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For the past 60 years, many of the innovations and ideas that shape our world were formulated, hatched and brought to reality in the wide swath of piney forest known as Research Triangle Park. The Park was founded as an attempt to keep the talent that was being churned out by Duke, UNC and N.C. State within the state. Now it draws talent from all over the globe, and holds unique prestige among technological and innovation hubs. (Durham Magazine, May, 2019).

Anchored by giants such as GlaxoSmithKline, Pfizer, Grifols, Biogen and BASF, some interesting small biotech’s are making a name for themselves.  My picks for the Raleigh-Durham area are:

Heat Bio, Chapel Hill, has a proprietary gp96 platform that activates the human immune system to combat cancer and infectious disease. Heat Biologics is developing a robust pipeline of first-in-class products that activate and / or modulate the immune system in non-small cell lung cancer, solid tumors and an infectious disease initiative with the military.

CoImmune, Durham, Acquired their RNA-loaded dendritic cell platform technology and assets of Argos Therapeutics in 2019.  They have a CAR-CIK platform technology in clinical trials and for Acute Lymphoblastic Leukemia; Non-Hodgkin’s Lymphoma; Chronic Lymphocytic Leukemia; Acute Myeloid Leukemia; and Metastatic Renal Cell Carcinoma.

G1 Therapeutics, RTP, G1 is advancing its mission by commercializing the first and only proactive multilineage myeloprotection therapy, COSELA™ (trilaciclib). G1 is also executing a tumor-agnostic development program, with late-stage clinical trials ongoing in multiple tumor types including colorectal, breast, lung, and bladder cancers.

StrideBio, RTP, StrideBio’s proprietary structure-inspired adeno-associated virus (AAV) vector engineering platform (STRIVE™) is a powerful discovery engine that creates unique and differentiated vectors that improve upon naturally occurring AAV serotypes to overcome current limitations of first-generation gene therapies and allow more patients to benefit from gene therapies with maximum efficiency.  They have products in the pipeline for Friedreich’s Ataxia, Angelman Syndrome, Rett Syndrome to name a few.

Arrevus, Raleigh, is a clinical stage biotechnology company developing novel therapies for orphan diseases.  They have products in the pipeline for pulmonary exacerbations of cystic fibrosis, CF and ID/oncology.

As a leading biomedical sciences hub at the heart of Asia, Singapore is the choice location for companies to develop and produce new products that better meet Asia’s healthcare needs. Companies benefit from our pro-business environment, infrastructure, talent and innovation ecosystem, as well as the proximity to the rest of Asia.

Companies like GlaxoSmithKline, MSD and Roche have based a range of commercial activities, including Supply Chain Management, Regulatory Affairs and Medical Affairs in Singapore. (EDB:  Singapore)

There were some very interesting choices for up-and-coming biotech companies.  Many of the companies I studied were focused on personalized oncology as well as neurodegenerative diseases.

Hummingbird Bioscience, Singapore, uses a unique Rational Antibody Discovery platform enables us to overcome many of the challenges of finding potent therapeutic antibodies and to generate antibodies precisely engineered for efficacy, safety and developability. This includes the discovery of functional agonists and antagonists, antibodies against transmembrane proteins as well as the conserved regions of rapidly mutating e.g., viral proteins.  Their current pipeline includes HMBD-001 a novel mechanism of action for inhibiting tumor growth and drug resistance in HER3-driven cancers, VISTA (V-domain immunoglobulin suppressor of T cell activation) is a co-inhibitory immune checkpoint that potently suppresses T cell activity in a variety of cancers and has been associated with a lack of response to other checkpoint therapies and HMBD-009 is our pioneering next-generation antibody program that targets both BCMA and TACI, proteins commonly overexpressed on tumor cells in multiple myeloma.

TauRx Pharmaceuticals, Singapore, has already completed three separate Phase 3 trials: two in Alzheimer’s disease and one in the rare neurodegenerative disorder, behavioral variant frontotemporal dementia (bvFTD).  In addition, TauRx evaluated its first-generation tau aggregation inhibitor (TAI), rember^®, in a large, double-blind, long-duration, Phase 2 clinical trial in 321 patients with mild or moderate Alzheimer’s disease. 

Tessa Therapeutics, Singapore, is leading the way in CD30 targeting using cell therapy. CD30 is a well validated lymphoma target with homogenous expression in Classical Hodgkin Lymphoma and multiple subsets of non-Hodgkin Lymphomas. Tessa’s approach to target CD30-positive cancers using CAR-T (Chimeric Antigen Receptor) cells has demonstrated excellent safety and efficacy in early clinical trials. Having received the RMAT and PRIME designation, Tessa is now advancing the therapy to a pivotal trial.

