St. Louis, Missouri is one of the fastest-growing and top-ranked emerging life sciences markets in the U.S., ranking sixth in a recent CBRE Research report. In terms of BioSpace’s hotbed, St. Louis falls into the very large BioMidwest, which includes Illinois, Indiana, Iowa, Michigan, Minnesota, Nebraska, Ohio, Wisconsin and, of course, Missouri.

St. Louis’s life sciences industry is built on the presence of several top academic institutions and commercial organizations, Washington University in St. Louis, BJC HealthCare, St. Louis University, Bayer (formerly Monsanto), University of Missouri-St. Louis, Donald Danforth Plant Science Center and others. (Source)

Here are 5 interesting biotech companies in STL:

Wugen, St Louis, MO, is developing off-the-shelf cellular therapies for cancer.  Their cell therapy products originate from healthy donors and are further manipulated in order to enhance their function to eliminate cancer cells. Our NK cell and CAR-T immuno-oncology therapies address the needs of patients with solid tumors, acute myeloid leukemia (AML) and T-cell malignancies.

They have NK cell therapies targeting solid tumors and hematologic malignancies and CAR-T cell therapies targeting T-cell malignancies.

Their issued and pending patents support and protect a strong pipeline of convenient, off-the-shelf therapies that are developed to treat patients with cancer.

APT Therapeutics™, St Louis, MO, is developing optimized human apyrases as safe, effective, and cost-competitive antiplatelet therapy for acute myocardial infarction, stroke and transplantation complications. Their business strategy is to out-license or co-develop the drug candidates with pharma partners.

APT’s core competencies address critical roadblocks that drug companies encounter during all phases of development including: 1) making choices on drug leads 2) rescuing drugs that failed and 3) extending the life of blockbuster drugs.

Accuronix Therapeutics, St Louis, MO, is a leader in discovering and developing a new class of drugs that work by selectively targeting the σ-2 (sigma-2) receptor on cancer cells to deliver cytotoxic payloads. The Accuronix team has established an innovative platform and a lead drug candidate based on advances in characterizing the molecular-targeted drug conjugate (MTDC) approach to cancer therapy.  The σ-2 receptors are highly expressed on the surface of rapidly proliferating cancer cells. They internalize upon ligand binding, allowing the delivery of therapeutic agents to intracellular targets. The result is a chemotherapeutic with increased effectiveness and reduction in toxicity risk.

Mobius Therapeutics, St. Louis, MO, is dedicated to developing sterile injectable pharmaceuticals optimized for ophthalmic applications. With a deep pipeline of products under development, we at Mobius continue our mission of improving the convenience, safety, and integrity of perioperative pharmaceuticals by seeking innovative methods of delivery on our mission to be your one-stop shop for FDA approved peri-operative drugs.

Vir Biotechnology, St Louis,MO, is a commercial-stage immunology company focused on combining immunologic insights with cutting-edge technologies to treat and prevent serious infectious diseases. Infectious diseases are one of the leading causes of death worldwide and the cause of hundreds of billions of dollars of economic burden each year. They believe that now is the time to apply the recent and remarkable advances in immunology to combat infectious diseases. Their approach begins with identifying the limitations of the immune system in combating a particular pathogen, the vulnerabilities of that pathogen and the reasons why previous approaches have failed. They then bring to bear powerful technologies that we believe, individually or in combination, will lead to effective therapies.

They are taking a multi-program, multi-technology platform approach, assembled through internal development, collaborations, and acquisitions.

Oregon is bordered by Washington State, Idaho, Nevada, California, and the Pacific Ocean.  It is known as the perfect state to enjoy the great outdoors.  From the famous Mt Hood to Crater Lake, the deepest in the U.S., Oregon is also a rising hub for biotech.

According to the Oregon Bioscience Association, the state’s biotech community boasts a total economic output of $10.7 billion, while its employee base stretches over 47,000. Evidently, Portland’s life sciences sector leads the state in job growth, further bolstering its burgeoning tech ecosystem.

