Category: General

The biotechnology industry is a major driver of San Diego’s innovation economy. Major pharmaceutical companies including Eli Lilly, Pfizer, and Takeda maintain a presence in San Diego to foster collaboration with major research institutes, universities, and small life sciences companies.

These unique assets, combined with one of the most talented workforces in the world, make San Diego one of the top three life sciences markets in the world. (Source)

Cardiff Oncology, Inc. San Diego, is a clinical-stage biotechnology company leveraging PLK1 inhibition to develop novel therapies across a range of cancers.  They recently announced two poster presentations for the European Society for Medical Oncology (ESMO) Congress 2022, which is taking place both virtually and at the Paris Expo Porte de Versailles in Paris, France from September 9 – 13, 2022.

The abstracts include data from an ongoing Phase 1b/2 trial evaluating onvansertib in combination with FOLFIRI/bevacizumab in second-line KRAS-mutated mCRC.  In addition, an Expanded Access Program (EAP) in which patients with KRAS-mutated metastatic colorectal cancer (mCRC) who failed or progressed on standard-of-care, including irinotecan, were treated with onvansertib in combination with FOLFIRI/bevacizumab.

Mark Erlander has assembled an impressive team to bring this therapy to patients.

Adcentrx,  San Diego, looks beyond a one-size-fits-all platform approach to developing protein conjugates, such as ADCs. Their goal is to develop first-in-class and best-in-class therapies that broaden the therapeutic window in treating cancer and other life-threatening diseases. We do this by optimizing key components of protein conjugate molecular design to solve specific challenges at hand.

Putting this into practice, they created and continue to build the Adcentrx “Toolbox” to intelligently address the issues that arose in previous generations of conjugates. The components of the Toolbox encompass the targeting moiety, the linker, the payload and the conjugation technology.

Led by Hui Li, CEO and Pia Chatille Eid, Chief Science Officer, they currently have projects in the pipeline for oncology and immunology.

DtX Pharma, San Diego, is using RNA medicines to address countless disease targets, currently undruggable by small molecules or biologics, by inhibiting or altering the expression of disease-causing genes.

Historically, this class of medications, which includes siRNA and antisense oligonucleotides, has been hampered by challenges with drug delivery.

DTx Pharma FALCON™ technology addresses the fundamental challenges associated with delivering RNA therapeutics by leveraging fatty acid receptors for cellular uptake and leveraging fatty acid albumin interactions to promote biodistribution.  Our team has created a platform called FALCON™ (Fatty Acid Ligand Conjugated OligoNucleotide) that promotes efficient non-toxic cellular uptake and enhanced systemic bioavailability. They combine the latest in RNA technology with their proprietary IP to create novel therapies that can efficiently reach targets beyond the liver.

Led by CEO Arthur Suckow and CSO Brian Laffitte, Dtx is taking RNA therapeutics to new heights.

Wonderful, wonderful Copenhagen!  Companies in Copenhagen have a strong focus on medical applications in areas like cancer and diabetes, as well as a lot of activity in industrial biotech. (labiotech.eu)  

With all the progress made in oncology therapeutics, cancer drug resistance remains a large unmet clinical need.  Shining a spotlight on a biotech company aiming to understand the molecular and cellular processes associated with resistance.

Scandion Oncology, based on Copenhagen, Denmark, have developed a framework for understanding drug resistance.   Resistance may be an intrinsic property of some cells or clones in a tumor. Over time upon treatment with a cancer drug, these cells are enriched in the tumor, such that the entire tumor becomes resistant to the treatment (this is called intrinsic resistance).

Another concept of resistance is known as acquired resistance. Here, tumor cells progress through a number of genetic events. Accumulation of defects and genetic changes will eventually make the tumor cells resistant to treatment. Over time and upon treatment, only resistant tumor cells will remain, and the entire tumor will become resistant to treatment.   

