Shionogi to Acquire IntraBio for $2B to Expand Rare Disease Portfolio

Shots: Shionogi announced a planned acquisition of IntraBio for $2.0B, adding AQNEURSA (levacetylleucine) and IntraBio’s rare disease capabilities to its portfolio; the transaction is expected to close between November and December 2026, subject to customary conditions and regulatory approvals AQNEURSA is approved in the US and EU for neurological manifestations of Niemann-Pick disease Type C…

ByPrince GiriOctober 6, 2026

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IntraBio Reports the US FDA Approval of Aqneursa for Ataxia-Telangiectasia

Shots: The US FDA has approved Aqneursa (levacetylleucine) for the treatment of ataxia in adult & pediatric pts (≥15kg) with ataxia-telangiectasia (A-T) Approval was backed by the P-III (IB1001-303) trial assessing Aqneursa vs PBO in 73 pts (4-50yrs.) with genetically confirmed A-T, which met its 1 & key 2EPs & showed that Aqneursa has a…

ByRidhi RastogiSeptember 21, 2026

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IntraBio Reports the US FDA’s sNDA Submission of Aqneursa for Ataxia-Telangiectasia

Shots: The US FDA has received sNDA of Aqneursa (levacetylleucine) for the treatment of Ataxia-Telangiectasia (A-T) sNDA was backed by the P-III trial assessing Aqneursa in adult and pediatric pts with A-T, which met its 1 & key 2EPs & showed that Aqneursa has a favorable safety profile Aqneursa was previously approved by both the…

ByRidhi RastogiMarch 23, 2026

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IntraBio Reports Topline P-III (IB1001-303) Trial Data on Aqneursa for Ataxia-Telangiectasia

Shots: IntraBio has reported the topline P-III (IB1001-303) trial data on Aqneursa (levacetylleucine) vs PBO in pediatric & adult pts with Ataxia-Telangiectasia (A-T) Trial met the 1EP with a significant –1.88 SARA improvement vs PBO (–1.92 vs –0.14) after 12wks.& achieved 2EPs, showing meaningful gains on ICARS (–4.22 vs –1.69) & CGI-I (–0.6 vs –0.2)…

ByRidhi RastogiJanuary 22, 2026

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Artios’ Alnodesertib Obtains the US FDA’s Fast Track Designation for ATM-negative Metastatic Colorectal Cancer

Shots: The US FDA has granted FTD to alnodesertib + low-dose irinotecan as the 3L treatment of adults with ATM-negative metastatic colorectal cancer (mCRC) Designation was based on the P-I/II (STELLA) trial (ongoing in the US) assessing the regimen in solid tumors with ATM deficiency, which showed responses in 3L mCRC & 7 additional solid…

ByRidhi RastogiSeptember 25, 2025

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Quince Therapeutics Completes Patient Enrolment in P-III (NEAT) Trial of eDSP for Ataxia-Telangiectasia

Shots: Quince Therapeutics has completed enrolment in its P-III (NEAT) trial to assess eDSP (6 infusions once every 21–30 days) vs PBO in Ataxia-Telangiectasia pts (n=105), incl. 83 pts (6-9yrs.) in primary analysis population & 22 pts aged ≥10yrs. Trial will assess change from baseline to the last efficacy visit using RmICARS as the 1EP; pts…

ByRidhi RastogiJuly 17, 2025

Shionogi to Acquire IntraBio for $2B to Expand Rare Disease Portfolio

IntraBio Reports the US FDA Approval of Aqneursa for Ataxia-Telangiectasia

IntraBio Reports the US FDA’s sNDA Submission of Aqneursa for Ataxia-Telangiectasia

IntraBio Reports Topline P-III (IB1001-303) Trial Data on Aqneursa for Ataxia-Telangiectasia

Artios’ Alnodesertib Obtains the US FDA’s Fast Track Designation for ATM-negative Metastatic Colorectal Cancer

Quince Therapeutics Completes Patient Enrolment in P-III (NEAT) Trial of eDSP for Ataxia-Telangiectasia

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