AbbVie Reveals Phase III TEMPO-2 Trial Positive Topline Results; FDA Accepts GSK’s NUCALA Submission in COPD; Novartis Boosts Huntington’s Disease Development Program with PTC518 In-Licensing; Chimerix to File for Accelerated FDA Approval of Dordaviprone for H3 K27M-Mutant Diffuse Glioma; MeiraGTx Receives FDA RMAT Designation for AAV2-hAQP1 in Grade 2/3 Radiation-Induced Xerostomia Treatment

AbbVie Unveils Positive Phase III TEMPO-2 Trial Findings for Tavapadon as Parkinson’s Disease Monotherapy

AbbVie announced favorable topline results from its pivotal Phase III TEMPO-2 trial, which assessed the investigational drug tavapadon as a flexible-dose monotherapy for early-stage Parkinson’s disease. Tavapadon is the first and only D1/D5 partial agonist being studied as a once-daily treatment for Parkinson’s disease.

The TEMPO-2 trial investigated the efficacy, safety, and tolerability of a flexible-dose regimen (5 mg to 15 mg once daily) of tavapadon as a monotherapy in adults with early Parkinson’s disease. The trial achieved its primary endpoint, showing that patients treated with tavapadon had a statistically significant improvement from baseline compared to those on placebo, as measured by the Movement Disorder Society – Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Parts II and III combined score at week 26 (placebo: -1.2; tavapadon 5-15 mg: -10.3; p-value <0.0001). 

Additionally, the TEMPO-2 trial met its key secondary endpoint, demonstrating a significant and clinically meaningful improvement in the motor aspects of daily living (MDS-UPDRS Part II) in the tavapadon group (5-15 mg) versus placebo at week 26.

“The encouraging outcomes from all three Phase III TEMPO trials highlight tavapadon’s potential as a groundbreaking D1/D5 partial agonist for treating Parkinson’s disease,” stated Primal Kaur, M.D., MBA, senior vice president of immunology, neuroscience, eye care, and specialty development at AbbVie. “With these results, we are eager to collaborate with regulatory authorities to determine the next steps, bringing us closer to offering tavapadon to those affected by this chronic and debilitating condition.”

US FDA Moves Forward with GSK’s NUCALA (mepolizumab) Submission for COPD

GSK plc announced that the FDA has accepted for review data from the MATINEE study to support a new indication for NUCALA (mepolizumab) as an add-on maintenance treatment for patients with chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype. The Prescription Drug User Fee Act (PDUFA) date is set for May 7, 2025.

The submission is based on data from the MATINEE study, which assessed the efficacy and safety of mepolizumab in 804 COPD patients exhibiting evidence of type 2 inflammation, as indicated by blood eosinophil count. The trial included a wide range of COPD patients, including those with emphysema-only, chronic bronchitis-only, or a combination of both. The MATINEE study met its primary endpoint, demonstrating a statistically significant and clinically meaningful reduction in the annualized rate of moderate/severe exacerbations compared to placebo, with patients treated for 52-104 weeks.

Chronic obstructive pulmonary disease (COPD) is a progressive lung condition characterized by obstructed airflow, making breathing difficult. It primarily encompasses two main conditions: chronic bronchitis and emphysema, often coexisting. COPD typically develops due to long-term exposure to irritants such as cigarette smoke, air pollution, or occupational dust.

As per DelveInsight analysis, the diagnosed prevalent population of COPD in the 7MM was around 44 million in 2023. These cases of COPD in the 7MM are expected to increase at a significant CAGR of 1.4% throughout the study period (2020–2034). 

NUCALA is approved in the US for four IL-5-mediated conditions. These include two respiratory indications: as an add-on maintenance therapy for patients aged 6 years and older with severe asthma and an eosinophilic phenotype, and as an add-on maintenance therapy for adults with chronic rhinosinusitis with nasal polyps (CRSwNP) who have not responded adequately to nasal corticosteroids. Additionally, NUCALA is indicated for the treatment of adults with eosinophilic granulomatosis with polyangiitis (EGPA) and for patients aged 12 years and older with hypereosinophilic syndrome (HES) lasting six months or longer, without a known non-hematologic secondary cause. NUCALA is not approved for the treatment of COPD anywhere in the world.

Novartis Strengthens Neuroscience Pipeline with PTC518 in-licensing for Huntington’s Disease

Novartis Pharmaceuticals Corporation, a subsidiary of Novartis AG, has announced a collaboration with PTC Therapeutics to develop what could become the first oral disease-modifying therapy for Huntington’s disease.

Under the global license and collaboration agreement, the focus will be on PTC Therapeutics’ PTC518 program for Huntington’s disease. PTC will receive an upfront payment of $1 billion, with the potential for up to $1.9 billion more, contingent on meeting specific milestones related to development, regulatory approval, and sales.