Led by President and CEO Bo Rode Hansen and COO Maj Hedtjarn, Scandion has two products currently in development.  CORIST, for the treatment of drug resistant metastatic colorectal cancer is a clinical Phase II study. The second program, PANTAX, for the treatment of unresectable or metastatic pancreatic cancer is a clinical Phase Ib study.  In fact, this month they announced a  better-than-expected tolerability of its potential new cancer treatment SCO-101 in the dose-finding PANTAX phase 1b-trial.  Thus, dosing is now escalated to higher levels than expected based on the initial findings in the CORIST trial

Using their proprietary DEN50-R platform they can efficiently screen non-resistant and resistant cancer cells simultaneously, providing unprecedented insights into cancer drug resistance mechanisms in a streamlined fashion. 

Looking forward to more products from Scandion Oncology to accelerate effective cancer treatments.

The Eiffel Tower, Louvre, and Arch d’Triumph attract tourists to Paris from all over the world.  But did you know that Paris, City of Lights, has cultivated some interesting and potentially life changing emerging biotech companies.

For decades, the French biotech scene has been catching up to key strongholds such as the U.S. and U.K. In the aftermath of the COVID-19 pandemic, the French government last year bet €7.5 billion on an ambitious project to turn France into Europe’s health innovation magnet by 2030. 

Acticor Biotech, Paris, is a clinical stage biopharmaceutical company, a spin-off from INSERM (the French National Institute of Health and Medical Research).  The company are developing innovative drugs for the treatment of cardiovascular emergencies, in particular stroke.  They recently announced that they have received “PRIority Medicines” status from the European Medicines Agency (EMA) for its drug candidate, glenzocimab, for the treatment of patients with stroke.

Led by Gilles Avenard, CEO and Sophie Binay, GM and CSO, this strong team is filling an unmet need for promoty treatment of ischemic stroke.

EG 427, Paris, is a French biotechnology company that is pioneering a new approach in gene therapy called pinpoint gene therapy.  By exploiting the natural properties of Herpes Simplex Virus 1 (HSV-1), notably its ability to establish lifelong latency in peripheral neurons, EG 427 ensures highly specific and durable expression of the transgene to treat, in the long run, patients with severe, chronic and localized diseases, starting with peripheral nervous system disorders.  

Scientific Founder Charles Joussain, MD, PhD, Post-doctoral position as visiting researcher at King’s College London, University of Versailles Saint-Quentin, Paris-Saclay, France says, “HSV-1 virus naturally drives infection towards peripheral neurons, one of the many unique characteristics of this virus. We are taking this property to our advantage, using HSV-1-based gene therapy vectors to naturally target diseases that affect the peripheral nervous system, with the first on our list being neurogenic bladder.”

PEP-Therapy, Paris, is a French biotechnology company developing first-in-class peptides as targeted therapies in oncology.  PEP-010, first drug candidate, is a pro-apoptotic agent which has demonstrated anti-tumor efficacy in several pre-clinical models and a good safety profile.

PEP-010 is currently evaluated in a Phase Ia/b clinical trial in patients with recurrent and/or metastatic solid tumors. 

PEP-Therapy and Institut Curie are conducting a first-in-human clinical trial of PEP-010 for the treatment of advanced solid tumors and in particular Triple-Negative Breast Cancer (TNBC) and Ovarian Cancer (OC).

Led by CEO Antoine Prestat, the company is open to collaborations and partnerships with pharma and biotech partners, clinical organizations, academic institutions and investors to accelerate the development of PEP-010, as well as its pipeline of peptide-based products through co-development or licensing-in programs.

What does the Senate’s new drug pricing legislation mean to clinical stage emerging biotechs?   Here is a synopsis of the key changes to drug pricing by year, a Q & A about the legislation, implications to clinical stage emerging biotech’s and “my take” on it all. 

Note, the details of the legislation are scant right now so I’ll post an update in 30-60 days as more detail emerges. 

Implementation Dates

Below are some (not all) of the provisions of the just passed Senate bill by year of implementation. 

