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Subject:  Gamida, biotech hidden gem? Date:  1/23/2019  2:12 AM
Author:  Fuma102 Number:  108219 of 115850

(opens pdf)

two products:
NiCord®: Product candidate for bone marrow transplant with curative potential
NAM-NK Immunotherapy (we'll ignore this for now as it's still early)

-"NAM causes metabolic reprogramming, leading to cell proliferation while preserving stem cells"
---> basically, you mix NiCord with cord blood, and you require way less cord blood / donations.
-addresses 80% of the Allogeneic stem cell transplant Candidate Patient Segmentation (total allogenic transplants = 5,000/yr in US)

Phase 1/2 Study of NiCord® in Patients with Hematologic Malignancies:
• Primary endpoint: Neutrophil engraftment
----> NiCord: 11 days vs 21 days

• Secondary endpoints: Platelet engraftment, acute GvHD, chronic GvHD, infections, hospitalization, non-relapse mortality, overall survival, disease-free survival
-----> Platelet engraftment NiCord: 34 days vs 46 days
-----> decreased infection rate, decrease hospitalization rate
-----> no word no survival / disease free survival

Currently in P3 study for allogenic transplant patients with hematologic malignancies
• Expected enrollment completion: 2H19; Topline data anticipated 1H20

The bad:
- primary end points that are novel and that are NOT morbidity / mortality do not fair as well as trials with morbidity/mortality as the primary endpoint.
-This has been around for awhile when browsing, but has had poor recruitment, or in some instances hasnt panned out.

The good:
-good potential for better patient outcomes here.
-less patient time in hospital
-one time infusion

The financial:
-TAM about 4,000 patients for hematologic malignancy.
-this might increase the number of transplants since donations can stretch further; also may encourage more transplants.
-about 300-600 new patients a year for aplastic anemia (currently in P1/P2 trials).

I have no idea how much this will cost; but whats the cost of 1-2 weeks in the hospital?? Might be worth looking more into.
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