IN treating Cancerous tumors, TransCode's technology is designed to target dysregulated microRNAs implicated in cancer progression, metastasis, and resistance to existing therapies.

 

Investor Report: TransCode Therapeutics Inc. (NASDAQ: RNAZ)

Introduction

TransCode Therapeutics Inc. is a clinical-stage biopharmaceutical company specializing in RNA-targeted therapeutics for the treatment of cancer and other serious diseases. Leveraging its proprietary platform technology, TransCode aims to overcome historical challenges associated with RNA delivery, offering innovative solutions that could transform the oncology landscape.

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Technology Advantage

Proprietary Delivery Platform: TTX

TransCode's principal technological advantage lies in its proprietary delivery platform known as TTX (TransCode Therapeutics Technology). This platform is engineered to enable the systemic delivery of RNA-based therapeutics directly to tumor sites, addressing significant hurdles in RNA therapy such as stability, specificity, and efficient cellular uptake.

Key Advantages:

• Targeted Delivery: TTX facilitates precise delivery of therapeutic agents to tumor cells, minimizing off-target effects and enhancing efficacy.
• Versatility: The platform is adaptable to various RNA modalities, including microRNA (miRNA), small interfering RNA (siRNA), antisense oligonucleotides, and messenger RNA (mRNA).
• Non-Immunogenic Profile: Designed to reduce immune system activation, thereby increasing the safety profile of the therapeutics.
• Overcoming Delivery Barriers: Addresses challenges like nuclease degradation and poor cellular uptake that have historically limited the effectiveness of RNA therapeutics.

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Technology Purpose and Pipeline

Focus on MicroRNA in Cancer Treatment

TransCode's technology is designed to target dysregulated microRNAs implicated in cancer progression, metastasis, and resistance to existing therapies.

Lead Candidate: TTX-MC138

• Mechanism of Action: TTX-MC138 targets microRNA-10b, a master regulator of metastatic cell viability in solid tumors.
• Clinical Development: (Completed positive Phase I) clinical trial, aiming to demonstrate safety and preliminary efficacy in patients with advanced solid tumors.
• (Received Government grant of $2,000,000 to fast track phase 2)

Pipeline Expansion

The company is also exploring additional candidates targeting other microRNAs and RNA targets associated with various cancer types, intending to build a robust oncology portfolio.

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Competitors

TransCode operates in a competitive field with several companies focusing on RNA-based therapeutics:

• Alnylam Pharmaceuticals: A leader in RNA interference (RNAi) therapeutics, though primarily focused on genetic disorders.
• Ionis Pharmaceuticals: Specializes in antisense oligonucleotide therapies across multiple indications, including oncology.
• Moderna Inc.: Utilizes mRNA technology for vaccines and therapeutics, including potential cancer treatments.
• Arrowhead Pharmaceuticals: Develops RNAi-based therapies, with some oncology programs in its pipeline.
• Viridian Therapeutics (formerly miRagen Therapeutics): Focused on microRNA-targeted therapies, though with a current emphasis outside oncology. 
  
  
  

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Partners and Potential Clients

Current Partnerships

While specific partnerships may not be publicly disclosed, TransCode is likely collaborating with:

• Academic Institutions: For preclinical research and clinical trial collaborations.
• Clinical Research Organizations (CROs): To support the execution of clinical studies.

Potential Partners and Clients

• Pharmaceutical Companies: Interested in licensing opportunities or co-development of RNA therapeutics.
• Biotechnology Firms: For synergistic collaborations leveraging complementary technologies.
• Healthcare Providers and Cancer Centers: As sites for clinical trials and eventual adoption of approved therapies.

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Financials

Stock Information

• Ticker Symbol: RNAZ
• Exchange: NASDAQ

Financial Highlights (as of latest available data up to October 2023)

• Market Capitalization: Subject to market fluctuations; investors should refer to current financial platforms for the latest figures.
• Revenue: Limited or no revenue from product sales due to the clinical-stage status.
• Research and Development Expenses: Significant investment in R&D reflecting the advancement of the pipeline.
• Cash Position: Adequate cash reserves are crucial for funding ongoing and future clinical trials.
• Recent Financing Activities: May include public offerings, private placements, or grant funding to extend the operational runway.

