SRPT Sarepta Therapeutics Inc.

Sarepta Therapeutics Announces Third Quarter 2021 Financial Results and Recent Corporate Developments

Sarepta Therapeutics Announces Third Quarter 2021 Financial Results and Recent Corporate Developments

  • Net product sales for the third quarter of 2021 reached $166.9 million, a 37% increase over the same quarter of prior year
  • In light of its continued over-performance, Sarepta raises its full-year product revenue guidance by $40 million to between $605 million to $615 million

CAMBRIDGE, Mass., Nov. 03, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the third quarter of 2021.

“We are pleased to report another quarter of strong performance serving the Duchenne community with our three currently approved therapies and on that basis have once again raised our full-year product revenue guidance. In total, we have raised guidance by some $70 million this year and are now guiding to $605 million to $615 million. This represents our 20th straight quarter of strong revenue growth and we anticipate this growth continuing in 2022,” said Doug Ingram, president and chief executive officer, Sarepta. “We have now initiated Part B of MOMENTUM, our pivotal trial for SRP-5051, our next-generation PPMO candidate for exon 51 skip amenable Duchenne patients as well as EMBARK, our pivotal trial for SRP-9001, our micro-dystrophin gene therapy for Duchenne. Also, this quarter we shared additional compelling data across three studies for SRP-9001, providing additional conviction as we execute on EMBARK and prepare to unblind and release Study 102 Part 2 results in the first quarter of next year. As we track out of 2021 and into a milestone-rich 2022, we are delivering on our approved therapies, seeing successes across our programs, and as of today, with greater than $2 billion of cash and cash equivalents on our balance sheet and a first-in-class team of genetic and rare disease professionals, have the resources and talent to deliver on the promise of our multi-platform pipeline.”

Third Quarter 2021 and Recent Corporate Developments:

  • At SRP-9001 Micro-dystrophin R&D Day Sarepta showed sustained functional improvements in multiple studies of individuals with Duchenne: In October of 2021, the Company presented new analyses and functional data from its SRP-9001 development program and details of EMBARK (Study SRP-9001-301); its global pivotal Phase 3 trial of SRP-9001 for the treatment of Duchenne. SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. The results presented highlight the breadth, depth and strength of the clinical evidence to date for SRP-9001 in treating Duchenne.
    • SRP-9001-101 (Study 101): Results from participants treated with SRP-9001 in Study 101 (n=4, ages 4 to 7) found that participants improved 8.6 points on the North Star Ambulatory Assessment (NSAA) compared to a matched natural history cohort three years following a single administration of SRP-9001 (p<0.0001).
    • SRP-9001-102 (Study 102): The results from Study 102 Part 1 found that SRP-9001-treated participants (n=12, ages 6 to 7) had a positive 2.9-point difference on NSAA compared to a matched natural history cohort one year after treatment (p=0.0129).
    • ENDEAVOR, SRP-9001-103 (Study 103): The first functional results presented from Study 103, which uses commercially representative SRP-9001 material, the first 11 participants in Cohort 1, ages 4 to 7, demonstrated a 3.0-point improvement from baseline on NSAA six months after treatment.
    • Across three clinical studies, the tolerability profile remains consistent in all treated patients.



  • Initiated EMBARK (Study SRP-9001-301), the first global pivotal study of SRP-9001 for the treatment of Duchenne: The EMBARK study is a multi-center clinical trial initiating in the US, Europe and Asia. It is the first global, randomized, double-blind, placebo-controlled clinical trial of commercially representative SRP-9001 material and will enroll 120 participants with Duchenne between the ages of 4 to 7. The primary endpoint will assess the change in NSAA total score from baseline to week 52 compared to placebo. Key features include stratification of participants by age and baseline NSAA, with a minimum of 50 percent of patients ages 4 to 5 to be enrolled. Inclusion criteria include a Time to Rise from Floor of less than 5 seconds, a stable daily dose of oral corticosteroids for at least 12 weeks before screening and rAAVrh74 antibody titers of less than 1:400. Participants with mutations between or including exons 1-17 or mutations fully contained within exon 45 (inclusive) are not eligible. The Company expects the trial to fully enroll in the first half of 2022.