1. 2023: Companies will be taxed for annual price increases greater than the inflation rate, (begins Oct 1, 2022). Note, this applies only to drugs used for Medicare patients and does NOT apply to private health insurance.
2. 2024: Eliminates 5% co-insurance for Medicare Part D catastrophic coverage
3. 2025: Caps annual out-of-pocket Part D costs at $2,000 per year.
4. 2026: Medicare sets price on 10 Part D drugs.
5. 2027: Medicare sets price on another 15 Part D drugs.
6. 2028: Medicare sets price on another 15 Part D drugs and 15 Part B drugs.
7. 2029: Medicare sets price on another 20 Part D & Part B drugs.

Q&A

1. Will patients see any drug price savings before 2025? Effectively none what so ever and the 2025 savings are only on Medicare Part-D, (e.g., not private pay for <65y.o.). The new legislation does not require firms to offer negotiated prices to private payors.
2. In 2025, how will the reduction in the “donut-hole” cap of approximately $7,050 per year to $2,000 in Medicare Part D be paid for? Answer to this still pending and I will provide in ~30-60 days with other updates as more of the bills details emerge. 
3. What is earliest time we will see any reduction in expensive IV cancer, specialty, or rare disease drug prices? 2028
4. How many new drugs won’t come to market due to impacts of this legislation? The CBO estimates 15 which is not believable. The real impact will be at the venture capital and biopharma company budget level and risk tolerance will surely change – and dampen investment.  
5. What benefits can people less than 65 expect? Effectively nothing. The current form of the bill allows for larger price increases for private insurance patients so their drug prices could in fact increase.  

Implications to Clinical Stage Emerging Biotech’s:

1. API no more than 4%: If your revenue assumptions use an annual price increase greater than 4%, you should reduce it to 3-4% unless the target audience is primarily a <65 year old population. This “schism” between Medicare and private pay annual price increases will force companies to revise pricing strategy and also force precise metrics to understand how much drug is used in these two different patient populations with two different payors (e.g. Medicare and private pay).
2. Higher launch price: With potential for less revenue in the out years of the drugs lifecycle, companies will seek higher launch prices to get more value out of the early years. 
3. Currently in pivotal trial: If your lead agent is currently in its pivotal trial, you should engage Market Access & Pricing Reimbursement experts – I can get you started on that and a broader view of commercialization, email me at russ@bridge1.net.  

My Take: 

Increasing patient access to innovative drugs is critical to impacting their disease and well-being. Many lifesaving drugs are not taken because the cost to the patient is too expensive. While a $2,000 annual cap in Medicare Part D is a step forward, it does not apply to those with private insurance and under 65 years of age. 

The new inflation measure that begins in 2023 will likely push companies to launch with higher than otherwise expected prices to get the most value out of the asset until it hits expiry of Medicare price control.

If the bill’s provisions push BioPharma companies to increase R&D budgets to develop novel drugs in diseases with high unmet needs rather than developing the 15^th PD-1 inhibitor, I think most people (including me) think that is a good thing.  

Finally, implementing the provisions of the new legislation will take a massive amount of regulatory input and “rule writing” and you can expect BioPharma to make every effort to influence that outcome.   So, how exactly today’s new legislation is enacted in 2025-29 is yet to be determined. Stay tuned!

One More Thing

For additional insight about the Medicare pricing changes, read Rachel Sachs’ outstanding piece Understanding The Democrats’ Drug Pricing Package, featured in the August 8 issue of Endpoints News.

With the ASCO meeting just around the corner, I took a look at some interesting and innovative oncology companies in China.  

Two key Chinese government initiatives, both launched in 2016, have been key milestones in advancing the oncology treatment landscape and growth opportunities for advanced oncology therapeutics.

The Precision Medicine Initiative was announced created as part of a plan to pump ~$9 billion for research of human genetics and biology (40x the size of a similar initiative in the United States). Today, Shenzhen’s Beijing Genomics Institute (BGI) is already the world’s largest sequencer and repository of genetic material in cancer and beyond.

Complementing the precision medicine investments, Healthy China 2030 covers five focus areas to ensure improved health: public health services, environment management, the medical industry, and food and drug safety. Goals for this include increasing Chinese citizens’ average life expectancy from 77.3 to 79 years by 2030. Infant mortality decreased from to 9.5 deaths per 1000 live births in 2020 and 6.0 deaths per 1000 live births in 2030. (Source)

Here are my top emerging picks for China:

Ranok Therapeutics – Hangzhou, China and Waltham, MA – is a privately held

pre-clinical stage biopharmaceutical company that is pioneering an innovative targeted protein degradation technology for the discovery and development of novel therapeutics.