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Prospects

Growth Opportunities

• Advancement of Clinical Programs: Successful progression of TTX-MC138 through clinical trials could significantly enhance company value.
• Pipeline Diversification: Expansion into other microRNA targets and cancer indications broadens market potential.
• Technological Licensing: Opportunity to out-license the TTX platform to other biotech or pharmaceutical companies.

Market Potential

• Addressing Unmet Needs: Targeting metastatic cancers with limited treatment options positions TransCode to fulfill critical medical needs.
• Global Oncology Market: The growing demand for effective cancer therapies presents substantial commercial opportunities.

Risks and Considerations

• Clinical and Regulatory Risks: The success of product candidates is contingent upon favorable clinical trial outcomes and regulatory approvals.
• Financial Sustainability: Ongoing capital requirements may necessitate additional financing, potentially leading to dilution.
• Competitive Landscape: Rapid advancements by competitors could impact market share and adoption.

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Conclusion

Increased Attractiveness to Potential Acquirers

De-risked Clinical Asset

• Validation of Technology: Successful Phase 1 trials demonstrate that TTX-MC138 is safe and potentially effective in humans, reducing the clinical risk associated with the asset.
• Enhanced Value Proposition: A de-risked asset is more appealing to larger pharmaceutical companies seeking to bolster their pipelines with promising candidates that have cleared early-stage hurdles.

Accelerated Development Timeline

• Government Support: The $2 million grant not only provides non-dilutive funding but also signals government endorsement of the technology, which can be attractive to acquirers.
• Fast-Track Potential: Accelerated Phase 2 trials can shorten the time to market, offering a quicker return on investment for potential buyers.

Strategic Alignment

• Expanding Oncology Portfolio: Companies looking to enhance their oncology offerings might find TTX-MC138's novel approach to targeting microRNA-10b appealing.
• RNA Therapeutics Interest: With growing interest in RNA-based therapies, acquiring TransCode could provide immediate entry into this innovative field.

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Market Dynamics Favoring Acquisition

Competitive Advantage

• First-Mover Status: TransCode's advancements could position it ahead of competitors in the microRNA-targeting space.
• Intellectual Property: Ownership of proprietary technology and patents can create barriers to entry for others, increasing the company's strategic value.

Industry Consolidation Trends

• Biotech Mergers and Acquisitions: The biotech sector frequently experiences consolidation, with larger firms acquiring smaller companies to access new technologies and candidates.
• Pipeline Augmentation: Acquisitions are a common strategy for big pharma to replenish their pipelines, especially as patents on existing drugs expire.

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Financial Considerations

Improved Financial Position

• Non-Dilutive Funding: The government grant enhances the company's cash position without affecting shareholder equity.
• Resource Allocation: Additional funds allow for more aggressive development and potentially better trial results, increasing the company's valuation.

Investor Sentiment

• Stock Performance: Positive clinical results and funding news often lead to stock appreciation, which can impact acquisition negotiations.
• Market Visibility: Success attracts attention from analysts and investors, raising the company's profile in the industry.

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Potential Impact on Takeover Likelihood

Negotiation Leverage

• Stronger Bargaining Position: Positive developments give TransCode more leverage in discussions, possibly leading to better acquisition terms.
• Interest from Multiple Suitors: Success may attract several potential acquirers, creating a competitive bidding environment.

Risk Mitigation for Acquirers

• Reduced Uncertainty: Advancements in clinical trials lower the risk for acquirers concerned about the viability of the drug candidate.
• Synergy Realization: An acquisition could allow for resource pooling, accelerating development and commercialization.

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Considerations and Risks

Regulatory Hurdles Remain

• Phase 2 and Beyond: While Phase 1 success is promising, subsequent trials must also demonstrate efficacy and safety.
• Approval Timelines: Regulatory approval processes can be lengthy and uncertain, which may affect acquisition timing.

Valuation Challenges

• Increased Valuation: While a higher valuation is positive, it may also make potential acquirers cautious due to the higher acquisition cost.
• Due Diligence Findings: Acquirers will conduct thorough evaluations, and any unforeseen issues could impact their interest.

