Israel is a showcase for thriving biotech

The Land of Israel, also known as the Holy Land or Palestine, is the birthplace of the Jewish people, the place where the final form of the Hebrew Bible is thought to have been compiled, and the birthplace of Judaism and Christianity. (Wikipedia)  It also has a thriving biotechnology sector, fueled by an educated workforce and strong government funding initiatives.

Blockbuster prescription drugs sold worldwide that treat multiple sclerosis, cancer, Alzheimer’s and Parkinson’s diseases derive from Israeli biotechnology. Israel creates more medical devices per capita than any other country, and its life sciences exports earn more than $3 billion a year. (Madein-Israel.com)

Looking through the vast array of Biotech’s in Israel, I had many stellar companies to choose from.  I looked for innovative companies that have not yet set up operations in the U.S. to showcase Israeli-based companies.

My choices are:

Biosight Pharma, Israel, is developing innovative therapeutics for hematological malignancies and disorders.  Their lead product, aspacytarabine (BST-236), is an innovative proprietary anti-metabolite designed to enable high-dose therapy with reduced systemic toxicity.  It may serve as an ideal therapy for leukemia, particularly for delivering high doses of cytarabine to medically unfit or older adults who otherwise cannot be treated by effective doses of chemotherapy.

BST-236 is being investigated as a single-agent in a Phase 2b for first-line treatment of acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS):

OSSIO, Israel, has created bio-Integrative OSSIOfiber®, a breakthrough fixation material technology that provides the first credible solution to the shortcomings of permanent metal hardware, conventional resorbable and allograft implants.

Strong and Bio-Integrative OSSIOfiber® implants provide a unique matrix of fibers made from minerals like those already found in bones to support strong fixation and enable early bone attachment, gradual bone and tissue ingrowth, and complete integration, all without local or systemic adverse response.

OSSIOfiber ® offers surgeons and patients the confidence and certainty of a fully Bio-Integrative solution that utilizes existing reimbursement and surgical techniques and provides a more natural healing environment.

Immunovative Therapies, Ltd, Jerusalem, is a clinical stage Israeli biopharmaceutical company specializing in the translation of proven immune mechanisms into immunotherapy products and protocols for treatment of cancer and infectious diseases. Our patented “Mirror EffectTM” technology provides an immunological platform for the development of drugs that elicit the same powerful anti-tumor and anti-HIV effects of bone marrow/stem cell transplant procedures while eliminating the devastating side effects of the procedure and the need for a tissue matched donor. We have two products in clinical development: AlloStim®, a bioengineered living cell allograft; and Chaperone-Rich Cell Lysate (CRCL), a patient-specific heat shock protein vaccine. AlloStim combines with a tumor cryoablation (killing by freezing) method to create an in-situ cancer vaccine called CryoVaxTM and combines with CRCL to create a personalized cancer vaccine called AlloVaxTM.

Vaxil™ Bio Ltd., is an Israeli immuno-oncology biotechnology company developing novel immunotherapies including neoantigen-like peptide products, and antibodies to treat cancer and infectious diseases. Vaxil’s products are derived from its fully owned proprietary platform VaxHit™, which in effect allows for the identification, isolation, and production of uniquely specific antigen-based immunotherapy products. Vaxil’s products are derived from its fully owned proprietary technology VaxHit™, which uses the Signal Peptide (SP) domain in selected targets as core antigens (see technology section). Vaxil’s lead product, ImMucin, which has received FDA Orphan Drug Status, was designed for the treatment of multiple myeloma (MM) cancer. Immucin™ is composed of the entire SP domain of the MUC1 tumor associated antigen (TAA), and has successfully completed a Phase I/II clinical trial in 15 MM patients demonstrating a high safety profile, robust immunity and initial hints for clinical efficacy. Vaxil™ is currently performing an additional Phase I/II study with ImMucin in metastatic breast cancer patients being treated with 1st line hormonal therapy. Vaxil also isolated SPmAb-2.1 and SPmAb-6, the only anti-MUC1 SP antibodies harboring superior therapeutic and diagnostic properties vs. other MUC1 antibodies. Vaxil further validated its VaxHit technology for anti-infective indications, isolated MTBuVax™, a multi-antigenic sub-unit LP vaccine against mycobacterium Tuberculosis (MTb) currently in pre-clinical animal studies.

