The life sciences sector is currently navigating a complex landscape of evolving and interconnected risks. According to Aon’s recent Global Risk Management Survey, the most pressing concerns for life sciences organizations include supply chain disruptions, cyber attacks, and regulatory changes. These risks are exacerbated by the industry’s heavy reliance on external partners and the need to continuously adapt to new scientific developments and patient needs.
Current Risks
The survey highlighted that supply chain or distribution failure is the top risk facing the industry today. Recent global events have disrupted trade and exposed vulnerabilities in supply chains. The life sciences industry depends heavily on a network of external partners, making it essential for organizations to adopt robust supply chain risk management practices. This includes regular reviews of critical suppliers and comprehensive business continuity planning.
Cyber attacks and data breaches are also a significant concern, ranking as the second-highest risk. The increasing use of digital technologies such as data analytics, the Internet of Things (IoT), and artificial intelligence (AI) in the industry has amplified these risks. Organizations are likely to lose billions globally to cyber attacks in the coming years, underscoring the need for a comprehensive cyber resilience strategy that includes assessment, mitigation, risk transfer, and recovery.
Business interruption, which was the top concern during the height of the COVID-19 pandemic, remains a critical risk but has now fallen to the third position. This shift reflects the ongoing challenges related to supply chain disruptions and the need for organizations to enhance their resilience against such interruptions.
Regulatory or legislative changes are another top concern, ranking fourth among current risks. Changes in government policies, such as those aimed at reducing drug prices or enhancing innovation, can significantly impact the business models of life sciences companies. For instance, recent legislative efforts in the EU and the US are forcing companies to rethink their commercial strategies and prioritize compliance with new regulations.
Failure to attract or retain top talent has emerged as a new critical risk, ranking fifth. The industry is facing a shortage of skilled professionals, particularly in digital fields such as AI and data science. This talent gap is a significant barrier to growth and innovation, highlighting the need for organizations to invest in talent acquisition and retention strategies.
Top 10 Current Risks
Supply Chain or Distribution Failure
Cyber Attack or Data Breach
Business Interruption
Regulatory or Legislative Changes
Failure to Attract or Retain Top Talent
Damage to Brand or Reputation
Product Liability or Recall
Failure to Innovate or Meet Customer Needs
Cash Flow or Liquidity Risk
Capital Availability
Recent global events have pushed these risks to the forefront, making strategic planning and risk management essential components of organizational resilience. Life sciences organizations must continuously monitor and adapt to these evolving risks to maintain their operational and financial stability.
Future Risks
Looking ahead, life sciences organizations anticipate that cyber attacks and data breaches will continue to be a top risk. The increasing digitalization of the industry, coupled with geopolitical volatility, means that cyber threats are likely to remain a persistent challenge. Additionally, the failure to attract or retain top talent is expected to intensify, ranking as the second most significant future risk.
Regulatory or legislative changes are predicted to remain a key issue, rising to the third position in the future. This reflects concerns related to government efforts to manage rising healthcare costs. Supply chain or distribution failure, which is currently the top risk, is expected to drop to the fourth position in the future, potentially due to ongoing efforts to mitigate this risk through improved supply chain resilience practices.
Top 10 Future Risks
Cyber Attack or Data Breach
Failure to Attract or Retain Top Talent
Regulatory or Legislative Changes
Supply Chain or Distribution Failure
Business Interruption
Failure to Implement or Communicate Strategy
Failure to Innovate or Meet Customer Needs
Commodity Price Risk or Scarcity of Materials
Cash Flow or Liquidity Risk
Merger, Acquisition or Restructuring
As the life sciences industry continues to evolve, so too will the risks it faces. Organizations must be proactive in their risk management strategies, ensuring they have the capabilities to assess and mitigate potential losses. This includes adopting comprehensive cyber resilience strategies, improving supply chain risk management, and investing in talent acquisition and retention.
Aon’s survey provides invaluable insights into the current and future risks facing the life sciences sector. For a more detailed exploration of these risks and strategies for mitigating them, read the full article on Aon.com.
Key Points: – Candel Therapeutics’ stock surged 172% after its Phase III trial of CAN-2409 for localized prostate cancer met its primary endpoint. – The trial showed a 14.5% relative improvement in disease-free survival compared to placebo, with promising long-term results. – The company plans to use Phase III data to seek regulatory approval from the FDA for CAN-2409.
Candel Therapeutics (CADL) has seen its stock price surge by 172% following the announcement of positive results from its Phase III clinical trial of CAN-2409, a viral immunotherapy designed for localized prostate cancer. This breakthrough was announced on December 11, 2024, signaling the potential of CAN-2409 as a new treatment option for patients battling prostate cancer.
The Phase III trial, conducted under a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA), tested CAN-2409 in combination with radiation therapy and the antiviral drug valacyclovir. The trial showed a statistically significant improvement in disease-free survival, with patients experiencing a 14.5% relative increase in survival compared to the placebo group after 54 months of observation. These results demonstrate the treatment’s ability to improve long-term outcomes for prostate cancer patients.
In addition to the survival benefit, the study also found an increased proportion of patients achieving a prostate-specific antigen (PSA) level associated with remission, further supporting CAN-2409’s potential as a promising treatment. The therapy works by stimulating the immune system to attack prostate cancer cells, offering a novel approach compared to traditional treatments, which often rely on chemotherapy or radiation alone. Importantly, the treatment demonstrated no new safety concerns, with a safety profile similar to that of existing therapies.
The company’s CEO, Paul Peter Tak, expressed confidence in the results, emphasizing that the Phase III trial validated earlier observations of CAN-2409’s effectiveness in hard-to-treat tumors. Tak noted that the study’s design, agreed upon by the FDA, could allow Candel to seek regulatory approval for CAN-2409 as a treatment for localized prostate cancer.
Dr. Glen Gejerman, the principal investigator of the study, highlighted the clinical significance of the results, noting that the improvement in disease-free survival could mark a major advancement in prostate cancer care. Gejerman also pointed out that CAN-2409 offers a treatment option without introducing substantial toxicity, which is a key concern for many prostate cancer therapies.