Chemically induced, targeted protein degradation (TPD) has recently emerged as an exciting new modality for small molecule drug development. Ranok has developed an innovative, next-generation TPD technology that engages different underlying biological processes and has unique advantages over competing TPD approaches. Ranok’s pipeline includes both well-validated and novel drug targets that are implicated in cancer and other diseases with significant unmet medical needs.

ImmVira Therapeutics, Shenzhen, is a biotechnology company focused on genetically modified oncolytic viruses as potential cancer therapeutics. The company has developed science, technology and know-how to support ongoing research, development and commercialization of oncolytic viruses on the OvPENS (OV+ Potent, Enabling, Novel & Safe) platform. The OvPENS platform comprises of research, patents, gene-recombinant know-how, manufacturing technology and commercialization analytics to develop next-generation oncolytic viruses that reach drug development targets and that are potent, enabling, novel and safe.

Abbisko Therapeutics, Shanghai, was founded in 2016 to address the #1 cause of death in China, cancer.  They have strategically designed and developed a rich pipeline of 10+ small molecule programs focused on oncology.  They have developed a strong pipeline of candidates in both precision oncology and immuno-oncology.

InnoCare Pharmaceuticals, Beijing, Nanjing, Shanghai, Guangzhou, New Jersey and Boston, are committed to discovering, developing and commercializing best-in-class and/or first-in-class drugs for the treatment of cancer and autoimmune diseases​. Led by a world-class management team of seasoned industry executives, we have built a fully integrated biopharmaceutical platform with strong in-house R&D capabilities, including drug target identification and verification, preclinical evaluation, clinical trial design and execution, drug manufacturing and quality control, and commercialization.

They currently have robust pipelines in liquid cancer, solid tumors and autoimmune diseases.

Hrain Biotechnology, Shanghai, is driven by the goal of relieving patients from diseases, focusing on R&D and application of tumor immunotherapy technologies and the transformation of the latest foreign tumor immunotherapy technologies.  They have always been an active promoter for innovative tumor immunotherapy technologies and a provider of advanced precision treatment solutions for cancer patients.

Hrain develops CAR-T technology as the center while CAR-NK, TCR-T and other technologies are being promoted in parallel. They have several CAR-T clinical trials registered on ClinicalTrials.gov, an authoritative platform for exchanges of clinical experience in America, and we also have multiple clinical centers located in Beijing, Shanghai, Guangzhou, etc.

Elpiscience, Shanghai and Germantown, MD, has a robust pipeline of globally innovative cancer immunotherapies, covering a wide range of oncology targets. Through internal R&D expertise and partnerships with global biopharmaceutical companies, Elpiscience endeavors to advance at least one innovative program into the clinic each year.

Elpiscience aims to transform cancer treatment by developing immunotherapies to turn “cold” tumors “hot.” They strive to find differentiated biology to enable effective crosstalk between innate immunity and adaptive immunity, harnessing the full potential of the immune system to fight cancer.

The Cambridge–Oxford–London Golden Triangle is now firmly established as one of the world’s leading concentrations of biotechnology and life sciences.  (Source)  In a recent article highlighting the Top 20 biotechs to watch in the EU, four were from Cambridge and Oxford.

My top picks for innovative biotechs in the Golden Triangle follow:

Excienta, Oxford, has a mission to design and develop novel, precision engineered drugs with an improved probability of clinical success for the benefit of patients.  Combining high-precision information from viable patient tissue, screened in their own laboratories, with carefully engineered CentaurAI systems, empowers target selection, precision design & experiment, as well as enhanced clinical assessment.

Arctoris, Oxford, Ulysses system enables peerless efficiency, accuracy, reproducibility, and above all full data and metadata capture from idea to IND.  They are automating the drug discovery development pipeline from Target Validation to Hit Finding to Hit-to-Lead to Lead Optimization to Preclinical.

