ABCD1

DSMB has recommended dose-escalation First patient in cohort 2 has received SBT101 Majority investor Syncona providing new financing for further clinical development PHILADELPHIA--(BUSINESS WIRE)-- SwanBio Therapeutics, a gene therapy company developing AAV-based therapies for the treatment of devastating, inherited neurological conditions, today announced it has begun the second dose-escalation cohort of its first interventional clinical study, PROPEL. PROPEL is a Phase 1/2 first-in-human clinical trial assessing the safety and efficacy of SBT101, an investigational gene therapy designed to compensate for the ABCD1 mutation that causes adrenomyeloneuropathy (AMN). An independent Data Safety Monitoring Board (DSMB) recommended progressing the study to the higher-dose cohort following the review of the initial safety data readout in August. Patients in the first cohort received a single intrathecal administration of the lower of two planned doses and were assessed for treatment safety and tolerability. Dosing procedures were completed successfully. No serious adverse events related to the procedure, nor the gene therapy product, have been reported to date. “We’re proud to share that our lead candidate – the only gene therapy in clinical development for the treatment of AMN – has been successfully administered to and well-tolerated by men living with this destructive, degenerative disease,” said David Weiner, chief medical officer, SwanBio. “Today’s milestone of progressing to our second cohort takes us one step closer to our goal of bringing a safe and therapeutically meaningful drug to men and families impacted by this disease.” Lead investor and majority shareholder, Syncona Investment Management Limited, has made an additional $10 million commitment to fund the second cohort of PROPEL. “Following a review of the patient data from the first cohort, we are encouraged that the DSMB has recommended the initiation of the higher-dose cohort and we look forward to seeing the data generated from this cohort,” said Chris Hollowood, chief executive officer, Syncona. “Our further investment of $10M will enable the company to deliver additional data to deliver its long-term potential.” Currently active in the United States, PROPEL is expanding into Europe following the recent approval of its Clinical Trial Application (CTA) from the Central Committee on Research Involving Human Subjects (CCMO) in the Netherlands. SwanBio is already active in Europe with multiple clinical sites participating in CYGNET, a five-year natural history study of men with AMN, that completed enrollment in 2022. SwanBio’s PROPEL study leverages learnings from CYGNET (NCT05008874), which is piloting the use of wearable technologies to reduce onsite patient visits and is exploring more efficient ways to capture disease progression. Visit the SwanBio website for more information about PROPEL (NCT05394064), which is actively recruiting patients. About SBT101 SBT101 is the first clinical-stage adeno-associated virus (AAV)-based gene therapy candidate for people with adrenomyeloneuropathy (AMN). In preclinical studies, treatment with SBT101 demonstrated dose-dependent improvement of disease markers and functional improvement in AMN mouse models. SBT101 was also shown to be well-tolerated and persistent in non-human primates through twelve months post-treatment. The clinical program for SBT101 builds on this positive preclinical data, in addition to SwanBio’s deep understanding of the underlying pathophysiology of AMN and the AMN patient experience, including new insights being gathered in the ongoing, proprietary natural history study, CYGNET. SBT101 has been granted Fast Track and Orphan Drug Designation from the U.S. Food and Drug Administration and Orphan Drug Designation from the European Medicines Agency. About Adrenomyeloneuropathy Adrenomyeloneuropathy (AMN) is a progressive and debilitating neurodegenerative disease caused by mutations in the ABCD1 gene that disrupt the function of spinal cord cells and other tissues. AMN is characterized by loss of mobility in adulthood, incontinence, pain, and sexual dysfunction, which all affect quality of life. Patients often experience adrenal gland dysfunction as well. Between 8,000-10,000 men in the United States and EU5 (France, Germany, Italy, Spain, and the United Kingdom) are living with AMN. There are no approved therapies for the treatment of the disease; current standard of care is limited to symptom management. About SwanBio Therapeutics SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions. SwanBio’s initial focus is on gene therapies designed to be delivered intrathecally to address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications – spastic paraplegias, monogenic neuropathies, and polygenic neuropathies. SwanBio is supported by long-term, committed investment partners, including its primary investors Syncona, Ltd. and Mass General Brigham Ventures. For more information, visit SwanBioTx.com.