Candel Therapeutics now plans to use the data from this Phase III trial to advance its marketing application to the FDA. If approved, CAN-2409 could provide a much-needed treatment alternative for patients with localized prostate cancer, transforming the current treatment paradigm.
This success positions Candel as a leader in the prostate cancer space, with investors reacting positively to the trial’s results. The company’s stock price has risen significantly, reflecting growing confidence in its future prospects.
Candel’s success comes at a time when other companies in the prostate cancer field, such as Arvinas and Pfizer, are also advancing their own treatments. However, the dramatic stock increase following the Phase III results highlights the excitement surrounding CAN-2409 and its potential to change the landscape of prostate cancer treatment.
As the company moves toward FDA approval, the oncology community will be watching closely. If successful, CAN-2409 could become a game-changing option for prostate cancer patients, offering new hope and a more effective treatment strategy.
Key Points: – Poseida Therapeutics to be acquired by Roche for up to $1.5 billion, advancing the field of allogeneic CAR-T therapies. – Poseida’s non-viral, TSCM-rich CAR-T therapies aim to enhance clinical outcomes and expand treatment access. – Roche’s capabilities in late-stage development and commercialization will bring Poseida’s advancements to patients worldwide.
Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company pioneering allogeneic cell and genetic therapies, announced that it has entered into a definitive agreement to be acquired by Roche Holdings, Inc. in a transaction valued at up to $1.5 billion. Under the agreement, Poseida stockholders will receive $9.00 per share in cash upon closing, along with a contingent value right (CVR) for up to $4.00 per share upon achieving specified milestones. The acquisition has been unanimously approved by Poseida’s Board of Directors, which recommends that stockholders tender their shares.
The merger will bring Poseida’s advanced platform technologies and innovative therapies into Roche’s Pharmaceuticals Division. This acquisition marks a significant expansion of Roche’s capabilities in allogeneic cell therapy, a cutting-edge area of biotech innovation focused on creating “off-the-shelf” treatments. This collaboration builds on an ongoing strategic partnership between Poseida and Roche, particularly in developing CAR-T therapies for hematologic malignancies. The expanded scope will also include CAR-T programs targeting solid tumors and autoimmune diseases.
Poseida’s proprietary technology platform, which utilizes non-viral, T stem cell memory (TSCM)-rich CAR-T therapies, is at the forefront of innovation in cell therapy. TSCM cells are considered highly advantageous due to their long-lived, self-renewing, and multipotent properties, enabling potentially safer and more effective therapies. These characteristics differentiate Poseida’s therapies from other CAR-T approaches, which often rely on more differentiated cells with reduced regenerative capacity.
Highlighting Poseida’s potential, interim clinical data for P-BCMA-ALLO1, a CAR-T candidate for multiple myeloma, have shown compelling results. This reinforces the transformative potential of Poseida’s platform in addressing high unmet needs across various therapeutic areas. Kristin Yarema, Ph.D., President and CEO of Poseida Therapeutics, emphasized the importance of the acquisition: “Poseida has demonstrated the unique ability of its non-viral platform to deliver TSCM-rich CAR-T therapies with the potential to improve clinical outcomes and expand access. Joining Roche will allow us to accelerate development and bring our therapies to patients worldwide.”
The transaction terms include a tender offer for all outstanding Poseida shares, priced at $9.00 per share in cash at closing. The CVR, valued at up to $4.00 per share, will be contingent upon achieving specific clinical, regulatory, and commercial milestones. Roche plans to acquire any remaining shares not tendered during the initial offer through a second-step merger, ensuring full ownership of Poseida.
The deal is expected to close in the first quarter of 2025, subject to regulatory approvals and customary closing conditions. Centerview Partners LLC and Cooley LLP advised Poseida on the transaction, while Citi and Sidley Austin LLP served as advisors to Roche.
This acquisition represents a major milestone for Poseida and Roche alike. For Roche, it establishes a new core capability in allogeneic cell therapy and genetic medicine, areas that hold immense promise for the future of personalized healthcare. For Poseida, the merger offers an opportunity to leverage Roche’s global resources in late-stage development, commercialization, and market access, enabling its therapies to reach patients worldwide.
As the biotech industry increasingly focuses on transformative therapies, this deal underscores the growing importance of partnerships in accelerating innovation. With Poseida’s cutting-edge technology and Roche’s global expertise, the acquisition is poised to reshape the landscape of cell and genetic therapies, delivering groundbreaking treatments to patients across the globe.
The Department of Government Efficiency (DOGE), spearheaded by Donald Trump with the involvement of Elon Musk and Vivek Ramaswamy, promises sweeping changes to federal operations, including deregulation efforts targeting agencies like the FDA. For the biotech industry, these potential reforms could significantly impact drug development, approval processes, and market dynamics.
Faster Approvals: A Biotech Catalyst
The FDA’s rigorous drug approval process ensures safety and efficacy but often takes years and billions of dollars to navigate. By streamlining these regulations, DOGE could shorten timelines for bringing therapies to market. This shift would lower barriers for smaller biotech firms, enhance their ability to innovate, and reduce costs for large pharmaceutical companies.
For investors, expedited approvals would translate to quicker revenue streams for new drugs, driving valuations and fueling confidence in the sector. This environment could spur a rally in biotech stocks, particularly for companies with promising pipelines awaiting FDA clearance.
Risks of Deregulation
However, deregulation comes with significant challenges. The FDA exists to ensure public safety, and relaxing oversight could lead to unintended consequences. If subpar drugs enter the market, public trust in the industry might erode, and companies could face litigation or reputational damage. These risks could temper investor enthusiasm, particularly if deregulation is perceived as compromising safety.
Additionally, deregulation might benefit well-established players disproportionately. Large pharmaceutical firms with the resources to navigate even a reduced regulatory framework may thrive, while smaller biotech startups could struggle to compete despite faster approvals.
Investor Implications
DOGE’s initiatives might catalyze investment in biotechnology, particularly in areas like gene therapy, oncology, and rare diseases. Sectors with high unmet needs and long development cycles stand to gain the most from expedited approvals. Furthermore, the promise of reduced R&D costs could attract private equity and venture capital to early-stage biotech firms.
