What does new drug pricing legislation mean to clinical stage emerging biotechs?

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 15th 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.

Houston’s Life Science Hubs have produced many innovative companies

Having recently returned from BIO 2022, I came across an interesting publication on the Life Science market in Houston, TX.  They have divided the market into the following HUBS based on population:

-Pearland (Population 125,828)
-League City (Population 116,701)
-Sugar Land (Population 120,000)
-The Woodlands (Population 120,000)
-Conroe (Population 101,582)

These Hubs surround the Texas Medical Center anchored by MD Anderson Cancer Center and Texas Children’s Hospital.

I have chosen an innovative biotech company from each Hub.  My picks include:

Adient Medical, Pearland, is leading the development of an absorbable vascular filter for the prevention of pulmonary embolism (PE), a leading cause of death in the US claiming the lives of over 100,000  Americans annually — more than breast cancer, AIDS, and traffic fatalities combined.  The filter is inserted post PE or DVT and remains for 12 weeks to capture clots, then dissolves into carbon dioxide and water.

Jupeng Bio, League City, develops advanced bioenergy technology which takes wastes and sustainably turns them into advanced biofuel and renewable power.

Backed by decades of experience in developing and licensing industrial-scale technology, Jupeng Bio creates more sustainable communities and transportation by converting waste and non-food crop biomass into advanced biofuel. The Jupeng Bio technology solution uses a flexible approach that is safe, fast and reliable, allowing it to be commercialized wherever there is waste. By taking this localized approach, Jupeng Bio reduces landfill and air pollution, creates jobs, generates tax revenue and safely produces renewable fuel and clean energy.

Hope Biosciences, Sugar Land, develops adult stem cell-based therapeutics for a variety of indications  and is the only clinical grade stem cell banking facility in the nation that banks both adults and newborns.

Their core technology is the ability to make accurate copies of your own stem cells to unprecedented quantities. We start with one tablespoon of fat or approximately 100,000 mesenchymal stem cells and can culture (grow) these cells in a way that not only generates high amounts; but, maintains their original characteristics such as identity, purity, genetic stability, potency, differentiation capacity and phenotype.

In a nutshell, they have the ability to generate unlimited quantities of your stem cells that are the same as those we took from the body.

Lexicon Pharmaceuticals, The Woodlands, is a biopharmaceutical company that is applying a unique approach to gene science based on Nobel Prize-winning technology to discover and develop precise medicines for people with serious, chronic conditions. Using a patient driven approach, we are working to discover and develop innovative medicines to safely and effectively treat disease and improve patient lives.

Their robust pipeline includes candidates for heart failure, Type 1 diabetes, diabetic neuropathy, post-herpetic neuralgia, metabolism and immune system disorders.

VGXI, Conroe, is a full-service Contract Development and Manufacturing Organization (CDMO) that produces DNA plasmids for human clinical trials, virus production, toxicology studies and pre-clinical research.

VGXI delivers high purity pre-clinical plasmid preparations suitable for all stages of pre-clinical DNA vaccine.  They also provide a Highly Documented (HD) Plasmid Production Service provides high supercoil, low endotoxin plasmid.  Finally, they offer cGMP Production Service provides injectable-grade plasmid DNA suitable for use in human clinical trials.

China initiatives advance oncology therapeutics

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 an EU biotech stand-out

The CambridgeOxfordLondon 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’s “medical alley” leads in healthcare innovations

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 phenotypesHungry 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.

Colorado – Beauty Meets Biotech Hotspot

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 HyperthermiaTM (THT), an elegant, safe and effective cancer therapy which generates therapeutic heat within solid tumors using SivaRodsTM gold nanorods and a SivaLumTM 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

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.

Emerging Biotech Companies in Austin Texas

For the past ten years, Austin, Texas has been swiftly growing as a high-tech hub.  Oftentimes referred to as the Silicon Valley of the South, Austin has attracted giants in technology including Google, Apple and of course Dell was founded there.  In the past few years, Austin won the contract for the new Tesla Mega Factory.  Recent reports state that Tesla electric cars will begin rolling out of the new factory by the end of the year, bringing thousands of jobs to the area.

Another sector to see rapid expansion in the Austin area is biotechnology.  With a highly educated workforce, a major university, a new medical school and the desire for people to live in Austin, there is no shortage of skilled employees.  In addition, the state of Texas has dedicated funds to support cancer called the Cancer Prevention and Research Institute of Texas (CPRIT).  Since 2008, CPRIT has funded $2.8B in grants for Research, Prevention and Product Development Research for cancer in Texas.