UbiVac – Portland, OR, is a clinical stage immunotherapy & cancer target discovery company, with first-in-human combination immunotherapies that include the newly discovered non-mutated shared cancer neoantigens 

UbiVac has unique and disruptive immune educating and activating vaccine technology. UbiVac’s lead agent, DPV-001, contains the newly discovered non-mutated shared cancer neoantigens and more than 300 antigens for the majority of patients with adenocarcinoma or squamous cell cancers. Due to its unique platform vaccine technology, novel targets are being discovered that might be used for ADC, CAR-T/NK, and TCR gene therapy. UbiVac postulates that by applying machine learning, artificial intelligence, and generative adversarial networks to the analysis of the extensive monitoring data from these ongoing first-in-human trials, new insights will allow future trials to tailor treatment regimens and further improve outcomes of patients with cancer.

AgonOx -Portland, OR, is in the process of identifying new immunotherapy targets by understanding the complexity of human tumor infiltrating lymphocytes (TIL) isolated from several different tumor types. Through our close alignment with the Earl A Chiles Research Institute at the Providence Cancer Center we have evaluated over a hundred tumor specimens obtained from surgery. We process these samples as soon as they are obtained from the operating room in order to keep the immune phenotypes intact, which avoids changes that can occur during freezing or resting. Differences in RNA array data have identified genes that are upregulated in either the activating and suppressive immune populations within the tumor microenvironment. From this information we design and develop recombinant proteins, antibodies, or small molecules that block and/or activate novel immune-specific pathways and generate tumor-specific TIL to target tumors.

Veana Therapeutics – Portland, OR has developed VIMO technology which is based on orally bioavailable anti-cancer agents that selectively attack and destroy the energy powerhouses (mitochondria) of cancer cells in a fashion that subsequently stimulates the immune system. 

Veana’s VIMO platform of α-TEA salt form-based products can be combined with other forms of immunotherapy to further improve anti-tumor activity.

Enosi Therapeutics – Eugene, OR, has developed an AI-driven (in silico) method capable of optimizing affinity of ligands to their binding site(s) to receptor:Fc fusion proteins capable of making their ligand-trapping comparable to the cell membrane-associated receptors, and therefore better ligand traps than those existing today. Enosi’s TRAP technology can be applied to soluble or cell-associated targets (or both) in a monospecific or multispecific format. TRAPS avoid the common toxicity associated with receptor-targeted antibody therapeutics which occurs when the receptor is not overexpressed on the target tissue. This occurs via ADCC or because of inappropriate shut-down of receptor function in normal tissues. This hurdle has been particularly encountered for all anti-EGFR antibodies and limits their utility.

Single-Armed Antibodies: Enosi is developing single-armed antibodies in cases where bivalent antibody-mediated receptor clustering is not wanted because of potential activation. Whereas transient activation of receptor function is commonplace with antagonist antibodies, like trastuzumab/Herceptin, in the case of TNFR1 this may be disastrous if the cytokine cascade/storm is activated. Thus, a TNFR1 molecule must be designed that does not trigger clustering and is not a significant agaonist.

SIGA – Corvalles, OR, is s a public, commercial-stage pharmaceutical company focused on providing solutions for unmet needs in the health security market that comprises medical countermeasures against chemical, biological, radiological, and nuclear (CBRN) threats, as well as emerging infectious diseases. The company is headquartered in New York City, with research and development facilities in Corvallis, Oregon.

Their product, TPOXX® (tecovirimat) that is available in oral and intravenous formulations, is approved by the U.S. Food and Drug Administration (FDA) for the treatment of smallpox. The European Medicine Agency (EMA) has approved oral TPOXX (under the name Tecovirimat SIGA) for the treatment of smallpox, monkeypox, cowpox, and vaccinia complications following vaccination against smallpox.

The BioCapital Biotech Hub is a cluster of high performing biotech companies in Delaware, Maryland, Washington D.C., and Virginia.  The BioCapital™ community ranks third in the life sciences industry. It is home to the second most biotechnology, medical device, and pharmaceutical companies of all major groups. This region is 3rd in NIH funding as well as patents but 4th in terms of lab space and 9th in terms of jobs. ( https://www.excedr.com/)

Here are 5 companies to watch in the BioCapital Hub:

Vanda Pharmaceuticals, Washington, D.C., is a global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs.  They have two products on the market for psychosis and sleep-wake disorders.  They also have a robust pipeline for a variety of indications including sleep disorders (including jet lag), bipolar and Parkinson’s psychosis, hematologic malignancies, social anxiety, and atopic dermatitis.