ImmunoBrain Checkpoint (IBC), Israel, is a biopharmaceutical company developing novel neuroprotective immune therapies for neurodegenerative diseases. IBC licensed its platform IP from Yeda, the commercialization company of the Weizmann Institute of Science in Israel. IBC developed a proprietary fully human anti-PD-L1 antibody, engineered based on its mechanism of action to treat Alzheimer’s disease and dementia. IBC plans to start its first-in-human clinical trial in early 2022.

Emosis, Israel, addresses thrombosis, a localized clotting of the blood leading to the obstruction of the arterial or the venous circulation, is a pan-vascular issue that crosses the borders of all medical specialties. To address hemostasis and thrombosis complexity in a clinically useful way, Emosis relies on flow cytometry, a powerful technique that revolutionized clinical hematology (e.g. phenotyping of leukemia) and immunology (e.g. HIV).Conventional assays, whether related to coagulation or platelet function, are bulk assays measuring average values over heterogeneous sets of molecules or populations of cells.  In contrast, flow cytometry enables a granular assessment of platelet activation through the measurement of 10 to 20 variables per single cells (e.g. platelets), over tens of thousands of cells (platelets) within second.

Biopharma has a major presence in the Boston-Cambrige area.

Eighteen of the top 20 Biopharmas have a major presence in Boston/Cambridge, and the region continues to welcome companies of all sizes. Boston/Cambridge ranks number-one in NIH funding (4,735 awards totaling $2.457 billion), VC funding ($6.162 billion in 156 deals), and lab space (26.8 million square feet). [Source.]  

Boston/Cambridge is the home to some of the best universities and medical facilities in the world.  Mass General, Brigham and Women’s and Beth Israel Deaconess are all top ranked nationally.  For higher education, there is Harvard, MI, Boston College and Tufts University.  It is no wonder that the Boston/Cambridge area is one of the top biotech hubs in the world.

My picks for interesting and innovative biotechs are:

Entrada Therapeutics, Boston, has developed a proprietary Endosomal Escape Vehicle (EEV™) Platform that is highly versatile and modular, designed to enable the efficient intracellular delivery of a wide range of therapeutics into a variety of organs and tissues with an improved therapeutic index.  Their novel approach is designed to address challenges associated with current therapeutics and engage targets previously considered inaccessible and undruggable.

Their current pipeline highlights trials for Duchenne Muscular Dystrophy, Myotonic Dystrophy Type 1, with preclinical products for Pompe and Neurodegenerative Diseases.

Karuna Therapeutics, Boston, strives to deliver more effective and better-tolerated treatments for large and underserved patient populations. Their lead product candidate, KarXT, is currently under development as an investigational treatment for schizophrenia and dementia-related psychosis (DRP).  KarXT, an oral modulator of muscarinic receptors that are located both in the central nervous system (CNS) and various peripheral tissues. KarXT is our proprietary product candidate that combines xanomeline, a novel muscarinic agonist, with trospium, an approved muscarinic antagonist, to preferentially stimulate muscarinic receptors in the CNS.

Checkmate Pharmaceuticals, Cambridge, is investigating the efficacy and safety of vidutolimod in combination with checkpoint inhibitors in patients with various types and stages of cancer, including melanoma and squamous cell carcinoma of the head and neck. Vidutolimod works by two complementary mechanisms that together have a unique ability to drive a strong systemic anti-tumor T cell response. First, the VLP activates an immune response to the VLP, leading to the production of antibodies that deliver the VLP into plasmacytoid dendritic cells (pDC) and other immune cells via specialized receptors called FcR . This provides an initial stimulatory signal to pDC and brings the CpG-A to TLR9 (the receptor for CpG DNA) inside the (pDC). Second, CpG-A stimulates TLR9 in a manner that induces significantly higher levels of type I interferons (IFN-α and others) in pDC resulting in a stronger anti-tumor T cell response, as compared to other innate immune activators.

