Avro­bio shares blast­ed af­ter tri­al up­date sours out­look on gene ther­a­py for Fab­ry dis­ease

Avro­bio made the leap on­to Nas­daq a few months ago and then pro­ceed­ed to run the ta­ble large­ly on a snap­shot of ef­fi­ca­cy for its lead gene ther­a­py to treat Fab­ry dis­ease. On Mon­day, though, an up­dat­ed look at a key da­ta point sent in­vestors run­ning, col­laps­ing the share price.

By the end of the day on Mon­day Avro­bio’s stock $AVRO had been beat­en down 52%, wip­ing out hun­dreds of mil­lions of dol­lars in mar­ket cap.

Ge­off MacK­ay

On the sur­face, Avro­bio’s press re­lease in­di­cat­ed noth­ing less than con­tin­ued suc­cess with AVR-RD-01, with CEO Ge­off MacK­ay say­ing he was “en­cour­aged” by the ther­a­peu­tic ef­fect re­searchers were see­ing in sev­er­al pa­tients. Their lentivi­ral treat­ment ap­peared to be do­ing the job it was de­signed to do — in­sert­ing healthy copies of the GLA gene that en­codes for a de­fi­cient en­zyme in Fab­ry dis­ease and see­ing a rise in en­zyme ac­tiv­i­ty.

But the slide show for the day al­so in­clud­ed a dis­turb­ing sight, with the vec­tor copy num­bers in pe­riph­er­al blood drop­ping in 4 out of 4 cas­es over a span of any­where from 1 to 18 months. 

The VCN is a key mark­er for the trans­fer of healthy genes in­to cells. And as any­one who fol­lows blue­bird bio can tell you, if the VCN isn’t right, then no­body’s hap­py. Blue­bird went back to the draw­ing board af­ter track­ing VCN num­bers of 0.3 and 0.6 in the first gen­er­a­tion of its gene ther­a­py for be­ta-tha­lassemia, herald­ing a spike to 1.5 and 2.1 for two pa­tients in a Lenti-Glo­bin study. And blue­bird knows full well that a high­er, or ris­ing, VCN breeds con­fi­dence.

So what spooked in­vestors Mon­day? Here are the de­tails:

Pa­tient #1 start­ed ther­a­py with a VCN of 0.7 and then re­searchers tracked a steady drop to 0.1 18 months post-treat­ment. Pa­tient #2 dropped from 1.4 to 0.4 in 6 months. Pa­tient #3: 0.8 to 02 in 1 month. And pa­tient #4 went from 0.7 to 0.5 in a few months.

In gene ther­a­py, com­pa­nies are push­ing to cre­ate new once-and-done treat­ments. Any in­di­ca­tion that their prod­uct can’t stand up over time plays hav­oc with the busi­ness mod­el. So in­vestors bailed on the stock.

Avro­bio still has plen­ty of op­por­tu­ni­ties to do bet­ter. But next time you can ex­pect a lot of peo­ple to move straight to the VCN as the com­pa­ny talks about ther­a­peu­tic ef­fects.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aromatic amino acid decarboxylase (AADC) deficiency is an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, such as dopamine and serotonin, usually leading to developmental delays, weak muscle tone and inability to control the movement of the limbs. It can also lead to multiple organ failure.

To date, there have been no treatments approved for AADC deficiency, which has been identified in less than 150 patients.

Ather­sys tries to post-hoc-an­a­lyze its way out of an­oth­er tri­al fail for stroke stem cell ther­a­py

Athersys’ stem cell therapy has failed yet again.

In a 206-person trial conducted in Japan, Athersys’ stem cell therapy for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores.

While a greater percentage of patients in the treatment group reached the primary endpoint compared to placebo, that difference was not statistically significant.

Castle Creek Biosciences chair Jeff Aronin

Scoop: Af­ter pulling IPO am­bi­tions last De­cem­ber, Jeff Aron­in's Cas­tle Creek turns to pri­vate back­ers

Jeff Aronin’s cell and gene therapy biotech Castle Creek Biosciences has raised $112 million in equity, Endpoints News has learned.

The Exton, PA, biotech secured the financing from 54 investors, according to an SEC filing dated May 2. The late-stage startup had last year considered a $100 million Nasdaq debut, but in a sign of the bear market that has plagued hundreds of newly minted public biotechs, Castle Creek pulled those ambitions in the last few weeks of 2021.

Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.