Company's Strategic Goals

• Desire for Independence: TransCode's management may prefer to continue independently to fully realize the company's potential.
• Shareholder Expectations: Shareholders might expect higher returns, influencing the acceptance of any takeover offers.

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Conclusion

The successful Phase 1 trial results for TTX-MC138 and the government grant to expedite Phase 2 development enhance TransCode Therapeutics' profile in the biotech industry. These achievements:

• Increase the company's visibility and attractiveness to larger pharmaceutical firms seeking innovative oncology assets.
• Strengthen TransCode's negotiating position by demonstrating clinical success and securing non-dilutive funding.
• Potentially accelerate acquisition interest, especially from companies aiming to expand into RNA-based therapies or bolster their cancer treatment pipelines.

While these factors could increase the chances of a takeover, it's important to recognize that acquisitions depend on a complex interplay of strategic fit, financial considerations, and market conditions. There is no guarantee that these developments will lead to a takeover, but they certainly position TransCode more favorably in the eyes of potential acquirers.

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Disclaimer: This analysis is for informational purposes only and does not constitute investment advice or a recommendation to buy or sell any securities. Investing in biotechnology companies involves significant risks, including the potential loss of investment. Always conduct thorough research and consult with a qualified financial advisor before making investment decisions. Therapeutics Inc. offers a compelling proposition in the field of RNA-based cancer therapeutics. Its innovative TTX platform addresses key challenges in RNA delivery, potentially unlocking new treatment modalities for hard-to-treat cancers. While the company operates in a high-risk, high-reward segment typical of clinical-stage biotechs, its technology and strategic focus present significant upside potential for investors willing to navigate the inherent uncertainties.

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Note: Investors are encouraged to review the most recent financial statements, press releases, and consult financial advisors for the latest information and personalized investment advice.

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Targeting deadly forms of cancer, here are two microcaps we own that have developed unique medical technology available now!

These "Microcap" companies operate in innovative and emerging sectors, which may position them for significant growth.

"The rapid and complete resolution of aggressive MSS colorectal cancer tumors observed in this study is unprecedented in the field" says the author Dr. Kasi

 On June 28th Agenus Inc. ("Agenus") (Nasdaq: $AGEN), announced results from an investigator-sponsored trial (IST) of botensilimab and balstilimab (BOT/BAL) in the neoadjuvant setting for colon cancer. 

Data were presented at the 2024 European Society for Medical Oncology (ESMO) Gastrointestinal Cancers Congress in Munich, Germany.

Dr. Pashtoon Kasi, the originator of this groundbreaking study, stated, 

"The rapid and complete resolution of aggressive MSS colorectal cancer tumors observed in this study is unprecedented in the field. The exceptional activity of the BOT/BAL combination therapy in the neoadjuvant setting offers new hope for patients facing this challenging cancer subtype. Furthermore, the pattern of response and the lack of clinical recurrence speaks to the curative potential of one's own body to fight cancer."

Study Highlights:

   --  Enrollment: 20 patients were evaluable at the data cutoff with 
      available pathology results, 17 microsatellite stable (MSS) and 3 high 
      microsatellite instability (MSI-H). 
 
   --  Treatment Regimens: Both cohorts received one dose of botensilimab with 
      balstilimab. The NEST-1 cohort received one additional dose of 
      balstilimab two weeks later, whereas the NEST-2 cohort received up to 3 
      additional doses of balstilimab. 

Clinical Findings:

   --  Pathologic Response: In the NEST-2 cohort, 78% (7/9) of MSS patients 
      achieved pathologic responses of at least 50% tumor regression, with 56% 
      (5/9) reaching complete pathologic responses. 
 
   --  Surgical Outcomes and Safety: No surgeries were delayed due to adverse 
      events, and no patients had unresolved immune related adverse events. 
      Side effects were manageable, and no new safety concerns emerged. 

Agenus Inc. is emerging as a significant player in the treatment of colorectal cancer, particularly with their innovative combination therapy of botensilimab and balstilimab. Their recent clinical trial results have shown unprecedented tumor shrinkage and robust biomarker responses, making them a promising leader in this field.