Rivus Pharmaceuticals, Charlottesville, VA, is a leader in mitochondrial biology, is advancing a new class of medicines designed to address a primary driver of cardio-metabolic diseases: obesity. Harnessing the natural process of mitochondrial uncoupling, Rivus’ Controlled Metabolic Accelerators (CMAs) provide a new, measured approach to reducing fat throughout the body. By doing so, CMAs can reduce the risk of cardio-metabolic disease with the aim of enabling healthier lives for millions.

Indivior Pharma, North Chesterfield, Virginia, has a mission is to discover and develop innovative medications that help transform patient lives through our industry-leading understanding of addiction and the chronic conditions and co-occurring disorders of addiction.

Indivior, is pioneering the development and discovery of non-opioid treatment strategies for OUD, as well as exploring new therapies to potentially address the needs of people suffering from alcohol, stimulant and cannabis use disorders. 

Indivior’s R&D has led to a range of daily and monthly treatment options for patients with moderate-to -severe opioid use disorder. We also developed a monthly treatment option for the treatment of schizophrenia in adult patients. 

Diffusion Pharmaceuticals, Charlottesville, VA, is an innovative biopharmaceutical company developing novel therapies that enhance the body’s ability to deliver oxygen to the areas where it is needed most.

Their lead product candidate, Trans sodium crocetinate (TSC), is a first-in-class, small molecule therapeutic with a novel mechanism-of-action that enhances the diffusion of oxygen to hypoxic tissues.

Adaptive Phage Therapeutics (APT), Gaithersburg, MD, is a clinical-stage company founded to provide an effective therapeutic response to the global rise of multi-drug resistant (MDR) pathogenic bacteria.APT’s approach arms us with the unique capability of adapting to the emergence of future antibiotic-resistant superbugs. Their phage bank’s precision targeted, genomically screened, and highly purified phage collection is dynamically expanding in response to the emergence of new strains of bacterial superbugs.

Salt Lake City, Utah

According to their web site, BioHive works closely with BioUtah to foster community, progress, innovation and transparency as the trusted source for the life science community.

Here are some interesting companies exploring new treatments in SLC.

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

Their robust pipeline includes candidates for the following disease states:  

Cerebral Cavernous Malformation, Neurofibromatosis Type 2,
Clostridioides difficile Colitis, Familial Adenomatous Polyposis,
AXIN1 or APC Mutant Cancers, Cancer Immunotherapy, Target Delta,
HR-Proficient Ovarian Cancer, RBM39,
Cancer Immunotherapy Target Alpha, MYC-Driven Oncology

JSK Therapeutics – JSK Therapeutics (JSKT) was founded in order to develop new anti-cancer therapeutics with fewer side effects.

The lead compound they will bring to market is JS-K, a first-in-class nitric oxide (NO)-generating compound of the arylated diazeniumdiolate class. JS-K was developed through collaboration between the team of Dr. Larry Keefer at the National Cancer Institute (NCI) and Dr. Paul Shami’s laboratory at the University of Utah.

KNOWN Medicine – Has developed the ODIN™ platform, a predictive engine built to understand how any cancer patient will respond to any drug treatment. They have received hundreds of patient samples into their lab and created thousands of organoids from each one. These organoids– the M3DUSA™ models– are dosed with anticancer drugs (chemo-, targeted-, and immuno-therapy) to identify the best treatment course for each cancer and to select the most effective drugs to advance through clinical trials.

Q Therapeutics – Has adopted a unique approach to developing human cell-based therapies for debilitating – and currently incurable – diseases of the brain and spinal cord (the central nervous system, or CNS). Our revolutionary approach focuses on glial cells – non-neuronal cells in the CNS that are essential for supporting, repairing and maintaining normal neuron function and health.

Pyrexer Medical – The global leader in superficial and deep regional hyperthermia systems, developed as an adjunct solution, for the treatment of solid cancer tumors. Pyrexar RF uses non-ionizing radiation to excite surrounding cellular tissue to “fever” temperatures to increase effectiveness when used with radiotherapy.