In the stock market, the potential for reform could trigger bullish sentiment in biotech ETFs and sector-specific indices. Companies with late-stage trials or pending FDA decisions might see the greatest benefit. However, volatility is likely as the industry adapts to new regulations, and investors should be cautious of overvaluations driven by speculative enthusiasm.
Industry Innovators at the Forefront
As these potential regulatory shifts emerge, a select group of biotech pioneers are positioning themselves to capitalize on the DOGE initiative’s promising landscape. At the upcoming NobleCon conference, a plethora of healthcare companies will showcase their innovative approaches and strategic vision, offering investors and industry observers a glimpse into how they might leverage potential FDA streamlining and accelerated approval processes. These organizations represent the cutting edge of biotech innovation, each poised to potentially benefit from the proposed regulatory reforms.
Broader Impacts on Healthcare
Beyond the biotech sector, DOGE’s focus on efficiency could reshape the broader healthcare landscape. Cheaper and faster drug approvals might improve patient access to innovative treatments. Yet, balancing speed with safety will be critical to achieving long-term success.
The initiative also highlights the growing intersection of politics and business, with leaders like Musk and Ramaswamy advocating for entrepreneurial approaches to governance. Whether DOGE delivers on its promises remains to be seen, but its potential to disrupt traditional industries, including biotechnology, is undeniable.
DOGE represents both opportunity and risk for biotech investors. If executed thoughtfully, its reforms could unlock unprecedented growth in the sector by fostering innovation and reducing costs. However, ensuring safety and maintaining public trust will be critical to its long-term success. Investors should monitor regulatory developments closely, as the outcomes of these reforms will shape the biotech landscape for years to come.
The biotech rally might already be on the horizon—if DOGE achieves its ambitious goals, this sector could see transformative growth.
DENVER, Nov. 07, 2024 (GLOBE NEWSWIRE) — Medicine Man Technologies, Inc., operating as Schwazze, (OTC: SHWZ) (Cboe CA: SHWZ) (“Schwazze” or the “Company”), today announced preliminary financial and operational results for the third quarter ended September 30, 2024.
“We continued to generate momentum from our retail growth and optimization initiatives in Q3, reflected by our ability to outpace two highly competitive markets while generating sequential improvements in profitability and positive cash flow from operations,” said Forrest Hoffmaster, Interim CEO of Schwazze. “Our efforts to sharpen our pricing and promotional strategy, elevate the in-store experience, and improve product quality, assortment and in-stock positions are yielding positive results. I’m proud of our team’s hard work and dedication to drive these improvements in the overall customer experience, which has led to increased store traffic in both Colorado and New Mexico. Over the past year, our focused efforts to optimize our operations have built a solid foundation, setting the stage for sustained growth and enhanced levels of profitability in the year ahead.
“Due to our ongoing re-review process with our new auditor, Baker Tilly, we expect a delay in filing our Form 10-Q for the quarter ended September 30, 2024. Our team, in close collaboration with Baker Tilly, is making every effort to complete this re-review promptly. We expect to release our unaudited third quarter results and host a conference call in the coming weeks to discuss our financial and operational performance in greater detail.”
Q3 2024 Preliminary Financial Results
Based on preliminary and unaudited results, the Company expects to report the following for the third quarter ended September 30, 2024:
Revenue of approximately $42 million
Adjusted EBITDA of approximately $11 million
Cash flow from operations of approximately $0.2 million
At quarter end, the Company held cash and cash equivalents of approximately $11 million with principal amount of debt outstanding of approximately $196 million.
Update on Delayed Filing
As previously announced on April 8, 2024, Schwazze dismissed BF Borgers CPA PC (“BF Borgers”) as its independent registered public accountant and engaged Baker Tilly US, LLP (“Baker Tilly”) as its new independent accountant. Subsequent to the transition, on May 3, 2024, the SEC issued an Order against BF Borgers for systemic failures in meeting PCAOB standards, which impacted over 1,500 SEC filings and affected at least 75 percent of BF Borgers’ 369 clients.
As a result of the SEC Order, Schwazze’s new auditor needs additional time to complete its prior period review, which includes re-auditing the Company’s fiscal year 2023 financial statements and re-reviewing the closing of its 2022 balance sheet prior to filing its 2024 Annual Report. Baker Tilly is actively working to re-audit the Company’s financial statements for the associated periods.
Due to the ongoing re-review process, Schwazze anticipates a delay in filing its Quarterly Report on Form 10-Q for the three- and nine-months ending September 30, 2024. Moreover, the Company expects to simultaneously file its Quarterly Report on Form 10-Q for the three months ending March 31, 2024, and June 30, 2024.
About Schwazze
Schwazze (OTC: SHWZ) (Cboe CA: SHWZ) is building a premier vertically integrated regional cannabis company with assets in Colorado and New Mexico and will continue to explore taking its operating system to other states where it can develop a differentiated regional leadership position. Schwazze is the parent company of a portfolio of leading cannabis businesses and brands spanning seed to sale.
Schwazze is anchored by a high-performance culture that combines customer-centric thinking and data science to test, measure, and drive decisions and outcomes. The Company’s leadership team has deep expertise in retailing, wholesaling, and building consumer brands at Fortune 500 companies as well as in the cannabis sector.
