Biomarin already markets a drug for PKU known as Kuvan but its efficacy is limited. Peg-Pal is more effective and could be used by a larger swath of PKU patients.
3. The most intriguing drug in Biomarin's pipeline is BMN-111, which is under development for achondroplasia (dwarfism.)
Dwarfism is caused by a mutation in the FGFR3 gene, a negative regulator of growth. BMN-111 is designed to work by dimming the "always on" signal sent by the mutated FGFR3 gene, thereby helping patients grow. The trick for Biomarin is to get BMN-111 to reduce the effects of the FGFR3 gene but not shut it down completely because complete blockage of the gene can lead to abnormal growth and mental retardation.
Biomarin is expected to announce soon results from a phase I safety study in adult patients. While primarily designed to assess the safety and blood levels of BMN-111, the study may also offer some hints about the drug's efficacy. A larger 12-month study of BMN-11 is being planned for early next year.BMN-111 is exciting for Biomarin investors because the dwarfism population is many times greater than any of the orphan diseases the company typically focuses on. There are over 25,000 patients with dwarfism in the world and at least 500 are born every year. If the phase 1 safety study is positive, Wall Street will start including BMN-111 in the company's valuation. 4. Finally, before year's end, Biomarin will release data from its only non-orphan drug program: BMRN-763, a very potent PARP inhibitor. Early data from an ovarian cancer trial looked very promising. Updated results will be out in December. With four data events in the next three months, the rest of the year could be a very exciting time for Biomarin shareholders. Excitement doesn't always mean positive. If the GALNS phase III study fails, Biomarin's stock price will take a huge hit. I'll be very surprised if GALNS fails, but positive results in clinical trials can never be taken for granted. Consider using options to mitigate the downside risk in Biomarin just in case GALNS doesn't work. Rosenblum is long Biomarin.