A positive readout on Valrox, the late-stage hemophilia A therapy being developed by Biomarin Pharmaceutical, could have a significant impact on potential M&A opportunities for the company.
Valrox is on track to be approved as the first gene therapy option to treat hemophilia A. The hemophilia market is currently estimated at roughly $30 to 90 billion, based on approximately 117,000 patients worldwide.
In an investor call, Biomarin’s worldwide research head, Dr. Henry Fuchs, informed participants that the company would provide a top-line update on Valrox before June 7. While Valrox has demonstrated promising results in Phase 1 and 2 trials in the past, the upcoming data release at the three year mark will determine if the drug has long-term durability and meets safety standards.
If Valrox reports positive data, chances of regulatory approval from the FDA could be accelerated, meaning that Valrox could enter the market by 2020.
In this context, Valrox’s success could potentially attract significant M&A interest in Biomarin. Moreover, other companies working on gene-therapy drugs for hemophilia could also become more attractive for strategic acquirers.
In addition to Valrox, Sangamo Therapeutics’ SB-525 and Spark Therapeutics’ SPK-8011 are other leading candidates in the space.
Hemophilia A is a bleeding disorder caused by a deficiency of coagulation factor VIII (FVIII) function due to mutations in the F8 gene. Therefore, the success of these drugs is essentially determined whether they increase and then safely maintain high FVIII levels for longer periods of time.
Recently, Sangamo announced positive early results associated with SB-525. The results indicate early success for the treatment, but Sangamo will still has a long way to go in proving that SB-525 has a high durational impact on patients. It is likely that the efficacy of drugs such as SB-525 or Valrox would be assessed on the basis of durability and a high safety score.
In addition to SB-525 and Valrox, Spark Therapeutics’ SPK-8011 has also demonstrated early success in treating patients with Hemophilia A. Despite a few safety setbacks, SPK-8011 could be another serious player in the market.