Shire ($SHPG) is having a mixed 24 hours after yesterday posting positive ADHD data for its long-delayed SHP465--which saw its shares rise by 6%--but this morning saw the failure of a Phase II treatment for extremely premature infants.
First, to the good news: Its once-daily SHP465 met its primary endpoints in both the low and higher doses by besting placebo in ADHD symptom improvement.
Ireland’s Shire already markets an ADHD drug in the form of Vyvanse (lisdexamfetamine dimesylate), a stimulant that’s FDA-approved in patients 6-years and up with ADHD, as well as for treating moderate to severe binge eating.
It also markets the aging Adderall franchise, a type of amphetamine for ADHD, although it has now begun to see generic competition.
SHP465 (triple-bead mixed amphetamine salts) is an investigational oral stimulant, and is targeting adult patients who only need to take the pill once a day in the hope of shoring of the lost revenue that will eventually come from its older meds.
It’s not been all plain sailing, however, and SHP465 has been hit by FDA knockbacks. It was set to submit last year, but was required to give the Agency more data. The company has said however that it has now fulfilled the “requirement to evaluate the safety and efficacy of SHP465 in children and adolescents prior to filing a Class 2 resubmission of the medicine for FDA approval.”
The biopharma said in a statement that it was “on track” for a 2H 2017 submission to the FDA. Shire was up 6% yesterday on the news.
But it's not been all good news after the company today posted new midstage data for its experimental SHP607, which was aiming to reduce the severity of retinopathy (a disease of the retina which can cause blindness) of prematurity, but missed this primary endpoint.
The company was at pains to stress that the drug--an investigational protein replacement--did however show “clinically relevant effects in secondary endpoints,” although it still managed to miss a secondary endpoint as well.
On the positive side, the secondary endpoints it did hit were those related to the development of severe bronchopulmonary dysplasia (BPD) and severe intraventricular hemorrhage (IVH), a type of brain injury.
Top-line data showed a 53% reduction in the incidence of severe BPD as compared to untreated infants. The data also showed a 44% reduction in the incidence of severe IVH in all assessed patients that received SHP607, as compared to untreated infants.
But there was bad news here too as one of the secondary endpoints--that of time to discharge from neonatal intensive care--was not met.
Shire, naturally, is trying to look on the bright side with Philip Vickers, head of research & development at the company, saying: "Although the study did not meet its primary endpoint, we are extremely encouraged by the topline secondary endpoints related to lung and brain.
“For severe complications related to the lung and brain, there are no approved treatment options, and these data support our commitment to further investigate the potential systemic benefits of SHP607 in this population where the unmet patient need is substantial."
Shire said it now “expects to begin discussions with regulatory authorities about a Phase III clinical program focusing on clinically relevant complications of prematurity,” which may see it re-jig its endpoints in the next stage of testing.
Analysts at Bernstein were cautiously optimistic. In their initial thoughts on the data, the firm said in a note to clients that Shire was overall able to provide premature babies with a “broad-effect growth factor they were missing (which usually supplied by the mother) and measure all potential outcomes.” While noting the company’s trial failures, it added that Shire was still “able to influence other conditions associated with very premature birth.”
But the analysts believe that, while still a risk, the negative results may not be as bad as it first seems. “This is the first test of IGF-1 in pre-term babies and the trial was 'hypothesis generating'--put the drug in and see what you can improve (one could argue that if you measure enough things, something will come up). However, it should not be too surprising IGF-1 has beneficial effects in pre-term babies and there appears to be a correlation between achieving blood level and outcomes.”
Bernstein expects the drug to go into a “high risk p3,” but are still touting it as a blockbuster drug. A similar sentiment was shared by Leerink, who said that the program “remains viable.”
And its seems the market agrees, with Shire's shares up 1.3% by 10am ET.
Shire's long-delayed ADHD drug hits another FDA speed bump