Portfolio

ALNARA Pharmaceuticals

Improving nutritional status and pill burden in patients with pancreatic diseases

Alnara Pharmaceuticals, Inc. (Alnara) is dedicated to developing and commercializing novel protein therapeutics for the treatment of metabolic diseases. The company’s innovative approach focuses on designing effective protein therapies that can be orally delivered directly to the gastrointestinal tract without being absorbed into the bloodstream. Alnara’s lead product is liprotamase, a novel, non-porcine pancreatic enzyme replacement therapy, which has completed Phase 3 clinical development in collaboration with the Cystic Fibrosis Foundation Therapeutics, Inc. Alnara was co-founded in 2008 by Alexey Margolin, Rich Aldrich and Christoph Westphal and sold to Eli Lilly in 2010 for $180M upfront.

Verastem

Novel drugs targeting cancer stem cells

Verastem, Inc. (Verastem) (NASDAQ: VSTM) is discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, an underlying cause of tumor recurrence and metastasis. Verastem is developing small molecule inhibitors of signaling pathways that are critical to cancer stem cell survival and proliferation including focal adhesion kinase (FAK) and PI3K/mTOR. For more information, please visit the company's website at www.verastem.com.

Ovascience

Improving fertility through science

OvaScience, Inc. (OvaScience) (NASDAQ: OVAS) is a global life sciences company dedicated to improving fertility for women around the world. OvaScience is discovering, developing and commercializing new fertility treatments. Each OvaScience treatment is based on the company's proprietary technology platform that leverages the breakthrough discovery of egg precursor (EggPCSM) cells – immature egg cells found inside the protective ovarian lining. For more information, please visit the company's website atwww.ovascience.com.

IlluminOss Medical

minimally invasive solutions for the treatment of bone fractures

IlluminOss Medical (IlluminOss) is a privately-held, commercial stage medical device company focused on designing, developing and marketing orthopedic fracture repair products that leverage its proprietary bone stabilization technology. This minimally invasive technology allows for fracture fixation using a light-curable polymer, contained within an expandable balloon catheter, to achieve bone stabilization. The company currently markets and sells its products internationally under CE Mark approval for clinical applications. For more information, please visit the company’s website at www.illuminoss.com.

PTC Therapeutics

Focused on the discovery, development and commercialization of proprietary small-molecule drugs

PTC Therapeutics, Inc. (PTC) (NASDAQ: PTCT) a global biopharmaceutical company focused on the discovery, development and commercialization of orally administered, proprietary small molecule drugs targeting an area of RNA biology we refer to as post-transcriptional control. Post-transcriptional control processes are the regulatory events that occur in cells during and after a messenger RNA is copied from DNA through the transcription process. PTC has received conditional marketing authorization in the European Economic Area for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. PTC's internally discovered pipeline addresses multiple therapeutic areas, including rare disorders, oncology and infectious diseases. PTC has discovered all of its compounds currently under development using its proprietary technologies. PTC plans to continue to develop these compounds both on its own and through selective collaboration arrangements with leading pharmaceutical and biotechnology companies. For more information, please visit the company’s website www.ptcbio.com.

Mitobridge

developing drugs that can treat a wide range of serious diseases by modulating mitochondrial function

Mitobridge, Inc. (Mitobridge), formerly known as Mitokyne, is a biopharmaceutical company focused on mitochondria-related drug discovery and development. The company is utilizing its innovative screening platform to identify and develop novel therapeutics that improve mitochondria function and provide treatments for mitochondrial genetic diseases, musculoskeletal and neurodegenerative disorders as well as diseases and conditions of aging. Mitobridge’s scientific founders and advisors include world-renowned leaders in biology and mitochondrial function. For more information about Mitobridge, please visit the company's website at www.mitobridge.com.

