FierceBiotech Unveils 2016's 15 Most Promising Biopharma Companies Amid IPO Filings
In the biopharmaceutical industry, media outlets typically focus on issues such as substantial price hikes for products with no innovative value, the rising costs of drugs from companies like Mylan and Valeant, and the restriction of FDA-approved biologics due to intellectual property disputes.
A summary of these reports highlights one crucial point: while prices for other consumer goods are falling, drug prices continue to rise. For a long time, the U.S. biopharmaceutical industry and the government have maintained an implicit understanding: unlike European countries, the U.S. government rarely engages in price negotiations for pharmaceuticals, resulting in higher drug expenditures in the United States. This, in turn, enables Americans with comprehensive health insurance to access the most innovative drugs and clinical trials more rapidly than anywhere else.
This is somewhat analogous to superpowers maintaining standing armies: although it comes at a cost, it is an obligation that must be fulfilled. It is logical that if the United States provides financial support for a certain biopharmaceutical product, people in other parts of the world will ultimately benefit from it. One theory holds that if Americans are willing, the United States will allocate more funding to support drug development, thereby ensuring continued progress in global biopharmaceuticals.
Throughout President Obama’s healthcare reform, the aforementioned fundamental premise remained largely unchallenged. However, starting around last year, issues such as rising prices for non-innovative drugs sparked widespread public discontent in the United States and became a national political issue. The American public has also gradually come to recognize a long-standing reality: the lack of incentive among wealthy Americans, who spend lavishly, to negotiate drug prices has enabled several major biopharmaceutical companies to reap substantial profits. All of this has made the biomedical industry an easy target for criticism, particularly in a presidential election year.
The biopharmaceutical industry typically raises three counterarguments, none of which currently resonates with Americans: 1) Medications account for only a small fraction of healthcare costs and, when used appropriately, generally alleviate patient suffering while reducing overall expenses. 2) Drug development is exceedingly costly and unpredictable, with some candidates requiring billions of dollars in investment yet failing to recoup their costs. 3) Innovation!
The third point is particularly compelling. FierceBiotech recently announced the list of the 14th Annual Fierce 15 Companies. VCBeat (WeChat ID: vcbeat) has compiled a summary for you. It is evident that the biopharmaceutical industry is conducting remarkable R&D work, some of which holds the potential to achieve breakthroughs. Immuno-oncology may cure cancer, or at least transform it into a chronically manageable condition; microbes, once a source of psychological distress, are now being evaluated for their therapeutic potential; and gene therapy offers the promise of addressing certain genetic disorders while providing a framework for the rational development of pharmaceuticals.
Therefore, the companies on the Fierce 15 list bear significant responsibility, as their innovative efforts are working to turn these visions into reality.
The company is designing next-generation immune cell therapies.
CEO: Aya Jakobovits
Address:Menlo Park, California
Establishment Date:2014
Clinical Research Focus:Designing Immune Cell Therapies
Investor:OrbiMed, Novartis Venture Fund, and Pontifax
Adicet has disclosed limited details regarding its technology or objectives. The company aims to leverage controlled antigen receptors (CARs) and T-cell receptors to engineer existing immune cells. Although immunotherapy has achieved some success in treating hematologic malignancies, Adicet is currently targeting the more challenging area of solid tumor therapies and is also exploring its potential for treating autoimmune disorders.
In the field of immunotherapy, Adicet has successfully attracted the attention of several major biopharmaceutical companies. Just six months after entering the public eye, the company completed a $51 million Series A financing round in January, with participation from Novartis Venture Funds, and Adicet entered into a secured transaction with Regeneron Pharmaceuticals ($REGN).
Adicet Bio will receive an upfront payment of $25 million from its transaction with Regeneron to develop existing cell therapies, as part of a five-year R&D collaboration. The biopharmaceutical company holds the option to secure development and commercialization rights for an unspecified number of investigational products, while Adicet Bio may either participate in the development and marketing processes or opt to receive royalties.
The company has adopted new approaches to better understand and mitigate neurodegenerative autoimmune diseases.
CEO: Douglas Love
Address:South San Francisco, California
Establishment Date:2011
Clinical Research Focus:Alzheimer's Disease, Huntington's Disease, Glaucoma, and Autoimmune Diseases
Investors:NEA, Correlation Ventures, Novartis Venture Fund, Clarus Ventures, and Satter Investment Management
This preclinical biotechnology company is led by Doug Love, a retiree from Genentech and Elan. The company plans to bring its classic complement inhibitor antibodies into clinical use starting next year to treat a range of conditions, including central nervous system disorders, autoimmune diseases, and ocular diseases. Annexon has two main candidate products: ANX005 for the treatment of central nervous system disorders and autoimmune diseases, and ANX007 for ocular diseases. Both drugs are designed to inhibit C1q, the initiating molecule of the classical complement pathway, thereby blocking complement activation during neurodegeneration.
If a biotechnology company were researching Alzheimer’s disease, you would inevitably express admiration, given that 99% of such research efforts over the past decade have failed. Since 2014, Love has served as CEO of Annexon. He acknowledges the numerous failures in Alzheimer’s research and admits that merely contemplating the challenge of confronting this disease naturally evokes fear. Nevertheless, he remains confident that their approach is bound to succeed.
While the majority of pharmaceutical and biotechnology companies are currently focused on clearing taurine or amyloid proteins from the brains of patients with Alzheimer’s disease, Annexon is attempting to prevent synaptic degeneration, rather than clearing certain debris from the brain after the memory disorder has been brought under control.
This startup biopharmaceutical company is striving to establish a hub for immuno-oncology research, providing rational approaches for combination therapies.
CEO: Terry Rosen
Address:Hayward, California
Establishment Date:2015
Clinical Research Focus:Immuno-oncology
Investors:Founders, Column Group, Foresite Capital, Novartis ($NVS), Celgene ($CELG), GV, Invus, Taiho Venture Capital, DROIA Oncology Venture Capital, and Stanford University
Terry Rosen exited the largest private biotechnology company, selling Flexus Biosciences, which had $1.25 billion in sales, to Bristol-Myers Squibb ($BMY). He then founded Arcus, with his team aiming to build a completely new biopharmaceutical company rather than seeking to benefit from a quick exit strategy.
Arcus is dedicated to the research of small-molecule and antibody-based immuno-oncology therapies, with the aim of internally integrating these modalities. Inspired by combination therapies for hepatitis C, Rosen believes that combining these two therapeutic approaches will yield favorable outcomes.
Since its founding in May 2015, Arcus has raised $120 million through two rounds of financing. The company intends to independently conduct clinical development without relying on large partners or acquirers, as it aims to retain ownership and enhance its valuation. Arcus seeks to achieve best-in-class results in three areas: blockade of active immunosuppressive mechanisms, enhancement of antigen-presenting cell function, and augmentation of effector activities in T cells, NK cells, and macrophages.
The startup is also conducting preclinical work on monoclonal antibodies, which could potentially enable the treatment of bone cancer, all to complement the efficacy of small-molecule therapies. Arcus is not focused on researching individual candidate products but rather on seeking synergistic effects among them and in combination with drugs from other companies. They anticipate having up to three antibodies enter clinical treatment by 2018.
Developing Antibody- and T Cell Receptor-Based Therapies and Vaccines by Harnessing Effective Patient Immune Responses
CEO: Tito Serafini
Address:Redwood City, CA
Establishment Date:2010
Clinical Research Focus:Immunotherapy
Investor:An undisclosed U.S. healthcare-focused fund, the Bill & Melinda Gates Foundation, Mission Bay Capital, and GSK.
Pharmaceutical companies like Atreca boast broad technology platforms. This startup relies not only on its investors but also on a wide array of partners, including GlaxoSmithKline ($GSK), Johnson & Johnson’s ($JNJ) Janssen, Sanofi ($SNY), and Novartis ($NVS).
They, along with the Gates Foundation and several other academic centers, hope that Atreca’s highly precise reverse-engineering approach to drug development will make advances across multiple fields. This technology can be used to develop vaccines for preventing infectious diseases or to reduce excessive activity of the autoimmune system.
Atreca focuses on immuno-oncology research and has achieved results in other fields through collaborations with other companies. Its technology can deconstruct the immune response of individual patients, and through measurement and analysis, identify antibodies and T-cell receptors, which are key to treating patients. It can also identify and generate these functional human antibodies and T-cell receptors without prior knowledge of the antigens.
Atreca aims to develop a pipeline of drugs targeting shared tumor antigens, which are widely present in cancer patients, unlike certain specific antigens that lack universality. Last November, the company raised $56 million and plans to file its first investigational new drug application for clinical trials in 2018.
Developing new therapies for fibrotic diseases to treat various functional disorders.
CEO: Dr. Wendye Robbins
Address:South San Francisco, California
Establishment Date: 2015
Clinical Research Focus:Fibrotic Diseases
Investors:Deerfield Management, Pfizer Venture Investments, Novartis Institutes for BioMedical Research, Bristol-Myers Squibb, MPM Capital, and Osage University Partners.
After years of closely observing the development of fibrosis research, Luke Evnin committed himself to this field. His investment was substantial, as his firm, MPM Capital, has backed Blade Therapeutics in this area.
Blade, founded last year, leverages registered technologies from Johns Hopkins University in the United States, particularly the extensive laboratory achievements of Dr. Harry Dietz, a Professor of Genetics and Pharmacology at the university and the company’s founder. The professor’s research focuses on two fibrotic disorders—Marfan syndrome and stiff skin syndrome—laying the foundation for Blade’s novel biological therapies targeting tissue fibrosis and dysfunction.
Three pharmaceutical companies acquired early stakes in Blade: Pfizer Ventures, the Novartis Institutes for BioMedical Research, and Bristol-Myers Squibb ($BMY), collectively providing $45 million in Series B financing to the startup.
The company hopes that these funds will be sufficient to help advance its drug into the clinical stage. It is expected that in the second half of next year, they will submit an investigational new drug application for a candidate drug, with the aim of initiating clinical trials in the second half of 2018. Blade Therapeutics also aims to develop another drug for the treatment of fibrotic diseases; however, the specific indication and development targets have not yet been disclosed.
A novel small-molecule targeted protein degradation therapy is employed to combat disease-causing proteins, promoting their rapid degradation and clearance from cells.
President & Chief Scientist:Andrew Phillips
Address:Cambridge, Massachusetts
Date of Establishment:2016
Clinical Research Focus:Targeted protein degradation using degronimid technology, with the initial goal of treating cancer.
Investors:Cobro Venture Capital, Roche, Novartis, Cormorant Asset Management, Kraft Group, and EG Capital Group.
C4 Therapeutics is one of the few companies employing small-molecule binders, known as degronimids, which target, destroy, and clear proteins via the ubiquitin-proteasome system. In the preclinical stage, the company is confident in its approach, believing it can be effective against cancer and a range of other diseases.
Jason Fisherman is the CEO of this company. He previously worked in venture capital and served as a researcher at the National Cancer Institute. He told FierceBiotech, “We are rapidly positioning C4 Therapeutics as a leader in the protein degradation industry. Our mission is to explore and develop drugs for the treatment of cancer and other life-threatening diseases. Founded in early 2016, our company was established by distinguished scientists and physicians from Dana-Farber Cancer Institute in Boston. Our technology builds on the research of Jay Bradner and Ken Anderson; Bradner now works at Novartis, an investor in C4, while Anderson is a pioneer in the use of IMiDs (immunomodulatory drugs) for the treatment of multiple myeloma.”
“They came together at the beginning of the year, bringing in $75 million in investment and securing a $75 million deal with Roche. This is a substantial transaction amount that has been tangibly realized by our company. I am not sure if many other startups have had such a fortunate start.”
However, their drugs have not yet entered the clinical stage; preliminary estimates suggest that first-in-human trials will not begin until 2018 at the earliest. According to Fisherman, in the near future, these investigational drugs will combine cell activation therapy with immuno-oncology to treat related diseases.
Plan to strengthen research on cancer immune responses and fulfill the previous commitment to anti-CD47 therapy.
CEO: Jonathan MacQuitty
Address:Menlo Park, California
Date of Establishment: 2016
Clinical Research Focus:Early Trials of the Anti-CD47 Antibody Hu5F9-G4 for Treating Various Cancers.
Investors:GV (Google’s early-stage venture capital arm), Lightspeed Venture Partners, Sutter Hill Ventures, and Clarus Ventures.
Originating from Irv Weissman’s laboratory at Stanford University, Forty Seven has targeted CD47, a protein that suppresses an immune response which helps tumors evade phagocytosis (the process by which phagocytes engulf cancer cells). Dating back to 2012, early research data from Stanford University were compelling, and now its spin-off company is investigating a novel immune response to combat cancer cells.
However, Forty Seven Inc. has no intention of following the crowd by engaging in checkpoint inhibitor research; instead, it is more inclined to leverage its anti-CD47 compound to develop a new generation of immuno-oncology drugs. MacQuitty explained, “Currently, all internal and external research efforts are focused on the adaptive immune system, which comprises cellular components and processes that eliminate or inhibit pathogen growth. At the same time, we also have the innate immune system, which is regulated by a type of white blood cell known as macrophages.”
The company raised $75 million in its Series A financing round at the beginning of the year, with support from GV, Google’s investment arm. “Google’s contributions go far beyond capital,” but MacQuitty stated that details are not yet ready for public disclosure.
Research into Gene Regulation Science, and Exploring the Therapeutic Benefits of Gene Switching Through Stem Cells and CRISPR-Cas9
CEO: Robert Gould
Address:Cambridge, Massachusetts
Establishment Date:2015
Focus of Clinical Research:Two Monogenic Diseases: Fragile X Syndrome and Facioscapulohumeral Muscular Dystrophy
Investor:Third Rock Ventures
Adopting this approach, fulfilling the promise of gene regulation and developing new drugs is no easy task, but the recently established Fulcrum Therapeutics is attempting to do just that for severe monogenic diseases with very limited treatment options. Led by former Epizyme CEO Robert Gould, the company’s strategy centers on modulation: using patient-derived cells and CRISPR-Cas9 (a cutting-edge biotechnology developed by the company) to attempt to silence specific genes.
What Makes Fulcrum Therapeutics So Impressive? In a Word, Its Mission and Scientific Research. With this mission established, it is noteworthy that there are currently no approved treatments for Fragile X Syndrome or Facioscapulohumeral Muscular Dystrophy (FSHD). Patients with FSHD eventually become wheelchair-bound and progressively debilitated, with no means to slow or reverse this decline. Fragile X Syndrome in children is an unavoidable genetic disorder caused by learning disabilities, affecting 1 in 4,000 males and 1 in 8,000 females, and leading to a range of learning, social, language, attentional, emotional, and behavioral issues. While certain approved medications can address some of these symptoms, the underlying causes remain undiscovered.
Gould summarized, “The uniqueness of Fulcrum lies in its integration of iPSC-based mimetic gene biology with an understanding of the regulatory mechanisms governing human gene expression, combined with CRISPR-Cas9 technology and the use of patient-derived cells from those we aim to treat. These represent unprecedented approaches and opportunities.”
Bringing Small Molecules to the Forefront of Immuno-Oncology
CEO: Yujiro Hata
Address:South San Francisco, California
Date of Establishment: 2015
Clinical Research Focus:Immuno-oncology
Investors:5AM Ventures, Canaan Partners, Celgene, WuXi Healthcare Ventures, Novartis Institutes for BioMedical Research, and Alexandria Real Estate Equities
In the field of immuno-oncology, with its growing emphasis on combination therapies, and in targeting synthetic lethal interactions driven by cancer mutations (beyond BRCA genes), Ideaya aims to be the first company to bring small molecules to the forefront. The company is also dedicated to inducing DNA damage to enhance the efficacy of immunotherapies.
“We have engaged with our investors and potential pharmaceutical partners, who are highly interested in the intersection of DNA damage and immunotherapy,” Yujiro Hata, co-founder and CEO of Ideaya, told FierceBiotech.
Ideaya’s $46 million Series A financing round in May included three strategic investors: Celgene, Novartis, and WuXi. Robert Hershberg, Chief Scientific Officer at Celgene, also serves as a board observer for the startup and has contributed his expertise, said Hata.
Although it has not yet entered the clinical stage, Ideaya is engaging in discussions with potential partners. Hata anticipates that more substantive negotiations will take place within the next year. The company is currently selecting candidate projects from dozens of different initiatives, which encompass research in immunotherapy and synthetic lethality.
The company is dedicated to researching the efficacy of tumor antigens.
Interim CEO:Cary Pfeffer
Address:Cambridge, Massachusetts
Date of Establishment: 2015
Clinical Research Focus:Personalized Cancer Therapeutic Vaccines
Investor:Third Rock, Clal Biotechnology Industries, and Access Industries.
When people think of cancer vaccines, they cannot help but recall the failures encountered in laboratories and during production. However, Neon Therapeutics is seeking next-generation treatments for a broad range of tumors. The company is currently led by CEO Dr. Cary Pfeffer from Third Rock Ventures. Their initial clinical trial will be conducted this fall, combining their investigational therapy with Bristol-Myers Squibb’s blockbuster PD-1 inhibitor Opdivo (a monoclonal antibody) to enhance therapeutic efficacy in patients.
The company’s flagship product is NEO-PV-01, a personalized cancer vaccine for which it has recently submitted an application, with clinical use slated for this year. “This will commence this autumn, coinciding with the first anniversary of our company’s founding. Such a short timeframe is truly remarkable.”
Pfeffer stated that the company has a four-pronged plan. One aspect involves personalized cancer therapeutic vaccines utilizing tumor antigens—exogenous substances identified in cancers—which can be customized for each patient to elicit specific immune system attacks, thereby treating the disease.
The second is to seek shared tumor antigens that can be used for existing therapeutic vaccines, where the antigens are present in a group of patients.
The third focuses on personalized T-cell strategies, which involve identifying T cells capable of attacking cancer cells, extracting them from the patient’s body, enhancing their efficacy, and expanding their use within the patient. The final approach involves off-the-shelf T-cell products that can be used in a broader population.
Treating Refractory Malignant Solid Tumors Using Tumor Tissue Self-Limiting Technology
CEO: Mitchell Finer
Address:Cambridge, Massachusetts
Establishment Date:2015
Clinical Research Focus:Oncolytic Virus
Investors:Celgene ($CELG), MPM Capital, Deerfield Management Company, Arkin Bio Ventures, Excelyrate Capital, and Long March Investment Company.
This startup is developing next-generation oncolytic viruses, offering a more cost-effective and potent immuno-oncology therapy compared to autologous CAR-T cell therapies currently under development. They are tackling hard-to-treat malignant solid tumors, including glioblastoma, a type of brain cancer.
Oncorus was co-founded by Mitchell Finer, former Chief Scientific Officer of bluebird bio, and a Managing Director at MPM Capital. Their approach targets specific tumor tissues, thereby eliminating the risk of damage to non-tumor tissues associated with immuno-oncology therapies.
What Makes Oncorus So Impressive? The latest and most innovative cancer therapies are typically limited to the U.S. and European markets due to their high costs. Oncorus aims to provide a one-time, durable treatment that makes immuno-oncology accessible to more patients.
“I don’t think our product is expensive,” said Finer. “This drug can be distributed globally and provides immunotherapeutic effects lasting up to seventeen months. It elevates efficacy and versatility to the level of a vaccine, thereby replacing cell therapy. However, all treatments must still be combined with surgery, chemotherapy, and radiotherapy.”
Aims to become the first gene therapy product approved in the United States
CEO: Currently managed by the F-Prime Capital team
Address:Los Angeles, California
Date of Establishment:2016
Clinical Research Focus:Rare and Ultra-Rare Genetic Diseases
Investors:Supported by F-Prime Capital and UCL Business, with additional support from the UCL Technology Fund.
Orchard is poised to recruit talent from GlaxoSmithKline ($GSK) to enhance Strimvelis, its recently approved therapy for “bubble boy syndrome,” by leveraging cutting-edge technologies used in treating fewer than 100 patients worldwide, with the aim of making the treatment better, more effective, and safer.
Orchard’s development programs focus on research into autologous hematopoietic stem cell gene therapies to restore normal gene function in primary immunodeficiencies, metabolic disorders, and hematopoietic system diseases.
This cutting-edge technology utilizes samples of a patient’s own stem cells ex vivo, repairs the missing or defective genes, and then transplants them back into the patient’s body. Using the patient’s own (autologous) cells eliminates the need for stem cell matching, thereby saving months or even years of time.
The early candidate product from this small company is an ex vivo autologous lentiviral stem cell gene therapy for severe combined immunodeficiency (SCID). This condition, also known as "bubble boy syndrome," renders affected children highly susceptible to infections, necessitating continuous protection through placement in sterile environments. Orchard Therapeutics is also exploring the application of ex vivo autologous lentiviral stem cell gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA, or Sanfilippo syndrome type A).
