Re­genxBio ral­ly fades fast af­ter biotech re­ports set­backs, hos­pi­tal­iza­tion in gene ther­a­py study

Re­genxBio $RGNX has some ex­plain­ing to do to­day.

In its quar­ter­ly re­port the gene ther­a­py com­pa­ny re­port­ed that it ex­pe­ri­enced sev­er­al set­backs dur­ing its Phase I/II study of RGX-501, one of 4 pipeline pro­grams at the biotech that’s be­ing de­vel­oped for ho­mozy­gous fa­mil­ial hy­per­c­ho­les­terolemia (HoFH) in a tri­al un­der­way at the Uni­ver­si­ty of Penn­syl­va­nia. In ad­di­tion to re­port­ing ad­verse events, the in­ter­im analy­sis failed to pro­duce ev­i­dence of LDL re­duc­tion in one co­hort — and may not be able to in the oth­er.

The biotech’s stock ini­tial­ly popped 10%, then be­gan to slide back down as news of the set­backs spread. With­in min­utes, the stock was 11% in the red.

Ken Mills, Re­genxBio CEO

Re­searchers say that fol­low­ing an ear­li­er re­port­ed im­mune re­sponse in a pa­tient ac­com­pa­nied by tran­sient hy­poten­sion and el­e­va­tion in transam­i­nas­es, a sec­ond pa­tient was hos­pi­tal­ized to treat transam­i­nas­es while all three in that group “ex­pe­ri­enced an el­e­va­tion in transam­i­nas­es 4-6 weeks post-dos­ing. The peak ALTs were 165, 388 and 1469 IU/L in the three sub­jects. All three sub­jects were asymp­to­matic and re­spond­ed rapid­ly to the ini­ti­a­tion of pred­nisone fol­lowed by a slow ta­per, with nor­mal­iza­tion of the transam­i­nas­es.”

This ther­a­py us­es an AAV vec­tor to de­liv­er the hu­man low-den­si­ty lipopro­tein re­cep­tor gene to liv­er cells.

It was Co­hort 1 that failed to pro­duce signs of ef­fi­ca­cy, while for Co­hort 2:

Re­genxBio be­lieves that the abil­i­ty to as­sess LDL-C lev­els at 12 weeks in the three sub­jects in Co­hort II may be con­found­ed by the po­ten­tial ef­fects on the liv­er and the re­sult­ing steroid ther­a­py.

As a re­sult of the prob­lems, the com­pa­ny says it will change up the study de­sign, go­ing in ear­ly with steroids and push­ing back the ef­fi­ca­cy time­line from 12 weeks to 26 weeks while adding more pa­tients to the tri­al.

The biotech much pre­ferred to re­port on some mixed but pos­i­tive ear­ly re­sults from an­oth­er ef­fort it has un­der­way for RGX-314 for wet, age-re­lat­ed mac­u­lar de­gen­er­a­tion. The ther­a­py is de­signed to re­place an­ti-VEGF in­jec­tions, and af­ter in­jec­tions 3 pa­tients in one co­hort were free of the in­jec­tions with some ear­ly signs of vi­su­al im­prove­ments among the pa­tients in the ear­ly-stage study.



John Hood [file photo]

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The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

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UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

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As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

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Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

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