Medicine Man Technologies, Inc. was Schwazze’s former operating trade name. The corporate entity continues to be named Medicine Man Technologies, Inc. Schwazze derives its name from the pruning technique of a cannabis plant to enhance plant structure and promote healthy growth. To learn more about Schwazze, visit https://schwazze.com/.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These forward-looking statements include financial outlooks; any projections of net sales, earnings, or other financial items; any statements of the strategies, plans and objectives of our management team for future operations; expectations in connection with the Company’s previously announced business plans; any statements regarding future economic conditions or performance; and statements regarding the intent, belief or current expectations of our management team. Such statements may be preceded by the words “may,” “will,” “could,” “would,” “should,” “expect,” “intends,” “plans,” “strategy,” “prospects,” “anticipate,” “believe,” “approximately,” “estimate,” “predict,” “project,” “potential,” “continue,” “ongoing,” or the negative of these terms or other words of similar meaning in connection with a discussion of future events or future operating or financial performance, although the absence of these words does not necessarily mean that a statement is not forward-looking. We have based our forward-looking statements on management’s current expectations and assumptions about future events and trends affecting our business and industry. Although we do not make forward-looking statements unless we believe we have a reasonable basis for doing so, we cannot guarantee their accuracy. Therefore, forward-looking statements are not guarantees of future events or performance, are based on certain assumptions, and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company’s control and cannot be predicted or quantified. Consequently, actual events and results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, risks and uncertainties associated with (i) regulatory limitations on our products and services and the uncertainty in the application of federal, state, and local laws to our business, and any changes in such laws; (ii) our ability to manufacture our products and product candidates on a commercial scale on our own or in collaboration with third parties; (iii) our ability to identify, consummate, and integrate anticipated acquisitions; (iv) general industry and economic conditions; (v) our ability to access adequate capital upon terms and conditions that are acceptable to us; (vi) our ability to pay interest and principal on outstanding debt when due; (vii) volatility in credit and market conditions; (viii) the loss of one or more key executives or other key employees; and (ix) other risks and uncertainties related to the cannabis market and our business strategy. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company’s filings with the Securities and Exchange Commission (SEC), including the Company’s Annual Report on Form 10-K and its Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC’s website at http://www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise except as required by law.
Investor Relations Contact Sean Mansouri, CFA or Aaron D’Souza Elevate IR (720) 330-2829 ir@schwazze.com
Key Points: – Astrana will acquire Prospect Health to expand its U.S. provider network across four key states. – The transaction includes a $1,095 million bridge financing, backed by major financial institutions. – The acquisition aligns with Astrana’s mission to provide localized, high-quality healthcare, benefiting 1.7 million members.
Astrana Health, Inc. (NASDAQ: ASTH), a technology-driven healthcare provider, has entered a definitive agreement to acquire Prospect Health, a healthcare system with a robust network in California, Texas, Arizona, and Rhode Island. This acquisition is valued at $745 million and aims to expand Astrana’s reach across critical U.S. markets, enabling coordinated, high-quality care for approximately 1.7 million Americans. Expected to close by mid-2025, the transaction will mark a significant expansion for Astrana in the U.S. healthcare sector.
Astrana’s acquisition of Prospect Health includes an array of healthcare assets such as the Prospect Health Plan, medical groups in four states, a pharmacy (RightRx), and Foothill Regional Medical Center in California. Prospect currently serves around 610,000 members across Medicare Advantage, Medicaid, and Commercial plans through its 3,000 primary care providers and 10,000 specialists. The acquisition will allow Astrana to strengthen its position as a leading U.S. healthcare delivery platform, focused on providing accessible, high-value care.
Astrana will fund the purchase with a combination of cash and a $1,095 million senior secured bridge commitment from Truist Bank and J.P. Morgan. The transaction includes an extended closing timeline, aiming for regulatory approvals and completion by mid-2025. The combined network will also bring substantial integration risks, given the complexity of merging operations across multiple states and entities. However, Astrana anticipates that its investment in infrastructure improvements will help ensure local, personalized care in each region.
CEO Brandon K. Sim noted that the acquisition represents a union of two organizations with a shared mission of patient-centric care. Prospect’s established presence in markets like Southern California will allow Astrana to expand beyond its current regions, particularly into Orange County, where Astrana has limited operations. This geographic expansion, coupled with Astrana’s technology-enabled healthcare model, will provide a scalable solution for accessible healthcare in diverse communities.
Astrana expects Prospect to generate approximately $1.2 billion in revenue, with adjusted EBITDA of around $81 million for 2024. This acquisition aligns with Astrana’s strategy to grow through value-based care and increase its reach across new markets while ensuring continuity of care for Prospect’s patients. According to CFO Tom Holloway, Astrana projects the transaction to be immediately accretive to earnings per share, excluding expected synergies, thus enhancing shareholder value over the long term.
Jim Brown, CEO of Prospect, expressed optimism about the partnership, highlighting shared cultural values and operational synergies between the companies. He emphasized that the acquisition will create a larger, more coordinated care network that offers improved access, quality, and efficiency for patients. The integrated healthcare system will enable Astrana to expand its end-to-end technology capabilities and support local healthcare infrastructure with continued investment in infrastructure and patient services.
Key Points: – AbbVie acquires Aliada Therapeutics, adding ALIA-1758 and its unique drug-delivery platform. – Expands AbbVie’s neuroscience pipeline with advanced Alzheimer’s treatments. – Aliada’s MODEL platform enhances drug delivery across the blood-brain barrier.
AbbVie has strategically bolstered its Alzheimer’s portfolio by acquiring Boston-based Aliada Therapeutics in a deal valued at $1.4 billion. The acquisition brings AbbVie ALIA-1758, a Phase I anti-amyloid antibody targeting Alzheimer’s disease, along with Aliada’s novel Modular Delivery (MODEL) platform. This technology aims to improve the delivery of therapeutics across the blood-brain barrier (BBB), a significant challenge in developing drugs for the central nervous system.
With Alzheimer’s becoming a critical area for biotech and pharma innovation, AbbVie’s acquisition comes amid heightened interest in anti-amyloid therapies. The recent successes of Biogen and Eisai’s Leqembi and Eli Lilly’s Kisunla, the first FDA-approved disease-modifying treatments for Alzheimer’s, have demonstrated the potential of anti-amyloid treatments, though they come with risks. ALIA-1758 is designed to target pyroglutamate amyloid beta, an epitope similar to that in Kisunla, and leverages Aliada’s MODEL platform to improve therapeutic delivery.
The MODEL platform is engineered to transport therapeutic agents across the BBB by targeting transferrin and CD98 receptors, both of which are abundantly expressed in brain endothelial cells. The technology effectively carries antibodies across the BBB, allowing higher therapeutic concentrations in the brain to address amyloid plaques associated with Alzheimer’s. This targeted approach has the potential to provide superior treatment efficacy compared to previous approaches.
This acquisition aligns with AbbVie’s strategy of expanding its presence in neuroscience. The company already has a robust portfolio that includes experimental therapies like ABBV-916, another anti-amyloid antibody; ABBV-552, which targets nerve terminals to enhance synaptic function; and AL002, an antibody developed in partnership with Alector Therapeutics. With the addition of ALIA-1758, AbbVie strengthens its position in the field and continues to invest in innovation that could transform the treatment landscape for neurodegenerative diseases.
While the Alzheimer’s market is promising, AbbVie’s expansion comes with some caution. Analysts have noted that investor sentiment in anti-amyloid drugs is mixed, given the high cost and developmental challenges. However, AbbVie’s investment signals confidence in the MODEL platform’s potential to enhance drug delivery, particularly in addressing diseases with significant unmet needs like Alzheimer’s. AbbVie is optimistic that Aliada’s technology will complement its existing assets and support long-term growth in the neuroscience sector.
Expected to close by the end of 2024, the acquisition of Aliada Therapeutics is subject to regulatory approvals and standard closing conditions. The deal underscores AbbVie’s ongoing commitment to innovation and its mission to bring novel treatments to patients suffering from Alzheimer’s and other neurological disorders.
Key Points: – Upstream Bio raised $255 million to fund advanced trials for its respiratory disease drug verekitug, achieving a valuation of $830 million. – Ceribell raised $180 million to further develop its AI-powered EEG platform, focusing on diagnosing serious neurological conditions. – CAMP4 Therapeutics raised $75 million to continue its work in gene expression therapies, pricing its IPO below expectations at $11 per share.
Three biotech companies priced their initial public offerings (IPOs) on Thursday, securing substantial funding to advance their innovative therapies and technologies. The companies—Upstream Bio, Ceribell, and CAMP4 Therapeutics—collectively raised millions, with plans to use the proceeds for various clinical trials and product developments.
Upstream Bio: Raising $255 Million for Respiratory Therapies Upstream Bio led the day by raising $255 million, with its shares priced at the higher end of the expected range. Initially planning to sell 12.5 million shares between $15 and $17 each, the company increased the offering to 15 million shares due to high demand, pricing them near $17. This could further rise if underwriters exercise their option to purchase an additional 2.25 million shares. The company, which now has a valuation of around $830 million, will trade on the NASDAQ under the ticker symbol UPB.
Upstream Bio is using the capital to advance clinical trials for its lead drug, verekitug, which is being tested for severe asthma and chronic rhinosinusitis with nasal polyps. The drug targets a receptor for thymic stromal lymphopoietin, a key player in inflammatory diseases. Proceeds will also help initiate a phase 3 trial for severe asthma.
Ceribell: Secures $180 Million for AI-Powered Neurological Diagnostics Ceribell, a commercial-stage medical technology company, raised $180 million through its IPO, with 10.6 million shares priced at $17 each, giving the company a valuation of $578 million. Like Upstream, Ceribell also granted underwriters the option to purchase additional shares—up to 1.6 million more. The company will trade on the NASDAQ under the symbol CBLL.
Ceribell specializes in AI-powered neurological diagnostic tools, notably its point-of-care electroencephalography (EEG) platform, designed to help in the diagnosis and management of critical neurological conditions. The technology is expected to revolutionize the way healthcare providers address neurological emergencies.
CAMP4 Therapeutics: Raises $75 Million Despite Pricing Below Expectations CAMP4 Therapeutics saw its IPO priced below expectations, at $11 per share, compared to an initial range of $14 to $16. Despite this, the company managed to sell 6.8 million shares, surpassing its original goal of 5 million, for total gross proceeds of $75 million. CAMP4 will begin trading on the NASDAQ under the ticker symbol CAMP.
CAMP4 focuses on regulatory RNA-targeting therapeutics, aiming to upregulate gene expression in genetic diseases. The proceeds will be used to further develop its drug pipeline, including its recent collaboration with BioMarin to target RNA sequences for therapeutic applications.
OVERLAND PARK, Kan.–(BUSINESS WIRE)– SelectQuote, Inc. (NYSE: SLQT) today announced that SelectRx, the company’s full-service medication management pharmacy that serves all 50 states, will open a third fulfillment facility in Olathe, Kansas, to serve its rapidly expanding customer base, alongside its existing facilities in Monaca, Pennsylvania, and Indianapolis, Indiana.
SelectQuote launched the SelectRx business in 2021 as part of its Healthcare Services Division after acquiring two smaller pharmacy operations. In just over three years, SelectRx has grown its membership from under 5,000 to over 75,000 members who receive regular prescription shipments in adherence-friendly packaging. The new 54,000 square foot state-of-the-art facility at 404 W. Frontier Lane in Olathe, Kansas, is expected to open during the first half of calendar 2025 and should significantly expand the pharmacy’s capacity.
SelectQuote plans to add over 50 new positions at the location and is seeking experienced pharmacists, pharmacy fulfillment technicians (to assist in filling, processing, and shipping prescriptions), and various leadership and support roles such as shipping and inventory management, facilities management and information technology.
In 2023, SelectRx was recognized as a Patient-Centered Pharmacy Home (PCPH) by The Compliance Team. PCPH is based on TCT’s CMS-recognized Patient-Centered Medical Home program, which puts special emphasis on health maintenance, preventative screening, medication management, multi-specialty medical services, patient experience reporting, and benchmarking for chronically-ill patients.
SelectQuote CEO Tim Danker commented, “We are thrilled with the rapid growth of our SelectRx business over the past three years. With this new facility, we will be able to extend this highly convenient and adherence-friendly pharmacy solution to many more Seniors who can benefit from it. We are also thrilled to build out this beautiful new facility in our home market of Kansas City.”
About SelectQuote:
Founded in 1985, SelectQuote (NYSE: SLQT) provides solutions that help consumers protect their most valuable assets: their families, health, and property. The company pioneered the model of providing unbiased comparisons from multiple, highly-rated insurance companies allowing consumers to choose the policy and terms that best meet their unique needs. Two foundational pillars underpin SelectQuote’s success: a strong force of highly-trained and skilled agents who provide a consultative needs analysis for every consumer, and proprietary technology that sources and routes high-quality leads.
With an ecosystem offering high touchpoints for consumers across Insurance, Medicare, Pharmacy, and Value-Based Care, the company now has four core business lines: SelectQuote Senior, SelectQuote Healthcare Services, SelectQuote Life, and SelectQuote Auto and Home. SelectQuote Senior serves the needs of a demographic that sees around 10,000 people turn 65 each day with a range of Medicare Advantage and Medicare Supplement plans. SelectQuote Healthcare Services is comprised of the SelectRx Pharmacy, a specialized medication management pharmacy, and Healthcare Select which proactively connects its members with best-in-class healthcare services that fit each member’s unique healthcare needs. The platform improves health outcomes and lowers healthcare costs through proactive engagement and access to high-value healthcare solutions.
This post was written and published as a collaboration between the in-house editorial team at Benzinga and Tonix Pharmaceuticals Holding Corp. with financial support from Tonix. The two organizations work to ensure that any and all information contained within is true and accurate as of the date hereof to the best of their knowledge and research. This content is for informational purposes only and not intended to be investing advice.
CHATHAM, NJ / ACCESSWIRE / August 23, 2024 / Following an upsurge of cases in the Democratic Republic of the Congo and several other countries in Africa, the World Health Organization (WHO) declared mpox a public health emergency of international concern on Aug. 14 – the second such declaration in two years. The WHO has called on vaccine manufacturers to step up efforts to curb the spread of a new, more deadly strain of the virus.
The WHO also is asking companies and organizations to bring in their vaccines, inviting developers of mpox vaccines to submit an Expression of Interest for Emergency Use Listing (EUL).
Amid this backdrop, Tonix Pharmaceuticals (NASDAQ:TNXP) is developing a vaccine candidate, called TNX-801, that could play a role in combating this escalating global health crisis. Tonix has successfully completed non-human primate studies showing protection from challenge with lethal doses of the Clade 1 monkeypox virus that is driving the new epidemic.
Clearly more testing is ahead for TNX-801, but the technology behind it is rooted in the science of Edward Jenner’s smallpox vaccine of the late 1700s, the only vaccine to successfully eradicate a contagious viral pathogen.
These credentials have earned the vaccine technology platform upon which TNX-801 is based a competitive spot in the NIH’s NextGen program for a more effective, single-dose COVID-19 vaccine to potentially provide durable protection instead of the every-six month booster strategy of mRNA vaccines.
Last month, another of Tonix’s technologies was awarded a contract for up to $34 million by the U.S. Department of Defense to develop a single broad spectrum antiviral that would work against multiple potential biowarfare agents.
An Historical Approach to Modern Challenges The current mpox crisis can be traced back to a decision made in the 1970s to discontinue routine smallpox vaccinations after smallpox was successfully eradicated. Research shows that the smallpox vaccine also provided immunity against mpox. In retrospect, smallpox vaccination kept mpox out of the human population in Africa. So an unintended consequence of stopping vaccinations for smallpox meant that mpox, then known as monkeypox (which is still the name of the virus), was able to reemerge in the human population.
Tonix’s mpox vaccine is believed to be closely related to the original smallpox vaccine invented by British physician Edward Jenner in 1796. Jenner’s vaccine is credited with being the first and only vaccine to successfully eradicate a viral pathogen. This triumph depended on several important attributes of the vaccine, including that it was generally well-tolerated, provided long-term immunity with a single dose, prevented forward transmission and provided a simple biomarker confirmation of protective immunity known as a “take”.
TNX-801 is designed to have these same attributes; it is delivered in a single dose that provides protective immunity to animals that is marked by a “take”. It is believed to have a high likelihood of providing durable immunity and preventing forward transmission. Furthermore, unlike many other vaccines, TNX-801 does not require complex ultra cold-chain storage, making it a strong candidate for global distribution.
The Science Behind TNX-801 Unlike mRNA vaccines, which primarily elicit an antibody response that requires frequent booster shots, TNX-801 is designed to trigger a robust T-cell response. T-cells have the ability to recognize and remember internal parts of viral proteins, not just those on the surface. T-cell memory lasts for years – even decades – providing long-term immunity and potentially eliminating the need for repeated boosters.
In preclinical trials, Tonix says TNX-801 demonstrated the ability to prevent death, clinical disease and lesions caused by a lethal challenge of monkeypox virus in non-human primates. Similar to Jenner’s smallpox vaccine, TNX-801 also significantly reduced viral shedding, suggesting that it can block forward transmission,. Tonix is rushing to manufacture vaccine suitable for Phase 1 human trials.
Beyond Mpox: A Platform for Future Pandemics The vaccine platform on which TNX-801 is based has potential applications beyond mpox. The company says the TNX-1800 version of the platform, for example, is designed to protect against COVID-19 and has shown promise in preclinical studies. The U.S. National Institute of Health has recognized the potential of Tonix’s platform by selecting it for Project NextGen, aimed at developing next-generation COVID-19 vaccines and pandemic platform technologies.
As the world faces the growing threat of mpox and other infectious diseases, Tonix Pharmaceuticals is not only attempting to address the current crisis with TNX-801, but also potentially contributing to a future where pandemics can be more predictably contained.
PONTE VEDRA, Fla., Aug. 22, 2024 — Cadrenal Therapeutics, Inc. (Nasdaq: CVKD), a biopharmaceutical company developing tecarfarin, a late-stage, next-generation Vitamin K Antagonist (VKA) oral and reversible anticoagulant (blood thinner) designed to prevent heart attacks, strokes, and deaths due to blood clots in patients with implanted cardiac devices and those with rare cardiovascular conditions, announced today that it will be engaging with the U.S. Food and Drug Administration (FDA) in early September for a Type-B meeting to discuss its clinical trial for tecarfarin in LVAD patients.
“This upcoming meeting with the FDA is a crucial step in developing tecarfarin as we prepare for our pivotal trial. We look forward to discussing the development program for tecarfarin in LVAD patients,” said Quang Pham, Chief Executive Officer of Cadrenal Therapeutics.
ABOUT LVAD PATIENTS
Left Ventricular Assist Devices (LVADs) are mechanical pumps to support heart function in patients with advanced heart failure. These devices are vital for patients awaiting heart transplants or those who are ineligible for transplants. However, LVAD patients face an increased risk of thromboembolic events, such as strokes, which necessitates ongoing anticoagulation therapy. The current anticoagulation therapy, warfarin, presents challenges, including variability in dosing, a narrow therapeutic window, and potential interactions with other medications, making effective management crucial to reducing complications and ensuring patient safety.
ABOUT CADRENAL THERAPEUTICS, INC. Cadrenal Therapeutics is developing tecarfarin for unmet needs in anticoagulation therapy. Tecarfarin is a late-stage novel oral and reversible anticoagulant (blood thinner) to prevent heart attacks, strokes, and deaths due to blood clots in patients with implanted cardiac devices and those with rare cardiovascular conditions. Tecarfarin has orphan drug designation for the prevention of thrombosis and thromboembolism in patients with ventricular assist devices. Tecarfarin also has orphan drug and fast-track designations from the FDA for the prevention of systemic thromboembolism (blood clots) of cardiac origin in patients with end-stage kidney disease and atrial fibrillation. Cadrenal is also pursuing additional regulatory strategies for unmet needs in anticoagulation therapy for patients with thrombotic antiphospholipid syndrome (APS). Tecarfarin is specifically designed to leverage a different metabolism pathway than the oldest and most commonly prescribed Vitamin K Antagonist (warfarin). Tecarfarin has been evaluated in 11 human clinical trials and more than 1,000 individuals. In Phase 1, Phase 2, and Phase 2/3 clinical trials, tecarfarin has generally been well-tolerated in both healthy adult subjects and patients with chronic kidney disease. For more information, please visit: www.cadrenal.com.
SAFE HARBOR STATEMENT
Any statements contained in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements.” These statements include statements regarding the Company engaging with the FDA in early September for a Type-B meeting to discuss its clinical trial for tecarfarin in LVAD patients and the planned pivotal trial. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the ability of tecarfarin to improve anticoagulation treatment in patients, the success of the Type-B meeting, the ability of the Company to commence and complete a pivotal trial and commercialize tecarfarin with patients with left ventricular assist devices (LVADs), and the other risk factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, and the Company’s subsequent filings with the SEC, including subsequent periodic reports on Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Any forward-looking statements contained in this press release speak only as of the date hereof and, except as required by federal securities laws, the Company specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise.
For more information, please contact:
Cadrenal Therapeutics: Matthew Szot, CFO 858-337-0766 press@cadrenal.com
Investors: Lytham Partners, LLC Robert Blum, Managing Partner 602-889-9700 CVKD@lythampartners.com
Analysis features the growing need to evolve anticoagulation therapy beyond warfarin to avoid gastrointestinal bleeding, a significant and common complication for left ventricular assist device (LVAD) patients resulting in expensive hospitalizations
Tecarfarin, a novel vitamin K antagonist (VKA), uses a different metabolic pathway than warfarin, the most commonly used anticoagulant for LVAD patients, and may offer more stable and effective anticoagulation
PONTE VEDRA, Fla., Aug. 20, 2024 — Cadrenal Therapeutics, Inc., (Nasdaq: CVKD), a biopharmaceutical company developing tecarfarin, a late-stage, next-generation VKA oral and reversible anticoagulant (blood thinner) designed to prevent heart attacks, strokes, and deaths due to blood clots in patients with implanted cardiac devices or rare cardiovascular conditions, today highlighted a manuscript that was recently peer-reviewed and published in the Journal of Cardiac Failure evaluating the relationship between time in therapeutic range, or TTR, management quality and LVAD patient clinical outcomes. These findings focus on the potential role of investigating new VKA agents beyond warfarin in improving clinical outcomes in LVAD patients.
In the manuscript co-authored by Mandeep R. Mehra, MD, MSc, FRCP, Brigham and Women’s Hospital Heart and Vascular Center, Center for Advanced Heart Disease, titled “Antithrombotic Strategies with Left Ventricular Devices,” the findings conclude that although the current generation of LVAD pumps has largely overcome hemocompatibility-related adverse events there is a continuing need to evolve the anticoagulant therapy to avoid persistent gastrointestinal bleeding, which leads to frequent hospital admissions, procedures, blood transfusions, decreased quality of life and increased cost of care.1
Dr. Mandeep Mehra, who chaired the ARIES-HM3 study, commented, “Tecarfarin could potentially be an important therapy for patients with LVADs who all require chronic anticoagulation since it does not get affected by drug-drug interactions or changes in kidney function like warfarin and deserves further study.”
A secondary analysis of the ARIES-HM3 trial, sponsored by Abbott, evaluated the relationship between VKA management quality and clinical outcomes, wherein a median TTR for VKAs of 56% was achieved. This analysis found an inverse relationship between TTR and bleeding events, with a 47% reduction in bleeding risk for patients with a TTR above the median. Lower TTRs were associated with threefold more sub-therapeutic INRs than supra-therapeutic INRs, with no clear correlation between higher INRs and bleeding events.
“These findings provide us with increased confidence that tecarfarin has the potential to address the LVAD patients’ critical unmet anticoagulation needs more effectively,” said Quang X. Pham, Chairman and Chief Executive Officer of Cadrenal Therapeutics. “We are excited to work with the LVAD community to potentially bring our next-generation VKA to patients.”
About Tecarfarin Tecarfarin is the only oral anticoagulant in development worldwide for patients with implanted cardiac devices and other rare cardiovascular conditions. It has been uniquely designed to overcome many of the challenges patients experience with warfarin. Tecarfarin is metabolized using carboxyl esterase rather than the cytochrome P450 system, which allows the drug to avoid interactions with many other commonly used medications and may offer more stable anticoagulation, including those patients with renal dysfunction, which is common in LVAD patients. In a Phase II study with 66 patients with atrial fibrillation switched from warfarin to tecarfarin, the mean interpolated TTR was 71.4% within three weeks, with minimal time spent in extreme INR ranges (<1.5 and >4.0). If approved, tecarfarin has the potential to be the only on-label drug for LVAD patients in the U.S.
