With the ‘worst’ FDA head out and Huntington’s disease advocates pressing for bipartisan reform, uniQure announces new plan to seek gene therapy approval

  

On June 17, uniQure announced a new plan to apply to the U.S. Food and Drug Administration (FDA) to seek approval of its gene therapy for Huntington’s disease  – a dramatic shift after the agency had last year blocked the drug despite results showing, for the first time, that HD progression could be slowed.

 

The announcement comes in the wake of the May 12 resignation of the head of the crisis-ridden FDA, which has clashed with uniQure.

 

In a press release, uniQure reported that, at a recent meeting with the FDA, the agency had accepted that the three-year analysis of the drug, AMT-130, which the company has presented as demonstrating efficacy against HD, can serve as the “primary basis” for a drug approval application.

 

uniQure aims to apply in the third quarter of this year. In alignment with the FDA, the company will also conduct a “confirmatory study” to further test the efficacy of AMT-130. According to early-stage clinical trial results reported by uniQure, AMT-130 had demonstrated a 75 percent slowing in HD over a three-year period – a historic achievement.

 

For the confirmatory study, uniQure will work with the FDA to determine how to evaluate a “concurrent control,” that is, a placebo or comparator to measure drug efficacy in those not receiving the drug. According to the press release, this study would not include a sham surgery as a placebo – a requirement introduced in March by the FDA but considered by many to be unethical because of the already harmful symptoms of HD.

 

According to the press release, the FDA has agreed that uniQure can, for a comparator, still use the patient information from the important Enroll-HD database of affected individuals – a major point of contention in the FDA’s surprise reversal of its promises regarding the study of AMT-130.

 

“Today's announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA’s genuine commitment to addressing the unmet need of Americans living with Huntington’s disease,” Matt Kapusta, uniQure CEO, stated in the release. “The FDA has agreed that our current clinical data can support a near-term BLA [biologics license application] submission and has committed to work expeditiously with us to align on the design of the required confirmatory study.”

 

‘A meaningful step forward’

 

The new understanding with the FDA represents a significant shift, because, after previously clashing with uniQure, the FDA recommended in March that the firm conduct a full-blown Phase III clinical trial, including the use of a sham surgery.

 

“Today’s announcement from uniQure represents an encouraging and meaningful step forward for the Huntington’s disease community,” Amy Gray, president and CEO of the Huntington’s Disease Society of America (HDSA), stated in a press release. “I applaud the leadership at the FDA for allowing uniQure to take this important step forward in the development of its investigational treatment for Huntington’s disease.

 

Gray added that “this progress did not happen in isolation.” Following “regulatory hurdles,” the HD community “united like never before.” HD advocates delivered to the FDA two petitions with more than 48,000 signatures in favor of AMT-130. According to Gray, more than 11,000 messages to Congress, participation in legislative meetings, and sharing of personal stories added to the impact.

 

“I am deeply grateful to the Members of Congress who stood with Huntington’s disease patients and families during this effort,” Gray said. “Their willingness to engage with the FDA, ask important questions, and advocate for a regulatory framework that reflects the realities of rare disease research helped ensure that the voices of our community were heard.”

A whirlwind of developments

 

The uniQure announcement about AMT-130 came in at the end of a whirlwind of recent developments. The HD community has regrouped in support of improved drug discovery policy at the crisis-ridden FDA.

 

On April 30, uniQure took the case for its drug to the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) as a first step to seek approval in that country. The company plans to submit an application in the third quarter of 2026.

 

On May 12 FDA Commissioner Marty Makary, a Trump administration appointee, resigned after what STAT considered to be the “worst” leadership of the agency in 25 years because of “a fundamental lack of understanding of the nature of the role, of the functions of his agency, and of the needs of the employees who worked for him.” His departure prompted calls for rebuilding public trust in, and better leadership at, the FDA (click here and here to read more).

