FDA Rejects PTC's Friedrich's Ataxia Drug; Eli Lilly Raises Nearly $7B in Bond Sale

PTC Therapeutics Faces Setback in Friedrich's Ataxia Treatment

The Food and Drug Administration (FDA) has rejected PTC Therapeutics' experimental drug vatiquinone for the treatment of Friedrich's ataxia, a rare neuromuscular disorder. The agency determined that the data supporting the drug did not provide "substantial evidence of efficacy," according to a disclosure by PTC on Tuesday.

The FDA's decision requires PTC to conduct another positive study before resubmitting its application. This outcome was not unexpected by analysts, as PTC executives had recently indicated they were not in labeling discussions with the agency. The main study of vatiquinone failed to show a significant change in the trial's primary endpoint, although PTC claimed positive findings on a prespecified secondary measure.

Eli Lilly Secures Significant Funding Through Bond Sale

Eli Lilly has successfully priced nearly $7 billion in corporate bond issues, including a $1 billion long-dated tranche set to mature in 40 years. The pharmaceutical giant stated in its prospectus that the proceeds from the bond sales would be used for "general corporate purposes," potentially including the repayment of existing notes.

As of the end of June, Lilly reported holding approximately $3.4 billion in cash and cash equivalents, with total debt nearing $40 billion. The company's strong financial position is underscored by its net income of about $8.5 billion for the first six months of the year.

Regulatory Delays and Industry Collaborations

The FDA has extended its review deadline for Regenxbio's potential first-in-class gene therapy for Hunter syndrome. The decision date has been pushed back by three months, from November 9 to February 8, following the company's submission of additional longer-term data from the key trial supporting its application. Regenxbio maintains that the new data is consistent with previously submitted results and that no safety concerns have been raised by the agency.

In other industry news, Skyhawk Therapeutics has entered into a collaboration with Merck KGaA to develop RNA-targeting small molecules for unspecified neurological diseases. The deal, which could be worth more than $2 billion overall, will see Skyhawk leading discovery and preclinical work, with Merck taking charge of further development and commercialization for licensed drugs.

Lastly, Roche's Genentech division has announced the termination of its long-standing alliance with Adaptive Biotechnologies for the development of personalized cell therapies for cancer. The collaboration, which began in January 2019 with a $300 million upfront payment to Adaptive, will officially end on February 9, 2026. This marks the second recent cancellation of a cell therapy partnership by Genentech, following the termination of its collaboration with Adaptimmune last year.