Halia Therapeutics are discovering and developing novel therapies to improve the lives of patients with inflammatory disorders and neurological diseases. They do this by advancing innovative medicines that target the immune system’s response to resolve chronic inflammation and eliminate the damage caused by aberrantly activated immune responses.

They have a robust pipeline for NEK7 Inhibitors for gastroenterology, ophthalmology, neuroinflammation, intervertebral disc disease and dermatology.  They also have LRRK2 inhibitors for Parkinson’s Disease and Alzheimer’s Disease.

I had a morning paper route as a kid and on Saturday’s I finished delivering papers by 7am and made bacon and eggs for breakfast – no one else was awake yet.

My grandfather loved food and used to call me into the kitchen and say, “hey, try this”. He subscribed to Gourmet and I paid attention. Ten thousand Saturday morning cooking shows later and a lot experimenting, I developed some skills and infinite quest to try new things.

When my wife and I were first married we went to see her grandmother in Henderson Texas and she would always make the best fried chicken for lunch and flan for dessert. She was in her late 80’s. The flan was always made when we got there and I didn’t quite pay enough attention to the fried chicken recipe albeit I stood there in the kitchen watching her make it. 

It was always something I knew I would come back to and recently, I’ve been trying to replicate the fried chicken and flan. 

Fabulous Fried Chicken 

I began tackling the chicken by surveying some of my favorite chefs fried chicken recipes including Emeril, Bobby Flay, Stephen Pyles, Dean Fearing and Cynthia Paulson. I found some common themes of using a marinade and either an egg or butter milk wash. But none of the early attempts hit the mark. Flavor was “good” not great and texture was off – oil wasn’t hot enough.

Marinade

I use a product called “Better Than Boullion” to flavor short ribs and when I discovered they had a chicken stock, I tried that on the Fried Chicken, (6-7 hours marinading). Flavor went from good to “great”!

Chicken Size

I did some fried chicken “marketing research” at bay area restaurants and noticed a theme of smaller pieces than the full bone in chicken breasts I had been using. So, I cut those

same breast in two and also increased the depth of the cooking oil (Crisco of course) and made sure the cooking temp staying very close to 350 degrees. Those changes created perfection – several times now! 

Physics of Flan

I found a great recipe but it was designed for small individual portions and I wanted to make a large portion that could be served at any size.

With that I had to explore different types of “flan pans” and ultimately landed on a nine inch square ceramic dish (2 inches deep). 

I then had to up the volume of the recipe by 50% and also figure out the cook time, (75 min at 325).

Lastly, perfecting the caramel took some attempts to understand how much time to continue cooking after the first signs of caramel color appears (about 1 min). 

After the all experimenting above, my wife finally proclaimed “ this is as great as Grandma Cheek’s” and that’s when I knew I got it! I wrote the recipes down….

In 20 plus years working exclusively in the emerging biotech space, screening hundreds and hundreds of companies each year, I’ve seen only 1-2 “Commercial Advisors” listed on the company websites.

Some Tough Love for a Tougher Emerging Biotech World

There are too many capital constrained companies out there trying to develop the 20^th PD-1 inhibitor, 25^th BTK inhibitor and 30th CAR-T cell therapy targeting CD-19/20 lymphoma. What is the probability of technical success that their agent will succeed in achieving a new standard of care (SOC) vs the other 10-15 agents trying to do so?  

And if the agent does not achieve a new SOC, how will it compete for market share vs Merck, BMS, Genentech BioOncology, AZ, AbbVie etc. and navigate the brutal world of market access and formulary positioning as a “Small-co”? Who onboard is helping them avoid those pitfalls?

IRA Impacts on Oral Formulations

The IRA is imposing new limitations on orphan drugs and oral compounds for indications in the Medicare population especially cancer, diabetes, autoimmune and Alzheimer’s diseases. Companies need to understand how the value equation has shifted with the new legislation. If your company is focused on small molecules and/or orphan indications, how has your company responded to the IRA legislation over the past 9 months?

First Time CEO’s

While not a 100% of the time, the overwhelming number of first time CEOs (many times a PhD founder) have never been anywhere near commercialization or a commercial launch. And with that, it is striking to see such a lack of curiosity about commercial assessments informing portfolio decisions and commercialization – they don’t know what they don’t know but aren’t asking any questions. And why isn’t the board coaching the CEO to gain those insights?