Calithera Biosciences

Developing oncology therapeutics that inhibit pathways critical to tumor growth and survival

Calithera Biosciences, Inc. (NASDAQ: CALA) (Calithera) is a clinical-stage pharmaceutical company focused on discovering and developing novel small molecule drugs directed against tumor metabolism and tumor immunology targets for the treatment of cancer. Calithera's lead product candidate, CB-839, an orally bioavailable inhibitor of glutaminase, is currently being evaluated in three Phase 1 clinical trials in solid and hematological cancers. For more information, please visit the company’s website www.calithera.com.

Flex Pharma

Novel Treatments for Muscle Cramps and Spasms

Flex Pharma, Inc. (NASDAQ: FLKS) is a biotechnology company developing innovative and proprietary treatments for nocturnal leg cramps and spasms associated with severe neuromuscular conditions. In three randomized, blinded, placebo-controlled, cross-over studies, Flex Pharma's proprietary treatment has shown a statistically significant reduction in the intensity of muscle cramps in healthy normal volunteers. Flex Pharma was founded with National Academy of Science members Rod MacKinnon, M.D. (2003 Nobel Laureate), and Bruce Bean, Ph.D., recognized leaders in the fields of ion channels and neurobiology, both serve as board members for the company. For more information, please visit the company's website http://www.flex-pharma.com.

Colorescience

Scientifically based, proprietary products that treat sun damaged skin & protect from the damaging effects of the sun, environment and aging

Colorescience is a skincare company that develops and markets scientifically based, proprietary products that treat sun damaged skin, as well as protecting the skin from the damaging effects of the sun, the environment and aging. The products include high-quality, dermatologist-recommended formulations with high SPF. The Colorescience team is led by Mary Fisher who brings more than 25 years of experience in the pharmaceutical and biotechnology industries. Ms. Fisher currently serves as Chief Executive Officer and Director at Colorescience, a former division of SkinMedica, Inc., where she served as Chief Executive Officer from 2008 until 2012, when she led the successful sale of the company to Allergan, Inc. for $375 million. For more information, please visit the company's website http://www.colorescience.com.

KalVista Pharmaceuticals

OPHTHALMOLOGY COMPANY FOCUSED ON ON DIABETIC MACULAR EDEMA AND DISCOVERY & DEVELOPMENT OF SERINE PROTEASE INHIBITORS

KalVista Pharmaceuticals (KalVista) is an ophthalmology company with a primary focus on Diabetic Macular Edema (DME) and deep expertise in the discovery and development of serine protease inhibitors.  KalVista's novel plasma kallikrein inhibitors represent a new approach to the treatment of DME, a leading cause of adult visual loss in developed countries.  Although VEGF inhibitors (existing treatment) can benefit DME, a significant number of patients do not respond fully to these agents and have limited treatment options.  Plasma kallikrein inhibitors target a distinct molecular pathway and have the potential to offer those patients an effective treatment option.  KalVista has a clinical and advanced pre-clinical product pipeline targeting both intravitreal injection and oral administration, and its plasma kallikrein inhibitors can be applied to additional indications.  KalVista's founders include world experts in diabetic retinopathy, Dr. Lloyd Paul Aiello, Professor of Ophthalmology at Harvard Medical School and Director of the Joslin's Beetham Eye Institute, and Dr. Edward P. Feener, Associate Professor of Medicine at Harvard Medical School and Joslin Diabetes Center.  For more information, please visit the company's website: http://www.kalvista.com/.

Channel Medsystems

Endometrial ablation device for women

Channel Medsystems was founded to bring innovation to the delivery of women's healthcare. Channel Medsystems is committed to ensuring patients have access to high quality outcomes that can be delivered in the convenience and comfort of their healthcare provider's office. Our novel cryothermic technology and streamlined delivery system's first application focuses on global endometrial ablation for women with heavy menstrual bleeding. The cryothermic platform at the core of this technology has the potential for multiple future applications. For more information, please visit the company's website: www.channelmedsystems.com.

Companies Founded by Members of the Longwood Team

Ovascience Verastem ALNARA Pharmaceuticals Flex Pharma
Sirtris Pharmaceuticals Momenta CoNCERT Pharmaceuticals Alnylam Pharmaceuticals Acceleron