BridgeBio Pharma’s Origin Biosciences Enters Partnership with Medison To Commercialize BBP-870 In Israel for MoCD Type A

BOSTON, December 18, 2019 – BridgeBio Pharma, Inc. (Nasdaq: BBIO) subsidiary Origin Biosciences, a company focused on developing and commercializing a treatment for Molybdenum Cofactor Deficiency (MoCD) Type A, and Medison Pharma Ltd., a leading commercial biotech partner that operates in Israel, Canada and Central and Eastern European countries, have entered into an exclusive license agreement under which Medison received rights from Origin to distribute, market, sell and otherwise commercialize Origin’s drug product known as fosdenopterin (BBP-870/ORGN001) in Israel.

Fosdenopterin is a cPMP replacement therapy designed to treat patients with MoCD Type A. Origin recently initiated the rolling submission of a New Drug Application (NDA) with the US Food and Drug Administration (FDA) for fosdenopterin. Subsequent to FDA approval, Medison will be responsible for seeking the requisite regulatory approval and, once approved, commercializing fosdenoptrin in Israel. Origin will receive an upfront cash payment and will receive milestone payments upon the achievement of certain milestone events and ongoing royalties on net sales of fosdenopterin in Israel.

“Our partnership with Medison marks an important step in Origin’s global commercial strategy and strengthens our ability to make fosdenopterin available to infants and children suffering from MoCD Type A,” said Matt Outten, Chief Commercial Officer at BridgeBio. “Medison has a strong commercial organization in Israel with a proven track record of successfully marketing orphan disease products. Its commercial infrastructure is uniquely suited to support patients suffering from rare diseases like MoCD Type A. We look forward to collaborating with Medison to enhance our efforts to help patients by targeting MoCD Type A at its source.”

“Our promise is to assure that every patient under our responsibility has access to the best available treatments,” said Meir Jakobsohn, the founder and CEO of Medison Pharma. “The partnership with Origin Biosciences echoes our shared internal values of scientific excellence.”

Fosdenopterin has received Orphan Drug Designation in the US and Europe, and Rare Pediatric Disease Designation and Breakthrough Therapy Designation in the United States. Since the NDA for fosdenopterin is seeking approval of treatment for patients with a serious and life-threatening disease with no other treatment options (MoCD Type A), it is also eligible for Priority Review Designation, which, if granted, may further expedite the NDA review time for the potential approval of this new medicine in the United States.

About Molybdenum Cofactor Deficiency (MoCD) Type A

MoCD Type A is an ultra-rare, autosomal recessive, inborn error of metabolism caused by disruption in molybdenum cofactor (MoCo) synthesis that is vital for sulfite oxidase (SOX) activity. Patients are often infants with severe encephalopathy and intractable seizures. Disease progression is rapid with a high infant mortality rate.1,2 Those who survive beyond the first few months experience profuse developmental delays and suffer the effects of irreversible neurological damage, including brain atrophy with white matter necrosis, dysmorphic facial features, and spastic paraplegia.1,4 Clinical presentation that can be similar to hypoxic-ischemic encephalopathy (HIE) or other neonatal seizure disorders may lead to misdiagnosis and underdiagnosis.2,3 Immediate testing for elevated sulfite levels and S-sulfocysteine in the urine and very low serum uric acid may help with suspicion of MoCD Type A.2,4

About Origin Biosciences

Origin Biosciences, a subsidiary of BridgeBio Pharma Inc., is a biotechnology company focused on developing and commercializing a treatment for MoCD Type A. Origin is led by a team of veteran biotechnology executives. Together with patients and physicians, the company aims to bring a safe, effective treatment for MoCD Type A to market as quickly as possible. For more information on Origin Biosciences, please visit the company’s website at www.origintx.com.

About Medison

Medison, is one of the world’s largest commercial partners of leading global biotech companies, providing the complete spectrum of integrated services for international companies looking to enter or expand their presence in Israel, Canada and CEE markets. Medison operates a corporate venture arm with a dedicated research and evaluation team boasting deep scientific and commercial backgrounds. Medison also operates a scouting program to cater its partners and is an active investor in life science projects around drug development and digital health.

About BridgeBio Pharma

BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio’s pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development.

BridgeBio Pharma Forward Looking Statements

This press release contains forward-looking statements. Statements we make in this press release may include statements which are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to Origin Biosciences’ clinical development plans, including its clinical development plans for BBP-870 (ORGN001), clinical trial results, timing and completion of clinical trials and regulatory submissions, competitive environment and clinical and therapeutic potential of BBP-870, the potential approval of BBP-870 by the FDA and subsequent development and commercialization by Medison of BBP-870 under the exclusive license agreement and our ability to receive milestone and royalty payments under the agreement, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, Origin Biosciences’ ability to continue its planned clinical development and regulatory submissions for BBP-870 and the timing and success of any such continued clinical development and planned regulatory submissions, as well as those set forth in the Risk Factors section of BridgeBio Pharma Inc.’s most recent Quarterly Report on Form 10-Q and our other SEC filings. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

  1. Spiegel R, Schwahn B, Scribner C L, Confer N. A natural history study of molybdenum cofactor (MoCo) and isolated sulfite oxidase deficiencies (ISOD). https://origintx.com/posters/
  2. Mechler K, Mountford WK, Hoffmann GF, Ries M. Ultra-orphan diseases: a quantitative analysis of the natural history of molybdenum cofactor deficiency. Genet Med. 2015;17(12):965-970.
  3. Durmaz MS, Özbakır B. Molybdenum cofactor deficiency: neuroimaging findings. Radiol Case Rep. 2018;13(3):592-595.
  4. Schwahn BC, Van Spronsen FJ, Belaidi AA, et al. Efficacy and safety of cyclic pyranopterin monophosphate substitution in severe molybdenum cofactor deficiency type A: a prospective cohort study. Lancet. 2015;386(10007):1955-1963.

For more information:

Origin Biosciences
Grace Rauh
[email protected]
(917) 232-5478

Medison Pharma LTD.
Gil Gurfinkel
VP Corporate Development
[email protected]

Source: BridgeBio Pharma, Inc., Medison Pharma Ltd.

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