1. Clinical Trial Success: The NEST-1 and NEST-2 studies demonstrated significant tumor regression in patients with both MSS and MSI-H colorectal cancer, with manageable side effects and no delays in surgical procedures. This positions Agenus at the forefront of developing effective and less invasive treatment options​ (Agenus Bio)​​ (Agenus Bio)​.

2. FDA Fast Track Designation: The FDA has granted Fast Track designation to the botensilimab and balstilimab combination for non-MSI-H/dMMR metastatic colorectal cancer, recognizing the high unmet medical need in this population and potentially expediting the development and review process​ (Cancer Network)​​ (Pharmaceutical Technology)​.

3. Innovative Approach: Botensilimab, a multifunctional CTLA-4 immune activator, is designed to boost both innate and adaptive anti-tumor immune responses, targeting "cold" tumors that typically respond poorly to standard therapies. This innovative approach extends the benefits of immunotherapy to a broader range of tumor types, further establishing Agenus as a leader in immuno-oncology​ (Agenus Bio)​​ (Pharmaceutical Technology)​.

Overall, while Agenus is not the only company working in the immunotherapy space, their recent advancements and strategic milestones indicate that they are making significant strides and establishing themselves as a leader in colorectal cancer treatment.

The outlook for Agenus Inc. (AGEN) over the next 36 months appears promising according to several analysts and recent company developments.

1. Analyst Ratings and Price Targets:

   • Analysts have a generally positive outlook on Agenus, with a consensus price target significantly higher than its current price. The average 12-month price target is around $70, with high estimates reaching up to $100, indicating a potential upside of over 350% from current levels​ (Stock Analysis)​​ (MarketBeat)​.
   • Recommendations are predominantly "Strong Buy," reflecting confidence in the company's future performance and growth potential.

2. Clinical and Regulatory Progress:

   • Agenus has reported encouraging results from its Phase 1 and Phase 2 trials, particularly in the development of its BOT/BAL combination therapy for metastatic MSS colorectal cancer. These trials have shown promising overall survival rates, which strengthens the company's potential in the oncology market​ (Agenus Bio Investor Relations)​.
   • The company plans to submit a Biologics License Application (BLA) to the FDA and present further data at a major medical conference later this year. Successful regulatory approvals and positive trial results could significantly boost the stock's value.

3. Financial Projections:

   • Revenue for 2024 is projected to increase to approximately $224.75 million, up from $156.31 million in 2023, reflecting strong growth. However, there is a slight expected decrease in revenue for 2025, followed by a return to growth in subsequent years​ (Stock Analysis)​.
   • Earnings per share (EPS) are expected to improve, though the company is still projected to incur losses in the near term. Improvements in EPS and revenue growth are key indicators of the company's financial health and market potential.

Overall, the combination of strong analyst ratings, positive clinical trial outcomes, and promising financial forecasts contribute to an optimistic long-term outlook for Agenus Inc. 

However, as with any biotech company, there are inherent risks related to clinical trial results, regulatory approvals, and market conditions that should be considered.

Update: July 18, 2024

The FDA has discouraged Agenus from submitting BOT/BAL for accelerated approval and the stock sold off over 54%.   Regulatory approval is often an obstacle for smaller healthcare companies!

For more detailed and up-to-date information, you can refer to sources like Stock Analysis and MarketBeat​ (Stock Analysis)​​ (MarketBeat)​​ (Agenus Bio Investor Relations)​.

Agenus Inc. (formerly known as Agenus Therapeutics) is not claiming to be close to a cure for any form of cancer, but they are making significant strides in developing innovative cancer therapies.

In Bio Science, there is a race for better Gene sequencing and genomics technology!

Recursion Pharmaceuticals is at the forefront of AI-driven drug discovery!

 Recursion Pharmaceuticals has caught the attention of major pharmaceutical companies due to its innovative approach to drug discovery. More recently, nVidia bought $70 Million of shares in $RXRX

Here’s why it’s worth knowing about:

1. AI-Driven Drug Discovery:

   Recursion collaborates with major biopharma companies to reduce research and development (R&D) costs and timelines. It employs AI-enabled methods to identify promising biological targets and molecules. By using digital chemistry and machine learning, Recursion calculates potential drug development targets efficiently.