LYON, France--(BUSINESS WIRE)-- Regulatory News: POXEL SA (Euronext: POXEL - FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, today announced its participation at the ULF (United Leukodystrophy Foundation) Scientific Symposium and Family Conference on June 23rd and 24th in Itasca, Illinois, USA. Sophie Bozec, Poxel Senior Vice President, R&D Pharmacology and Scientific Communication will present Poxel’s status and plans for PXL770 and for the deuterium-modified TZD platform, using PXL065, two proprietary promising candidates, focused on the treatment of adrenoleukodystrophy (ALD), a rare and genetic disease in adults, based on robust scientific rationale and a complete preclinical package. Poxel remains committed to discover, develop, and commercialize innovative therapies for patients suffering from serious chronic and rare diseases with underlying metabolic pathophysiology, starting with ALD. PXL770 and PXL065 have demonstrated preclinically their efficacy in adrenomyeloneuropathy (AMN) and cerebral ALD (c-ALD) in mouse and human models1. These two compounds are prepared to advance, subject to additional financing, into Phase 2 biomarker proof-of-concept (POC) clinical trials in male patients with AMN, the most common ALD subtype. In line with his mission to bring therapeutic options to treat ALD, Poxel recently supported the Alex Leukodystrophy Charity during their Community Weekend, which took place from April 28th to May 1st in Birmingham, England. This event brings together leukodystrophy sufferers and their families, alongside doctors, researchers and scientists from around the world to discuss leukodystrophies. About ALD X-linked adrenoleukodystrophy (ALD) is an orphan neurometabolic disease caused by mutations in the ABCD1 gene which encodes for a key protein that is required for metabolism of very long chain fatty acids (VLCFA) by peroxisomes (cellular organelles). ALD is the most common leukodystrophy with a prevalence similar to hemophilia – up to 1/10,000 individuals in the general population have ALD [ ]. Forms of this disease include cerebral ALD (C-ALD) and adrenomyeloneuropathy (AMN) which is the most common form – typically occurring in adolescence through adulthood. AMN is characterized by chronic and progressive distal axonopathy involving the long tracts of the spinal cord and to a lesser extent the peripheral nerves resulting in progressive stiffness and weakness in the legs, impaired gait and balance, incontinence, and loss of sensation. Nearly all men with a diagnosis of ALD will develop AMN, and many women also present with features of AMN with a later onset. C-ALD is characterized by inflammatory demyelination of cells in the brain and typically afflicts children, but many men with AMN may also develop cerebral disease; these white matter brain lesions lead to severe neurologic deficits and death. There are no approved medicines for ALD (other than glucocorticoid supplements for associated adrenal insufficiency). C-ALD when first detected in early childhood, can be treated with hematopoietic stem cell transplantation. HSCT is currently limited to early stage of C-ALD and this procedure is at risk of severe adverse reactions. About Poxel SA Poxel is a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare disorders. For the treatment of NASH, PXL065 (deuterium-stabilized R-pioglitazone) met its primary endpoint in a streamlined Phase 2 trial (DESTINY-1). In rare diseases, development of PXL770, a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator, is focused on the treatment of adrenoleukodystrophy (ALD) and autosomal dominant polycystic kidney disease (ADPKD). TWYMEEG® (Imeglimin), Poxel’s first-in-class product that targets mitochondrial dysfunction, is marketed for the treatment of type 2 diabetes in Japan by Sumitomo Pharma and Poxel expects to receive royalties and sales-based payments. Poxel has a strategic partnership with Sumitomo Pharma for Imeglimin in Japan, China, and eleven other Asian countries. Listed on Euronext Paris, Poxel is headquartered in Lyon, France, and has subsidiaries in Boston, MA, and Tokyo, Japan. For more information, please visit: All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by or including words such as “target,” “believe,” “expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements. The Company does not endorse or is not otherwise responsible for the content of external hyperlinks referred to in this press release. 1 Preclinical study results were published in prestigious journals: In The Journal of Inherited Metabolic Disease (“JIMD”): “Therapeutic potential of deuterium-stabilized (R)-pioglitazone - PXL065 - for X-linked adrenoleukodystrophy”. In The Journal of Pharmacology and Experimental Therapeutics (“JPET”): “Beneficial effects of the direct AMP-Kinase activator PXL770 in in vitro and in vivo models of X-Linked Adrenoleukodystrophy”. View source version on businesswire.com: Contacts Investor relations / Media Aurélie Bozza Investor Relations & Communication Senior Director aurelie.bozza@poxelpharma.com +33 6 99 81 08 36 Elizabeth Woo Senior Vice President, Investor Relations & Communication elizabeth.woo@poxelpharma.com NewCap Emmanuel Huynh or Arthur Rouillé poxel@newcap.eu +33 1 44 71 94 94 Source: Poxel View this news release online at:

