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Specialty pharmaceutical company Santhera is drawing a line in research efforts with the candidate Puldysa (idebenone).
An initial analysis of the Phase III SIDEROS study shows that the objectives set are barely being met. Therefore, the program will be suspended, the company announced Tuesday.
In this study, the active ingredient was used to treat Duchenne muscular dystrophy (DMD) patients who were also being treated with glucocorticoids. The objective was to find out if the candidate Santhera would delay the loss of respiratory function that occurs in these patients. DMD is a genetically determined muscle disease that occurs in childhood and mainly affects children. Loss of respiratory function is one of the leading causes of death from this disease.
“Now we have pulled the tightrope at Puldysa,” explained Santhera CEO Dario Eklund in an interview with AWP. “These results are also a shock to us.” However, it must be honestly said that the candidate’s development story was anything but simple and rather shaped by the setbacks of recent years, the manager added.
It is important to look ahead in this situation and see the reverse as a new perspective. For the Santhera company, it is now about positioning itself for the future and moving forward in working with the candidate Vamorolone, Eklund continued.
Goals can hardly be achieved
Specifically, Santhera announced in the morning that the interim analysis was carried out by the independent Data and Security Monitoring Board (DSMB). According to the press release, the results indicate that the study will hardly achieve the objectives set.
Therefore, the study will be canceled, the European application for approval will be withdrawn and the Puldysa global development program will be terminated. Santhera intends to initiate a restructuring plan that will focus on maintaining key roles in bringing the DMD drug candidate Vamorolone to patients and continuing other ongoing programs, according to the announcement. These other programs reportedly include Lonodelestat for cystic fibrosis and other lung diseases and the gene therapy approach for developing congenital muscular dystrophy.
“For Vamorolone, we expect main data from the VISION-DMD registry study in the second quarter of 2021,” Eklund announced. Vamorolone is also used to treat DMD. “However, the approach with this candidate is completely different from Puldysa,” added the manager. The mechanism of action is different.
Number of job cuts is not yet clear
What exactly the restructuring will be like, he couldn’t say at this point. You have to redistribute the resources. “Ultimately, we will not be able to retain all of our employees,” Eklund said. However, he still cannot say for sure how many jobs will ultimately need to be cut.
According to the CEO, there are still questions regarding Santhera’s financial resources. First, you need to assess how much funding the restructuring will need, including adjustments to the number of employees.
The news surprised many analysts and market participants. At Mirabaud they are clearly disappointed. They would have deemed a negative interim analysis unlikely, especially after the additional data submitted for the now-suspended European review was absolutely supportive, experts say.
Price drop
The company now has the other major value driver, Vamorolone. Mirabaud analysts estimate the sales potential here at more than 500 million francs.
“Until then, we expect the company to advance its business development efforts to find a partner to develop vamorolone in additional indications beyond DMD, and the same applies to Lonodelestat for its potential in non-cystic fibrosis indications where we have potential. It is not only seen in various forms of bronchiectasis, but also as a new checkpoint inhibitor in the treatment of lung cancer. “
The price reaction, however, was clearly negative. However, after an initial price drop of nearly 50 percent, the shares rallied and halved the negative percentage to 25 percent.
According to traders, there is speculation in the market that partner Idorsia could further increase its stake in Santhera. Late last week Idorsia reported that it now owned 22.34 percent of Santhera. By November 2018, the biotech company had invested 13.3 percent in Santhera. The bet was part of Vamorolone’s agreement that Santhera replaced Idorsia.
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