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Efmody® is a modified-release preparation of hydrocortisone that has been specifically designed for the treatment of patients with CAH, a rare condition caused by a genetic deficiency of adrenal enzymes.


Lead Product(s): Hydrocortisone

Therapeutic Area: Genetic Disease Product Name: Efmody

Highest Development Status: ApprovedProduct Type: Small molecule

Recipient: EffRx Pharmaceuticals

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Licensing Agreement April 26, 2022

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Efmody® is a preparation of hydrocortisone that has been specifically designed for patients with CAH, a rare condition caused by deficiency of adrenal enzymes, most commonly 21-hydroxylase.


Lead Product(s): Hydrocortisone

Therapeutic Area: Genetic Disease Product Name: Efmody

Highest Development Status: ApprovedProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable September 13, 2021

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The $1.25 million milestone payment has been triggered as a result of the recent European Commission’s approval for Efmody® (hydrocortisone modified-release hard capsules) and the attainment by Citrine of certain development milestones in China for Alkindi.


Lead Product(s): Hydrocortisone

Therapeutic Area: Genetic Disease Product Name: Efmody

Highest Development Status: ApprovedProduct Type: Small molecule

Partner/Sponsor/Collaborator: Citrine Medicine

Deal Size: $2.2 million Upfront Cash: $1.0 million

Deal Type: Partnership June 01, 2021

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Efmody® is a preparation of hydrocortisone that has been specifically designed for patients with CAH, an orphan condition caused by deficiency of adrenal enzymes, most commonly 21-hydroxylase.


Lead Product(s): Hydrocortisone

Therapeutic Area: Genetic Disease Product Name: Efmody

Highest Development Status: ApprovedProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable May 28, 2021

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The Phase 3 study results published by the JCEM found that although the standard-deviation-score-focused primary endpoint of the study was missed, Chronocort® improved morning and early afternoon biochemical control for adults with CAH over standard glucocorticoid therapy.


Lead Product(s): Hydrocortisone

Therapeutic Area: Genetic Disease Product Name: Chronocort

Highest Development Status: Phase IIIProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable February 02, 2021

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Citrine will be responsible for obtaining registration for Alkindi® as a treatment for paediatric patients in China and for all commercialisation activities, including pricing and reimbursement.


Lead Product(s): Hydrocortisone

Therapeutic Area: Genetic Disease Product Name: Alkindi

Highest Development Status: ApprovedProduct Type: Small molecule

Partner/Sponsor/Collaborator: Citrine

Deal Size: $13.2 million Upfront Cash: $0.5 million

Deal Type: Licensing Agreement January 27, 2021

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Eton Pharmaceuticals Canadian rights to ALKINDI® SPRINKLE from Diurnal Group plc. Eton Pharmaceuticals currently commercializes ALKINDI® SPRINKLE in the United States as a replacement therapy for Adrenocortical Insufficiency (AI) in children under 17 years of age.


Lead Product(s): Hydrocortisone

Therapeutic Area: Endocrinology Product Name: Alkindi Sprinkle

Highest Development Status: ApprovedProduct Type: Small molecule

Partner/Sponsor/Collaborator: Eton Pharmaceuticals

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Acquisition January 14, 2021

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The submission for the MHRA is based on the data from the Company’s Phase 3 study, an open-label safety extension study of Chronocort® and written formal Scientific Advice received in April 2019 confirming the clinical and regulatory pathway for Chronocort® for CAH treatment.


Lead Product(s): Hydrocortisone

Therapeutic Area: Genetic Disease Product Name: Chronocort

Highest Development Status: Phase IIIProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 12, 2021

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Under the terms of the agreement EffRx has received the exclusive rights to register and commercialize Alkindi® in Switzerland.


Lead Product(s): Hydrocortisone

Therapeutic Area: Endocrinology Product Name: Alkindi Sprinkle

Highest Development Status: ApprovedProduct Type: Small molecule

Partner/Sponsor/Collaborator: EffRx

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Licensing Agreement October 21, 2020

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The acquisition advanced Eton’s leadership in pediatric rare diseases products providing a $100 million market opportunity.


Lead Product(s): Hydrocortisone

Therapeutic Area: Endocrinology Product Name: Undisclosed

Highest Development Status: Phase IIIProduct Type: Small molecule

Partner/Sponsor/Collaborator: Eton Pharmaceuticals

Deal Size: $7.5 million Upfront Cash: $3.5 million

Deal Type: Licensing Agreement March 27, 2020

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