Lucence, Singapore, is making cancer testing easier.  LiquidHALLMARK® is an ultrasensitive, next-generation sequencing assay for genomic profiling of a patient’s unique cancer.  Requiring only a single blood sample, it provides important information for cancer care, especially when tissue by invasive biopsy is insufficient or inaccessible.

Curiox Biosystems, Singapore, pioneered centrifuge-free cell washing to improve data and simplify workflows.  Their Laminar Wash™ Systems deliver higher quality data through better cell retention, improved preservation of cellular physiology and viability, and exceptional study-to-study and operator-to-operator reproducibility.  

According to the Silicon Republic, Ireland is a home for 24 of the world’s top biotech and pharma companies.  AstraZeneca, J&J, Pfizer, Shire and AbbVie all have homes in Ireland.  Why?  Pharma companies enjoy the benefits of an English-speaking population, friendly business environment, a favorable tax environment and a well-educated workforce.  In fact, Ireland has one of the world’s lowest corporate tax rates at just 12.5%. (Innopharma Education).

Read my top 5 up and coming Irish companies below:

Iterum Therapeutics, Dublin, is marketing Sulopenem, the first oral and IV penem antibiotic demonstrating a potent spectrum of activity against multi-drug resistant gram-negative infections in both the hospital and community settings.  The main target is UTIs and intra-abdominal infections.

Perfuze, Galway, have developed the Millipede 088 to address the Unmet Clinical Need for a simple, repeatable way to completely reperfuse an occluded brain blood vessel in a single attempt in stroke patients. 

Trinity Biotech, Dublin, develops high quality assays for the Point of Care (POC) setting. Their POC range allows healthcare providers to give fast and accurate results as an aid in patient diagnosis for HIV, Infectious Diseases, Drugs of Abuse and Fertility.

Organic Vaccines, Dublin, is a life sciences company at the clinical stage of development, dedicated to delivering personalized therapies utilizing one’s own cells (one blood sample equals 10 years of vaccines) Organic Vaccines is positioned for First-in-Man in 12 to 18 months, followed by Phase III multi-centric clinical trials.  Organic Vaccine’s technology addresses uncovered therapeutic aspects for HPV and influenza, and provides an answer to the demand for « green » vaccines, with no side effects.

Avadel, Dublin, has developed FT218 is an investigational formulation of sodium oxybate leveraging our proprietary drug delivery technology and designed to be taken once at bedtime for the treatment of EDS and cataplexy in adults with narcolepsy.

Philadelphia, the city of brotherly love, Rocky running up the “Rocky Steps” at the Philadelphia Museum of Art, and the Liberty Bell. Oh, and we cannot forget the world-famous delicacy aptly known as Philly Cheesesteak!  

Philly has been growing as a biotech hub anchored by UPenn.  More than 30 cell and gene therapy development companies and roughly 80 percent of all pharmaceutical and biotech companies in the U.S. have offices in Greater Philadelphia, according to data from Select Greater Philadelphia.

Here are my top 5 picks for interesting and growing biotechs in Philadelphia:

Amicus Therapeutics, Philadelphia, has a wide array of innovative products for Fabry Disease, Pompe Disease, Batten Disease and products for CNS disorders.  

Carisma Therapeutics, Philadelphia, uses CAR Macrophages combined with a unique set of characteristics, which they believe are the key to success in solid tumor treatment: recruitment and access to the solid tumor TME, ability to survive in the hostile solid tumor milieu, maintenance of an anti-tumor phenotype in the presence of immunosuppressive factors, capacity to selectively destroy cancer cells, and activation of an adaptive immune response by presenting engulfed tumor material.  Their current pipeline includes CT-0508 (Her-2), CT-0729 (PSMA) and CT-1119 (Mesothelin)

Passage Bio, Philadelphia, are accelerating the delivery of AAV gene therapies for central nervous system (CNS) disorders using next-generation science.  They have three main product lines as follows:  GMI gangliosidosis, Krabbe disease and Frontotemporal dementia.

Treovir, Philadelphia, was founded in 2019 to develop and commercialize G207 for recurrent glioblastoma in children.  G207 is a targeted oncolytic immunotherapy that has demonstrated improved survival in a phase 1 clinical study.    In 2021, Treovir will launch a Phase 2 study designed to support registration of G207 for treatment of pediatric glioma.   If successful, G207 would be the first-ever approved therapy for pediatric glioma.

