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Home » PKU

Articles Tagged with ''PKU''

Handshake with DNA, molecules

Otsuka to buy Boston chemoproteomics biotech for up to $1.1B

Aug. 6, 2024
By Marian (YoonJee) Chu
Tokyo-headquartered Otsuka Pharmaceutical Co. Ltd. said Aug. 1 that it will acquire Boston-based Jnana Therapeutics Inc. through a potential $1.125 billion M&A deal. Under the terms, Otsuka will work to close the transaction by the third quarter of 2024, with $800 million paid out to Jnana shareholders, along with up to $325 million in additional development and regulatory milestones payments.
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Handshake with DNA, molecules

Otsuka to buy Boston chemoproteomics biotech for up to $1.1B

Aug. 2, 2024
By Marian (YoonJee) Chu
Tokyo-headquartered Otsuka Pharmaceutical Co. Ltd. said Aug. 1 that it will acquire Boston-based Jnana Therapeutics Inc. through a potential $1.125 billion M&A deal. Under the terms, Otsuka will work to close the transaction by the third quarter of 2024, with $800 million paid out to Jnana shareholders, along with up to $325 million in additional development and regulatory milestones payments.
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Layoff illustration

Synlogic pulls plug after peek at phenylketonuria phase III data

Feb. 9, 2024
By Randy Osborne
Synlogic Inc. jolted Wall Street with news that the firm is scrapping for futility Synpheny-3, its pivotal study with labafenogene marselecobac (SYNB-1934) for phenylketonuria, and will evaluate strategic options. Shares of the Cambridge, Mass.-based firm (NASDAQ:SYBX) fell 48.7%, or $1.68, to end Feb. 9 at $1.77. Synlogic will cease operations and reduce its workforce by more than 90%, retaining only certain employees to help with the wind-down.
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Coming through in PKU: PTC’s sepiapterin weighed against Kuvan post-phase III

May 17, 2023
By Randy Osborne
With PTC Therapeutics Inc.’s positive results in phase III with sepiapterin for pediatric and adult patients with phenylketonuria (PKU), attention turned to the would-be showdown with Kuvan (sapropterin dihydrochloride), the drug from Biomarin Pharmaceuticals Inc. that was approved in December 2017.
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Endocrine/Metabolic

SLC6A19 inhibition as a strategy for PKU treatment

March 22, 2023
Phenylketonuria (PKU) is an autosomal recessive disorder where the primary catabolic pathway for phenylalanine (Phe) is disrupted due to mutations in the gene encoding PAH. Elevated Phe levels lie behind several neuropathologic anomalies that can lead to severe and irreversible mental retardation, if untreated.
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Gold dollar sign

Jnana closes $107M series C round, banks $50M up front in second discovery Roche pact

Nov. 15, 2022
By Cormac Sheridan
Momentum is building at Jnana Therapeutics Inc. The firm has raised $107 million in a series C round and banked another $50 million up front from a new drug discovery and development pact with Roche Holding AG, which could potentially deliver significant near-term milestones and more than $2 billion in future milestone payments. It also commenced recruitment onto a first-in-human study of its lead drug candidate, JNT-517, an inhibitor of the phenylalanine transporter SLC6A19, which is in development for phenylketonuria.
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FDA puts Biomarin gene therapy trial on hold, following expert discussion on safety

Sep. 7, 2021
By Richard Staines
The FDA has put a hold on a clinical study of a rare disease gene therapy BMN-307 from Biomarin Pharmaceutical Inc. after several mice developed liver tumors following a high dose in a preclinical trial.
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3D dollar sign

Jnana closes $50M series B round, picks PKU as lead indication

Aug. 18, 2021
By Cormac Sheridan
Jnana Therapeutics Inc. closed a $50 million series B round and took the covers off its lead program, a small-molecule inhibitor of the solute carrier transporter SLC6A19, which it is prepping for clinical trials in phenylketonuria (PKU).
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RNA

lncRNA HULC linked to phenylketonuria prevention, HULC-mimetics show therapeutic promise

Aug. 13, 2021
By W. Todd Penberthy
Sometimes highly impactful serendipitous discoveries are made when performing genetic loss-of-function studies that were initially focused on putative tumor suppressors or other hypotheses.
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Biotech deal illustration

PTC lands Censa PKU program with $10M up front, plus stock

May 7, 2020
By Michael Fitzhugh

Passed up for acquisition by former partner Retrophin Inc., Censa Pharmaceuticals Inc. has found a new home in rare disease specialist PTC Therapeutics Inc., which has agreed to pay $10 million up front for the opportunity to develop CNSA-001 (sepiapterin), a candidate for orphan metabolic diseases, starting with phenylketonuria (PKU). The proposed transaction also includes up to 850,000 shares of PTC common stock (NASDAQ:PTCT), valued around $40 million, plus additional rewards for achieving development, regulatory and commercial milestones. Shares of PTC closed at $46.91 on May 7, down $1.31.


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