2. Massive Data Repository:

   Recursion maintains an extensive database with over 3.1 trillion relationships between chemical and biological components. This data helps identify potential drug targets and streamline the drug discovery process.

3. Recent Acquisitions:

   Recursion recently acquired AI-related biotechs, Cyclica and Valence, which will enhance its drug discovery capabilities. These acquisitions aim to make Recursion even more effective at identifying drug targets and collaborating with pharmaceutical companies1.

4. Existing Collaborations:

    As we pointed out, powerful players like Roche and Bayer are already among Recursion’s patrons. Collaborating with Recursion allows these companies to leverage AI-driven approaches for drug discovery2.

5. Rare Disease Pipeline:

   Recursion also has its own development pipeline, including three programs in phase 2 clinical trials for rare diseases. For example, one program targets neurofibromatosis type 2, a condition affecting only 33,000 people worldwide. These pipeline projects could significantly boost Recursion’s revenue in the long term

6. Recursion Pharmaceuticals (RXRX on the Nasdaq)
7. offers a unique blend of AI-driven drug discovery, a vast data repository, and promising collaborations, making it an attractive partner for big pharmaceutical companies

Recursion Pharmaceuticals collaborates with NVIDIA to accelerate drug discovery using AI technology. Their newest supercomputer, BioHive-2, powered by 504 NVIDIA H100 Tensor Core GPUs, delivers 2 exaflops of AI performance—nearly 5x faster than their previous system.

Latest news:

Recursion Pharmaceuticals, Inc. - Recursion Announces Completion of NVIDIA-Powered BioHive-2, the Largest Supercomputer in Pharmaceutical Industry

Recursion ($RXRX)

Has developed an impressive artificial intelligence system called BioHive-2, which is powered by NVIDIA AI.  

 This supercomputer is specifically designed to accelerate drug discovery in the field of biology.

 

Let me share some details about it:

1. BioHive-2: Recursion’s BioHive-2 is the largest system in the pharmaceutical industry. It’s located at Recursion’s headquarters in Salt Lake City.

   This supercomputer ranks No. 35 on the latest TOP500 list of the world’s fastest supercomputers. 

2. It’s a significant improvement over its predecessor, BioHive-1, and delivers an impressive 2 exaflops of AI performance1.

3. AI in Drug Discovery: Recursion uses AI models on BioHive-2 to accelerate drug discovery. The field of biology is incredibly complex, and finding new drug candidates often involves years of wet-lab experiments.

   Recursion’s scientists can now run over 2 million experiments per week! 

4. With AI assistance, they can focus on the most promising areas, significantly reducing the need for extensive wet lab work. The goal is to achieve 80% of the value with only 40% of the experiments, thanks to AI-guided decision-making.

5. Massive Biological Dataset: Recursion collaborates with biopharma companies like Bayer AG, Roche, and Genentech. Over time, they’ve built a 50-petabyte database containing biological, chemical, and patient data. 

6. This vast dataset fuels powerful AI models that accelerate drug discovery. In fact, Recursion believes it’s one of the largest biological datasets globally, intentionally spanning both biology and chemistry.

7. Phenom Models: Using BioHive-1, Recursion developed a family of foundation models called Phenom. These models transform microscopic cellular images into meaningful representations for understanding underlying biology. 

8. Phenom-Beta, a member of this family, is now available as a cloud API and the first third-party model on NVIDIA BioNeMo, a generative AI platform for drug discovery1.

In summary, Recursion’s AI-driven approach is revolutionizing drug discovery by leveraging massive datasets, powerful AI models, and BioHive-2’s computational prowess. 

Their work has the potential to transform our understanding of biology and improve patients’ lives through better drug treatments

Disclosure: we are long $RXRX

"The rapid and complete resolution of aggressive MSS colorectal cancer tumors observed in this study is unprecedented in the field" says the author Dr. Kasi

Adaptive Biotechnologies (ADPT on Nasdaq) is making significant advancements in biotechnology, focusing on harnessing the adaptive immune system for diagnosing and treating diseases

The Human body is highly "Adaptive" in fighting desease, and these two companies are developing technology right now to help that system fight Cancer!