Vibe Pharmaceuticals, Philadelphia, is an early-stage drug discovery company, spun out of the University of Pennsylvania, developing effective therapeutics for musculoskeletal disorders which lead to bone and muscle loss.  Using a unique screening platform, the Vibe Pharmaceuticals team has isolated secreted vibration-induced bone-enhancing (Vibe) molecules, an example of which is the protein VB-102. 

Having launched dozens of biotech products, I have had a front row seat to good product launches, excellent product launches and those that one would never want to repeat.  Interestingly, there are five key areas that separate an excellent market entry from one that has issues (or doesn’t happen at all).

For many companies, hiring a commercial team early on is not fiscally feasible.  Hence, they tend to overlook some key areas or “fill in the blanks” without commercial guidance.  Both approaches are risky, but there is an alternative.  An Interim Chief Commercial Officer (#iCCO) can serve this key role at about 50% of the cost and less risk than investing in a full time Chief Commercial Officer.  An iCCO can help ensure an excellent market launch that brings success to the team, the investors and the researchers or patients who benefit from the product.

What are the five areas in need of commercial expertise?  They are:

KOL strategy – Identifying key opinion leaders and educating them to become advocates for the product is an essential step in the launch process.  KOLs serve as peer leaders who can provide the company with vital information on the market and customer needs.   In addition, KOLs serve as brand advocates, representing the company at conferences and industry trade shows.

Competitive Landscape – Knowing the players and their offerings is essential for market entry.  If you think about market entry as a board game, you must know the players and anticipate the moves they may make.  It is not enough to make a current assessment, you must think ahead, anticipate their moves, anticipate new entrants and ensure you have a strategy for this changing market landscape.

Forecasting – An accurate forecast requires a great deal of analysis and assumptions based on data and commercial experience.  A great forecast ensures that the product build is accurate, eliminating issues with making too much or too little inventory.  It also supports the sales forecast for each geography.

Pricing/Reimbursement – What are you charging?  Why are you charging that price?  How much are the payers getting reimbursed?  All three of these questions are equally important.  You can price yourself out of the market.  You can leave money on the table or you can miss the mark on reimbursements by not understanding all of the details.  

Education – This is two-fold.  Making sure you have the tools to educate the medical community and having the tolls to educate and train the sales team.  What is your product story?  How do you explain the science behind the product and how it is better that the current solution?  Preparing these tools to effectively educate your stakeholders is a step that cannot be missed or done as an afterthought.

Product commercialization requires the expertise of a commercial expert.  Someone who has the expertise to get the answers needed and expose the landmines that the executive team may not see.  

Australia has ranked in the top five globally for biotechnology three years running, outperforming nations such as the United Kingdom and Germany, according to Scientific American Worldview. The medical-biotechnology sector has benefited from a multibillion-dollar windfall of government funding, as well as from substantial tax breaks for companies investing in research and development. (Nature, May 9, 2018).

There were many great choices, yet I have narrowed down my top picks to the outstanding companies below:

Kazia Therapeutics, Sydney, are working on drug candidates designed to treat diseases such as brain cancer, renal cancer, and liver cancers which are poorly served by current therapies.   Using PAXALISIB (Genentech), Kazia is working on newly-diagnosed glioblastoma and advanced solid tumors.

Actinogen Medical, Sydney, is an ASX-listed, biotechnology company developing an innovative treatment for cognitive impairment associated with neurological diseases amenable to modifications of raised cortisol levels inside brain cells. ‘Cognition’ relates to how a person understands and acts in the world around them. Cognitive functions include memory, reasoning, awareness and decision-making, and to a large extent, influence our personality.  Their lead drug candidate, Xanamem®, has been specifically designed to block the production of cortisol – the stress hormone – in the brain. 

Mesoblast, Melbourne, is using its proprietary mesenchymal lineage cell technology platform to develop and commercialize innovative allogeneic cellular medicines to treat complex inflammatory diseases resistant to conventional standard of care.  They currently have three Phase 3 product candidates.  Graft vs. host disease in children and ARDS (acute respiratory distress syndrome) due to COVID-19.  Advanced chronic heart failure and chronic low back pain due to degenerative disc disease.

Imugene, Sydney, is a clinical stage immuno-oncology company developing a range of new treatments that seek to activate the immune system of cancer patients to identify and eradicate tumors.  Their product pipeline includes an Oncolytic Virus and B-Cell Immunotherapies aimed at treating a variety of cancers in combination with standard of care drugs and immunotherapies. We are supported by a leading team of international cancer experts with extensive experience in developing new cancer therapies.