Many are hoping for a buy out before they have to cross the commercial divide and only begin commercialization after the pivotal trial read out. Thus, investing too little too late in commercialization which inevitably leads to launch failure and silly statements to the analyst community like, “we didn’t anticipate the evolving market dynamics”.

On the Other Hand,…

To be clear, some companies incorporate commercial assessments and commercial strategy very early, and I’ve had the privilege of working with some of them. Recent examples include Revolution Medicines, Bicycle Therapeutics, Morphimmune, Onconova, ALX Oncology, KBP Biosciences and Steve Pott’ new stealth cancer company Anticipate Bioscience. All have strong leaders, are continuously problem solving in multiple dimensions, invest early for commercial success and most have a commercial expert (or two) on their board. 

So maybe in the not-too-distant future, a majority of emerging biotech companies will place a commercial expert on their board and/or add a commercial advisor to their ad board early in clinical development and leverage what is knowable about commercial value before they deploy their precious development capital and begin commercialization early enough to win.

…just advocating for a friend…

Several well-known institutions are based in the Seattle area, such as Fred Hutchinson Cancer Research Center, Allen Institute for Brain Science and the Institute for Systems Biology, along with startups including Adaptive Biotechnologies and Sana Biotechnology, launched recently by former executives from Juno Therapeutics, another Seattle company that was acquired by Celgene for $9 billion. The Bill & Melinda Gates Foundation and the UW also help bolster the growing local life sciences industry. (Source)

Here are some really cool biotech companies in Seattle.

Presage Biosciences – Their patented CIVO® (Comparative In Vivo Oncology) technology delivers microdoses of multiple drugs to different regions of a tumor, while the tumor is still in the patient. Observing molecular and cellular responses to drugs and combinations in a naturally occurring tumor has the potential to provide a more detailed understanding of tumor biology. 

ImmuSoft – Engineered B cells as biofactories for therapeutic protein delivery, a novel approach that they have pioneered. Their approach, called Immune System Programming (ISP™) entails collecting a type of the patient’s immune cells, called B cells. In response to immune stimulation, B cells can turn into biofactories (known as plasma cells), each of which manufactures and secretes thousands of antibodies per second. ImmuSoft harnesses this biofactory capability of the plasma cell by programming B cells to produce personalized protein therapeutics.

Shape Therapeutics – Using the unlimited potential of AI & RNA.  Combining Artificial Intelligence and RNA technology to make programmable RNA medicine available to everyone.

Cyrus Biotechnology – A pre-clinical-stage biotech company combining protein design and screening to create novel biologics for serious unmet medical needs. Using this approach, they are developing an early pipeline of innovative programs in multiple indications. They are partnering with leading biotech and pharma companies and research institutes to bring collaborative programs forward from discovery to the clinic.

In the pipeline, they currently have partnerships with Taktos Therapeutics for rare diseases, Selecta Biosciences for autoimmunity, CRISPR# with the Broad Institute as well as in-house programs.

LifeSci NYC is driving investments in the biotech sector for New York City.

Talent pool, technologies, academic centers—plus space, funding, and diversity. Together, these are the foundations of LifeSci NYC, the City’s initiative, led by New York City Economic Development Corporation (NYCEDC), to carry out the billion-dollar investment and establish NYC as the global leader in life sciences. (Source)

Here are some up-and-coming biotechs in NY, NY:

In8bio– Their DeltEx platform has enabled a deep pipeline of preclinical and clinical product candidates, which are designed to effectively target and potentially eradicate disease, to improve patient outcomes.  Key elements of our approach to treating cancer include:

• Advanced expertise in the manufacturing of ex-vivo, expanded, activated gamma-delta T cells
• First-in-class proprietary gamma-delta T cell engineering for chemotherapy resistance
• Advanced next-generation, closed-system, scalable, gamma-delta T cell manufacturing
• Broad applicability of our engineered DeltEx cells across multiple solid tumor indications

Inspirna –  Using a novel discovery platform, RNA-DRIVEr™, developed by Inspirna’s founding scientists at Rockefeller University, and now exclusively licensed to Inspirna, we are developing first-in-class small molecule and biologic therapeutics that target cancer drivers in high unmet need oncology settings.​

Their current pipeline includes novel drug candidates for colorectal cancer (RGX-202) as well as small cell lung cancer and non-small cell lung cancer (RGX-104).