 

On June 1, the FDA announced that Acting Commissioner Kyle Diamantas would meet with rare disease group leaders to seek to steady operations and mend fences with disease groups. That meeting took place on June 3.

 

Jeff Allen, CEO of Friends of Cancer Research, described the encounter as a “breath of fresh air.”

 

The FDA did not invite HDSA and other HD advocacy organizations to join the meeting. The FDA declined to answer questions. As of publication time, a message left at the National Organization for Rare Disorders seeking comment had not been returned.

 

A key briefing on rare diseases

 

However, on June 2, HDSA CEO Gray moderated a panel of five rare disease community representatives at a bipartisan congressional townhall briefing in Washington, D.C., titled “The Pathway to Cures and Treatments for Rare Diseases.”

 

“Rare disease families need a clear and sustainable pathway to research, treatments, and care,” said Gray in an HDSA press release about the event, available on YouTube. “This briefing is an important opportunity to bring patient advocacy organizations, scientific leaders, policy experts, and lawmakers together to discuss how we can advance meaningful progress for families impacted by rare diseases.”

 

With more than 40 people in attendance, the meeting highlighted the suffering of people with rare diseases, the urgent need for effective treatments, and the need to improve the FDA’s procedures. It included comments from both a Democratic and a Republican member of the House of Representatives.

 

The Pathway to Cures and Treatments for Rare Diseases congressional townhall briefing at the Rayburn House Office Building, Washington, D.C., June 2. From left to right, Kathryn Bryant Knudon, The Speak Foundation; Emily Gantman, Ph.D., CHDI Foundation; Lauren Moore, Ph.D., National Ataxia Foundation; Monet Stanford, PharmD, Washington Analysis; Tamara Maiuri, Ph.D., HDSA; Amy Gray, HDSA; and Rep. Jake Auchincloss (screenshot by Gene Veritas, aka Kenneth P. Serbin)

 

Bringing ‘smart’ leadership to the FDA

 

Rep. Jake Auchincloss, a Democrat from the greater Boston area and a member of a family of physician-scientists, said that he was “passionate” about the issue of rare disease drug development. He is on the Subcommittee on Health of the Committee of Energy and Commerce.

 

The subcommittee oversees the FDA. Auchincloss’s focus includes clinical trial reform, which the panelists agreed was important for rare disease drug development.

 

“While the science has never been better in trying to unlock those answers, the politics has never been worse,” Auchincloss said at the briefing.

 

Auchincloss spoke of the need for change on three fronts involving science and research: to bring U.S. research spending back to its previously high levels; to bring “smart” leadership back to the FDA; and to require insurance companies to cover the many new rare disease therapies on the horizon.

 

Auchincloss added that the “most urgent issue now” is to stop the Trump administration’s “unprecedented” cutting of National Institutes of Health grants without the time-honored standard peer review.

 

‘Cut the red tape’

 

Rep. Morgan Griffith, a Republican who represents far western Virginia, chairs the Subcommittee on Health. He supports the cause of the ALS (amyotrophic lateral sclerosis) community and pediatric cancer. He spoke about how people with rare diseases in remote areas have difficulty accessing clinical trials in urban areas.

 

Griffith also spoke movingly of the two HD families he has gotten to know.

 

He agreed with Auchincloss’s approach regarding the FDA and clinical trial reform. The two speak regularly on these issues.

 

“We have to figure out a better way to do it,” Griffith said. “It needs to be bipartisan.”

 

Auchincloss is not seeking more money from the government but simply to “cut through the red tape” regarding drug development, Griffith said, adding that people with rare diseases should not be required to “jump through the same hoops” as those participating in clinical trials for less difficult conditions such as nausea.

 

‘We were finally heard’

 

On September 17 and 18, news about AMT-130 dominated bioscience headlines after the uniQure announcement – and provided a needed boost to the HD community.

 

“I’m literally crying right now,” Lauren Holder, a Help4HD International Advocate and, like me, an HD gene carrier, told STAT. “I’m so happy that I don’t even know how to put what I’m feeling into words.”