Goldfinch Bio, Cambridge, is applying precision medicine to discover, develop and commercialize disease-modifying kidney disease treatments. Their approach enables them to uncover subsets of patients based on genetic and environmental factors within heterogenous kidney diseases. They have a robust pipeline of novel, clinical-stage, precision medicine product candidates, including or lead program, GFB-887 for TRPC5-mediated FSGS. All of our programs target kidney diseases with significant unmet medical needs.

oRNA, Cambridge, was built on groundbreaking research from Prof. Daniel Anderson’s lab at MIT, their oRNATM technology is the next revolution in RNA therapeutics: synthetic circular RNA efficiently generated by an elegant and proprietary molecular platform. oRNATM circular RNAs lack caps and tails and are always full-length, and are made by combining nature-driven insights with rational design. They are easily made, well-tolerated, and are engineered to express therapeutic proteins through original and proprietary mechanisms.

The potential applications of oRNA’s technology are far reaching, including cancer, autoimmune diseases, regenerative medicine, and genetic disorders. oRNAis currently evaluating where their medicines can make the biggest difference, with an initial focus on treating challenging diseases where other modalities have fallen short. As an initial focus, oRNA believes it can apply its oRNATM technology to potentially address the limitations of current immunotherapies by delivering chimeric antigen receptors (CARs) directly to patient’s immune cells within the body (isCARTM therapy).

Inhibikase Therapeutics, Boston, has a pipeline including multiple products developed from their proprietary RAMP™ drug innovation and prodrug technology engines. Their programs are focused on halting and reversing the effects of neurodegenerative diseases inside and outside of the brain.  They are also developing host-targeted therapeutics for neurological infections in the brain. Products in the current pipeline target Parkinson’s and the GI tract, chronic myeloid leukemia, progressive multifocal leukoencephalopathy

Toronto is one of the top cities for the human health sciences

Toronto is the largest city in Canada.  Whether ascending the CN Tower or visiting the Hockey Hall of Fame, there are many fun places to visit and great dining in the The 6.

Toronto is one of the top cities in North America for the human health sciences, combining research, clinical and business expertise to deliver new diagnostics and therapies. The city’s life sciences sector employs nearly 30,000 professional and contributes more than $2 billion to the local economy. (Toronto.ca/business-economy 2021).

Here are my picks for exciting biotech companies in Toronto:

Bresotec is a team of health experts and entrepreneurs that are alleviating the cost, complexity and health burden of sleep apnea. Their work provides healthcare professionals and their patients with a better, simpler sleep test that reduces the cost of sleep testing without compromising lab accuracy.

RNA Diagnostics has developed The RNA Disruption Assay™ (RDA™) that will provide physicians with an evaluation of how individual patients are responding to cancer therapy. If the cancer drugs are likely to provide the patient with a benefit, the physician and patient can continue treatment with confidence. If the cancer drugs are not likely to benefit the patient, physicians may consider alternate treatments. For patients, this means avoiding harmful side effects and increasing the possibility of improved survival outcomes.

Mannin Research, is developing a unique set of molecules to treat vascular related diseases, such as Primary Open Angle Glaucoma, Pediatric Glaucoma, Cystic Kidney Disease, and Inflammation via their proprietary research platform.  Their primary indication, MAN-01, is for treatment of Primary Open Angle Glaucoma, wherein they are developing a small molecule in the form of an easy to administer eye drop to reduce intra-ocular eye pressure (IOP), a key risk factor in the progression of Glaucoma.

Notch Therapeutics is improving on current T cell therapies that require a complex, patient-specific manufacturing process. The “vein-to-vein” processing of a CAR-T therapy leads to delays and variability that negatively impact patient outcomes. To fulfill the promise of a cell therapy that can be delivered as an off-the-shelf, fully characterized medicine, Notch Therapeutics is manufacturing T cells from induced pluripotent stem cells (iPSCs).  Their unique technologies and capabilities are rooted in foundational discoveries in developmental biology.

NoNO has long believed in the promise not only of reducing ischemic damage but to unlock the brain’s own capacity to recover from disease.  Their therapeutics have demonstrated potential to improve the lives of patients following ischemic stroke, traumatic brain injury, and other chronic neurological and neurodegenerative diseases.

