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

MILAN, Italy and SAN MATEO, Calif, Feb. 9, 2022 /PRNewswire/ -- Dompé farmaceutici S.p.A. and Dompé U.S. Inc. (collectively Dompé) announced today 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. This trial is part of a program that includes two randomized, 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 (pronounced SHOW-grins) 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," said Janet Church, CEO of the Sjögren's Foundation. "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."  said Melissa Toyos, MD, lead investigator and Partner and Research Director of the Toyos Clinic. "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. "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 [email protected] or visit www.clinicaltrials.gov.

About Oxervate® (cenegermin-bkbj Ophthalmic Solution)

Oxervate is a topical solution 0.002% of cenegermin-bkbj (20 mcg/mL) currently approved by the FDA for the rare disease, Neurotrophic Keratitis (NK).  Its active ingredient cenegermin-bkbj, is a recombinant form of human nerve growth factor and is structurally identical to the NGF protein made naturally by the body^{5, 6}. Nerve growth factor (NGF), is involved in the differentiation and maintenance of neurons which acts through specific high-affinity and low-affinity receptors in the anterior segment of the eye to support corneal innervation and integrity.⁷

Oxervate is not yet approved by the FDA for the treatment of severe Sjögren's-related dry eye disease. More information on Oxervate for neurotrophic keratitis can be found in the full prescribing information on the product: www.oxervate.com

Important Safety Information

Contact lenses should be removed before applying OXERVATE because the presence of a contact lens (either therapeutic or corrective) could theoretically limit the distribution of cenegermin-bkbj onto the area of the corneal lesion. Lenses may be reinserted 15 minutes after administration.

OXERVATE may cause mild to moderate eye discomfort such as eye pain during treatment. The patient should be advised to contact their doctor if a more serious eye reaction occurs.

The most common adverse reaction in clinical trials that occurred more frequently with OXERVATE was eye pain (16% of patients). Adverse reactions included corneal deposits, foreign body sensations in the eye, ocular hyperemia (enlarged blood vessels in the white of the eyes), swelling (inflammation) of the eye, and increase of tears (1-10% of patients). ⁷

Dompé in Ophthalmology

Dompé farmaceutici S.p.A. is a private, rapidly scaling global biopharmaceutical company founded in Milan, Italy, with a 130-year legacy of medical innovation. For more than a decade, Dompé has been a leader in the research and development of innovative therapies based on neurotrophins, proteins derived from Nerve Growth Factor (NGF). The company's expertise in NGF began in ophthalmology and led to a breakthrough-designation followed FDA approval for Oxervate® for the rare disease, Neurotrophic Keratitis (NK). Ongoing research in ophthalmology includes post-marketing research in NK, studies in severe Sjögren's-related dry eye disease, and additional undisclosed programs.  Dompé's R&D is anchored by EXSCALATE, a structure-based virtual screening platform developed in-house that leverages one of the most powerful supercomputing and artificial intelligence platforms in the world. Dompé employs more than 800 employees worldwide and maintains U.S. commercial operations hub in the San Francisco Bay Area as well as an R&D presence in Boston.

Forward Looking Statements

This press release refers to certain information that may not coincide with expected future results. Dompé firmly believes in the soundness and reasonableness of the concepts expressed. However, some of the information is subject to a certain degree of indetermination in relation to its research and development activities and the necessary verifications to be performed by regulatory bodies. Therefore, as of today, Dompé cannot guarantee that the expected results will be consistent with the information provided above.

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

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