Solid Biosciences Inc. provided a clinical update on SGT-001 and reported that the FDA has notified the company that IGNITE DMD, its Phase I/II study of SGT-001, has been placed on clinical hold. To date, six patients have been dosed with SGT-001, Solid’s gene transfer candidate under investigation for Duchenne. This includes three patients in the first cohort at a 5E13 vg/kg dose, who continue to do well and are being followed per the study protocol. Three patients were subsequently dosed in the second cohort at a 2E14 vg/kg dose. The first two of these patients are also doing well and being followed per study protocol.

The third patient in the 2E14 vg/kg cohort, dosed in late October, experienced a serious adverse event (SAE) deemed related to the study drug that was characterized by complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury, and cardio-pulmonary insufficiency. Neither cytokine- nor coagulopathy-related abnormalities were observed. Currently the patient is being closely followed by his care team. He is recovering and continues to improve.

While we are disappointed by this news, we appreciate the swift action of the FDA and Solid. Our hearts and prayers are with the young man recovering and his family. As our community knows all too well, there is risk and uncertainty whenever new therapies are in development and we are so grateful to the families who are willing to participate in clinical trials.

Solid’s Letter to the Duchenne Community:

Dear Duchenne Community,

Since day one, we have had one clear objective at Solid Biosciences – to discover, develop and deliver meaningful new therapies for patients with Duchenne muscular dystrophy. Our singular focus has been on Duchenne, and on improving the lives of patients and their families.

This morning we issued a press release providing an update on IGNITE DMD, our Phase I/II clinical study with our microdystrophin gene therapy, SGT-001. In our ongoing commitment to the Duchenne community, we would like to provide you with this update.

The latest child dosed has experienced a serious adverse event (SAE), and thankfully, he is now improving. We have reported the event to the FDA and the IGNITE DMD Data Safety Monitoring Board. Following notification, we received a phone call from the FDA informing us that the IGNITE DMD study has been placed on hold and we expect to receive formal written communication from the Agency with additional information. We are committed to working with the FDA to resolve the hold.

To date, six patients have been dosed with SGT-001. This includes three patients in the first dose cohort at the 5E13 vg/kg dose. These patients are all doing well. As you may recall, in February of this year, we reported initial data from this first cohort – and we announced our plan to increase the dose to 2E14 vg/kg.

Three patients have now been dosed in this second cohort. The first two of these patients are also doing well. As mentioned above, the third patient in the 2E14 vg/kg cohort was dosed late in October and experienced an SAE. The SAE led to low platelet count, a decrease in red blood cell count, acute kidney injury, and cardiopulmonary decline. As last reported, the patient is being closely followed by his care team. He is recovering and continues to improve. We are working hard to better understand this event and we will continue to monitor this patient with Dr. Barry Byrne and his exceptional team at the University of Florida.

Before year end, we look forward to reporting initial biomarker data from the first two patients dosed at 2E14. We will have an update on the status of patient’s health and continue to work to better understand the adverse event and how to best manage administration of SGT-001. Our ultimate goal is to better understand the functional benefit that may result from our treatment and how to effectively manage its administration.

For years now we have been combating this horrible disease and believe we are on the edge of what’s possible. This requires hard work, perseverance and the ability to address and overcome challenges along the way.

Our thoughts are with the last patient dosed and his family. We had, of course, hoped for a different clinical trial experience for this boy and are extremely grateful for his ongoing recovery from the SAE. We are hopeful the child will soon leave the hospital – and that he will have experienced meaningful clinical benefit from this therapy.

We remain as dedicated as ever to getting this right and helping the thousands of young people suffering from Duchenne. As always, we will continue to work with urgency, passion, scientific rigor and the utmost regard for the safety of our patients.

Sincerely,

Your Solid Biosciences team

Click here to read the full letter.

Read Solid’s Press Release:

Solid Biosciences Provides SGT-001 Program Update

Company to hold conference call at 8:30 AM ET today

CAMBRIDGE, Mass., Nov. 12, 2019 (GLOBE NEWSWIRE) — Solid Biosciences Inc. (Nasdaq: SLDB) today provided a clinical update on SGT-001 and reported that the U.S. Food and Drug Administration (FDA) has notified the company that IGNITE DMD, its Phase I/II study of SGT-001, has been placed on clinical hold. The company will hold a webcast conference call this morning to discuss this update.

To date, six patients have been dosed with SGT-001, Solid’s gene transfer candidate under investigation for Duchenne muscular dystrophy (DMD). This includes three patients in the first cohort at a 5E13 vg/kg dose, who continue to do well and are being followed per the study protocol. Three patients were subsequently dosed in the second cohort at a 2E14 vg/kg dose. The first two of these patients are also doing well and being followed per study protocol.

The third patient in the 2E14 vg/kg cohort, dosed in late October, experienced a serious adverse event (SAE) deemed related to the study drug that was characterized by complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury, and cardio-pulmonary insufficiency. Neither cytokine- nor coagulopathy-related abnormalities were observed. Currently the patient is being closely followed by his care team. He is recovering and continues to improve.

The company reported the event to the FDA and the study Data Safety Monitoring Board (DSMB). The FDA has notified the company that the study has been placed on clinical hold. Solid will work with the FDA in an effort to resolve the hold and determine next steps for IGNITE DMD. The company continues to plan to report additional biomarker data from the study before year end.

“We are encouraged that this patient is recovering. I would like to thank both the patient and his family for their participation in our study, as well as the team at the University of Florida for the excellent care they provide,” said Ilan Ganot, Chief Executive Officer, President and Co-Founder of Solid Biosciences. “We remain committed to bringing meaningful new therapies to the Duchenne community and continue to believe in the differentiated construct of SGT-001 and the potential benefits it may offer to patients. In the coming weeks, we anticipate that we will have a better understanding of the biological activity and potential benefit of SGT-001. We look forward to sharing this additional data and working with the FDA to resolve the clinical hold and determining next steps for the program.”

Conference Call Information
The company will host a conference call and webcast at 8:30 a.m. ET today to discuss the program update. Participants are invited to listen by dialing +1 866-763-0341 (domestic) or +1 703-871-3818 (international) five minutes prior to the start of the call and providing the passcode 2277849. A listen-only webcast of the conference call can also be accessed through the “Investors” tab on the Solid Biosciences website, www.solidbio.com, and a replay of the call will be available for approximately six weeks after the call.

About SGT-001
Solid’s lead candidate, SGT-001, is a novel adeno-associated viral (AAV) vector-mediated gene transfer under investigation for its ability to address the underlying genetic cause of DMD, mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase (nNOS). Data from Solid’s preclinical program suggests that SGT-001 has the potential to slow or stop the progression of DMD, regardless of genetic mutation or disease stage.

SGT-001 is based on pioneering research in dystrophin biology by Dr. Jeffrey Chamberlain of the University of Washington and Dr. Dongsheng Duan of the University of Missouri. SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, in the United States and Orphan Drug Designations in both the United States and European Union.

About Solid Biosciences
Solid Biosciences is a life science company focused solely on finding meaningful therapies for Duchenne muscular dystrophy (DMD). Founded by those touched by the disease, Solid is a center of excellence for DMD, bringing together experts in science, technology and care to drive forward a portfolio of candidates that have life-changing potential. Currently, Solid is progressing programs across four scientific platforms: Corrective Therapies, Disease-Modifying Therapies, Disease Understanding and Assistive Devices. For more information, please visit www.solidbio.com.

Click here to read the full press release.