PHILADELPHIA--(BUSINESS WIRE)-- SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, presented preliminary data from primate studies of AAV capsids that outperform the commonly used AAV9 capsid. The findings illuminate SwanBio’s vector engineering capabilities and ongoing pursuit of novel capsids that deliver enhanced transgene expression, biodistribution, and safety pro “Our vector engineering platform, along with our proprietary insights into delivery and biodistribution, and expertise in cassette design, form the three pillars of how we approach gene therapy,” said Karen Kozarsky, co-founder and chief scientific officer, SwanBio. “This research, conducted with our partners at the Mass General Research Institute’s Maguire Lab, indicates the potential of our innovative approach to capsid optimization. We are excited by the potential to incorporate these next-gen capsids into our pipeline products and future portfolio.” Key findings presented at the 2023 ASGCT Annual Meeting include: Screening of a capsid library delivered intrathecally in primates resulted in the identification of a pool of 13 new capsid candidates, which displayed enhanced biodistribution compared to AAV9 at all levels of the spinal cord, ranging from 2- to 265-fold. Four of seven candidate capsids demonstrated enhanced transgene expression in the spinal cord (up to 2.4-fold). Several capsids displayed significant reduction in off-target distribution, including lower leakage to the liver (up to 1,250-fold) compared to AAV9. Additional research in mice will evaluate transduction performance of the most promising capsids following intrathecal delivery, for application in new disease models. SwanBio is developing a pipeline of gene therapy candidates designed to be delivered intrathecally to targets in both the central and peripheral nervous systems, with an initial focus on spinal cord-related disorders. The company’s lead candidate, SBT101, is currently being investigated for the treatment of adrenomyeloneuropathy (AMN) in a Phase 1/2 clinical trial, PROPEL. SwanBio is also presenting a poster at ASGCT showcasing a recent healthcare resource use study of men and women living with AMN. The research indicates that AMN imposes a substantial and previously under-recognized health burden, pointing to the value of developing targeted genetic therapies. To view SwanBio’s final 2023 ASGCT posters, visit swanbiotx.com/investors-and-media/events-and-presentations/. About Adrenomyeloneuropathy Adrenomyeloneuropathy (AMN) is a progressive and debilitating neurodegenerative disease caused by mutations in the ABCD1 gene that disrupt the function of spinal cord cells and other tissues. AMN is characterized by loss of mobility in adulthood, incontinence, pain, and sexual dysfunction, which all affect quality of life. Patients often experience adrenal gland dysfunction as well. Between 8,000-10,000 men in the United States and EU5 (France, Germany, Italy, Spain, and the United Kingdom) are living with AMN. There are no approved therapies for the treatment of the disease; current standard of care is limited to symptom management. About SBT101 SBT101 is the first clinical-stage adeno-associated virus (AAV)-based gene therapy candidate for people with adrenomyeloneuropathy (AMN). In preclinical studies, treatment with SBT101 demonstrated dose-dependent improvement of disease markers and functional improvement in AMN mouse models. SBT101 was also shown to be well-tolerated in non-human primates through six months post-treatment. The ongoing clinical program for SBT101 builds on this positive preclinical data, plus SwanBio’s deep understanding of the underlying pathophysiology of AMN and the AMN patient experience, including new insights being gathered in a proprietary natural history study, CYGNET. SBT101 has been granted Fast Track and Orphan Drug Designation from the U.S. Food and Drug Administration and Orphan Drug Designation from the European Medicines Agency. About SwanBio Therapeutics SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions. SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications – spastic paraplegias, monogenic neuropathies, and polygenic neuropathies. SwanBio’s lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). SwanBio is supported by long-term, committed investment partners, including its primary investors Syncona, Ltd. (lead investor and majority shareholder) and Mass General Brigham Ventures. For more information, visit SwanBioTx.com. View source version on businesswire.com: Contacts Media: Lara Furst +1-703-946-0183 media@swanbiotx.com Investor: Jan Case investors@swanbiotx.com Source: SwanBio Therapeutics View this news release online at: