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.



Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

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Albert Bourla, AP

UP­DAT­ED: Where's the Pfiz­er ef­fi­ca­cy read­out? CEO Bourla says 'soon,' but you're go­ing to have to wait for it

Pfizer CEO Albert Bourla had promised repeatedly that the pharma giant would know if its leading Covid-19 vaccine is effective by the end of this month — now just a few days away.

Instead, the company reported early Tuesday that it has yet to conduct any interim efficacy analyses. And it won’t now until sometime next month.

The news was included in a slide for their Q3 report.

In the morning Q3 call with analysts, Bourla says that they expect efficacy data “soon,” but noted that they wouldn’t be able to say anything until all the administrative work was done on the interim, which would take about a week. And he added that Pfizer isn’t going to say anything else about that hot topic until they have the data in hand.

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Charles Baum, Mirati CEO

UP­DAT­ED: Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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Yung Chyung, Scholar Rock CMO (Business Wire)

A dark horse en­trant in­to the spinal mus­cu­lar at­ro­phy field dou­bles its val­ue on some PhII da­ta

The last four years have seen a sudden explosion in treatments for spinal muscular atrophy, a neurodegenerative condition that once led patients — often young ones — with a grim prognosis and no options. The prognosis still isn’t rosy, but now there are three FDA-approved options, enough to make the choice of one difficult.

Now a fourth potential option has entered the mix. Today, Scholar Rock announced the results from a proof-of-concept testing their SMA drug by itself and in combination with Ionis’ Spinraza, showing that all patient cohorts improved on standard scales used for measuring motor function in people with SMA.

Three years in­to a PhI­II pro­gram for a failed Duchenne MD drug, Cataba­sis hauls down the flag and ad­mits de­feat

Three years ago, Catabasis CEO Jill Milne and the crew insisted they had found good reason for great cheer once they plumbed the data from their failed study for the Duchenne MD drug edasalonexent. Plunging into the extended open-label data, they said, you could find solid evidence of efficacy. And that justified a try in Phase III.

But they were wrong.

Monday, after the bell, the little biotech acknowledged that their pivotal attempt following the mid-stage flop was another failure. The primary, change in baseline on the North Star Ambulatory Assessment, and the secondary on timed function tests both came up short of statistical significance.

Ul­tragenyx slams the brakes on rare dis­ease study af­ter all 5 pa­tients are hit with a se­ri­ous set­back

Ultragenyx $RARE and its partners at GeneTx have been forced to halt early-stage study for a drug to treat rare cases of Angelman syndrome after all 5 of the patients being treated for the first time experienced a severe adverse event at the highest dose.

The patients in the study experienced “lower extremity weakness believed to be related to local inflammation due to GTX-102,” triggering a red light on dosing and enrollment.

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Gary Glick, Keith Flaherty

What can a biotech en­tre­pre­neur and a top sci­en­tist come up with on a cou­ple of shared flights? Well...

Just weeks before the pandemic hit the states, Gary Glick and Keith Flaherty had a chance to spend 12 hours together, talking craft and trade on board shared flights as they headed to and from a board meeting together.

It may have been the most fruitful 12 hours of Glick’s productive life. They certainly found plenty to talk about.

Glick, a well known biotech entrepreneur and inflammatory specialist, had bumped up to executive chair at IFM Therapeutics just weeks before, after running through a slate of sizable deals with some major pharma players like Novartis.