Certa Therapeutics, Melbourne, is a clinical stage biotechnology company developing innovative therapies for the treatment of fibrotic diseases.  Certa’s novel drugs block a receptor that is a key driver of fibrosis (scarring), a progressive condition which can lead to organ failure and ultimately in some diseases, the need for organ transplantation. Certa Therapeutics is focused on the emerging field of medical science which uses genetic information to identify which patients will best respond to a specific treatment, commonly referred to as precision medicine.

Did you know that Salt Lake City,Utah ranks as the number three genomics market in the nation, based on innovation, talent and growth metrics. In addition, Utah leads the nation in job growth in the life sciences industry.  Companies such as ARUP Laboratories, BD, BioFire Diagnostics, Biomerics, Edwards Lifesciences, Fresenius Medical Care, GE Healthcare, Merit Medical, Myriad Genetics, Nelson Laboratories, Recursion Pharmaceuticals, Stryker, Tolero Pharmaceuticals, Varex Imaging, and others have facilities in Utah. (BioUtah)

Salt Lake City is the capital of Utah.  Utah has a competitive advantage in the life sciences because of our trained workforce, intellectual capital, historical expertise, and government support. These components create fertile ground for the state’s innovation ecosystem as we collaborate and work together to build a world-class life sciences hub. (BioUtah)

Here are my top 5 picks:

IONIQ Sciences uses the body’s electrical properties change in the presence of cancer to non-invasively detect malignancy in its earliest stages.  The IONIQ System utilizes Electrical Impedance Analytics (EIA) to take up to 13,125 electrical readings of the body over a 20-minute period. Their proprietary algorithm processes this data to determine the likelihood of malignancy in its earliest, most-treatable stages.

IONIQ is integrating Artificial Intelligence (AI) to further improve performance and expand from its “Breakthrough” ProLung Test to a Multi-Cancer Screen.

Recursion Pharmaceuticals manufactures an integrated Recursion Operating Systems that creates a closed-loop system combining proprietary in-house data generation and advanced computational tools to generate novel insights to initiate or accelerate therapeutic programs.  They iterate on this approach to create a virtuous cycle of learning within our system and progress programs at each stage of discovery and pre-clinical development.

Q Therapeutics is developing innovative cell therapies for debilitating CNS diseases by boosting the body’s natural support and repair mechanisms to restore neuron function and health.  Q Therapeutics is uniquely focused on a glial cell therapeutic approach to treating neurodegenerative diseases and damage to the brain

Q-Cells^® – our first cellular therapeutic candidate – are human glial progenitor cells that give rise to two types of specialized glial cells, astrocytes and oligodendrocytes.

PEEL Therapeutics began with a focus on cancer-fighting proteins in elephants, and quickly recognized the power of using evolution’s solutions to treat and prevent disease.  Our expanded drug development pipeline now includes other compounds that also originate from nature.  PEEL’s Neutrophil Targeting Peptide (NTP) comes from a small protein in newborns during the first 48 hours of life that blocks inflammation.  PEEL’s anti-cancer small molecule originates from the leaf of an ancient Chinese tree used in Eastern medicine for thousands of years. Our company exists at the intersection between nature and technology with a robust and growing drug development pipeline based on over 55 million years of evolution’s Research & Development.   

Stella Diagnostics, Inc. is a molecular diagnostics-based organization focused on improving patient management strategies for over 60 million people living with severe esophageal disease. Stella Diagnostic’s mass spec proteomic assays will offer physicians insight into the molecular properties of their patients’ tissue to determine if the disease is stable, progressing or turning cancerous. Stella Diagnostics is developing first-line diagnostic tools that provides superior molecular information for providers as compared to the current standard screening protocols.

Buffalo NY, the second largest city in New York State, is known as the home of delicious Buffalo chicken wings, the Buffalo Bills and legendary snow storms. I was a freshman at UB during the infamous “blizzard of ‘77” where we jumped off the third floor of the Katherine Cornell theatre into 15 foot show drifts (spread eagle landing). And Duff’s in Amherst, not the Anchor Bar, was our go-to wing joint, (suicide wings and Labatt’s beer).  

In the past few decades, it has also become the home to some interesting and innovative biotech companies.  With a special focus in immuno-oncology companies, I have highlighted some of the standouts below.

According to esd.ny.gov, New York is growing its life science industry through a broad range of economic development programs and public-private partnerships, supported by the state’s award-winning research talent. And through its $620 million Life Science Initiative, New York is expanding the state’s ability to commercialize research and spurring the growth of a world-class life science research cluster.