 

Holder added, “This is the best-case scenario for our community.”

 

“This happened because dedicated patient advocates refused to give up, because this community continued to show up, speak up, and fight, even when it felt like no one was listening,” she said. “Today, it feels like we were finally heard.”

As uniQure seeks to overcome ‘dysfunction’ at the FDA, Huntington’s disease community rallies in defense of gene therapy drug

As uniQure seeks to overcome a decision by the U.S. Food and Drug Administration (FDA) to roll back its consultations regarding the firm’s promising gene therapy for Huntington’s disease, the HD community has begun to rally by organizing two petitions asking the agency to support the remedy.

 

uniQure and others in the biotech sector believe that the FDA has become dysfunctional under the Trump administration.

 

In September uniQure announced that its drug, AMT-130, had slowed the progression of HD by 75 percent over three years.

 

On November 3, uniQure announced that, after a recent meeting with the FDA, it believes that the “FDA currently no longer agrees” that data from its clinical trial of AMT-130 "may be adequate to provide the primary evidence in support of” an application for approval.

 

The company’s plan to seek approval of AMT-130 in early 2026 and launch it into the market later that year may no longer be possible.

 

Although the FDA claims it will “unleash gene therapies,” the documented collapse of the agency under the Trump administration, a more conservative view of gene therapies, and “mistrust and paranoia” in the division in charge of those therapies set the stage for backtracking on AMT-130 (click here to read more).

 

On November 13, the key biotech site STAT reported on a private dinner held by uniQure CEO Matt Kapusta with investors on November 11. According to the report, uniQure hopes to find a way forward for AMT-130.

“We remain fully committed to people living with HD, who have no disease-modifying treatment options,” Tom Malone, uniQure’s senior director of communications, wrote me via e-mail on November 14. “We are wholly focused on working with the FDA to determine the best path forward to rapidly bring AMT-130 to patients and their families in the U.S.”

The company is withholding further public comment until it receives official final minutes of its most recent meeting with the FDA.

Two groups of HD advocates have launched petitions to the FDA to support the original uniQure timeline for AMT-130. They are discussed below.

 

 

The online petition to the FDA titled "Bring Hope to Huntington's Disease Families," on November 15, 2025 (screenshot by Gene Veritas, aka Kenneth P. Serbin)

 

Deep frustration with the FDA

 

The STAT report noted that Kapusta “doesn’t like all the drama” inside the FDA surrounding its upending of uniQure’s plans. They had involved extensive consultations with the FDA in 2024.

 

Aiming to stabilize the agency, the FDA has named Richard Pazdur, M.D., a leading cancer specialist and 26-year veteran of the entity, to run its center for regulating and approving new drugs.

 

Dr. Pazdur’s appointment is a sign the Trump administration is seriously addressing the “FDA dysfunction,” Kapusta said at the investor dinner, as reported by STAT.

 

According to the STAT report, Kapusta’s remarks at the dinner “reflected biotech’s frustration with volatility” at the FDA. uniQure was disturbed by the fact that the FDA’s new message on AMT-130 was “delivered by lower-level staffers” and not senior decision-makers, STAT reported.

 

The firm “feels like it was screwed over by the FDA, and rightfully so,” one investor told the STAT reporter.

 

Meanwhile, the FDA and some scientists have moved ahead with researching and approving the world’s first personalized gene-editing treatments for individuals with rare genetic conditions. It is unclear how much this might benefit rare disease communities like HD (with thousands of affected individuals) and impact the FDA’s thinking on AMT-130.

 

Two petitions: ‘AMT-130 could change everything’

 

As of November 15, the two online petitions to the FDA from HD advocates have already garnered almost 9,000 signatures.

 

These moving, persuasive petitions effectively portray the devastating impact of HD on patients and families and the historic breakthrough towards a treatment achieved with AMT-130. They effectively demonstrate the profound need for the drug and urgent action by the FDA.

