While RNA-medicine developer Wave Life Sciences Ltd. brought in a clinical data win, it also got knocked back a step as a major collaborator will go its separate way. The company’s ongoing phase Ib/IIa study of its A-to-I RNA editing oligonucleotide produced positive proof-of-mechanism data in treating alpha-1 antitrypsin deficiency, a genetic condition that can lead to lung and liver disease. However, in a separate development, Takeda Pharmaceutical Co. Ltd. said it will terminate its option for the Huntingtin target under the companies’ February 2018 collaboration and license agreement. Since then, Wave has received about $260 million from Takeda. Wave’s stock (NASDAQ:WVE) wasn’t hampered at midday as shares were up 75% to $14.98 each.
US FDA Rare Disease Hub an answer to regulatory challenges?
The U.S. FDA is getting an earful of advice to help it set up a new Rare Disease Innovation Hub that’s intended to bring more certainty to the development of rare disease treatments through enhanced communication and collaboration among the patient community, companies working in the rare disease space, and the agency’s drug and biological products centers. One piece of advice offered at today’s public meeting is that the hub also should fully integrate the Center for Device and Radiological Health into the collaboration. Other comments focused on the need for a new understanding of regulatory flexibility across the agency and the evidence required for approval of a new rare disease treatment.
Synthesis Bioventures founder wins Aussie innovation award
When Andrew Wilks invented the JAK inhibitor momelotinib in the late 1980s for myelofibrosis, he never would have imagined it would take more than 20 years to develop and eventually be acquired for $1.9 billion. Today he’s on a mission to ensure Australian inventors have more options than he did, telling BioWorld that he had to sell the molecule for around $10 million because he couldn’t get funding. Momelotinib, branded as Ojjaara, gained U.S. FDA approval in September 2023 for use in intermediate- or high-risk myelofibrosis patients with anemia regardless of prior administration with JAK inhibitors. Wilks is now leading venture fund Synthesis Bioventures and was honored this year with the Australian Prime Minister's Prize for Innovation in Science for inventing and commercializing momelotinib with co-inventor Chris Burns.
Biopharma financing drops in Q3, outpaces full-year 2023 and 2022
Biopharma companies secured $16.31 billion in financing during the third quarter (Q3) of 2024, marking a 29% decrease from the $23.07 billion raised in Q2 and a 65% drop from Q1’s $47.25 billion. Despite the quarterly decline, year-to-date financings through September reached $86.63 billion, surpassing the total annual figures of both 2023 ($70.97 billion) and 2022 ($60.81 billion). Only the record-setting years of 2020 and 2021 saw higher amounts raised.
Alternative splicing study reveals genetic variants across Indonesian archipelago
A new study helps explain the role of genetic variation in shaping gene regulation in the Indonesian archipelago, one of the most diverse regions in the world. “This study is the only study of splicing from Southeast Asian populations. There is basically no data from this part of the world,” study author Irene Gallego Romero told BioWorld. For drug discovery, most of the people that have historically participated in clinical trials are of European ancestry, and scientists are just beginning to study African populations to better understand genetic differences in these populations, said Romero, a population geneticist and biological anthropologist at the University of Melbourne.
AI drug developer Aigen Sciences raises ₩12B series A
Backed by AI technology, Aigen Sciences Inc. raised ₩12 billion (US$8.8 million) in a series A financing round to further advance its cancer and rare disease drug pipelines. Aigen said Oct. 16 that the series A round was joined by existing investors Partners Investment, Quad Investment Management and Medytox Venture Investment, as well new investors Premier Partners, K2 Investment Partners and Scale Up Partners. The latest financing adds to Seoul, South Korea-based Aigen’s pre-series A financing round that raised ₩4.2 billion in October 2022.