“This collaboration brings together PTC’s expertise in small molecule splicing therapies and Novartis’s global experience in neuroscience development and commercialization. We’re excited to work with Novartis to advance PTC518, aiming to provide a well-tolerated, effective disease-modifying therapy for the hundreds of thousands of Huntington’s disease patients worldwide. The proceeds will help us expand our splicing platform and support our commercial and development initiatives,” said Dr. Klein.

Dr. Matthew Klein, CEO of PTC Therapeutics, emphasized, “PTC518 is the leading oral disease-modifying therapy in development for Huntington’s disease, and the financial terms of this agreement reflect the promising potential of this treatment.”

PTC518 is currently under investigation in the ongoing Phase II PIVOT-HD trial. Interim results released in June indicated that PTC518 achieved a durable, dose-dependent reduction in mutant Huntingtin protein (HTT) levels in both blood and cerebrospinal fluid (CSF) after 12 months, as reported by PTC.

Novartis will oversee the development, manufacturing, and commercialization of the HTT mRNA splice modulator. The licensing agreement is anticipated to be finalized in Q1 of 2025, pending the fulfillment of standard closing conditions.

Chimerix Plans to Seek Accelerated FDA Approval for Dordaviprone in Recurrent H3 K27M-Mutant Diffuse Glioma Patients by Year-End

Chimerix announced that, after extensive discussions with the FDA, the company intends to submit a complete New Drug Application (NDA) for accelerated approval of dordaviprone as a treatment for recurrent H3 K27M-mutant diffuse glioma in the United States by the end of the year.

“We believe that, if approved, dordaviprone will significantly transform the treatment options for patients with this deadly form of brain cancer, who currently have very few alternatives. Throughout the year, we have worked closely with the U.S. FDA, disease experts, and patient advocates to potentially speed up access to dordaviprone for this community,” said Mike Andriole, CEO of Chimerix. “In preparation for potential approval, we have strengthened our commercial leadership team and are on track to launch in the U.S. as early as Q3 2025, assuming the application is accepted and receives Priority Review, if granted.”

“As a pediatric oncologist, this program holds great significance, especially considering the profound impact that a potential approval would have on children and young adults affected by this disease. We are confident that the data collected so far could support an expedited approval to address this urgent, unmet medical need,” said Allen Melemed, M.D., Chief Medical Officer of Chimerix. “H3 K27M mutant gliomas are highly aggressive and affect more than 2,000 patients annually in the United States. If successful, dordaviprone would become the first FDA-approved treatment for this deadly disease, as well as one of the first molecularly defined approvals for any high-grade glioma.”

Dordaviprone has been granted a Rare Pediatric Disease Designation for H3 K27M-mutant glioma and is eligible for a Rare Pediatric Disease Priority Review Voucher (PRV). Chimerix plans to apply for this PRV with the upcoming NDA submission.

FDA Grants RMAT Designation to MeiraGTx’s AAV2-hAQP1 for Radiation-Induced Xerostomia

MeiraGTx Holdings plc announced that the FDA has awarded Regenerative Medicine Advanced Therapy (RMAT) designation to AAV2-hAQP1 for treating Grade 2/3 radiation-induced xerostomia (RIX). The FDA had previously granted Orphan Drug Designation to AAV2-hAQP1.

“This RMAT designation highlights the strength of our data, which suggests that our AAV2-hAQP1 therapy has the potential to significantly improve the lives of patients suffering from xerostomia after radiation treatment. To qualify for RMAT designation, the drug candidate must be an advanced regenerative medicine—like our gene therapy—and target a serious condition, in this case, Grade 2 and Grade 3 late xerostomia caused by radiotherapy for cancers of the upper aerodigestive tract. Additionally, the applicant must present preliminary clinical evidence showing the therapy’s potential to address an unmet need. Given that less than half of RMAT applications are approved, we are thrilled to receive this designation for our AAV-hAQP1 program and look forward to working with the FDA to make this potentially life-changing therapy available to patients with no alternative treatments as quickly as possible,” said Alexandria Forbes, Ph.D., president and CEO of MeiraGTx.

Grade 2/3 radiation-induced xerostomia (RIX) is a severely debilitating side effect of radiation therapy for head and neck cancer, affecting about 30-40% of patients undergoing such treatment. This condition represents a significant unmet medical need, with no available treatment options, and a large potential market, including over 170,000 patients in the U.S. and an additional 15,000 new cases annually. AAV2-hAQP1, an investigational gene therapy, is administered in a small dose directly to the salivary gland through a non-invasive procedure. This treatment can be delivered in a dental office or oncology center, where patients regularly visit post-radiation. The low-cost, in-house production of AAV2-hAQP1 at MeiraGTx, combined with its potential for long-term effectiveness and ease of administration, makes this a promising commercial opportunity in a large, underserved market.