Olatec – A privately held biopharmaceutical company developing a platform of safe, oral NLRP3 antagonist therapeutics, to treat and prevent a broad spectrum of acute and chronic inflammatory diseases that are known to be mediated by Interleukin-1 (IL-1), including: arthritis, heart failure, asthma, Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, spinal cord injury, type-2 diabetes, melanoma and breast cancers, among others.
 
By selectively targeting NLRP3, the body’s first line response in the innate immune system, Olatec’s lead compound, dapansutrile (lab code: OLT1177®) inhibits the production of pro-inflammatory cytokines IL1β and IL18.  

Promontory Therapeutics is developing novel small molecule immunogenic anti-cancer agents. Our lead agent, PT-112, is a highly potent immunogenic cell death (ICD) inducer, currently in Phase 2 clinical development.

Tumors that originate in or metastasize to the bone are difficult to treat and may cause severe pain. This compromises quality of life and presents a drug development challenge.  PT-112’s pyrophosphate component has affinity for bone (osteotropism). This provides a rationale for addressing cancers, such as metastatic prostate, lung or breast cancer, and hematological malignancies, such as multiple myeloma.

Scopus Biopharma – Their lead development program is a novel, first-in-class, targeted immuno-oncology gene therapy for the treatment of multiple cancers. We have partnered with City of Hope for CpG-STAT3siRNA, or CO-sTiRNA™, a STAT3 inhibitor. In pre-clinical testing at City of Hope, CO-sTiRNA™ has successfully reduced growth and metastasis of various pre-clinical tumor models, including melanoma, and colon and bladder cancers, as well as leukemia and lymphoma. CO-sTiRNA™ is initially being developed for the treatment of B-cell non-Hodgkins lymphoma.

In collaboration with City of Hope, Phase 1 clinical trials for additional cancer indications are being contemplated for CO-sTiRNA™ in combination with immune checkpoint inhibitors and chimeric antigen receptor T-cells, or CAR-Ts.

This month, the FDA has announced the creation of a new Cell and Gene Therapy office to manage the number of new candidates coming into the agency. (scienceboard.net)

Of the 22 cell and gene therapies approved by the FDA, 14 are cord blood-based treatments. The remaining include two gene therapies, seven cell therapies, and five gene modified cell therapies. In total, cell and gene therapies represent just 7% of the 340 approved biologics.

There are currently over 1,000 ongoing clinical trials for cell and gene therapies registered with ClinicalTrials.gov. Of these, the vast majority are in Phase 1 and 2 trials, meaning that they are still far from commercialization, and most will not reach approval. (Source)

Here is a look at some interesting therapies in the approval pipeline.

Atara Biotherapeutics is a leading allogenic T-cell immunotherapy company pioneering the development of transformative therapies for patients with serious diseases by leveraging our novel allogenic Epstein-Barr virus (EBV) T-cell platform.

They currently have a clinical trial Phase 3 ongoing for Tabelecleucel (tab-cel®) for patients with Epstein-Barr virus associated post-transplant lymphoproliferative disease as well as other EBV-associated diseases.  

Treos Bio is a clinical stage company introducing a disruptive computational technology to change the paradigm of cancer immunotherapy development by resolving the dual challenges of patient and tumor heterogeneity. We use proprietary therapeutic peptides, produced through commercially scalable processes without need for tumor biopsy, to stimulate clinically effective cancer-specific immune responses in patients predicted by our diagnostic tests to respond.

Their PEPI Panel  is a unique approach for personalized cancer immunotherapy generation, their Peptide Library (PEPI Panel) currently contains 3,286 proprietary, immunogenic peptide fragments from 184 tumor antigens associated with 19 cancer indications that induce immune responses in the largest subpopulation of subjects, based on HLA genotype (both class I and II) of 16,000 subjects. PEPI Panel approach guides selection of 12 individual peptides tailored to the tumor type and HLA genotype of patients from a warehouse of off-the-shelf peptides permitting rapid, efficient manufacture of a personalized immunotherapy.