  • Initiated Part B of MOMENTUM (Study SRP-5051-201) in patients with Duchenne amenable to exon 51 skipping: In the fourth quarter of 2021, the Company initiated Part B of MOMENTUM, a global trial investigating the use of SRP-5051, the Company’s next-generation peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) to treat patients with Duchenne who are amenable to exon 51 skipping. The study plans to enroll between 20-40 patients between ages 7 to 21 amenable to exon 51 skipping who are naïve to SRP-5051. Additionally, those previously dosed in Study 5051-201, Part A or Study 5051-102 who meet the entrance criteria will be eligible to participate. Both ambulatory and non-ambulatory patients are eligible for participation. Sarepta anticipates Part B of MOMENTUM to serve as a pivotal study for SRP-5051 and plans to seek Accelerated Approval if the trial is successful.



  • Showcased data from gene therapy and RNA platforms at World Muscle Society (WMS) 2021 Virtual Congress: Sarepta’s presentations at WMS 2021 highlighted scientific leadership and innovation from across the Company’s deep, multi-platform portfolio and reflect a continued commitment to advancing life-changing therapies for those with rare genetic diseases. Notably, new research was presented on the prevalence of pre-existing antibodies to the AAVrh74 vector, which is used in several of Sarepta’s gene transfer therapy programs. The posters are available on the Investor Relations section of .

Conference Call

The Company will be hosting a conference call at 4:30 p.m. Eastern Time to discuss Sarepta’s financial results and provide a corporate update. The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The passcode for the call is 7131019. Please specify to the operator that you would like to join the “Sarepta Third Quarter 2021 Earnings Call.” The conference call will be webcast live under the investor relations section of Sarepta's website at  and will be archived there following the call for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

Financial Results

On a GAAP basis, for the three months ended September 30, 2021, the Company reported a net loss of $48.1 million, or $0.60 per basic and diluted share, compared to a net loss of $196.5 million reported for the same period of 2020, or $2.50 per basic and diluted share. On a non-GAAP basis, the net loss for the three months ended September 30, 2021 was $15.6 million, or $0.19 per basic and diluted share, compared to a net loss of $111.5 million, or $1.42 per basic and diluted share for the same period of 2020.

On a GAAP basis, for the nine months ended September 30, 2021, the Company reported a net loss of $296.8 million, or $3.72 per basic and diluted share, compared to a net loss of $364.8 million reported for the same period of 2020, or $4.70 per basic and diluted share. On a non-GAAP basis, the net loss for the nine months ended September 30, 2021 was $259.2 million, or $3.25 per basic and diluted share, compared to a net loss of $309.2 million, or $3.98 per basic and diluted share for the same period of 2020.

Revenues

For the three months ended September 30, 2021, the Company recorded total revenues of $189.4 million, compared to total revenues of $143.9 million for the same period of 2020, an increase of $45.5 million. For the nine months ended September 30, 2021, the Company recorded total revenues of $500.4 million, compared to total revenues of $395.0 million for the same period of 2020, an increase of $105.4 million.

For the three months ended September 30, 2021, the Company recorded net product revenues of $166.9 million, compared to net product revenues of $121.4 million for the same period of 2020, an increase of $45.5 million. For the nine months ended September 30, 2021, the Company recorded net product revenues of $433.7 million, compared to net product revenues of $333.2 million for the same period of 2020, an increase of $100.5 million. The increase primarily reflects the launch of AMONDYS 45 in the first quarter of 2021 and the continuing increase in demand for the Company’s other two products in the U.S.

For both the three months ended September 30, 2021 and 2020, the Company recognized $22.5 million of collaboration revenue. For the nine months ended September 30, 2021 and 2020, the Company recognized $66.8 million and $61.7 million of collaboration revenue, respectively. For all periods presented, collaboration revenue primarily relates to the F. Hoffman-La Roche Ltd. (Roche) collaboration arrangement.

Cost and Operating Expenses

Cost of sales (excluding amortization of in-licensed rights)

For the three months ended September 30, 2021, cost of sales (excluding amortization of in-licensed rights) was $23.4 million, compared to $15.0 million for the same period of 2020, an increase of $8.4 million. The increase in cost of sales is primarily due to increasing demand for the Company’s products. For the nine months ended September 30, 2021, cost of sales (excluding amortization of in-licensed rights) was $65.3 million, compared to $41.0 million for the same period of 2020, an increase of $24.3 million. The increase is primarily due to increasing demand for the Company’s products and the write-offs of certain batches of the Company’s products not meeting the Company’s quality specifications for the nine months ended September 30, 2021, with