 Let’s compare Adaptive BioTechnologies and Agenus Inc. in terms of how they utilize the adaptive immune system to combat cancer:

1. Adaptive BioTechnologies:

   • Immunosequencing: Adaptive BioTechnologies provides an end-to-end immunosequencing solution called Immunosequencing. 
   • 
     
   • This solution helps researchers discover the breadth and depth of the adaptive immune system. Here are some key features:
     • Quantitative Abundance Data: 
     • Immunosequencing uses a multiplex PCR-based assay that enables sequencing directly from genomic DNA. Unlike RNA-based assays, it provides precise and quantitative abundance data on cell populations.
     • 
       
     • Synthetic Immune Templates: 
     • Rigorously designed synthetic immune templates act as in-line controls, ensuring accurate, quantitative, and unbiased results.
     • 
       
     • High-Throughput Sequencing: 
     • By sequencing the CDR3 variable chain of T- and B-cell receptors, Immunosequencing captures the depth and breadth of the immune repertoire, allowing insights into disease dynamics and treatment response.
     • 
       
   • Collaborations: 
   • Adaptive BioTechnologies collaborates with companies like AstraZeneca to map the immune response to cancer antigens, potentially informing the development of novel diagnostics and therapeutics.
   • 
     
   • Recent Highlights from Q1 2024
     

        -- Revenue for the first quarter of 2024 was $41.9 million. The MRD business, 
           which contributed 78% of revenue, grew 52% versus the first quarter of 
           2023. 
      
        -- clonoSEQ test volume in the first quarter of 2024 grew 41% to 17,040 
           tests delivered versus the first quarter of 2023. 
      
        -- Recognized $4.5 million within MRD revenue upon the achievement of 
           regulatory milestones from biopharmaceutical clinical studies. 
      
        -- The FDA's Oncologic Drug Advisory Committee (ODAC) voted unanimously in 
           favor of the use of MRD as a primary endpoint to support the accelerated 
           approval of new therapies for patients with multiple myeloma. 
      
        -- Following a strategic review, MRD and Immune Medicine will operate under 
           Adaptive with dedicated resources, separate segment reporting and 
           disciplined capital allocation. 
      
        -- Initiated antibody discovery campaign in Immune Medicine for multiple 
           sclerosis and type 1 diabetes to discover, make and test select 
           antibodies to generate preclinical data during 2024. 

2. Agenus Inc.:

   • Botensilimab: Agenus has developed a next-generation CTLA-4 antibody called Botensilimab. Here’s how it works:
     • Cold Tumors: 
     • Botensilimab extends the curative benefits of immunotherapy (I-O) to cold tumors that do not respond to approved immunotherapies.
     • Hot Tumors:
     •  It also expands clinical benefit in hot tumors (e.g., melanoma), where CTLA-4 is approved but shows clinical benefit in only a third of patients.
     • Improved Tolerability: 
     • Botensilimab aims to reduce or eliminate side effects that cause treatment discontinuation.
     • 
       
   • Pipeline: 
   • Agenus discovers, manufactures, and develops immuno-oncology therapeutics, including checkpoint modulators (CPMs), patient-specific anti-cancer vaccines, and adjuvants for use with various vaccines.
   • 
     
   • Recent Trials: 
   • Agenus initiated global Phase 2 trials for botensilimab in advanced MSS colorectal cancer and advanced melanoma, activating both innate and adaptive immune responses. 
   • Additionally, the combination of botensilimab and balstilimab showed unprecedented activity in microsatellite-stable colorectal cancer.

   • "The rapid and complete resolution of aggressive MSS colorectal cancer tumors observed in this study is unprecedented in the field" says the author Dr. Kasi

In summary, both companies leverage the adaptive immune system through innovative approaches to combat cancer, with Adaptive BioTechnologies focusing on immunosequencing and Agenus developing next-generation antibodies like botensilimab. 

Keep in mind that their specific strategies may vary based on their respective pipelines and collaborations.

These two small cap companies can be found on the Nasdaq under the symbols

$ADPT - $AGEN

From the "American College of Surgeons" report!

Agenus Inc. (formerly known as Agenus Therapeutics) is not claiming to be close to a cure for any form of cancer, but they are making significant strides in developing innovative cancer therapies.