Progress Now: Drug Approvals and Clinical Trial Updates

Amyotrophic lateral sclerosis (ALS)

Phase 3 Clinical Trial: Enrolling

PREVAiLS is a phase 3 trial testing an investigational therapy called pridopidine in adults with early, rapidly progressing ALS. The main goal is to evaluate whether pridopidine can slow the disease progression.

Inclusion criteria include:

• Definite or probable ALS diagnosis
• Between 18 and 80 years old
• ALS symptoms began within the past 18 months

Overview

Pridopidine is designed to activate a nerve protein called sigma-1 receptor (S1R). This protein helps turn on systems that protect nerve cells. Pridopidine is delivered as a capsule that is swallowed.

Timeframe

The study begins with a 48-week, randomized, double-blind period, during which some participants receive pridopidine, and others receive a placebo (an inactive substance). This is followed by a 48-week open-label extension, during which all participants receive pridopidine. The total duration is about 2 years.

Location

Massachusetts, Nebraska, Texas

Learn more

Visit ClinicalTrials.gov and enter NCT07322003 in the “Other terms” search box.

Contact

Prilenia, Executive Director Clinical Operations, 857-574-5755, MedInfo@prilenia.com

[H4] Phase 2 Clinical Trial: Results

AL-S Pharma released positive results from a phase 2 study of the experimental therapy AP-101. In the trial, early treatment with AP-101 was associated with extended survival and delayed need for respiratory support in people with ALS.

Overview

Superoxide dismutase 1 (SOD1) is a protein that helps protect cells from damage. In some people with ALS, SOD1 proteins do not fold correctly, causing them to clump together. These clumps can damage motor neurons, which is thought to contribute to ALS progression. AP-101 is designed to bind to misfolded SOD1 proteins and help the body’s immune system remove the toxic clumps.

Key points

• The phase 2 clinical trial included 73 adults with sporadic or SOD1-related familial ALS.
• It was a randomized, double-blind, placebo-controlled study.
• AP-101 extended survival and delayed the need for respiratory support in participants.
• Some trial participants also saw improvements in functional ability.
• AL-S Pharma plans to launch a phase 3 study later this year.

Learn more

Visit ClinicalTrials.gov and enter NCT05039099 in the “Other terms” search box.

Phase 1 Clinical Trial: Enrolling

This study, called ARMOR, is testing an experimental gene therapy for ALS, called INS1202. The goal is to evaluate the safety and tolerability of a single dose in adults with ALS.

Inclusion criteria include:

• Diagnosis of ALS with an SOD1 mutation or sporadic disease without a known ALS-causing mutation
• Between 18 and 79 years old
• ALS symptoms began less than 3.5 years ago

Overview

INS1202 is designed to reduce levels of misfolded SOD1 protein. Participants will receive a single dose of INS1202 via intrathecal injection (an injection into the fluid around the spinal cord in the lower back).

Timeframe

30-day safety monitoring period after dosing and 48 weeks of follow-up

Location

Missouri, Ohio, Pennsylvania

Learn more

Visit ClinicalTrials.gov and enter NCT07290062 in the “Other terms” search box.

Contact

Insmed Medical Information, 844-446-7633, MedicalInformation@insmed.com

Duchenne muscular dystrophy (DMD)

Phase 2 Clinical Trial: Enrolling

This trial, called BASECAMP, is testing the oral therapy SAT-3247 in boys with DMD. The goal is to evaluate the investigational drug’s safety, tolerability, and effect on muscle strength.

Inclusion criteria include:

• Genetic or clinical diagnosis of DMD
• Between 7 and 9 years old
• Ambulatory (able to walk)

Overview

In DMD, the lack of dystrophin protein leads to problems with muscle stem cells that grow new muscle tissue to repair damage. SAT-3247 is designed to restore stem cells’ ability to produce healthy muscle tissue.

This is a randomized, double-blind, placebo-controlled study, meaning some participants will receive SAT-3247 and others will receive a placebo (an inactive substance). SAT-3247 is administered in tablet form.

Timeframe

12 weeks

Location

25 global sites, including 11 in the US

Learn more

Visit ClinicalTrials.gov and enter NCT07287189 in the “Other terms” search box.

Contact

Satellos Medical Information, 647-660-1780, MedicalInfo@satellos.com

[H4] Phase 1 Clinical Trial: Enrolling

This trial tests an investigational stem cell therapy for DMD, called MyoPAXon, in combination with an immunosuppressant drug called tacrolimus. The goals are to evaluate whether this treatment is safe and well-tolerated in adults with DMD, and whether it may increase muscle growth and function.

Inclusion criteria include:

• Genetic or lab-supported diagnosis of DMD
• Age 18 years or older
• Nonambulatory (not able to walk)

Overview

MyoPAXon is a muscle stem cell product. Researchers believe it may help the body regenerate healthy muscle. Participants will start taking tacrolimus one week before MyoPAXon is administered. They will receive a one-time intramuscular (in the muscle) injection of MyoPAXon, then continue taking tacrolimus for three months.

Timeframe

3-month study, followed by monitoring for 15 years

Location

Minnesota

Learn more

Visit ClinicalTrials.gov and enter NCT06692426 in the “Other terms” search box.

Contact

Peter Kang, MD, 612-624-9452, mdstemcell@umn.edu

[H4] Phase 1/2 Clinical Trial: Results

Researchers shared encouraging results from the phase 1/2 trial of the exon-skipping therapy zeleciment rostudirsen (z-rostudirsen), formerly called DYNE-251. Dyne Therapeutics is asking the US Food and Drug Administration (FDA) for accelerated approval of the therapy after trial data showed early signs of multiple benefits for boys with DMD who are amenable to exon 51 skipping.

Overview

The dystrophin gene is made up of sections called exons, which are identified by numbers. In some cases of DMD, a genetic mutation in the dystrophin gene affects a specific exon. Exon-skipping drugs deliver instructions to cells to skip the affected exon, allowing the body to produce shorter but functional dystrophin protein.

This trial, called DELIVER, tested z-rostudirsen against a placebo in 86 participants with DMD amenable to exon 51 skipping.

Key points

• The phase 1/2 clinical trial included ambulatory and nonambulatory boys ages 4 to 16.
• It was a randomized, double-blind, placebo-controlled trial.
• Participants experienced notable increases in dystrophin protein levels after six months.
• Data also showed improvements in walking ability, upper limb strength, and lung function.
• Z-rostudirsen is administered once a month via IV infusion (a tube into the vein).

Learn more

Visit ClinicalTrials.gov and enter NCT05524883 in the “Other terms” search box.

Treatment News: Approval Pending

Capricor Therapeutics resubmitted an application for FDA approval of deramiocel, which targets heart disease in DMD.

Overview

In DMD, the lack of dystrophin protein affects heart muscle, as well as skeletal muscle. Deramiocel is a cell-based therapy designed to help the body repair heart muscle. In 2025, the FDA did not approve deramiocel and instead requested more data on its effectiveness. Capricor is including results from the phase 3 HOPE-3 study in its new submission. The HOPE-3 data show improvements in heart and arm muscle function over one year, compared to a placebo.

If approved, deramiocel would be the first cell-based therapy specifically targeting DMD-related cardiomyopathy. The FDA is expected to make a decision in August 2026.

Learn more

Visit ClinicalTrials.gov and enter NCT05126758 in the “Other terms” search box.

Idiopathic inflammatory myopathies (IIMs)

Phase 2 Clinical Trial: Enrolling

This trial, called AUTOGRAPH-IIM, aims to determine if the investigational drug rapcabtagene autoleucel is effective and safe for adults with severe myositis who have not responded to currently available treatment options.

Inclusion criteria include:

• A definite or probable myositis diagnosis
• Between 18 and 75 years old
• Inadequate response to prior therapy

Overview

Rapcabtagene autoleucel is a cell-based therapy (a CAR T-cell therapy) designed to target and eliminate B-cells, which contribute to autoimmune activity in IIMs. It is administered as a one-time IV infusion (a tube into the vein).

This is a randomized, open-label, controlled study, meaning participants will know whether they have been randomly assigned to a group receiving rapcabtagene autoleucel or to a comparison group receiving standard-of-care treatments.

Timeframe

Participants in the group receiving rapcabtagene autoleucel will begin with a 2-week hospital stay, followed by a 5-year follow-up period. Participants in the standard-of-care group will follow a 5-year schedule without the initial hospitalization.

Location

55 global sites, including 6 in the US

Learn more

Visit ClinicalTrials.gov and enter NCT06665256 in the “Other terms” search box.

Contact

Novartis Pharmaceuticals, 888-669-6682, novartis.email@novartis.com

Myasthenia gravis (MG)

Treatment News: Drug Approval

The US Food and Drug Administration (FDA) approved Amgen’s inebilizumab-cdon (Uplizna®) to treat generalized myasthenia gravis (gMG) in adults who are acetylcholine receptor (AChR) and muscle-specific tyrosine kinase (MuSK) antibody positive.

Overview

Uplizna is a B-cell-depleting therapy newly approved by the FDA for gMG. It was previously approved for two other conditions that share similar abnormal immune system responses with MG. Uplizna is a monoclonal antibody that targets a specific type of immune cell, called B cells. It reduces B cells to decrease the autoimmune response that leads to MG symptoms. It is administered via IV infusion (a tube into the vein).

Learn more

Visit uplizna.com.

Vyvgart Approval Expanded

In May, the FDA expanded approval of efgartigimod alfa-fcab (VYVGART®) and efgartigimod alfa and hyaluronidaseqvfc (VYVGART Hytrulo®) to all adults living with generalized myasthenia gravis (gMG). This includes AChR antibodypositive, MuSK antibody-positive, LRP4 antibody-positive, and triple seronegative subtypes of gMG.

Learn more in When ‘No Options’ Starts to Change: A New Chapter for Seronegative Myasthenia Gravis.

Phase 2 Clinical Trial: Enrolling

This trial, called SYNAPSE-MG, is testing an investigational therapy, called NMD670, in adults with gMG. The goals are to evaluate whether it is safe and effective, and whether it improves muscle strength, power, and endurance.

Inclusion criteria include:

• A confirmed diagnosis of gMG with a positive antibody test for AChR or MuSK autoantibodies
• Age 18 years or older

Overview

NMD670 targets skeletal muscle and helps muscles respond to nerve signals. Early research suggested it may help muscles activate more effectively, even when nerve signals are weak.

This is a randomized, double-blind, placebo-controlled study, meaning some participants will receive NMD670 and others will receive a placebo (an inactive substance). The drug will be administered as a tablet that is swallowed.

Timeframe

8 weeks

Location

40 global sites, including 11 in the US

Learn more

Visit ClinicalTrials.gov and enter NCT06414954 in the “Other terms” search box.

Contact

NMD Pharma A/S, contact@nmdpharma.com

Spinal muscular atrophy (SMA)

Treatment News: Drug Approval

The US Food and Drug Administration (FDA) approved a high-dose regimen of nusinersen (Spinraza®) for the treatment of SMA.

Overview

Since Spinraza was approved in 2016, its standard dose has been 12 mg, with a series of loading doses, followed by maintenance every four months. The new higher-‐dose regimen, developed by Biogen, involves a more rapid loading phase, with two 50 mg loading doses 14 days apart, followed by a maintenance dose of 28 mg every four months. The new regimen is designed to deliver a higher concentration of the drug, potentially leading to greater clinical benefits while maintaining safety.

The FDA based its approval on findings from the DEVOTE study, which showed that symptomatic infants given the high-dose regimen of Spinraza, rather than the original dosing regimen, experienced statistically significant improvements in motor function. The study also generated supportive data in older patients.

Learn more

Visit spinraza.com.

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Clinical Trial Terms to Know

Double-blind: Neither researchers nor participants know which participants are taking the drug or placebo.

Open-label: Participants know what treatment they are receiving.

Placebo-controlled: Some participants receive the treatment being tested, and some receive a placebo that looks like the real treatment but has no active ingredients.

Randomized: Participants are randomly assigned to groups taking the drug or placebo.