Participated · Series C · Jul 2021

Amylyx Pharmaceuticals is a Cambridge, Mass. pharmaceutical company developing novel therapeutics for ALS, Alzheimer’s disease and other neurodegenerative diseases. Its lead product candidate, AMX0035, is a co‑formulation of sodium phenylbutyrate (PB) and taurursodiol (TURSO/ursodoxicoltaurine) designed to reduce neuronal death by targeting endoplasmic reticulum and mitochondrial‑dependent degeneration pathways. Amylyx has published data on AMX0035 in the New England Journal of Medicine and Muscle and Nerve. The company filed a New Drug Submission (NDS) for AMX0035 with Health Canada in June and intends to submit a Marketing Authorization Application (MAA) to the EMA’s CHMP by the end of 2021. Amylyx plans to initiate a Phase 3 clinical trial and will continue to update the global ALS community, including plans involving the US FDA. The company closed an oversubscribed Series C to support late‑stage clinical development, potential launch planning for AMX0035, and further R&D in other neurodegenerative diseases. Amylyx Pharmaceuticals, based in Cambridge, Mass., is developing AMX0035, a first-in-class investigational therapy designed to reduce neuronal death by targeting mitochondrial and endoplasmic reticulum–dependent degeneration pathways. The company completed a Phase II ALS trial (CENTAUR) and has completed enrollment in a second Phase II Alzheimer’s trial (PEGASUS). CENTAUR was a 24-week, randomized, double-blind, placebo-controlled study assessing safety, tolerability and disease progression using ALSFRS-R and other measures; full CENTAUR data are anticipated for publication in the near term. PEGASUS is a biomarker-focused 24-week study in late mild cognitive impairment or early dementia due to Alzheimer’s, with topline data expected in the first quarter of 2021. Financially, Amylyx has closed an oversubscribed $30 million Series B financing led by Morningside Ventures to support ongoing clinical development. Company leadership states the proceeds will help the firm continue to advance trials and press ahead as rapidly as possible for patients with ALS, Alzheimer’s and other neurological conditions. Amylyx Pharmaceuticals is developing AMX0035, a proprietary fixed‑dose combination of sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA) for treatment of ALS. AMX0035 is designed to target neuroinflammation and nerve‑cell death; preclinical models showed synergistic activity and the individual agents have prior clinical testing showing safety, tolerability and preliminary signs of efficacy. The company plans to begin a Phase II clinical trial to evaluate safety, tolerability, functional outcomes, biomarkers of cell function/neuronal damage and inflammation, and a new objective measure of muscle strength. The IND for the Phase II trial is on schedule for the fourth quarter of 2016 and the trial is expected to start shortly thereafter, with over 20 U.S. clinical sites already expressing interest. Financially, Amylyx completed a $5.0 million Series A and received a $2.96 million grant for the trial; the company reports $10 million in combined grant funding and private financing to advance AMX0035. Investor board members have joined the company to support clinical development as Amylyx advances its candidate in a disease area with significant unmet need. Amylyx Pharmaceuticals is developing AMX0035, a combination of two existing small-molecule drugs intended to work in tandem to address nerve cell death and inflammation in neurodegenerative diseases. AMX0035 is designed to block stress on mitochondria and the endoplasmic reticulum, processes the company says mediate nerve cell death and neuroinflammation. The company has completed only preclinical testing to date and reports efficacy in disease-specific models of ALS, Alzheimer's and mitochondrial disease. Amylyx plans to launch clinical trials for ALS next year and will use the new funding to support IND-enabling studies for its lead candidate. According to an SEC filing it raised $1.3 million in a new equity round and additionally received $600,000 in grants from the ALS Finding a Cure Foundation and the Cure Alzheimer's Fund, totaling $1.9 million. Founded in 2013 and based in Cambridge, this is Amylyx's first round of equity financing; its scientific advisory board is chaired by Dr. Rudolph Tanzi.

