3 Things In Biotech, April 14: Organovo, Catalyst, And Aeglea Open Up

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Welcome to another edition of “3 Things In Biotech,” a daily digest dedicated to helping you keep pace with the fast-moving world of pharmaceutical and biotechnology research.

Organovo shows signs it can model liver damage

Company: Organovo (ONVO)

Therapy: 3D bioprinted liver tissue

Disease: Non-alcoholic fatty liver disease

News: ONVO presented preclinical findings at EASL 2018 demonstrating the feasibility of its bioprinted liver tissue as a model for NASH, wherein it was able to show the induction of steatosis upon chronic exposure to lipids and high glucose levels. This led to biochemical phenomena reminiscent of NASH in an environment that recapitulates the structure and function of liver tissue.

Looking forward: If successful, ONVO has on its hands a powerful tool for testing new drugs that might be effective in NASH, which is a major area of expansion for companies involved in liver disease therapy (you can find a whole series of workup on NASH and current prospects in the Total Pharma Tracker). It’s even possible that this system could be used to dig deeper, building patient-specific variations on the technique to more precisely screen for useful compounds. For a company trying to find footholds in the fallout of regenerative medicine’s failure to launch, this could be a useful road forward.

Still, this is still an early-stage development, and I would stress caution before buying.

Catalyst ramps up its early hemophilia study in Korea

Company: Catalyst Biosciences (CBIO)

Therapy: CB-FIX

Disease: Hemophilia B

News: CBIO announced that the Korean Ministry of Food and Drug Safety has approved the addition of a sixth cohort of patients to its phase 1/2 study investigating CB-FIX, a subcutaneous form of Factor IX that is used to treat hemophilia B. The sixth cohort will enroll a larger number of patients, providing firmer support for whatever safety findings are identified in the study.

Looking forward: It may not seem like much in the ever-evolving world of hemophilia therapy to attack hemophilia from the point of a new formulation of therapy. However, for patients who will not have access to gene therapies (or for those who do not respond), recombinant Factor IX will continue to be a needed option. And the subcutaneous nature of delivery means that longer-term dosing is possible compared with IV formulations currently on the market.

So this could be an important beginning for CBIO if its phase 1/2 study shows suitable safety and signs of efficacy.

Aeglea shares a peek of the promise of arginase replacement therapy

Company: Aeglea BioTherapeutics (AGLE)

Therapy: Pegzilarginase

Disease: Arginase 1 deficiency

News: AGLE presented very early findings from a phase 1/2 trial investigating the safety and efficacy of arginase replacement therapy in patients with arginase 1 deficiency. This report included findings from two patients (out of a total planned enrollment of ten patients), both of whom demonstrated no evidence of an immunologic reaction to the enzyme. Furthermore, both patients experienced an improvement in the six-minute walk test, increasing the distance the patients could walk by 23% to 31%. Furthermore, laboratory assessment suggested that arginine levels were indeed decreased in the serum, as expected.

Looking forward: While it’s early, and in a very small number of patients, these findings are encouraging, as they suggest some meaningful improvement in motor function in these patients. It will be critical, moving forward, to see the longer-term follow-up data to see if the improvements in function persist or if they decline, since the improvement was observed rather rapidly, which throws into question where the treatment benefit is coming from.

These findings are still very early, overall, and I would not insist on buying into AGLE on this alone. However, it should warrant a deeper dive.

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Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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