CRISPIR Therapeutics, Cambridge, are focused on developing transformative gene-based medicines for serious human diseases.

They are rapidly translating their specific, efficient and versatile CRISPR/Cas9 gene-editing platform into therapies to treat hemoglobinopathies, cancer, diabetes and other diseases

Their multi-disciplinary team of world-class researchers and drug developers works every day to translate our CRISPR/Cas9 technology into breakthrough human therapeutics. Their lead program targeting the blood diseases β-thalassemia and sickle cell disease has entered clinical testing, as has our first allogeneic CAR-T program targeting B-cell malignancies. They are also advancing additional blood stem cell, immuno-oncology, regenerative medicine and in vivo programs towards the clinic.

Bit.bio, Cambridge, has developed breakthrough technology that combines synthetic and stem cell biology, offering limitless possibilities. Enabling a new generation of cell therapies, providing the best human cells for research and drug discovery, and allowing the control of advanced synthetic biology circuits for biomanufacturing.

Their opti-ox™ is capable of producing batches of every cell in the human body at scale and gives them the power to precision engineer human cells. This is because it reliably activates specific transcription factors within the cells.

Achilles Therapeutics, London, is a biopharmaceutical company developing precision T cell therapies targeting neoantigens: protein markers unique to each individual that are present on the surface of a cancer cell and can be detected by the immune system. Their approach is based on the science of tumor evolution which allows them to identify neoantigens that arise early in the cancer’s development and are retained as each tumor grows. These ‘clonal’ neoantigens are present on all the cancer cells and are completely absent from a patient’s healthy tissue. By directing T cells therapies to clonal neoantigens we can for the first time target every cancer cell without attacking healthy tissue.

Genflow Biosciences, London, has the potential to extend a healthy lifespan with groundbreaking science.

The SIRT6 gene is recognized as a master regulator of healthy ageing.  SIRT6 overexpression has been shown to lead to a healthy lifespan extension in both male and female mice.  Genflow Biosciences intends to promote healthy ageing by providing extra copies of a variant of the Human SIRT6 gene found in centenarians. Genflow Biosciences is developing an ethical, safe and efficient gene delivery system to deliver this centenarian variant of SIRT6 gene.

Minnesota, Land of 10,000 Lakes, is home to the prestigious Mayo Clinic.  The 5th largest company on Fortune’s list, UnitedHealth Group, calls Minnesota home, and other global leaders like Mayo Clinic, 3M, Medtronic and Boston Scientific are headquartered or have major operations here.

Minnesota is known as Medical Alley – the #1 Health Tech Cluster in the world. It is home to the nation’s largest private health insurer and more than 1,000 health care technology companies. The Smithsonian has recognized six “Great Places of Invention” in the U.S., including only one for health care: Medical Alley.

Here are my picks for the top innovative biotechs in Minnesota. (Source)

Panbela Therapeutics, Waconia, has developed SBP-101, a proprietary polyamine analogue that accumulates in the pancreatic acinar cells due to its unique chemical structure. It was discovered by Professor Raymond J. Bergeron at the University of Florida College of Pharmacy. Laboratory studies suggest the primary mechanism of action of SBP-101 is driven by its enhanced uptake in pancreatic cancer cells and, potentially, other cancer cell types, resulting in disruption of normal polyamine metabolism. SBP-101 is also taken up preferentially by the exocrine pancreas, the liver and kidneys. Importantly, pancreatic islet cells, which secrete insulin and are structurally and functionally dissimilar to acinar cells, are not impacted by SBP-101. SBP-101 has demonstrated significant growth inhibition of transplanted human pancreatic cancer cells in animal models.

Phenomix Sciences, St Paul, uses data-driven precision medicine to individualize obesity treatment.  They can predict how you will respond to different weight loss interventions through biological samples, patient assessments and machine learning.

Leading physicians from Mayo Clinic, Drs. Andres Acosta and Michael Camilleri, studied over 700 patients and found that obesity can be categorized as four different diseases requiring different treatments. We refer to these as obesity phenotypes: Hungry Brain, Hungry Gut, Emotional Hunger, and Slow Burn.Obesity phenotyping empowers providers to use precision medicine to individualize treatment, and was shown to double the amount of weight lost.