Participated · Series B · Mar 2020

Akouos develops precision gene therapies for sensorineural hearing loss using an adeno-associated viral (AAV) vector-based platform. Its lead program, AK-OTOF, targets hearing loss caused by mutations in the otoferlin (OTOF) gene. The company plans to advance AK-OTOF to first-in-human clinical studies subject to IND filing and acceptance. Proceeds from its recent financing will also support establishing in-house GMP manufacturing at new offices in the Boston Seaport and accelerating multiple pipeline programs with well-defined mechanisms and gene targets. Akouos intends to expand its teams across research, clinical development, and manufacturing. The company has strategic partnerships with Massachusetts Eye and Ear and Lonza, Inc. Akouos is a precision genetic medicine company focused on developing targeted adeno-associated viral (AAV)-based gene therapies for sensorineural hearing loss, with an initial emphasis on monogenic forms that cause profound deafness. The company leverages a proprietary AAV gene therapy platform and has strategic partnerships with Massachusetts Eye and Ear and Lonza. Proceeds from a recent $50 million Series A will support advancement of its lead program to first-in-human clinical studies and accelerate multiple pipeline programs addressing other validated gene targets. Akouos has assembled clinical and scientific leadership, expanded its board, and formed a scientific advisory board composed of world-leading experts in gene therapy and hearing loss. The company states its approach aims to deliver one-time precision therapies to treat underlying genetic causes of hearing disorders. Akouos develops gene therapies aimed at restoring and preserving hearing and balance using in silico-designed Anc-AAV viral vectors. The company entered strategic license agreements with Lonza and Massachusetts Eye and Ear to obtain exclusive rights to the Anc-AAV gene therapy platform and broad sublicensing rights. Anc-AAVs were first developed in the laboratory of Dr. Luk H. Vandenberghe and are intended for application across hearing and balance disorders. Akouos was launched in 2017 by Dr. Manny Simons, who serves as CEO; its scientific founders and advisors include Drs. Michael McKenna, William Sewell, Richard Smith and Luk Vandenberghe. In conjunction with closing a $7.5M seed financing, the licensing and partnership framework is designed to accelerate AAV gene therapy development for hearing and balance conditions. Dr. Vandenberghe is a listed inventor on Anc-AAV intellectual property licensed to Lonza and Akouos and receives royalties.

Participated · Series C · Jul 2019

Frequency Therapeutics is a clinical-stage biotech harnessing its Progenitor Cell Activation (PCA) platform to activate progenitor cells in situ and regenerate functional tissue. Its lead program, FX-322, is a potential first-in-class regenerative therapeutic targeting hearing restoration and is moving into a Phase 2a study for sensorineural hearing loss. The company plans to use proceeds from its recent financing to support FX-322’s clinical development and to advance discovery programs in other therapeutic areas including muscle, gastrointestinal, skin, and bone. Frequency recently announced a collaboration with Astellas that included an $80 million upfront payment and up to $545 million in potential milestone payments to support global development and ex‑US commercialization of FX-322. The company emphasizes reducing regenerative‑medicine complexity by leveraging innate biology with small molecules rather than cell or gene therapies. Frequency is based in Woburn, Massachusetts and is positioning its PCA platform as a source of new treatments across multiple degenerative-disease indications. Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Mass., developing a new class of small-molecule drugs that stimulate dormant progenitor cells (Progenitor Cell Activation, PCA) to repair diseased or damaged tissue. Its lead candidate, FX-322, targets sensorineural hearing loss and is being advanced for hearing regeneration. The company planned to move FX-322 into Phase 2 studies in 2019 and expected top-line Phase 1/2 results in the first half of 2019. Frequency highlights that its PCA approach enables native tissue regeneration without genetic modification or removal of cells, avoiding risks of genetic engineering and manufacturing challenges of cell therapy. Proceeds from the financing will support advancement of FX-322 and continued expansion of the PCA Regeneration pipeline into new therapeutic applications. Frequency has raised a total of $87 million to date. Frequency Therapeutics develops small‑molecule drugs that transiently activate progenitor cells using its proprietary Progenitor Cell Activation (PCA™) platform; its lead program targets chronic noise‑induced hearing loss by stimulating regrowth of inner ear sensory hair cells. The company holds a worldwide exclusive license to related intellectual property from MIT and Partners Healthcare and cites foundational work by co‑founders Robert Langer and Jeffrey Karp. Frequency was founded in 2015 and lists David Lucchino as co‑founder, president and CEO. It plans to move its lead program into the clinic in the next 12–18 months and is positioning PCA for additional indications including skin disorders, muscle regeneration and gastrointestinal diseases. Financially, Frequency closed a $32 million Series A to support clinical development, with participation from U.S. and international investors.