Whither the Clinical Development of Iplex, Amy Harmon?
NYT science reporter Amy Harmon has neglected to provide important follow-up on Insmed’s clinical development of Iplex. In May, Harmon covered the story of Joshua Thompson, a victim of ALS, who was trying to obtain compassionate access to the experimental drug—a synthetic insulin-like growth factor that is linked to a binding protein.
Harmon’s unidimensional coverage stressed the cruel fallout of a corporate battle over drug ownership and FDA bureaucracy—both of which, she implied, impeded Thompson’s access to Iplex (for important background, go here). And Harmon downplayed the total absence of clinical data (including safety data) to support the use of Iplex in ALS; the promise of the drug lay solely in its theoretical ease of access to the central nervous system and in pumped-up anecdotes found on the World Wide Web. After publication of Harmon’s story, Insmed experienced a substantial increase in its share price.
The epilogue of Iplex development, for those who care and remember, can be found by searching Google News and by reading Insmed’s press releases. On June 25th, the company reported highly disappointing results from a phase 2 trial of the drug in myotonic dystrophy. Insmed’s low-priced stock plunged overnight, from about $2.25 to a dollar.
Then one month later, Insmed announced it would no longer supply Iplex to new patients. The company lost its capability to manufacture the “extremely complicated” drug following the sale of its Boulder, Colorado, facility to Merck (a sale which was announced in March).* Insmed also reported that it would discontinue the drug’s clinical development. Specifically a planned phase 2 study of Iplex in ALS would be postponed. (This, after a statement in May, in which Insmed reported that it was “working diligently” with the FDA to “make the necessary preparations” for a US clinical trial of Iplex in ALS.)
Insmed stated that the existing, limited supply of Iplex would be reserved for the 70 currently treated ALS patients (12 in the United States [including Thompson] and the rest in Italy through an expanded access program). The inventory would provide coverage for “no more than 24 months.”
In response to the news, the ALS Association issued, in part, the following statement:
The Association encourages the FDA and INSMED to establish partnerships with the ALS community to ensure that the program yields meaningful results that will guide the next steps in determining whether IPLEX is effective and safe for people with ALS.
Based on existing clinical and scientific evidence, The ALS Association cannot encourage or recommend the off-label use of this medication without substantive evidence of its efficacy through a rigorous clinical trial. The ALS Association is continuing to monitor and assess information about IPLEX as it becomes available to provide the public with the most up-to-date reports about its potential for ALS.
Since early August, Insmed can only offer penny stock. The company’s pipeline currently consists of 2 molecules, rhIGFBP-3 and INSM-18, which may have antitumor activity. The company hopes to find a corporate partner(s) for their clinical development.
ALS = amyotrophic lateral sclerosis.
* In mid-February, Insmed announced that it was selling its follow-on biologics platform to Merck for $130 million. The money would be used, in part, to finance the clinical development of Iplex. The sale was completed in early April.