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This branch of our Sponsor Atlas series focuses on young pharmaceutical and biotechnology companies that are in early stages or stealth mode. In the next edition of Sponsor Atlas: Discovering Biotech Startups, we’re looking at Praxis Precision Medicines, exploring the current affairs and future orientation of this emerging Cambridge - based stealth biotech company. To accomplish this, we will give a business overview of their current operations, summarize their outsourcing needs, map out their development goals and decision-makers, and highlight their current strategies for capturing innovation. If you haven’t already read our other blogs on new biotech startups, be sure to check them out here.
Praxis Precision Medicines, Inc., formerly known as EpiPM Therapeutics, Inc. is a stealth biotech startup focused on developing novel therapies to treat neurological, psychiatric, and movement disorders.
Praxis has a website domain registered, but when accessed, it just redirects to their Contact Us page. There also isn’t much online information about their company, so we had to do some old-fashioned research and gather clues from various sources for details about their research and potential products. Praxis’ principal place of business is located at One Broadway, 16th Floor, Cambridge, MA 02142, a large building also known as the Cambridge Innovation Center, which houses hundreds of other innovative startups, including bigger companies like HubSpot, Boehringer Ingelheim, and Instacart to name a few. Praxis Precision Medicines was registered in Delaware as a C-Corp with a filing date on 12/1/2016, and it was originally incorporated on 9/22/2015, with a registered agent address through CT Corporation System in Boston. Their business name was officially changed from EpiPm Therapeutics, Inc. on 10/26/2016. They also have a decent employee count with a number ranging anywhere from 21 - 30 on staff.
Praxis Precision Medicines raised a total of $21.9M in funding over the previous 2 rounds, with their latest Series C round of $10M on 6/27/2019 from 3 investors. President and CEO of Praxis, Dr. Kiran Reddy, played a key role in securing the recent investment and it will aid in aggressively expanding the company, as well as to broaden and accelerate product development. 
Praxis Precision Medicines doesn’t have a lot of information on their current outsourcing needs, but considering their recent rounds of venture financing, they are in a great position to fund a ton of outsourcing work. The company is still early in development but they do have a few lead candidates already in clinical trials, with more on that in the next section. Their prospective pipeline will consist of therapies for treating multiple types of neurodegenerative diseases. Any experience with these types of diseases and the technologies around it would be of tremendous benefit to Praxis at this point in time.
Some of the ways outsourcing partners can get their foot in the door would be to provide support in the following general areas: large molecule CMOs, gene therapy and genetic engineering CROs, preclinical CROs, bioanalytical service providers, and clinical support services.
Praxis’ Pipeline and R&D Focus
Praxis Precision Medicines is a company inspired by breakthroughs in the genetics of epilepsy and overlapping biology with broader neuropsychiatric disorders. They are currently developing medicines in the innovative space of neurology genetics, specifically with CNS AAV gene therapy from targeting specific genetic epilepsies caused by mutations in sodium and potassium channels, and a precision medicine approach to treating other neurological disorders. Praxis recently established their Asia Pacific headquarters and research and development center in Melbourne, Australia, where they’re already conducting several clinical trials. They’re focusing on two lead candidates, namely PRAX-330 for treating epilepsy, and PRAX-944 for treating generalized epileptic syndromes with absence seizures, and essential tremor conditions. Recruitment is complete for the most recent studies and clinical trials are currently underway in Australia, with a few already having been completed.
PRAX-330 is currently progressing through a Phase I First-In-Human studies to assess its safety. In a recent abstract, PRAX-330 was shown to reduce persistent and resurgent sodium channel currents and neuronal hyperexcitability of subiculum neurons in a mouse model of SCN8A epileptic encephalopathy.  The second lead candidate, PRAX-944, is an oral tablet dosage form and is currently in a Phase II open-label study (Australia) for assessing safety and tolerability of ascending doses.
We think business development teams in the realms of large molecule CMOs, engineering support of medical devices for local and systemic drug delivery research, preclinical CROs, and bioanalytical service providers with experience in research of gene therapy targets for CNS diseases should definitely have Praxis Precision Medicines on their radar over the next few years.