 

One petition is titled “Bring Hope to Huntington's Disease Families: Urge the FDA to Uphold Accelerated Approval.”

 

It is sponsored by the Huntington’s Disease Society of America, HD Reach, Help4HD International, Huntington’s Disease Foundation, and Huntington’s Disease Youth Organization. These organizations have also pledged to improve collaboration.

 

It notes that “the FDA is now wavering on its commitment” to AMT-130. It asks the agency to honor its previous guidance to uniQure, recognize the urgency of the unmet dire medical need of HD families, and to expedite the approval of AMT-130.

 

It urges people to sign: “We cannot allow procedural hesitation to become a death sentence.”

 

The other petition is titled “Accelerate Breakthrough Drug Approval for Huntington's Disease - UniQure AMT-130.”

 

“Our Plea: Turn Heartbreak Into Hope,” it states. “We are mothers and fathers, sons and daughters, brothers and sisters, husbands and wives, friends and caregivers – all united by love and by loss. AMT-130 could change everything.”

 

As an HD gene carrier and HD family member, I immediately signed the first petition upon learning about it. I urge all friends and supporters of the HD community to join us in our plea to the FDA.

Wonderful news: uniQure’s one-and-done drug slows Huntington’s disease

 

AMT-130, a one-time gene therapy developed by uniQure, has successfully slowed the progression of Huntington’s disease.

 

While that is not a cure, and the therapeutic process is hardly simple, it is the first evidence that scientific progress translates into meaningful results for those suffering from HD.

 

The treatment is far more complex than a pill: it involves 12 to 18 hours of delicate brain surgery. A neurosurgeon injects AMT-130 directly into the brain under the guidance of an MRI. As a gene therapy, AMT-130 requires just this one application. (Watch the uniQure video about how AMT-130 is administered here).

 

According to clinical trial results reported by uniQure on September 24, AMT-130 achieved its main goal (primary endpoint) and demonstrated a 75 percent slowing in the progression of the disease over a three-year period.

 

“High-dose AMT-130 also demonstrated statistically significant slowing of disease progression as measured by TFC, a key secondary endpoint, and favorable trends across additional clinical measures,” the release stated. TFC is total functional capacity. It refers to a person’s ability to function – a key loss in HD.

 

Other progress

 

The company, based in Lexington, MA, and Amsterdam, reported that AMT-130 also reduced the measure of a protein known as neurofilament light, a marker of disease that reveals stress on the brain.

 

The clinical trial showed “favorable trends” in other second measures of motor (movement) and cognitive function, the release stated. Movement disorders and cognitive loss are major HD symptoms. Trial results demonstrated that AMT-130 is safe and well-tolerated.

 

“I believe these groundbreaking data are the most convincing in the field to date and underscore potential disease-modifying effects in Huntington’s disease, where an urgent need persists,” Sarah Tabrizi, M.D., Ph.D., a leading HD specialist at University College London, stated in the release. “These data indicate that AMT-130 has the potential to meaningfully slow disease progression – offering long-awaited hope to individuals and families impacted by this devastating disease.”

 

 

David Margolin, M.D., Ph.D., uniQure's vice president for clinical development, presents data illustrating ATM-130's slowing of the progression in Huntington's disease at the 20th Annual HD Therapeutics Conference, Palm Springs, CA, February 25, 2025 (photo by Gene Veritas, aka Kenneth P. Serbin).

 

Transforming the HD landscape

 

AMT-130 seeks to lower the amount of harmful mutant huntingtin protein in the brain cells of patients. Whether the drug has actually done this has yet to be verified.

 

In early 2026, uniQure plans to apply to the U.S. Food and Drug Administration (FDA) for drug approval. Pending drug approval, the drug would be launched in the U.S. later in 2026.

 

“We are incredibly excited about these topline results and what they may represent for individuals and families affected by Huntington’s disease,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington’s disease, while also providing important evidence supporting one-time, precision-delivered gene therapies for the treatment of neurological disorders.”

 

Hopes for a life-long treatment

 

“This is the first time any drug has been shown to alter the course of HD in people in a clinical trial,” the scientist-written website HDBuzz stated. “uniQure believes that AMT-130 has the potential to be a treatment that lasts for life.”

 

uniQure officials told HDBuzz that, beyond the FDA, they plan to seek approval with other regulators, including the European Medicines Agency, which oversee drug approvals in Europe.

 

HDBuzz cautioned that key details need to be worked out before AMT-130 can be administered to a large group of people beyond the fewer than 30 people whose data were analyzed by uniQure.

 

Only some of those individuals received the high dose of the drug that proved effective. Also, because AMT-130 requires an operation, the company must find a way to provide access to the drug, and at an affordable level, to a larger number of people.

 

On the whole, despite the caveats and complexities, this is wonderful news for the HD community.

 

"We never in our wildest dreams would have expected a 75% slowing of clinical progression," Dr. Tabrizi told the BBC.

Exploring the unique qualities of INGREZZA, the newest FDA-approved drug for Huntington’s disease chorea

 

After the news last year that the U.S. Food and Drug Administration (FDA) had approved INGREZZA to treat chorea associated with Huntington’s disease, a debilitating movement disorder, I wanted to better understand the development of this drug and the unique qualities claimed by its creator.

 

On November 17 I interviewed company officials at Neurocrine Biosciences, Inc., which fashioned valbenazine – the chemical name for INGREZZA – in the early 2000s. In 2017, INGREZZA was approved by the FDA for the treatment of tardive dyskinesia, an irreversible involuntary movement disorder unrelated to HD. Neurocrine is in San Diego, one of the world’s leading biotech hubs and where I reside.

 

In an initial report on INGREZZA, including a Zoom interview with three Neurocrine officials, I noted the drug’s advantages over the two other FDA-approved chorea remedies, Xenazine (tetrabenazine) and Austedo (deutetrabenazine).

 

INGREZZA is easier to take, requiring just one daily dose in capsule form. Xenaxine and Austedo have long required multiple daily doses, although in May the FDA approved once-daily extended-release tablets for Austedo. Unlike the other drugs, INGREZZA also does not require titration, that is, slowly increasing the dosage over weeks. INGREZZA is always just one pill.

 

 

In contrast with the other drugs aimed at chorea, INGREZZA is a capsule – not a tablet – and is taken once daily even without an extended-release formulation. These characteristics potentially provide physicians and patients greater flexibility in dosing, because INGREZZA can be crushed and is available in three effective doses. As a result, Neurocrine’s drug, while indicated for oral administration, can also be crushed and mixed with food or provided through a feeding tube – often necessities for late-stage HD patients.

 

In April the FDA approved INGREZZA SPRINKLE capsules, a new formulation of the drug, in oral granules. Neurocrine developed this version of the drug for those with HD or tardive dyskinesia who experience difficulties in swallowing. It can be sprinkled on soft food. INGREZZA SPRINKLE offers the same three simple and effective dosing options (40 mg, 60 mg and 80 mg) as INGREZZA.

 

In last year’s interview, recognizing that INGREZZA only treats chorea, the Neurocrine officials stated that they plan to seek potential disease-modifying remedies that could potentially slow, halt, or reverse the progression of debilitating neurological conditions, which might include HD.

 

A substantial reduction in chorea

 

According to a Neurocrine press release (and also a June 2023 scholarly article in Lancet Neurology), INGREZZA decreased chorea severity three times better than a placebo.

 

So far researchers have not done a head-to-head study of Xenazine, Austedo, and INGREZZA. All three are VMAT2 inhibitors, designed to reduce involuntary movements of chorea. VMAT2 inhibitors help regulate dopamine, a chemical messenger in the brain that affects movements.

 

As my above-mentioned article stated, Dietrich Haubenberger, M.D., the executive medical director at Neurocrine and clinical lead for the firm on the successful Phase 3 KINECT-HD clinical trial leading to INGREZZA’s approval, called valbenazine a “unique molecule.”