Research on Rare and Ultra-Rare Genetic Diseases
CEO: Keith Gottesdiener
Address:Boston, Massachusetts
Date of Establishment:2008
Focus of Clinical Research:Rare Forms of Obesity
Investors:Pfizer, Ipsen, Third Rock, Deerfield, NEA, Orbimed, NEA, MPM, and two other undisclosed investors.
Obesity has become a major problem in Western countries, a phenomenon more prevalent than you might imagine. However, affected individuals often gain significant weight at a very young age, rarely due to familial genetic disorders. Currently, there are no effective treatments available to help these children and young adults, but two candidate drugs from Rhythm Pharmaceuticals for pro-opiomelanocortin (POMC) deficiency obesity and Prader-Willi syndrome are set to change this landscape.
This biological candidate therapy for pro-opiomelanocortin (POMC) deficiency obesity yielded impressive data this summer. For instance, in Phase II of the study, two patients in their twenties, each weighing approximately 340 lbs at baseline, achieved a weekly weight loss of 3.75 to 4.4 lbs over 42 consecutive weeks. Although only two patients were enrolled at the investigative site, this sample represents a substantial proportion given that only 50 such patients are registered in the database.
“They have no intention of studying more types of obesity,” Gottesdiener stated that his company would continue to focus on the treatment of rare diseases, “We want to concentrate on areas with significant unmet needs, but we did not find such demand in the general obesity treatment market. However, in the known areas of rare obesity treatments, we can make a difference.”
Secured the first major microbiome therapeutics deal with Johnson & Johnson, preparing to independently advance the drug into clinical trials next year
CEO: Bernat Olle
Address:Cambridge, Massachusetts
Establishment Date:2010
Clinical Research Focus:Microorganisms
Investors:PureTech Health, Johnson & Johnson, Rock Springs Capital, Invesco Asset Management, and Health For Life Capital (Seventure).
Microbes are complex, enigmatic, and sensitive. Many industry observers believe that developing microbial therapies for disease treatment will be an arduous process.
Vedanta, a startup founded by PureTech after years of deliberation on current research, focuses primarily on the study of the gastrointestinal microbiota. The company entered into a significant collaboration with Johnson & Johnson in 2014 and is now poised to advance an investigational drug for inflammatory bowel disease into clinical trials in the first half of next year. Vedanta also aims to advance an infectious disease program into clinical trials during the same period.
“Our relationship with Johnson & Johnson was facilitated by the team at JLABS Boston,” Bernat Olle, CEO of Vedanta Biosciences, told FierceBiotech. “They share common interests in immunology and inflammatory bowel disease (IBD). The company holds significant exclusivity rights for Remicade in IBD, possesses strong clinical development capabilities in this therapeutic area, and overall maintains a prominent position in immunology research.”
The deal includes investment from Johnson & Johnson, an upfront payment, and potential milestone payments totaling up to $339 million.
"Attempting to develop a gene therapy to treat catastrophic diseases that cause infant mortality worldwide."
CEO: Sean Nolan
Address:Bannockburn, Illinois
Establishment Date:2010
Clinical Research Focus:Gene therapy candidates for the treatment of Type 1 spinal muscular atrophy.
Investor:T. Rowe Price, Deerfield Management, Roche Venture Fund, Venrock, Janus Capital, RA Capital, and Foresite Capital.
AveXis focuses on research into spinal muscular atrophy (SMA), a condition that affects approximately 10,000 children worldwide. SMA has four types in total, and AveXis is studying Type 1, the most severe form. Approximately 90% of children with this type do not survive beyond one year of age; during their short lives, they are unable to sit, crawl, or walk. Furthermore, these children have a very low quality of life and require long-term ventilatory support to sustain life.
Like many companies on this year’s Fierce 15 list, AveXis is pursuing gene therapy for this devastating disease, for which only palliative care is currently available to affected children. Spinal muscular atrophy type 1 (SMA 1) occurs when the body fails to produce sufficient amounts of a protein called SMN due to deletions in the SMN1 gene. Without this gene, the body cannot generate the protein, and in its absence, motor neurons and other tissues, such as muscles, degenerate and die.
Sean Nolan, CEO of AveXis, believes that gene therapy is the best approach to combating this disease.
Prior to its initial public offering in February of this year, the company remained privately held. Given its cutting-edge technology and its focus on rare catastrophic diseases, FierceBiotech made an exception to its private-company criteria and included it among the winners.