- Dr. Kiran Reddy - is CEO of Praxis Precision Medicines and Venture Partner at Clarus Ventures. Prior to Clarus, Kiran was part of Biogen’s Corporate Strategy leadership where he focused on sourcing new technologies and product opportunities via acquisitions, partnerships, and equity investments. Prior to Biogen, Kiran was a Partner at Third Rock Ventures, where he supported and managed various portfolio companies and new investments. He was also part of the founding team and interim Chief Business Officer for SAGE Therapeutics through its IPO, and co-inventor of SAGE-54, which is in Phase 3 studies for the rare epilepsy disorder refractory status epilepticus. Kiran was part of the team that launched Foundation Medicine, as well as serving on the board for Alnara Pharmaceuticals, Rhythm Pharmaceuticals, and PanOptica Pharmaceuticals. Kiran holds MD and MBA degrees from Georgetown University, and completed his internship in medicine and his neurology residency at Harvard/Massachusetts General Hospital and is a board-certified neurologist. Kiran was previously a Howard Hughes science fellow and has authored several peer-reviewed scientific papers in the field of neuroimmunology and neurodegenerative diseases. 
- Dr. Nicholas Galakatos - is Director at Praxis and also Head of the Blackstone Life Sciences business arm. Prior to joining Blackstone, Dr. Galakatos was a co-Founder and Managing Director of Clarus since 2005. Nicholas has over 30 years of industry and investment experience in the healthcare sector and has led investments in biotechnology, pharmaceutical company partnerships, and diagnostics. Prior to Clarus, Dr. Galakatos was a General Partner at MPM Capital, a healthcare venture capital firm. Dr. Galakatos earned his Ph.D. in Organic Chemistry from MIT and performed his post-doctoral studies at Harvard Medical School. He earned his undergraduate degree at Reed College. 
- Alfred Sandrock, Jr., M.D. - Board Member at Praxis is also the Executive Vice President and Chief Medical Officer at Biogen and has served on the Executive Committee since 2015. He was named Chief Medical Officer in 2012 and since joining the company in 1998, he has held several senior executive positions, including SVP of Development Sciences, SVP of Neurology Research and Development, and VP of Clinical Development, Neurology. Dr. Sandrock received his B.A. in human biology from Stanford University, an M.D. from Harvard Medical School, and a Ph.D. in neurobiology from Harvard University. He completed an internship in medicine, a residency and chief residency in neurology, and a clinical fellowship in Neuromuscular Disease and Clinical Neurophysiology (electromyography) at Massachusetts General Hospital. 
- William Young - Mr. Young represents Clarus Ventures on the Board of Directors of Praxis Precision Medicine and is the Chairman of the Board of Annexon Biosciences and Nanostring. Mr. Young received his bachelor's degree in chemical engineering from Purdue University, and an MBA from Indiana University, as well as an honorary doctorate in engineering from Purdue University. In 1993 he was elected to the National Academy of Engineering for his leadership in research and manufacturing of recombinant proteins using recombinant DNA technology.
- Stuart Chaffee, Ph.D., M.B.A. - Executive officer at Praxis, is also an is an EIR at Atlas Venture, where he recently co-founded an early-stage therapeutics company focused on targeted protein degradation. He joined Atlas from Biogen, where he spent five years in a number of different roles including Finance, Business Development, Program Management, and Corporate Strategy. Prior to Biogen, Stuart was at Leerink Partners, a healthcare-focused investment bank, and was part of the founding team at Zafgen, a now publicly-traded biotech company developing therapies for obesity. Originally trained as a synthetic organic chemist, Stuart started his career as a medicinal chemist at Amgen where he led a number of drug discovery and development efforts in oncology and neurodegenerative disease. Stuart holds a B.S. from the College of William and Mary, a Ph.D. from Yale University, and an M.B.A. from the Wharton School at the University of Pennsylvania. 
With a substantial amount of investment backing, considerable progress in scientific benchwork, and deep technical knowledge from their executives, we believe that Praxis Precision Medicines will do very well in their early-stage developments and clinical trial studies. Praxis’ innovative gene therapy approach to using precision medicine in genetically defined neurodegenerative disorders will surely create some promising new treatments in the future. With their number of disease-targeting programs in development using these new technologies, Praxis is going to make some unprecedented discoveries in the epilepsy field and beyond.
While it’s still fairly difficult to determine what Praxis Precision Medicines will have to offer during stealth mode, their amount of venture funding is massive, and with the backgrounds of the executive team, we think they are on track to create some major breakthroughs in the biotech industry over the next few years. Be sure to keep Praxis on your contact list and look out for our next edition in this series coming soon.
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