In a PhI­II show­down with Spark, uniQure switch­es out its lead gene ther­a­py for he­mo­phil­ia B

Late last year, the strug­gling gene ther­a­py biotech uniQure $QURE did some painful cost cut­ting, drop­ping pro­grams, ax­ing staff and tai­lor­ing the pipeline to con­cen­trate on a he­mo­phil­ia B pro­gram for AMT-060, which they said could be hus­tled through a piv­otal pro­gram and in­to the mar­ket, rac­ing with a ri­val ther­a­py from in­dus­try leader Spark Ther­a­peu­tics.

Steven Pipe

This morn­ing, though, uniQure ex­e­cut­ed a quick switch on its lead ef­fort. AMT-060, which had been des­ig­nat­ed as a break­through drug at the FDA, has been re­placed by AMT-061. And the ex­ecs at uniQure say that both the FDA and the EMA have es­sen­tial­ly de­cid­ed to keep this new drug in their VIP drug pro­gram and on the fast track to a reg­is­tra­tion tri­al.

In­vestors loved it. UniQure’s stock shot up more than 60% af­ter the news hit.

The way uniQure makes this sound, re­searchers tweaked their orig­i­nal ther­a­py to come up with some­thing bet­ter. In their state­ment, the biotech says that AMT-060 and AMT-061 are iden­ti­cal ex­cept for “two nu­cleotide sub­sti­tu­tions in the cod­ing se­quence for FIX.”

The gene vari­ant, re­ferred to as FIX-Pad­ua, ex­press­es a pro­tein with a sin­gle amino acid sub­sti­tu­tion that has been re­port­ed in mul­ti­ple pre­clin­i­cal and non­clin­i­cal stud­ies to pro­vide an ap­prox­i­mate 8- to 9-fold in­crease in FIX ac­tiv­i­ty com­pared to the wild-type FIX pro­tein. All oth­er crit­i­cal qual­i­ty at­trib­ut­es of AMT-061 are ex­pect­ed to be com­pa­ra­ble to those of AMT-060, as AMT-061 uti­lizes the same AAV5 cap­sid and pro­pri­etary in­sect cell-based man­u­fac­tur­ing plat­form.

They al­so want to style this new AMT-061 as a much, much bet­ter ther­a­py.

Steven Pipe, pro­fes­sor of pe­di­atrics and pathol­o­gy and pe­di­atric med­ical di­rec­tor of the he­mo­phil­ia and co­ag­u­la­tion dis­or­ders pro­gram at the Uni­ver­si­ty of Michi­gan, had this to say:

“Based on the da­ta gen­er­at­ed to date, AMT-061 may be the first gene ther­a­py to pro­vide durable, cu­ra­tive ben­e­fits to near­ly all pa­tients with he­mo­phil­ia B, with­out the com­pli­ca­tions as­so­ci­at­ed with cap­sid-re­lat­ed im­mune re­spons­es.  I very much look for­ward to serv­ing as an in­ves­ti­ga­tor in this ex­cit­ing Phase III pro­gram.”

Once a leader in gene ther­a­py, uniQure had to drop Gly­bera — ap­proved in Eu­rope but nev­er pitched in the US — af­ter it was re­port­ed­ly used just one time. Now Spark ap­pears to be in the lead af­ter an FDA pan­el en­dorsed its gene ther­a­py for a rare eye dis­ease. And one-time play­er Di­men­sion Ther­a­peu­tics was forced out of he­mo­phil­ia B by weak re­sults, with Ul­tragenyx pick­ing up the rest of the pipeline in a buy­out.

The race goes on.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Tony Coles, Cerevel CEO

Cerev­el takes the pub­lic of­fer­ing route, with a twist — rais­ing big mon­ey thanks to ri­val da­ta

As public biotechs seek to climb out of the bear market, a popular strategy to raise cash has been through public offerings on the heels of positive data. But one proposed raise Wednesday appeared to take advantage not of a company’s own data, but those from a competitor.

Cerevel Therapeutics plans to raise $250 million in a public offering and another $250 million in debt, the biotech announced Wednesday afternoon, even though it did not report any news on its pipeline. However, the move comes days after rival Karuna Therapeutics touted positive Phase III data in schizophrenia, a field where Cerevel is pursuing a similar program.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,500+ biopharma pros reading Endpoints daily — and it's free.

Who are the women blaz­ing trails in bio­phar­ma R&D? Nom­i­nate them for End­points' 2022 spe­cial re­port

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

Bernat Olle, Vedanta Biosciences CEO

Cit­ing 'chal­leng­ing eco­nom­ic en­vi­ron­ment,' PhI­II-ready mi­cro­bio­me biotech lays off 20% of staffers

The market downturn isn’t just sweeping up public biotechs.

Vedanta Biosciences, a developer of oral drugs derived from the human microbiome, is laying off about 20% of its staff — an unfortunately common occurrence these days. But CEO Bernat Olle took the unusual step of sharing the decision on LinkedIn and offering to connect the employees being let go with any company that’s hiring in their areas.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,500+ biopharma pros reading Endpoints daily — and it's free.

Up­dat­ed: Amid mas­sive re­struc­tur­ing, Bio­gen looks to re­duce phys­i­cal pres­ence in Boston

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The subbleases are “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” Biogen spokesperson Ashleigh Koss wrote in a statement to Endpoints News, adding that the company has had subleases across several buildings for years.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,500+ biopharma pros reading Endpoints daily — and it's free.

Saurabh Saha, Centessa CEO (BIO19)

One of 2021's star biotech play­ers flags an­oth­er big set­back for the pipeline

Two months after scuttling their lead drug, Centessa’s executive team is back with the latest in a series of setbacks that have tanked its stock and blown holes in its strategic lineup of biotech subs.

The company reported in its Q2 post today that it has decided to scrap ZF874 after a patient demonstrated elevated liver enzymes — a classic red safety flag — in a Phase I study for alpha-1-antitrypsin (A1AT).

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,500+ biopharma pros reading Endpoints daily — and it's free.

Illustration: Kim Ryu for Endpoints News

Why non-opi­oid pain drugs keep fail­ing — and what's next for the field

In 1938, Rita Levi-Montalcini was forced to move her lab into her bedroom in Turin, as Mussolini’s facist government expelled Jewish people from studying or working in schools in Italy. Levi-Montalcini, then just a few years out of medical school and using sewing needles as scalpels in her makeshift lab, would soon discover nerve growth factor, or NGF, in chicken embryos.

Her discoveries formed the basis of our understanding of the peripheral nervous system and how cells talk to each other, and Levi-Montalcini went on to win the Nobel Prize in 1986. Much later, NGF was hailed as a promising target for new pain therapies, with some analysts quoting an $11 billion market. However, the latest anti-NGF candidate, Pfizer and Eli Lilly’s tanezumab, was rejected by the FDA last year because of a side effect that dissolved bone in some of its patients.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Hervé Affagard, MaaT Pharma CEO

One year in­to clin­i­cal hold, FDA has more ques­tions about 'pooled' mi­cro­bio­me ther­a­py

The FDA is still wary about a trial testing a microbiome therapy in patients with steroid-resistant acute graft-versus-host disease (aGVHD).

A year after MaaT Pharma’s IND application in the US was first met with a clinical hold, the French biotech said the agency is maintaining the hold. The crux of the matter, MaaT suggested, has to do with the way it puts together its drug candidate, which is administered as an enema (i.e. an injection of fluid into the bowel).

Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.