Health Canada Approves INCRELEX®, First and Only Recombinant Human Insulin-like Growth Factor-1 (IGF-1) Therapy in Canada for the Treatment of Severe Growth Failure Associated with Ultra-Rare Condition in Children and Adolescents

MISSISSAUGA, ON, March 16, 2021 /CNW/ - Ipsen Biopharmaceuticals Canada Inc. today announced the commercial availability of INCRELEX^® (mecasermin) for the treatment of growth failure in children and adolescents from 2 to 18 years with confirmed severe primary insulin-like growth factor-1 deficiency (SPIGFD), following its Health Canada approval in December 2020.¹

INCRELEX^® is the first and only recombinant human IGF-1 therapy approved in Canada for children and adolescents with SPIGFD, an ultra-rare condition that affects the growth of fewer than five people per 10,000 globally.²

"Until now, there has been no treatment for children and adolescents with SPIGFD in Canada, and now we can do more to help patients," said Dr. Cheri Deal, past Chief of Pediatric Endocrinology and Diabetes at CHU Ste-Justine. "Arriving at an accurate diagnosis can be complex and may take several trips to multiple physicians before a short stature disorder is diagnosed. It's critical we investigate the children at the lower end of the growth chart and diagnose early because treatment is ineffective once the growth plates close. INCRELEX^® is an important milestone, providing hope for families who have a SPIGFD diagnosis."

The approval of INCRELEX^® by Health Canada was based on results from a clinical study involving 81 treatment-naïve patients with SPIGFD, with a primary efficacy endpoint of height velocity, which was compared with pre-treatment levels.³ The trial showed that long-term INCRELEX^® treatment resulted in a sustained increase in height velocity above pre-treatment levels for up to eight years, with the largest response observed in the first year.⁴

IGF-1 is a hormone important for the growth of bones and muscles and is normally produced by the body.⁵ A synthetic form of IGF-1, INCRELEX^® stimulates statural growth in children who are IGF-1 deficient, enabling growth.⁶

"We are very pleased INCRELEX^® has received Health Canada approval, which is great news for the SPIGFD community," said Ed Dybka, General Manager, Ipsen Biopharmaceuticals Canada. "Our mission is to give hope to those with serious and difficult-to-treat diseases – no matter the size of the patient population – which is why Ipsen is proud to make INCRELEX^® available to Canadian children and adolescents with SPIGFD who have previously had no treatment available to them. With patients at the heart of everything we do, the approval of INCRELEX^® is an example of our commitment to meeting the needs of Canadians with rare and serious diseases and our drive to provide new therapies for the rare disease community."

About SPIGFD
SPIGFD is defined by a height standard deviation score ≤ –3.0; basal IGF-1 levels below the 2.5th percentile for age and gender; growth hormone sufficiency; and exclusion of secondary forms of IGF-1 deficiency, such as malnutrition, hypopituitarism, hypothyroidism, or chronic treatment with pharmacologic doses of anti-inflammatory steroids.⁷

Children with SPIGFD have abnormally low levels of IGF-1, a hormone made primarily in the liver. IGF-1 regulates growth by affecting development of body tissues such as the growth plates of the bones.⁸ SPIGFD presents as growth failure and can potentially be associated with metabolic and dysmorphic abnormalities which can vary in severity based on the underlying genetic issue.⁹

SPIGFD includes patients with mutations in the GH receptor (GHR) gene/Laron's syndrome, post-GHR signaling pathway, and IGF-1 gene defects; they are not GH deficient, and therefore, they cannot be expected to respond adequately to exogenous GH treatment.¹⁰

Short stature is defined as a height more than two standard deviations below the mean for age.¹¹ Studies show compared to those with normal height, children with short stature experience poorer health-related quality of life.¹² Their size can impact their social relationships, self-esteem, and cognitive functioning.¹³

About INCRELEX^® 
INCRELEX^® (mecasermin) is indicated for the treatment of growth failure in children and adolescents from 2 to 18 years with confirmed SPIGFD.¹⁴ INCRELEX^® is administered by subcutaneous injection.¹⁵

The most common adverse events (AEs) reported were hypoglycemia, hypersensitivity, injection site hypertrophy, headache and snoring. Hypoglycemia was the most frequently reported AE.¹⁶ There is an increased risk of benign and malignant neoplasia in children and adolescents treated with INCRELEX^®, since IGF-1 plays a role in the initiation and progression of benign and malignant tumours.¹⁷ INCRELEX^® is contraindicated for children or adolescents with active or suspected neoplasia, or any condition or medical history which increases the risk of benign or malignant neoplasia.¹⁸

About Ipsen in North America
Ipsen (Euronext: IPN; ADR: IPSEY) is a global biopharmaceutical company focused on innovation and specialty care. The company develops and commercializes innovative medicines in three key therapeutic areas – Oncology, Neuroscience and Rare Diseases. At Ipsen, we focus our resources, investments and energy on discovering, developing and commercializing new therapeutic options to provide hope for patients whose lives are challenged by difficult-to-treat diseases. Ipsen's North American operations are located in Cambridge, Massachusetts, one of the company's three global hubs. Based in the heart of Kendall Square, our fully integrated biopharmaceutical business includes Commercial, Research & Development, Manufacturing, and Global External Innovation and Partnering. Combined with Ipsen Biopharmaceuticals Canada Inc., our Canadian headquarters in Mississauga, Ontario, and other locations, Ipsen employs approximately 600 people in North America. For more information, please visit www.ipsenus.com or www.ipsen.ca.

