Opko Health Inc (OPK)

[terry hallinan]

Eloxx Pharmaceuticals Reports Full Year 2017 Financial and Operating Results and Provides Business Update

https://tinyurl.com/y9a88jej

Eloxx is an Israeli start-up that had heavy backing from Frost and other wealthy investors that now has a headquarters in Waltham, MA, as well as Israel. OPK has a significant minority interest as well as a Frost family fund.

So?

So this is just one of the minority "investments" of OPK that get largely overlooked by analysts and stockholders alike though potential is vast.

ELX-02 in Cystic Fibrosis and Cystinosis

ELX-02 is our lead asset and our current development programs are focused on cystic fibrosis and cystinosis patients with diagnosed nonsense mutations. To advance the program, we have held pre-clinical trial application (CTA) discussions with the Federal Agency for Medicines and Health Products (FAMHP) in Brussels, Belgium and pre-IND discussions with the U.S. Food & Drug Administration (FDA) for cystic fibrosis and cystinosis, respectively.

Currently, the European Medicines Agency (EMA) has designated ELX-02 as an orphan medicine for the treatment of mucopolysaccharidosis type I (MPS I), and the FDA has granted orphan drug designation to ELX-02 for the treatment of MPS I and for the treatment of Rett Syndrome.

We are on-track for an expected mid-2018 submission of our CTA in Belgium for cystic fibrosis.

We expect to initiate Phase 2 studies in cystic fibrosis and cystinosis, by the end of 2018, subject to regulatory review and clearance of our CTA and IND, respectively.

[I was surprised by word of Phase II trials being initiated though cystic fibrosis is a prime personal interest because of a cousin who died of that terrible "nonsense" disease in Ireland where it is most common.]

As part of our clinical program, we have completed a Phase 1 single ascending dose (SAD) study in a total of 60 healthy volunteers at sites in Israel (ClinicalTrials.gov Identifier: NCT02807961) and Belgium (ClinicalTrials.gov Identifier: NCT03292302). Currently ongoing is the Phase 1 multiple ascending dose (MAD) study in 45 healthy volunteers in Belgium (ClinicalTrials.gov Identifier: NCT03309605)....

The first I heard of Eloxx it was described as an association of researchers in Israel but obviously it went far beyond that.

For perspective, consider muscular dystrophy is another nonsense disease [name comes from antisense lingo] with some 60 variations, all of whom ELOX hopes to cure rather than just treat as in the case of the headline news from Sarepta only treating the Duchenne variation.

Frost doesn't go small. His targets are large and expensive with success elusive but tending to be very large.

I grant critics failure can also be very large.

Just a thought for you. I have no separate investment in ELOX for now.

Best, Terry