 

Comparing INGREZZA with competitors

 

In 2015 I reported on the key differences between tetrabenazine (Xenazine) and its derivative deutetrabenazine (Austedo), which was also developed in San Diego.

 

During my November 2023 interview with Dr. Haubenberger and other Neurocrine scientists, I sought to better understand INGREZZA’s uniqueness and its benefits for HD-affected individuals. How does valbenazine contrast with tetrabenazine and its derivative deutetrabenazine?

 

The basics were described by Dimitri Grigoriadis, Ph.D., a pioneer of valbenazine and today a semi-retired distinguished scholar at Neurocrine. A neuropharmacologist by training, Dr. Grigoriadis started with the firm at its inception in 1993 and previously served as chief research officer.

 

Understanding the chemistry

 

Dr. Grigoriadis explained how INGREZZA works in the brain.

 

The “unique part of INGREZZA,” he said, is that it gets broken down by the body, then produces a single “isomer” that is a key metabolite of tetrabenazine. A metabolite results from the breaking down of a chemical.

 

“That is the chemical that binds to VMAT2, the protein, and blocks the entrance of dopamine” into relevant parts of brain cells, Dr. Grigoriadis added.

 

The drug discovery that Neurocrine did for valbenazine involved a drug profile that was highly selective for the VMAT2 protein, Dr. Grigoriadis recalled. “And through our research, we were able to identify a molecule that provides only the high affinity, very selective isomer of [the metabolites] of tetrabenazine.”

 

Comparing hands

 

Dr. Grigoriadis illustrated the concept of an isomer by noting how left and right hands resemble each other but are positioned differently.

 

“An isomer is a molecule that is exactly the same, has the same structural components,” he said. “So, it's an identical molecule, but it's left-handed, right-handed orientation. Your hands are four fingers and a thumb. They're identical, but they are in a different orientation.”

 

Isomers work in a similar way, he continued.

 

“They fit differently,” he said. “Functionally, they are different, even though they look exactly the same. You could have two isomers of the same molecule, a left hand and a right hand, that fit into different proteins, that fit into different spaces because they are in a different orientation.”

 

As a result, the various qualities of a drug “could be different,” he continued, resulting in a “slightly different” way in which the chemicals produced by INGREZZA “function.”

 

Dimitri Grigoriadis, Ph.D. (left), Dietrich Haubenberger, M.D., and Gene Veritas (aka Kenneth P. Serbin) discuss INGREZZA at the Neurocrine offices (photo by Aimee White, Director, Corporate Communications, Neurocrine). (Click on an image to make it larger.)

 

Building on tetrabenazine

 

At the time of valbenazine’s discovery, Neurocrine did not know whether it could help patients with diseases like tardive dyskinesia or HD, because the research on valbenazine had not been done, emphasized Eiry Roberts, M.D., Neurocrine’s chief medical officer. No drug had been developed for tardive dyskinesia before valbenazine.

 

“So this was a totally new research project,” Dr. Roberts continued. “There were learnings from experience with tetrabenazine that made it important to find out how valbenazine could be a safe and effective treatment for patients with conditions not sufficiently addressed by other medications available at the time.” The company also did extensive research to prove valbenazine’s safety, she added.

 

In addition, Neurocrine determined that, besides showing promise in the lab and efficacy in clinical trials for the treatment of tardive dyskinesia and chorea associated with Huntington’s disease, valbenazine could be mass-produced, Dr. Grigoriadis said.

 

Benefits for patients

 

Dr. Haubenberger stressed that INGREZZA is unique because  it involves just “one capsule, once daily with no complex dose adjustments to get to an effective dose.”

 

The once-a-day quality results from valbenazine’s “very long half-life of its effectiveness” (the time it remains in the body), explained Grace Liang, M.D., a movement disorders neurologist and Neurocrine’s vice president of clinical development in neurology.