Ipsen's Forward Looking Statement
The forward-looking statements, objectives and targets contained herein are based on the Group's management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect the Group's future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words "believes", "anticipates" and "expects" and similar expressions are intended to identify forward-looking statements, including the Group's expectations regarding future events, including regulatory filings and determinations, and the outcome of this study or other studies. Moreover, the targets described in this document were prepared without taking into account external growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by the Group. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising product in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. The Group must face or might face competition from generic products that might translate into a loss of market share. Furthermore, the Research and Development process involves several stages each of which involves the substantial risk that the Group may fail to achieve its objectives and be forced to abandon its efforts with regards to a product in which it has invested significant sums. Therefore, the Group cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the product concerned. There can be no guarantees a product will receive the necessary regulatory approvals or that the product will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of 6 pharmaceutical industry regulation and health care legislation; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the Group's ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the Group's patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions. The Group also depends on third parties to develop and market some of its products which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to the Group's activities and financial results. The Group cannot be certain that its partners will fulfill their obligations. It might be unable to obtain any benefit from those agreements. A default by any of the Group's partners could generate lower revenues than expected. Such situations could have a negative impact on the Group's business, financial position or performance. The Group expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. The Group's business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to the Group's 2019 Universal Registration Document available on its website (www.ipsen.com).

References

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¹ INCRELEX Product Monograph. Ipsen Biopharmaceuticals Canada Inc. December 17, 2020

² Ipsen Endocrinology, https://www.ipsen.com/websites/IPSENCOM-PROD/wp-content/uploads/2016/07/14120205/2015-AR-Endocrinology.pdf. Last accessed: January 22, 2021

³ INCRELEX Product Monograph. Ipsen Biopharmaceuticals Canada Inc. December 17, 2020

⁴ INCRELEX Product Monograph. Ipsen Biopharmaceuticals Canada Inc. December 17, 2020

⁵ Wellspan Health. Mecasermin, https://www.wellspan.org/health-library/Document.aspx?id=d05636a1. Last accessed January 5, 2021

⁶ INCRELEX, About Increlex, https://www.increlex.com/about-increlex/. Last accessed January 27, 2021

⁷ INCRELEX Product Monograph. Ipsen Biopharmaceuticals Canada Inc. December 17, 2020

⁸ Child Growth Foundation, https://childgrowthfoundation.org/conditions/igf1-deficiency/. Last accessed January 5, 2021

⁹ Savage, Martin O. Phenotypes, Investigation and Treatment of Primary IGF-1 Deficiency.Endocr Dev. 2013;24:138-49. doi: 10.1159/000342578. Epub 2013 Feb 1

¹⁰ INCRELEX Product Monograph. Ipsen Biopharmaceuticals Canada Inc. December 17, 2020

¹¹ Barstow, C. and Rerucha, C. (2015) Evaluation of short and tall stature in children. American Family Physician. 1:92: 43-50

¹² Lundkvist J, Shohet S, Cookson C, Law LM, Ribeiro-Oliveira Jr A. Assessing the human and economic burden of short stature: a systematic literature review. Value in Health. Vol 22, S733

¹³ Lundkvist J, Shohet S, Cookson C, Law LM, Ribeiro-Oliveira Jr A. Assessing the human and economic burden of short stature: a systematic literature review

¹⁴ INCRELEX Product Monograph. Ipsen Biopharmaceuticals Canada Inc. December 17, 2020

¹⁵ INCRELEX Product Monograph. Ipsen Biopharmaceuticals Canada Inc. December 17, 2020

¹⁶ INCRELEX Product Monograph. Ipsen Biopharmaceuticals Canada Inc. December 17, 2020

¹⁷ INCRELEX Product Monograph. Ipsen Biopharmaceuticals Canada Inc. December 17, 2020

¹⁸ INCRELEX Product Monograph. Ipsen Biopharmaceuticals Canada Inc. December 17, 2020

SOURCE Ipsen Biopharmaceuticals Canada Inc.

Diana Robson, Senior Consultant, Proof Strategies, [email protected], [phone]; Maryann Quinn, Director, North America Product Communications, Ipsen Biopharmaceuticals, [email protected], [phone]

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