Tychon Biosciences, Minneapolis, is advancing precision cancer immunotherapy through a novel approach that transforms a patient’s immune cells into a “PAR-T” cell trained to find and eliminate cancer cells.

Despite its promise as a new cancer treatment, clinical adoption of T-cell based immunotherapy has been slow due to a variety of shortcomings. Our therapy delivers on this promise by addressing these shortcomings with a novel approach.

Our PAR-T technology can be used as a primary therapy or in combination with other therapies, opening up enormous untapped potential to treat early and late-stage cancers.

Their pipeline includes solid tumor breast and colon, cancer stem cells, leukemia/lymphomia and hybrid CAR/PAR.

Vyraid, Rochester, has created a new modality for the treatment of cancer.They have a broad portfolio of oncolytic viruses in discovery, translational and clinical phases of development.  The clinical program is focused on two engineered viruses: Voyager-V1, a vesicular stomatitis virus, and MV-NIS, an attenuated measles virus.  The clinical development program is designed to establish clinical proof of concept for oncolytic virotherapies as a monotherapy or in combination with immune checkpoint inhibitors.

A series of Phase 1 and 2 clinical studies are ongoing in multiple cancer indications with both Voyager-V1 and MV-NIS.  These trials are being conducted in partnership with leading pharmaceutical companies and top medical research centers, including Vyriad’s founding academic partners, Mayo Clinic and University of Miami.

They currently have products in the pipeline for ASSLC, head/neck, solid tumors, bladder cancer, multiple myeloma and endometrial cancer.

OX2 Therapeutics, Minneapolis has received clearance from the U.S. Food and Drug Administration to launch a phase 1 clinical trial with its new combination therapy for treatment of recurrent high grade brain tumors for which no curative therapy is available. OX2 Therapeutics developed the first of its kind peptide OX2 Therapeutics receives FDA approval for a Phase I Clinical trial to treat High Grade Glioblastoma platform that targets the activation receptor of the CD200 immune checkpoint. The peptide activates the immune system through a mechanism that modulates the suppressive effects of the CD200, PD-1/PD-L1 and CTLA4 immune checkpoints to allow a more robust anti-tumor response. “This single peptide has the potential to replace the toxic antibody therapies that are currently used to block these immune checkpoints,” said Drs. Moertel and Olin. OX2 Therapeutics intends to initiate a phase I single center, open-label, dose-escalation clinical trial in adult patients with recurrent glioblastoma. This will be followed by a pediatric trial for children with recurrent malignant brain tumors based on its safety and pharmacokinetic profile.

A recent report by professional services and investment management firm JLL found that Colorado—specifically the Denver and Boulder areas—are a rapidly growing area for biopharma. In fact, the JLL Life Sciences Report ranked the Denver and Boulder biotech markets as #13 in the country based on life sciences employment, employment growth, venture capital funding, National Institutes of Health funding and several other criteria. (Source)

My picks for cool biotechs in Colorado are:

Siva Therapeutics, Denver, is developing Targeted Hyperthermia^TM (THT), an elegant, safe and effective cancer therapy which generates therapeutic heat within solid tumors using SivaRods^TM gold nanorods and a SivaLum^TM infrared light device. THT has multiple beneficial effects on tumors, and it is more selective than chemotherapy, less destructive than radiation, and without the risks of surgical treatment.  This treatment promises to be minimally invasive, to reduce harmful side effects, and to stimulate the immune system.  In addition to being more affordable, this technology promises to deliver faster results than current cancer treatments.   

Edgewise Therapeutics, Boulder,  is a clinical-stage biopharmaceutical company focused on creating novel precision medicines for the treatment of rare muscle disorders. Their intimate knowledge of integrated muscle physiology at a whole-body level allows us to develop innovative solutions for patients with muscle disease where significant unmet medical need exists. By protecting and improving muscle health, their goal is to dramatically enhance the lives of people living with progressive muscle disorders.