 

“We know that the long half-life is important not only for the convenience this provides, but it allows patients to take it consistently without forgetting a dose,” said Dr. Liang. “Everybody has a life to live.” The “long duration” in the body and the selectivity – that it doesn’t act on other receptors of the brain that may cause other effects – make INGREZZA “special” for patients, she added.

 

INGREZZA “gives that nice, smooth, and steady coverage,” in contrast with the other chorea drugs, which have multiple daily doses and a shorter half-life, Dr. Liang continued.

 

INGREZZA also takes effect faster than Xenazine and Austedo, Dr. Haubenberger pointed out. He added that “with the first dose and as early as at two weeks of treatment, the clinical trial showed a greater reduction of chorea in patients on valbenazine compared to placebo.”

 

Unlike medicines that purposely have a material on their capsule to cause an extended release, valbenazine needs no such modification to achieve its “inherent,” smooth, once-daily effect, Dr. Roberts added.

 

Looking to the HD community

 

The FDA approved INGREZZA for use in adults with HD chorea. Neurocrine has no plans for a clinical trial of the drug in juvenile HD patients.

 

“We do recommend that people just use INGREZZA as it's labeled,” Dr. Liang said. “Obviously the care providers, the physicians would need to evaluate what the best treatment options are for those children, and we're hopeful that future developments can also support their needs as well.”

 

“Data is still being collected,” said Dr. Haubenberger, referring to an ongoing three-year study of more than 150 adults with HD taking INGREZZA. “There's lots more to learn about the compound itself and that's really where we want to invest in, where we can even learn more, share that with the community and even learn from that to inspire future avenues of research in HD and also other conditions.”

 

In these studies that reflect more “real-life settings, there’s much that can be learned from a broader data set than what could feasibly be done in a controlled clinical trial,” added Dr. Liang.

 

Other valbenazine projects

 

Previously, a Neurocrine clinical trial program of valbenazine for Tourette’s disorder did not show efficacy, Dr. Roberts said.

 

Neurocrine currently has a Phase 3 program for “valbenazine as an adjunctive treatment for schizophrenia, adjunctive to the antipsychotics that those patients take right now,” Dr. Roberts said. The drug is also in a Phase 3 study for the “commonest movement disorder in children, dyskinetic cerebral palsy,” she added.

 

In March Neurocrine announced the start of a Phase 1 clinical trial with their next-generation VMAT2 inhibitor, NBI-1065890, to study the safety and tolerability in healthy volunteers.

 

“We’re excited to bring this next-generation, internally discovered, highly potent, oral, selective VMAT2 inhibitor into the clinic with the hope of providing differentiated benefit in treating certain neurological and neuropsychiatric conditions,” Dr. Roberts stated in a press release. 

 

Gene Veritas (left) with Eiry Roberts, M.D., Chief Medical Officer, Neurocrine (photo by Aimee White, Neurocrine)

 

Aiming for disease-modifying therapies

 

Our November interview concluded with Dr. Roberts’ reflections on Neurocrine’s commitment to seek disease-modifying therapies for neurological conditions such as HD, including its new partnership with Voyager Therapeutics, a key player in the development of next-generation gene therapies. Voyager has experience in HD research.

 

“While symptomatic treatments are incredibly important for patients living with the diseases that we're seeking to serve, like Huntington's, for us to be a true innovator as well in this field, we need to be focused on understanding disease modification and cures,” Dr. Roberts said. “And so the collaboration with Voyager is one that gets us into the gene therapy space for potential curative or disease-modifying treatments. And we have other efforts that are very early in our research to look at different ways of coming upon disease modification.”

 

“It's really a focus area for us as we look at some of these novel platforms that we're coming forward with,” Dr. Roberts concluded.

 

Those research platforms are getting a boost: Neurocrine is moving to a new, state-of-the art 535,000-square-foot research campus. The lab space is scheduled for completion by year’s end.

 

Neurocrine's new research campus (photo courtesy of Neurocrine).