Some standouts in the Austin small pharma/biotech industry include Cassava SciencesLumos PharmaSavara PharmaceuticalsXBiotechGenPrex, and Curtana Pharmaceuticals.

CASSAVA Sciences
In today’s Austin market, Cassava is the clear stand out with simufilam in two Phase III trials for Alzheimer’s Disease.  Simufilam improves the function of multiple brain receptors and exerts powerful anti-neuroinflammatory effects. A small company with a potential blockbuster on the horizon and CEO Rami Barbier and CFO Eric Shoen has done a brilliant job financing the company over the past two years. Put this company on your watchlist!

Lumos Pharma is a clinical-stage biopharmaceutical company committed to identifying, developing and commercializing life-changing therapies for patients and families living with rare diseases.  Their current candidate, LUM-201 is for PGHD – is in a Phase II trial for pediatric growth hormone deficiency.  Treatment of growth hormone deficient children is currently limited to daily injections of growth hormone. Missed injections reduce therapeutic effectiveness, resulting in suboptimal growth. Four other growth-related indications are in the pipeline. CEO Hawkins is a serial entrepreneur having built and sold several companies. 

Savara Pharmaceuticals is a rare lung disease company.  Their leading program, Molgradex, is an inhaled granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase III development for autoimmune pulmonary alveolar proteinosis (aPAP). CFO Dave Lowrence secured financing with Bain Capital which extends their runway beyond the Phase III readout. Savara/Molgradex could be the first FDA approved therapy for aPAP. 

XBiotech manufactures True Human™ antibodies that are derived from individuals with the most potent natural immunity to certain diseases. The company identifies unique genetic information of immune antibodies from donor blood. This genetic information is used for production of antibody therapies without modification.   This genetic information is used for production of antibody therapies without modification.   

GenPrex technologies are designed to administer disease-fighting genes to provide new treatment options for large patient populations with cancer and diabetes who currently have limited treatment options. The lead product candidate, REQORSA, will be administered with targeted therapies and with immunotherapies for NSCLC – non small cell lung cancer. 

Curtana Pharmaceuticals is developing highly targeted therapies for the treatment of brain cancer, including glioblastoma in adults and pediatric high-grade glioma and medulloblastoma in children.  They are advancing a novel small molecule therapy, CT-179, which has been shown to significantly prolong survival in relevant animal models. The target is Olig2, a cancer stem cell-associated transcription factor that is critical for tumor initiation and growth. CT-179 will enter clinical development in H1 2021.

Austin remains attractive to growing biotech companies.  Does it have the capacity to become a biotech giant?  If we factor in the relatively close proximity of its sister cities of Dallas, Houston and San Antonio, the horsepower ramps up significantly. 

World renowned universities such as The University of Texas, Texas A&M, Baylor and Rice, the top cancer center in the world (M.D. Anderson), and large pharmaceutical companies such as Allergan, AstraZeneca, Pfizer and Novartis, Austin is poised to become the hub for small to medium pharma/biotech.  Dallas takes large pharma, Houston excels in patient treatment and San Antonio leads in government facilities. 

Austin wins by tapping in to all three and building a world class platform fostering growth for smaller biotechs.  Locals like to say, “keep Austin weird” but biotech entrepreneurs should take a hard look at Austin!

Houston is at the heart of Texas healthcare and life science research.

Health care and life sciences are major industry sectors in Houston driven by world-class institutions and professional talent. Houston is home to the largest medical complex in the world, the Texas Medical Center, which provides clinical health care, research and education at its 61 institutions. Houston has more than 1,760 life sciences companies, cutting edge hospitals, health facilities and research institutions with a workforce of more than 320,000 people in health care, biotech and related fields in the area. (Source)

Here are my picks for innovative biotech companies in Houston:

Coya Therapeutics, Houston, is developing disruptive, first in class, autologous regulatory T-cell (Treg) and allogeneic exosome therapies – leveraging the seminal discoveries from the laboratory of Stanley Appel M.D., in which dysfunctional Tregs modulate neurodegenerative and autoimmune diseases.  

Coya’s pipeline targets devastating diseases such as ALS, Dementia and Alzheimer’s.

CAVU Biotherapies, Houston, provides immune-based solutions to treat cancer and autoimmune diseases in dogs. Their vision is to change the world by keeping pet families intact longer while becoming the global leader in immune-guided personalized medicine.  Their initial focus is on canine cancer, and then translating the personalized immunotherapies to treat dogs, cats and horses living with allergies, cancer and autoimmune diseases.

Saranas, Houston, has developed the Early Bird Bleed Monitoring System that detects bleeds for endovascular access procedures the moment they happen, helping to preserve patient outcomes and provide an additional level of assurance for the patients most vulnerable to bleeding events.

Tvardi Therapeutics, Houston, is focused on the development of inhibitors of STAT3, a key signaling molecule positioned at the intersection of many pathways controlling gene networks integral to the survival and immune sequestration of cancer cells as well as to the pathogenesis of many inflammatory and fibrotic conditions.

The company’s lead product, TTI-101, is currently being studied in a first-in-man Phase 1 trial of patients with advanced solid tumors who have failed all lines of therapy. To date, TTI-101 has demonstrated an excellent safety profile and clinical activity.

Greenwich LifeSciences, Inc., Houston, is a public biopharmaceutical company that is developing GP2, a novel peptide immunotherapy, and is planning to commence a Phase III clinical trial.

GP2 is derived from the HER2/neu protein, which is expressed in a variety of common cancers, including breast cancer.  Tumors with elevated expression of HER2/neu protein are highly aggressive, resulting in an increased disease recurrence and a worse prognosis.  In a Phase IIb clinical trial in the HER2/neu 3+ adjuvant setting, no breast cancer recurrences were observed after median 5 years of follow-up if the patient was fully immunized.  In addition, GP2 treatment is well tolerated and no serious adverse events related to GP2 immunotherapy were reported.

ImmunoGenesis, Houston, is bringing hope to cold tumor treatment.  Nearly 40% of people will have a cancer diagnosis in their lifetime. Four of the most common cancers resulting in cancer death in the United States are those that are considered cold tumors. The few treatments that are available have poor efficacy. Their platform may change this.

Their novel PD-L1/PD-L2 dual-specific inhibitor is a platform molecule around which several promising treatments for cold tumors can be built. This is the first antibody to target PD-L2. This vision puts ImmunoGenesis at the forefront of scientific exploration.

Built utilizing the platform, their lead asset was engineered with robust effector function. Another PD-L1/PD-L2 antibody from this platform is being developed as a tumor-selective delivery vehicle for our potent stimulator of interferon genes (STING) agonist.

Japan rises to the top as a leader in Biotech and Pharma

Japan, the Land of the Rising Sun, holds a number-two ranking in Asia, and leads the continent in biotech and pharma patents (14,414 listing at least one Japanese inventor, according to WIPO), as well as a firm number-two in R&D. (Source)

Here are my top picks for innovative biotechs:

Chordia Therapeutics, Fujisawa, is a start-up company that develops next-generation anticancer drugs based on solid scientific evidence. Their anti-cancer drug pipeline focuses on RNA regulation abnormalities that have been found as a new feature of cancer. Recent studies have newly discovered that RNA regulation is altered in cancer, and show that this RNA regulation abnormality is significantly involved in the development and progression of cancer.

Their lead asset, CLK CTX-712, carries out Phase I of First in Human.

United Immunity, Kobe, develops novel combination immunotherapies utilizing our potent anti-tumor T cell activating nano-immunotherapy “T-ignite”, we challenge conquest of intractable solid cancer, especially “cold tumor”, where conventional therapies are not effective for. The same technology is being used for the development of a next generation SARS-CoV-2 nanoparticle vaccine.

xFOREST Therapeutics, Kyoto is creating a paradigm shift in drug discovery with its unique technological assets “FOREST”, setting RNA structures as drug discovery targets. FOREST are technology platforms that integrate massively parallel biochemical analysis systems of up to 1 million species of RNA structures and in silico analysis pipelines. These comprehensive analyses include various RNA structures, regardless of their source: organ, species, or virus. As a result, the technologies have enabled the search for highly specific RNA-binders, empowering the rapid and efficient discovery of RNA-targeting drugs.

Rena Therapeutics, Tokyo, markets HDO, Hetero-duplex oligonucleotide developed by Professor Yokota Takanori of Tokyo Medical and Dental University.  This was developed to solve the inherent issues with nucleic acid drugs including:  delivery issues, toxicological issues and blood stability.

By establishing the third platform technology next to Ionis Pharmaceuticals, Inc (ASO) and Alnylam Pharmaceuticals, Inc (siRNA), we will contribute to the creation of new nucleic acid drugs, and to deliver therapeutic drugs to patients as soon as possible 

CYTLIMIC, Cytotoxic T Lymphocyte Immunotherapy in Cancer, Tokyo, is a biotechnology company developing immunotherapy products for the treatment of cancer, including a cancer peptide vaccine. CYTLIMIC’s products aim to activate immune systems to attack cancer cells while enabling patients to maintain a high quality-of-life during treatment.