Their lead drug candidate, nerinetide, is a first in class PSD95 inhibitor that promotes cell survival and disrupts toxic cell signals following damage to neurons. NoNO’s core technology and therapeutic targets have been validated in multiple laboratories worldwide and published in leading scientific journals.

Helix Biopharma has developed DOS47, based upon a naturally occurring enzyme isolated from the jack bean plant called urease that breaks down a natural substance found in the body, urea, into metabolites that include ammonium and hydroxyl ions.  By doing so at the site of cancerous tissues in the body, the Company believes DOS47 can modify the micro environmental conditions of cancerous cells in a manner that leads to apoptosis (programmed cell death).

Germany is becoming one of the standout countries in biotech.

Germany is famous for being the Land of Poets and Thinkers. From vital inventions to Christmas traditions, sausages and beer, Germany is home to plenty of culture, history and quirky laws! Germany is also known for its major cities, the Black Forest, the Alps and Oktoberfest. (Hey Explorer, 2020).  

Germany is also becoming one of the standout countries in biotech.  With large players such as Qiagen, Miltenyi Biotec, BioNTech and Evotec serving as anchors, smaller biotech’s are enabled to thrive. 

Germany’s biotech clusters create a variety of opportunities to transfer innovation into products, which should be attractive for investors, particularly since the EU is harmonized from a regulatory point of view and is one of the most important markets in the world. (Pinset Masons, 2021).

My picks for Germany are:

ORYX Translational Medicine, Vaterstetten, has developed strategies that bridge the gap between academic research and the pharmaceutical industry for new cancer therapies. Today, the company is developing three clinical cancer immunotherapy projects.  They have an oncolytic virus to infect and lyse tumor cells and evoke a strong anti-tumor immune response. In addition, they develop therapeutic vaccines to treat cancers by enhancing a patient’s natural defenses against the tumor.

Altyas Therapeutics, Amtsgericht Jena, is a spin-off of the University of Jena with the aim to develop novel immunological therapy concepts for the worldwide treatment of obesity, a condition with a large unmet clinical need.  The company has the relevant expertise and patents on various antigen epitopes with proven relevance to novel therapeutic immunological regulation of excessive amounts of adipocytes.

Medigene AG, Munich, is a publicly listed biotechnology company. With its scientific expertise, Medigene is working on the development of innovative immunotherapies such as T cell receptor-modified T cells (TCR‑Ts) or dendritic cell (DC) vaccines to treat cancer in fields of high medical need. The first product candidates are in clinical development.

Affectis Pharmaceuticals AGDortmund, is a pharmaceutical company developing novel drugs for the treatment of neurodegenerative and neuroinflammatory diseases.  Their capabilities in drug discovery and medicinal chemistry enabled the company to develop drugs with innovative mechanisms of action based on pioneering findings in the field of P2X7 receptors.  They are currently working on two products, one for neuropathic pain for MS and one for depression.

T2cureFrankfurt, is dedicated to the development of novel regenerative therapeutics based on mononuclear cells obtained from the patient’s bone marrow. These cells have the potential to initiate repair processes in the tissue by improving blood flow and releasing messenger substances that promote cell regeneration. Such repair processes are of particular importance in patients with recent myocardial infarction or chronic cardiac insufficiency as well as in patients with general circulatory disorders, such as the arteries of the legs.

Silence Therapeutics, Berlin, are developing a deep pipeline of innovative siRNAs based on their mRNAi GOLD™ platform for diseases with a genetic basis. The depth and versatility of their liver-targeting technology gives them the opportunity to address a wide range of conditions in virtually any therapeutic area. Their wholly owned pipeline is currently focused in three therapeutic areas of high unmet need: hematology, cardiovascular disease, and rare diseases.

Raleigh-Durham and the Research Triangle Park in North Carolina Is A Unique Innovation Biotechnology Hub

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.

Singapore Shines as a Biotech Star in the Heart of Asia

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.  

Ireland is Home to 24 of the World’s Top Biotech and Pharma Companies

According to the Silicon Republic, Ireland is a home for 24 of the world’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.

PerfuzeGalway, 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 Biotech

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, Philadelphiawas 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. 

The Top Five Areas Requiring Commercial Expertise

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.  

Biotech in the land down under – Australia.

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 MedicalSydney, 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.