Sirnaomics is the first to achieve positive Phase IIa clinical outcomes in oncology clinical-stage RNA therapeutics biopharmaceutical company with a strong presence in both China and the U.S. that is discovering and developing innovative drugs for indications with significant unmet medical needs and large market opportunities. We have built a professional international team for the discovery and development of RNAi therapeutics and mRNA vaccines and therapeutics, based on our proprietary drug delivery technology platforms.

They have a variety of products in the pipeline including STP for basal cell carcinoma.  In addition, they have products in the pipeline for ZSCLC, multiple solid tumors and a pan cancer panel.  They are seeking partnerships for head and neck, bladder and liver cancer.

Istari Oncology – Immune checkpoint inhibitors have revolutionized cancer therapy, but their success is limited by a lack of preexisting anticancer T cells or the inability of anticancer T cells to overcome the immunosuppressive tumor microenvironment.

Lerapolturev is a unique immune activator with multiple mechanisms to stimulate a powerful anticancer response.  It is based on polio virus, the only immune activator to target solid tumors vis CD155.  It also targets key antigen presenting cells (APCs) within the tumor.  They currently have clinical trials for melanoma and bladder cancer.

Anyone who has been through a major surgery knows the importance of pain management.  We are all aware of the benefits and downsides of opioid pain medication.  Although extremely effective, opioids can also be highly addictive.  Thanks to some innovative biotech companies, there are new alternatives that provide the benefits without the downsides.

HHS.gov/opioids provided the following infographic to illustrate the problem.

Shining a spotlight on some innovative biotechs developing non-opioid alternatives to pain relief.

Pacira, San Diego, CA, The cornerstone of the Pacira product portfolio is DepoFoam^®, a proprietary drug delivery platform designed to extend a medication’s duration of action without altering its molecular structure. The DepoFoam carrier matrix is made up of multivesicular liposomes that encapsulate a drug. Each chamber is separated by lipid membranes that naturally erode to release the drug over a desired period of time.

In 2012, Pacira successfully launched EXPAREL^® (bupivacaine liposome injectable suspension), the first and only DepoFoam-based local analgesic. Indicated for administration into the surgical site to produce postsurgical analgesia, a single dose of EXPAREL provides non-opioid pain control with reduced opioid requirements for up to 72 hours* without the need for catheters or pumps.

WEX Pharmaceuticals Inc. Vancouver, Canada, is developing a new class of non-opioid analgesics. WEX’s proprietary platform and the lead product is Halneuron®. The active pharmaceutical ingredient in Halneuron is Tetrodotoxin (“TTX”) which is a proven sodium channel blocker.

Halneuron is in Phase III clinical development for the treatment of cancer-related pain and will commence Phase III clinical development for chemotherapy-induced neuropathic pain. WEX has conducted over 15 clinical trials with TTX and has treated more than 700 patients to date.

Regulonix, Arizona, has designed a drug that disrupts the interaction between a mediator protein called CRMP2 and an enzyme, Ubc9, which is important in the activity of NaV1.7. The drug reversed pain in six different rat models, the researchers showed in a study published in Science Translational Medicine.

The University of Arizona has licensed the drug which was co-founded by the study’s senior author, Rajesh Khanna, Ph.D., to develop ion channel-targeting non-opioid painkillers. Khanna, a professor of pharmacology, and his team have been working with the National Institutes of Health’s National Center for Advancing Translational Sciences to optimize the compound, according to the university’s health sciences department.

Concentric Analgesics, San Francisco, CA is a clinical stage biotechnology company committed to developing novel, non-opioid therapeutics for chronic pain management. The company’s lead drug candidate CA-008 is a water-soluble prodrug that is a potent TRPV-1 agonist and can selectively and reversibly desensitize pain conducting C-fiber nociceptors. It has the potential to eliminate the need for opioids in post-surgery recovery period and can offer pain relief up to a week after injecting.

The company is focused on three underserved markets namely management of post-surgical pain, chronic osteoarthritis pain, and intractable cancer pain. The company received a Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for CA-008 in post-surgical pain in September 2018.