ODD Approval Rates Jason Napodano: For indica

ODD Approval Rates

Quote:

---

Jason Napodano: For indications outside of oncology, the likelihood of eventual FDA approval for orphan drugs is higher than for non-orphan drugs at all stages of clinical development. The average non-oncology Phase 1 drug in the BioMedTracker database has a 10% chance of eventual approval; the subset that are in development for orphan indications has a historical approval rate of 30%, and those that have received orphan drug designation have a historical approval rate of 44%.

---

Orphan Drug’s Probability of advancing to the next phase / Liklihood Of Approval (LOA)
Phase 1 to Phase 2 64.5% / 10.4%
Phase 2 to Phase 3 32.4% / 16.2%
Phase 3 to NDA/BLA 60.1% / 50%
http://www.nature.com/nbt/journal/v32/n1/fig_...86_T9.html

-- I believe pre-clinical drugs that have survived the Lead Optimization phase have roughly a 65% chance of progressing to a Phase 1 trial.

All drugs/BLAs probability of advancing / LOA
Phase 1 to Phase 2 71% / 21%
Phase 2 to Phase 3 44% / 30%
Phase 3 to NDA/BLA 69% / 69%

- Data from DiMasi's 2003 study referenced in Valuation in Life Sciences

Quote:

---

Jan. 2014, Joboggi: Jason, what is the current likelihood that AMBS will get a product to market?

---

Quote:

---

Napodano: Still waiting for some long-term data validation on LymPro, but I would say as long as the existing data holds and compares well with the retrospective analysis and new data they plan to generate in the next few months, the likelihood of CLIA approval for LymPro is high, I’d say at least 50%.

MANF is a preclinical asset, and historically preclinical drugs make it to market at about 1-2% after the IND is filed. MANF may be a tad higher, say 5%, given that proof-of-concept has already been demonstrated with drugs like GDNF; plus they are looking at like 3-4 different indications, so there are multiple shots on goal.

Eltoprazine is moving into a phase 2b study. Odds from this level to commercialization are historically around 15-20%. Eltroprazine looks safe, so that’s a plus. The fact that the MJFF has highlighted the drug as one of its targets for LID also helps. AMBS also seems to think eltoprazine will have utility in ADHD, so we’ll see. I wouldn’t put more than 20% odds right now though.

The thing about AMBS is, really if only 1 of these 3 drugs makes it to market there’s still huge upside to the shares. High risk/high reward.
Jason.

---