PR99446

LAUSANNE, Switzerland, January 12, 2023, /PRNewswire=KYODO JBN/--

-  Acromegaly and gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are

rare diseases that require life-long chronic treatment. Monthly somatostatin

analog (SSA) injections are the standard of care as first-line medical

treatment.

-  Debiopharm is developing Debio 4126, a novel 3-month extended-release

octreotide formulation, to optimize acromegaly and GEP-NET patient care by

reducing injection frequency, which translates into an improved quality of life

and less time spent with medical visits.

-  Debio 4126 is currently being investigated in two clinical studies. One of

them (study Debio 4126-102) is a phase 1b study in patients with acromegaly or

GEP-NETs. This study was designed to characterize the pharmacokinetic,

pharmacodynamic, efficacy, safety, and tolerability profiles of Debio 4126

administered intramuscularly (IM) every 3 months over an extended period.

Debiopharm (www.debiopharm.com) [ http://www.debiopharm.com ], a Swiss-based,

global biopharmaceutical company, today announced that the first patient was

dosed with Debio 4126, a 3-month extended-release formulation of octreotide, in

their open-label, non-randomized, single-arm, multicenter phase 1b study (Debio

4126-102). The study is composed of two patient cohorts – 1 group of patients

with acromegaly and 1 group with functioning GEP-NETs Patients will receive 4

injections of Debio 4126 for a total treatment duration of 48 weeks.

Debio 4126 is currently the only 3-month SSA in clinical development. The

development of Debio 4126 arises from Debiopharm's wish to leverage their

modified-release Debiosphere™ technology to alleviate the treatment burden of

patients with rare diseases, such as acromegaly and GEP-NETs, by reducing the

injection frequency to 4 injections per year.

In healthy volunteers, Debio 4126, an extended-release formulation of

octreotide, exhibited good bioavailability and sustained release for up to 3

months.1 It has the potential to offer efficacy and safety similar to those of

marketed 1-month SSAs. If successful, Debio 4126 will equip healthcare

professionals with a new, more convenient option for treating acromegalic and

GEP/NET patients.

"Going into this 2-year long phase 1b trial, we look forward to ascertaining

the benefits of this extended-release formulation. We believe that patient

quality of life can be improved through the substantially reduced number of

injections, from 12 to 4/year," explained Dr Simona Ispas Jouron, Senior

Medical Director, Endocrinology & Rare Diseases at Debiopharm.

Prior to this compound, Debiopharm's extended-release formulation expertise

extends over more than 35 years with the development of multiple formulations

of agonist analogue triptorelin, benefiting patients affected by prostate

cancer, breast cancer, endometriosis, central precocious puberty (CPP), and

uterine myomas.

"The success of triptorelin is linked to our modified-release Debiosphere™

technology that has contributed to the improvement of the quality of life of

patients treated long-term, particularly with our 1, 3 and 6-month formulations

of triptorelin and now with Debio 4126. This formulation adds significant value

to an efficacious and safe standard of care." expressed Bertrand Ducrey, CEO of

Debiopharm.

About Acromegaly

Acromegaly is a rare chronic disorder caused by excessive GH secretion by

pituitary adenomas, with more than 95% of cases being benign.2 The condition

most commonly affects middle-aged adults, with a slight predominance of female

patients (52-60%)3 and equal distribution among ethnicities. The disease is

clinically diagnosed based on characteristic symptoms, including progressive

skeletal and soft tissue overgrowth, mainly at the extremities (hands and feet)

and head. Diagnosis is confirmed biochemically via increased serum

concentrations of GH and IGF-1. Acromegaly is associated with a twofold

increase in mortality relative to that expected in the general population,

mostly due to cardiovascular events. Associated risk factors include

hypertension, glucose metabolism abnormalities, dyslipidemia, abdominal

adiposity, and peripheral insulin resistance (metabolic syndrome). Untreated

acromegaly is associated with a reduced quality of life and life expectancy

shortened by approximately 10 years.2

About Gastro-entero-pancreatic neuroendocrine tumors (GEP-NET)

GEP-NETs are rare and complex malignant solid tumors, derived from

neuroendocrine cells, occurring in various sites along the gastrointestinal

tract. Although these tumors have been considered rare, the most recent data

from the US Surveillance Epidemiology and End Results show an increase of more

than 400% in the incidence of these diseases over the period from 1973 to

2004. 4 Age at diagnosis is generally younger than 50 years, and these tumors

may arise sporadically or because of hereditary predisposition. Survival of

patients with GEP-NETs depends on the stage and histology. Patients with well

and moderately differentiated metastases have a 5-year survival probability of

35%, while poorly differentiated metastases lead to a 5-year survival

probability of 4%4

About Debio 4126

Octreotide is a synthetic octapeptide that mimics the pharmacology of

endogenous somatostatin (SST). Currently, octreotide is available as

immediate-release (Sandostatin®) and 4-week long-release formulations

(Sandostatin LAR®). Debio 4126 is a novel, 3-month extended-release formulation

of octreotide being developed by Debiopharm.

Debiopharm's commitment to patients

Debiopharm aims to develop innovative therapies that target high unmet medical

needs in oncology and bacterial infections. We identify high-potential

compounds and technologies for in-licensing, clinically demonstrate their

safety and efficacy, and then select pharmaceutical commercialization partners

to maximize patient access globally.

For more information, please visit www.debiopharm.com [

https://www.debiopharm.com/ ]

We are on Twitter. Follow us @DebiopharmNews at

http://twitter.com/DebiopharmNews [ https://twitter.com/DebiopharmNews ]

Debiopharm Contact

Dawn Bonine

Head of Communications

dawn.bonine@debiopharm.com

Tel: +41 (0)21 321 01 11

References

[1] Bellon A, et al. ENEA. 2022.

[2] Chanson P, et al. Pituitary tumours: acromegaly. Best Pract Res Clin

Endocrinol Metab. 2009;23(5):555-74.

[3] Fleseriu M, et al. Acromegaly: pathogenesis, diagnosis, and management.

Lancet Diabetes Endocrinol 2022 Nov;10(11):804-826

[4] Yao, et al. Journal of clinical oncology : official journal of the American

Society of Clinical Oncology vol. 26,18 (2008): 3063-72.

Source: Debiopharm International SA