Dompé enrolls first patient in Phase 3 trial of cenegermin in patients with Sjögren's-related dry eye disease

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The NGF0121 study will enroll 100 patients and will use Schirmer's test and the Symptom Assessment Questionnaire in Dry Eye questionnaire as its co-primary endpoints to measure improvement in signs and symptoms of dry eye over time.

Dompé farmaceutici S.p.A. and Dompé U.S. Inc. announced this week that the first patient has been enrolled in its Phase 3 clinical trial to evaluate the efficacy and safety of cenegermin in patients with severe Sjögren's-related dry eye disease, a primary symptom of Sjögren's, which is often under-diagnosed1.

According to the company, this trial is part of a program that includes two randomly assigned, double-masked, vehicle-controlled studies to be conducted at more than 10 sites in the U.S. and Europe, 2,3 and was initiated following encouraging data from a Phase 2 study, results of which will be shared at a future medical meeting. That study evaluated the efficacy and safety of cenegermin ophthalmic solution versus vehicle in patients with moderate-to-severe dry eye, including a subset of patients diagnosed with Sjögren's without an accompanying rheumatic disease. 8

Sjögren's is an autoimmune disorder that can affect many parts of the body. In patients who are affected, the body's white blood cells attack certain glands, including those that keep our eyes and mouth lubricated. As a result, dry eye is common in people living with Sjögren's1. Dry eyes associated with Sjögren's can lead to vision problems including light sensitivity, blurred vision and corneal damage4.

"The majority of Sjögren's patients live with dry eye, and many of these patients will experience ocular symptoms that can often be quite severe," Janet Church, CEO of the Sjögren's Foundation, said in a statement. "We are grateful for Dompé's commitment to research that may lead to new treatment options for people with Sjögren's who suffer from dry eyes and encourage people to talk to their physician to learn more about participating in clinical trials."

There is currently no FDA-approved treatment for severe Sjögren's-related dry eye disease; it is managed by replacing moisture at affected glandular sites and suppressing the autoimmune response locally as well as systemically1.

"With cenegermin, Dompé blazed a trail, leveraging decades of research to better understand the way nerve growth factor (NGF) contributes to the overall health of the eye." Melissa Toyos, MD, lead investigator and partner and research director of the Toyos Clinic, said in a statement. "Studies have demonstrated a neurosensory component to some types of dry eye disease, which suggests that cenegermin may provide an innovative option for these patients." 9

The NGF0121 study (NCT 05133180) will enroll 100 patients and will use Schirmer's test and the Symptom Assessment Questionnaire in Dry Eye (SANDE) questionnaire as its co-primary endpoints to measure improvement in signs and symptoms of dry eye over time2. The NGF0221 study (NCT 05136170) will enroll 48 patients who are currently being treated for severe Sjögren's-related dry eye disease with cyclosporine A3 and will use Schirmer's test as its sole primary endpoint3. Patients in both studies will be randomly assigned (1:1) to receive either cenegermin 20 mcg/mL ophthalmic solution or vehicle, three times daily 2,3.

"These studies exemplify our commitment to exploring how the science of NGF can break new ground for ocular diseases with high unmet medical need." said Ashley Kline, Global Biotech Head, Dompé farmaceutici S.p.A and General Manager, Dompé U.S. Inc. in the news release. "There is no drug or treatment for severe Sjögren's-related dry eye disease that remains durable following the cessation of therapy. It is our hope that cenegermin may have a lasting impact on the ocular tissue and contribute to restoring the natural homeostasis of the eye." 10

For more information or a list of sites for the ongoing clinical trials evaluating the safety and efficacy of cenegermin in patients with severe Sjögren's-related dry eye disease, contact usmedinfo@dompe.com or visit www.clinicaltrials.gov.

References

1. Carsons SE, Patel BC. Sjögren Syndrome. [Updated 2021 Aug 9]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2021 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK431049/.

2. ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). Identifier NCT05133180, Study to Evaluate Safety and Efficacy of Oxervate® Ophthalmic Solution vs Vehicle in Severe Sjogren's Dry Eye Disease (NGF0121); Updated 2022 Jan 18 [accessed 2022 Jan 26]; [about 10 screens]. Available from: https://clinicaltrials.gov/ct2/show/NCT05133180?term=rhNGF&cond=dry+eye&draw=2&rank=5

3. ClinicalTrials.gov [Internet]. Bethesda (MD): National Library of Medicine (US). Identifier NCT05136170, Study to Evaluate Safety and Efficacy of Cenegermin (Oxervate®) 20 mcg/mL vs Vehicle, in Patients With Sjogren's Dry Eye (NGF0221); Updated 2022 Jan 18 [accessed 2022 Jan 26]; [about 10 screens]. Available from: https://clinicaltrials.gov/ct2/show/NCT05136170?term=rhNGF&cond=dry+eye&draw=2&rank=4

4. Kassan SS, Moutsopoulos HM. Clinical manifestations and early diagnosis of Sjögren syndrome. Arch Intern Med. 2004 Jun 28;164(12):1275-84. doi: 10.1001/archinte.164.12.1275. PMID: 15226160.

5. Dompé Farmaceutici S.p.A. (October 2019). OXERVATE™ (cenegermin-bkbj) ophthalmic solution 0.002% (20 mcg/mL): Highlights of prescribing information. Retrieved from https://oxervate.com/wp-content/uploads/2020/05/OXERVATE_Prescribing_Information_102019.pdf [Accessed 2022 Jan 26]

6. Lambiase A, Rama P, Bonini S, Caprioglio G, Aloe L. Topical treatment with nerve growth factor for corneal neurotrophic ulcers. N Engl J Med 1998;338:1174-80.

7. OXERVATE.com. 2019. Important Safety Information - OXERVATE™ (cenegermin-bkbj) ophthalmic solution 0.002% (20 mcg/mL) HCP. [online] Available at: <https://oxervate.com/hcp/#isi> [Accessed 2022 Jan 26].

8. Data on File. NGF0118

9. Villani E, Galimberti D, Viola F, Mapelli C, Ratiglia R. The cornea in Sjogren's syndrome: an in vivo confocal study. Invest Ophthalmol Vis Sci. 2007 May;48(5):2017-22. doi: 10.1167/iovs.06-1129. PMID: 17460255.

10. Zhang X, M VJ, Qu Y, et al. Dry Eye Management: Targeting the Ocular Surface Microenvironment. Int J Mol Sci. 2017;18(7):1398. Published 2017 Jun 29. doi:10.3390/ijms18071398