MOD-5023 / OPKO Health, LeaderMed - LARVOL DELTA

LeaderMed Group and OPKO Health Form Joint Venture to Develop and Commercialize Oxyntomodulin and Factor VIIa-CTP in China and Other Asian Territories (GlobeNewswire) - "LeaderMed Health Group Limited...and OPKO Health, Inc...announce the formation of a joint venture to develop...two of OPKO’s clinical stage, long-acting drug products in Greater China and eight other Asian territories. Under the terms of the agreements, OPKO will grant the joint venture exclusive rights to develop, manufacture and commercialize (a) OPK88003...and (b) Factor VIIa-CTP...in exchange for a 47% ownership interest in the joint venture. In addition, OPKO will receive an upfront payment of $1 million and will be reimbursed for clinical trial material and technical support it provides the joint venture. LeaderMed will be responsible for funding the joint venture’s operations, development and commercialization efforts and will, with its syndicate partners, initially invest $11 million in exchange for a 53% ownership interest. OPKO retains full rights to oxyntomodulin and Factor VIIa-CTP in all other geographies."

Licensing / partnership • Genetic Disorders • Hemophilia

Prolor Biotech to present new preclinical data on its long-acting clotting Factor VIIa at leading European Scientific Conference (Prolor-Biotech) - "Prolor Biotech...announced that the company will present new data on its long-acting clotting factor VIIa... at EAHAD...expect to initiate a Phase II clinical trial in hemophilia patients in late 2013 or early 2014."

Anticipated new P2 trial • Hemophilia

A long-acting FVIIa -CTP proposing an improved prophylactic and on demand treatment for hemophilic patients following SC and IV administration – evaluation in animal models (ISTH 2013) - Abstract #OC 83.6; "Attachments of CTP to FVIIa led to a markedly enhanced PK, increased exposure as reflected by AUC, improved recovery and a prolonged hemostatic effect in hemophilic mice with a comparable specific activity to rFVIIa."

Preclinical • Hemophilia

Assessment of MOD-5014, a long acting FVIIA, pharmacokinetic, pharmacodynamics and correction of hemophilic coagulopathy in dogs with hemophilia a as part of the preparation for first in human study (ISTH 2015) - Abstract #PO048-WED; "Infusions were well tolerated with no injection site reactions or significant changes in lab results. MOD-5014 demonstrated a dose dependent response as reflected by TEG, PK and PD parameters and normalized the response to maximal efficacy maintained for a long period of time. MOD-5014 performance was
compared to published data for commercial rhFVIIa further confirming MOD-5014 long acting properties (3–4 fold higher compared to rhFVIIa)."

Preclinical • Hemophilia

OPKO announces third quarter operating and financial results (Businesswire) - "OPKO’s development programs for long acting Factor VII and long acting oxyntomodulin continue to progress toward entering clinical trials during 2015."

Anticipated new trial • Hemophilia

A Single-dose, Dose-escalation Study of a Long-acting MOD-5014 in Healthy Adult Male (clinicaltrials.gov) - P1; N=32; Completed; Sponsor: Opko Biologics; Recruiting ➔ Completed; N=64 ➔ 32

Enrollment change • Trial completion • Biosimilar • Gene Therapies • Genetic Disorders • Hematological Malignancies • Hemophilia • Oncology

A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B (clinicaltrials.gov) - P1/2; N=24; Recruiting; Sponsor: Opko Biologics; Not yet recruiting ➔ Recruiting

Enrollment open • Biosimilar • Gene Therapies • Hematological Malignancies • Hemophilia

Factor VIIa-CTP: Initiation of P2/3 study for hemophilia in mid 2018 (Ladenburg Thalman Healthcare Conference 2017, OPKO health)

New P2/3 trial • Genetic Disorders • Hemophilia

Factor VIIa-CTP: Initiation of P2/3 study for hemophilia in mid 2018 (Ladenburg Thalman Healthcare Conference 2017, OPKO health)

New P2/3 trial • Genetic Disorders • Hemophilia

OPKO Health announces dosing of first patient in phase 2a clinical study of a long-acting factor VIIa for the treatment of hemophilia (Businesswire) - "OPKO Health...today announced dosing of the first patient in a Phase 2a study evaluating the safety of a long-acting Factor VIIa in hemophilia patients. 'Later this year, we expect to initiate a second Phase 2a trial for Factor VIIa-CTP in a subcutaneous formulation that, if successful, could provide prophylactic therapy to prevent bleeding episodes and thereby change the treatment paradigm for patients with hemophilia.'"

Anticipated new P2a trial • Enrollment open • Hemophilia

OPKO: Barrington Research Fall Investment Conference (OPKO health) - Anticipated initiation of P2a trial for hemophilia in Q4 2015

Anticipated new P2a trial • Hemophilia

Several value creating events ahead for OPKO health (SeekingAlpha) - "Factor VIIa-CTP for the treatment of bleeding episodes in hemophilia A or B. During Q1, the company initiated a phase 2a dose escalation study and expects to have interim data by year-end....Good efficacy and safety data could result in the market assigning more value to this candidate."

Anticipated P2a data • Hemophilia

A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B (clinicaltrials.gov) - P1/2; N=27; Completed; Sponsor: Opko Biologics; Active, not recruiting ➔ Completed; Trial completion date: Apr 2019 ➔ Sep 2018

Clinical • Trial completion • Trial completion date

A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B (clinicaltrials.gov) - P1/2; N=27; Active, not recruiting; Sponsor: Opko Biologics; Recruiting ➔ Active, not recruiting; Trial completion date: Sep 2018 ➔ Apr 2019

Clinical • Enrollment closed • Trial completion date