before joining Sarepta in 2011, he oversaw nine drug programs at Cambridge-based Genzyme Corporation. The drugs he oversaw included the recently approved Cerdalga, which treats Gaucher disease and a drug to treat Fabry disease.
4. Although new to helming a company, Kaye had experience working not only with the medical and clinical side of the business, but also with the commercial side, which provides additional insight into the mindset of patients and their families.
Not only does Kaye has a challenge to finally secure Sarepta’s first U.S. Food and Drug Administration (FDA) approval, the company will need to do so before competitor BioMarin Pharmaceutical Inc. secures approval for its own Duchenne muscular dystrophy treatment. In April BioMarin announced its own rolling NDA submission to the FDA for its drug candidate Drisapersen. Drisapersen has been granted Orphan and Fast Track status, as well as Breakthrough Therapy designation by the FDA.
At the same time Pfizer Inc. is developing a myostatin inhibitor, PF-06252616 to treat DMD. The pharmaceutical giant is currently conducting a mid-stage clinical trial with results expected in early 2017. Pfizer’s drug may have a wider reach than the treatments being developed by Sarepta and BioMarin, which will only impact about 15 percent of patients with DMD.
But, Sarepta has made great strides into finally breaking through the approval barrier. On May 20 stock prices soared 50 percent following the company’s meeting with the FDA regarding a new drug application for eteplirsen, Sarepta’s treatment for Duchenne muscular dystrophy, when the two entities agreed to initiate a rolling NDA submission – a long-awaited result for Sarepta. Since April 2014, federal regulators have been working intensively to help Sarepta provide the additional data and analyses needed to support an NDA, the FDA said in a statement. The FDA had been critical of Sarepta’s methods used to measure dystrophin.
The FDA consistently said it would be necessary to include data in its NDA demonstrating that eteplirsen increases production of the muscle protein dystrophin. Regulatory agents said they were concerned the measurements were not adequately robust to support an NDA submission. But the relationship between the company and the FDA took a huge step forward 11 days ago after the company presented a new and detailed data package to the regulatory agency. The company plans to submit total components required by the regulatory agency by mid-2015.
In a company statement, Kaye said the rolling submission is aimed at facilitating regulatory review.
“The initiation of our NDA submission for eteplirsen marks a significant milestone for the Duchenne community and we look forward to completing the submission by the middle of the year and continuing to work with the agency towards the goal of providing treatments to patients as quickly as possible,” he said.
Kaye took over the top spot at Sarepta two months ago following the abrupt resignation of former CEO Chris Garabedian, who recently saw his relationship with corporate investors and federal regulators became tense as he unsuccessfully pushed for approval from the FDA for eteplirsen. Before taking the CEO job, Kaye served as chief medical officer, where he was responsible for the company’s medical and clinical operations.
Sarepta came under withering criticism over the last year for failing to get timely approval from the FDA for eteplirsen. John Hodgman, Sarepta’s interim chairman of the board, said the change in company leadership “will facilitate the company’s clinical and regulatory discussions and relationships with the goal of meeting its stated timelines for bringing a potentially disease-modifying treatment to patients with DMD (Duchenne muscular dystrophy) as soon as possible.”
According to Salary.com, as Interim CEO, Kaye will earn $1,703,921 in total compensation, including $399,017 in salary, $131,676 as a bonus and $1,155,231 in stock.
When he took over as interim-CEO, the company board of directors tasked Kaye to focus on regulatory approval of eteplirsen and Sarepta’s other products, he said in an interview with Forbes. Although he said he would be interested in permanently taking on the job, his first duty is to focus on the regulatory work. Kaye has certainly been sticking to that directive, but other positives are happening as well. In April Sarepta Therapeutics increased its employment by 40 percent, raising its number of employees to 204.
Sarepta’s stock was down Monday from the opening of $25.8 per share.
Will PfizerKline Become the Next Pharma Player?
The speculation surrounding a possible bid from Pfizer Inc. for struggling GlaxoSmithKline is heating up, after one closely-watched biotech analyst said in a note last week that Pfizer buying the company would “unlock access to its balance sheet and improve its tax situation.”
Gregg Gilbert, a biotech analyst at Deutsche Bank, wrote in a note to investors “Introducing PfizerKline” that he thinks a deal would be “materially accretive” for both companies. Gilbert estimated that a bid priced at $29.86 a share, via half stock and half cash, which would push up Pfizer’s earnings per share by 10 percent to 16 percent beginning in 2016.
“We believe that the company has a sense of urgency to create value by leveraging the power of its balance sheet to do needle-moving deals,” Gilbert wrote. “Since media reports in the past have pointed to the potential for a Pfizer/GSK combination, we are revisiting that theme.”
We want to know, dear readers, if you agree? Should Glaxo continue going it alone, or might Pfizer buy it and create one of the world’s largest pharma players in history?