OmniSeq, is a company helping cancer patients find and access the drug or trial that’s right for them. They do this through molecular sequencing and first-of-its-kind comprehensive immune profiling, as well as pharmaceutical services.  With LabCorp, they have launched OmniSeq INSIGHT℠. This innovative tool – which is offered exclusively to U.S.-based physicians by Integrated Oncology, a member of the LabCorp Specialty Testing Group, and biopharmaceutical customers globally through Labcorp Drug Development – enables medical professionals to identify appropriate treatments specific to a patient’s cancer and immune profile.

For-Robin, Inc, is named in honor of the founder’s sister who died at age 31 of breast cancer.  It is an antibody immunotherapy company whose primary mission is developing treatments for breast and lung cancer patients. Their proprietary technology (monoclonal antibody, JAA-F11 and humanized variants, hJAA-F11) targets all breast and lung cancer cell subtypes including triple negative breast cancer where no targeted therapy currently exists. For-Robin is developing hJAA-F11 for adjunct therapy of breast and lung cancer either directly or as antibody-drug conjugates.

Athenex is a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer. Their mission is to become a global leader in bringing innovative cancer treatments to the market and improve health outcomes.  The company has a robust pipeline for solid tumors, neuroblastoma, actinic keratosis, metastatic breast cancer, angiosarcoma, neoadjuvant breast cancer and gastric cancer.

Tactiva Therapuetics –  Current ACT approaches in immunotherapy focus primarily on developing anti-tumor CD8 T cell responses while overlooking the critical role of helper CD4 T cells. Tactiva utilizes a unique class of CD4 T cells that not only enhance the anti-tumor function of CD8 T cells, but also exhibit a capacity to directly target the tumor cells.

OncoTartis, Inc. Founding IP came from Prof. Andrei Gudkov’s laboratory at Roswell Park Cancer Institute (Buffalo, NY). OncoTartis is exploring a proprietary drug discovery approach called “TARTIS” (TARTIS = TARgeting TISsue).  OncoTartis develops a novel category of anti-cancer drugs directed against tissue-specific targets for a subset of cancers (breast, prostate, ovarian, melanoma and hematological malignancies). Its leading product OT-82 is a nicotinamide phosphoribosyltransferase (NAMPT) inhibitor being developed to treat hematological malignancies.

The Dallas-Fort Worth area of Texas is a thriving metropolis.  When visiting, one must make sure to go to the top of Reunion Tower, visit the 6^th Floor Museum at Dealy Plaza and wander about the George W. Bush Library.  Although Houston holds the states honor of being the top city for medical excellence, Dallas is nipping at its heels.  Home of UT Southwestern Medical Center (and the many companies that have spun out of UTSW) and large pharmaceutical companies such as Alcon, Novartis and Pfizer, the DFW area is growing.  In addition, the State of Texas has a generous funding body, Cancer Prevention and Research Institute of Texas, CPRIT, that provides funding for academic research, product development and prevention. 

Below is my list of top picks for emerging biotech companies in the DFW area:

Gradalis, Carrollton, TX is a biotechnology company focused on the development and commercialization of novel personalized therapeutics to treat cancer.   They have invented the the Vigil® platform, a fully personalized, patient-specific cancer immunotherapy that can be applied to virtually any cancer.

Lantern Pharma, Dallas, TX has developed RADR®, or Response Algorithm for Drug Positioning & Rescue.  It is Lantern’s proprietary integrated data analytics, experimental biology, biotechnology, and machine-learning-based platform. Their robust pipeline includes products for prostate cancer, solid tumors, NSCLC, glioblastoma and CNS cancers.

Reata Pharmaceuticals, Irving, TX has developed investigational Nrf2 activators, including bardoxolone methyl, omaveloxolone, and next generation molecules, modulate genes involved in cellular metabolism and mitochondrial function to regulate the cellular inflammatory response. They are developing bardoxolone methyl and omaveloxolone for treatment of chronic kidney disease and Friedreich’s ataxia, respectively. Their novel small molecule inhibitors of HSP90 and RORγt are in development for treatment of neurological and autoimmune diseases, respectively.

Nacuity Pharmaceuticals, Ft Worth, TX is focused on developing a treatment for retinitis pigmentosa (RP) and leveraging our diverse library of novel antioxidant molecules to address other ocular diseases involving oxidative stress. 

Arog Pharmaceuticals, Dallas, TX is a Phase 3 biopharmaceutical company dedicated to developing their lead investigational drug candidate, crenolanib, and a related class of benzimidazole-based compounds to become treatment options for cancer indications with high unmet medical need. Current targets include AML and multiple solid tumors.