In addition to Cubist, another stock you might want to check out is Biomarin. It's something of a turnaround story - after a major acquisition blunder (Orapred) by the previous CEO, they now have new management and the pipeline looks very interesting. They've had an orphan drug business model (ala Genzyme), and have several approved drugs (Aldurazyme - 50/50 venture with Genzyme, and the recently approved Naglazyme which they own 100% of). The near term driver going forward is Phenoptin for PKU (phenylketonuria), a larger and more lucrative orphan indication. These orphan programs move along very quickly, and they expect the Phase 3 results around late March. This trial looks like a virtual slam dunk, since the only patients in the Phase 3 are those who responded well in the earlier Phase 2 (the same PKU patients who were in the Phase 2 moved right into Phase 3, but poor-responders in the Phase 2 were excluded from the Phase 3. This sounds like cheating, but that's how the trial was designed with the FDA, and it has to do with the nature of PKU and Phenoptin). The big wildcard is that Phenoptin (also known as 6RBH4) deficiency is also implicated in vascular disorders, including those associated with diabetes and cardiovascular disease generally (listen to the last several presentations for the mechanisms involved - very interesting). So it looks like Phenoptin is a shoe-in for approval for PKU (a lucrative orphan indication), and if the vascular trials look good (vascular Phase 2 initiates in mid-06), the upside is absolutely gigantic. Anyway, it looks like one to watch closely over the next several years.
Recent RSPI News
- Form 8-K - Current report • Edgar (US Regulatory) • 05/29/2024 01:03:20 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 02/02/2024 01:30:27 PM
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- Form 8-K - Current report • Edgar (US Regulatory) • 12/11/2023 01:45:18 PM
- Form 10-Q - Quarterly report [Sections 13 or 15(d)] • Edgar (US Regulatory) • 11/17/2023 09:06:03 PM
- Form NT 10-Q - Notification of inability to timely file Form 10-Q or 10-QSB • Edgar (US Regulatory) • 11/14/2023 09:05:14 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 10/12/2023 01:00:21 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 10/02/2023 12:45:26 PM
- Form 10-Q - Quarterly report [Sections 13 or 15(d)] • Edgar (US Regulatory) • 08/21/2023 08:05:55 PM
- Form NT 10-Q - Notification of inability to timely file Form 10-Q or 10-QSB • Edgar (US Regulatory) • 08/14/2023 08:15:24 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 08/09/2023 12:52:24 PM
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