Edgewise is currently focused on developing novel myosin targeted therapies for the treatment of Duchenne, Becker, Limb-Girdle muscular dystrophies, and McArdle disease.

Triopsy, Aurora, the Triopsy™ approach will create opportunity across three key market segments: medical device, pathology systems, and mapping systems for more accurate diagnosis. Our novel biopsy and pathology technology will assess more accurately the precise tumor location, exact size, disease type and stage, genomic analysis, and pathway to personalized medicine.

They believe their work will initially improve the lives of millions of men with prostate issues, and we will ultimately expand our work into many other areas where better accuracy, fewer errors, and lower costs can have a positive impact on health outcomes.

Brickell Bio, Boulder, has a current pipeline aims to disrupt existing treatment paradigms and features new molecular entities with first-in-class and best-in-class potential as treatments for autoimmune, inflammatory, and other high unmet need conditions.

Their current pipeline includes products for autoimmune diseases, Rheumatoid Arthritis, Type 1 Diabetes, Atopic Dermatitis, Neuroinflammation and Rare Genetic Diseases.

ARCA Biopharma, Westminster, Gencaro™ (bucindolol hydrochloride) is a pharmacologically unique beta-blocker and mild vasodilator that has potential for the treatment and prevention of recurrent atrial fibrillation, or AF, in patients with heart failure with mid-range ejection fraction, or HFmrEF.

Switzerland is one of the best and most innovative locations for biotechnology in Europe. Local companies hold leading positions throughout many sectors and thus attract capital and researchers from all over the world. Switzerland offers an outstanding framework for procuring capital. (https://www.s-ge.com/en/publication/fact-sheet/biotechnology).  Anchored by Roche and Novartis, Switzerland is perfectly poised to support innovative biotech companies.

My top picks for Switzerland:

FoRx Therapeutics, Basel, are developing compounds against targets involved in the repair of collapsed DNA replication forks.  Synthetic lethality refers to the genetic principle in which the combination of two genetic perturbations is lethal, where each individually is not. Oncogene-induced DNA replication stress, a hallmark of cancer⁷, makes cancer cells dependent on repair pathways for collapsed DNA replication forks. FoRx Therapeutics is developing small chemical inhibitors of these repair pathways to specifically target cancer cells, while sparing normal cells.

Scailyte, Basel, focuses on the discovery and development of biomarkers for precision medicine using single-cell data and AI fully exploits the power of cutting-edge technologies in translational research. They are paving the way for the development of new life-saving diagnostics and next-generation therapeutics for complex diseases.

AC BioScience, Ecublens, is developing innovative approaches in the way they develop and apply new vascular normalizers that help drugs to better access solid tumors and oxygenation, thereby increasing the efficacy of all types of chemo and radiotherapy.

Secondly, with a Beta-carboline derivative showing outstanding pre-clinical results, their aim is to revert the tumor phenotype in order to improve MHC-1 mediated antigens presentation, and thereby potentiate the efficacy of anti PD-1 therapy.

AMAL Therapeutics, Geneva, have developed a proprietary KISMA® Platform, that can uniquely and simultaneously provide all the parameters essential for an efficient therapeutic vaccine.  It can induce helper and cytotoxic T cells, promote immunological memory and target a broad patient population (different antigen and HLA restriction).

KISIMA^® immunization is shown to work in synergy with different immune checkpoint inhibitors in several tumor models.  KISIMA^® technology can be used to deliver tumor-associated antigens, neo-antigens and modified epitopes.

HAYA Therapeutics, Lausanne, has developed long, non-coding RNAs, IncRNAs, which are a unique class of regulatory molecules that bridge the analog world of proteins with the digital world of nucleic acids both DNA & RNA to regulate disease associated cellular processes in response to the environment.  HAYA has developed proprietary tools and methods to interrogate lncRNA biology, identifying exquisitely tissue- and cell-type specific therapeutic targets.

Their proprietary drug discovery engine enables us to discover and generate a pipeline of lncRNA targeting anti-fibrotic candidates for many tissues including lung, kidney, liver and the tumor microenvironment. These lead targets have the potential for significantly greater efficacy, safety and accessibility than existing treatments.