In addition, tecarfarin may prove valuable for other patients where warfarin is not providing recommended anticoagulation because of genetic warfarin resistance or renal impairment making warfarin metabolism difficult. These include individuals with end-stage kidney disease and atrial fibrillation or those with mechanical heart valves and hard-to-control International Normalized Ratio, which measures how long it takes the blood to clot.
On August 6, 2024, Cadrenal Therapeutics announced that it had been in discussions with Abbott about Cadrenal’s planned pivotal study of tecarfarin in patients with recently implanted LVADs. LVAD patients require lifelong anticoagulation to protect against thromboembolic events. The only LVAD available in the U.S. is Abbott’s HeartMate 3TM.
On April 9, 2024, Cadrenal Therapeutics announced that the U.S. Food and Drug Administration (FDA) had granted tecarfarin Orphan Drug Designation for the prevention of thromboembolism and thrombosis in patients with an implanted mechanical circulatory support device, which includes the LVAD.
About Cadrenal Therapeutics, Inc. Cadrenal Therapeutics Is Developing Tecarfarin for unmet needs in anticoagulation therapy. Tecarfarin is a late-stage novel oral and reversible anticoagulant (blood thinner) to prevent heart attacks, strokes, and deaths due to blood clots in patients with implanted cardiac devices and those with rare cardiovascular conditions. Tecarfarin has an orphan drug designation for the prevention of thrombosis and thromboembolism in patients with ventricular assist devices (VADs). Tecarfarin also has orphan drug and fast-track designations from the FDA for the prevention of systemic thromboembolism (blood clots) of cardiac origin in patients with end-stage kidney disease and atrial fibrillation. Cadrenal is also pursuing additional regulatory strategies for unmet needs in anticoagulation therapy for patients with thrombotic antiphospholipid syndrome. Tecarfarin is specifically designed to leverage a different metabolism pathway than the oldest and most commonly prescribed Vitamin K Antagonist (warfarin). Tecarfarin has been evaluated in 11 human clinical trials and more than 1,000 individuals. In Phase 1, Phase 2, and Phase 2/3 clinical trials, tecarfarin has generally been well-tolerated in both healthy adult subjects and patients with chronic kidney disease. For more information, please visit: www.cadrenal.com.
Safe Harbor Statement Any statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements.” These statements include statements regarding tecarfarin offering more stable and effective anticoagulation than warfarin, the potential role of investigating new VKA agents beyond warfarin in improving clinical outcomes in LVAD patients, the continuing need to evolve the anticoagulant therapy to avoid persistent gastrointestinal bleeding and tecarfarin potentially being an important therapy for patients with LVADs who all require chronic anticoagulation since it does not get affected by drug-drug interactions or changes in kidney function like warfarin, tecarfarin having the potential to address the LVAD patients’ critical unmet anticoagulation needs more effectively and potentially bringing the Company’s r next-generation VKA to patients The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the ability of tecarfarin to improve anticoagulation treatment in patients, the ability of the Company to advance tecarfarin with patients with left ventricular assist devices (LVADs), and the other risk factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, and the Company’s subsequent filings with the SEC, including subsequent periodic reports on Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Any forward-looking statements contained in this press release speak only as of the date hereof and, except as required by federal securities laws, the Company specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise.
For more information, please contact:
Cadrenal Therapeutics: Matthew Szot, CFO 858-337-0766 press@cadrenal.com
Investors: Lytham Partners, LLC Robert Blum, Managing Partner 602-889-9700 CVKD@lythampartners.com
CATALYST is a Phase 2 single-blind, placebo-controlled, proof-of-concept study in patients presenting to the emergency department
More than 27,569 individuals in the U.S. died from drug overdose deaths involving cocaine in 2022; there is currently no FDA-approved product for cocaine intoxication
Topline results are expected in the first half of 2025
CHATHAM, N.J., Aug. 20, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, today announced the first patient has been dosed in the Phase 2, single-blind, placebo-controlled, proof of concept trial of TNX-1300 (double-mutant cocaine esterase 200 mg, i.v. solution) for the treatment of acute cocaine intoxication in the emergency department (ED). TNX-1300 is a recombinant enzyme that rapidly and efficiently degrades and metabolizes cocaine in cocaine users, as demonstrated in a prior Phase 2a randomized, double-blind, placebo-controlled, laboratory-based clinical study, providing support for the use of TNX-1300 as a treatment for life-threatening cocaine intoxication.1
Tonix has been awarded a Cooperative Agreement Grant from National Institute on Drug Abuse (NIDA), part of the National Institutes of Health (NIH), to support development of TNX-1300 for the treatment of cocaine intoxication. In addition, TNX-1300 has been granted Breakthrough Therapy designation by the U.S Food and Drug Administration (FDA).
“Cocaine abuse and dependence are major problems in the U.S. However, there is currently no FDA-approved treatment indicated for cocaine intoxication, a life-threatening state characterized by acute symptoms including agitation, hyperthermia, tachycardia, arrhythmias, hypertensive crisis, myocardial infarction, stroke, and seizures,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “In 2022, the number of overdose deaths involving cocaine reached 27,569 individuals.2 With approximately 505,000 emergency room visits annually involving cocaine use and approximately 61,000 of the visits involving detox services to treat cocaine overdose,3,4 we believe TNX-1300 has the potential to help address the morbidity and mortality caused by cocaine intoxication. By targeting the cause rather than the symptoms of cocaine intoxication, TNX-1300 may offer significant advantages to the current standard of care for cocaine overdose.”
The Phase 2 trial is a single-blind, open-label, placebo-controlled, randomized study comparing the safety of a single 200 mg dose of TNX-1300 to placebo injection plus standard of care alone for the treatment of signs and symptoms of acute cocaine intoxication. The study is being conducted in the EDs of six academic medical centers in the U.S. It will include approximately 60 subjects presenting to the ED with cocaine intoxication. During the treatment period, subjects randomized to receive TNX-1300 will receive a single i.v. injection of TNX-1300 administered over two minutes or less; whereas subjects randomized to receive standard of care alone will receive a single i.v. saline injection over two minutes or less. For both study arms, signs and symptoms of cocaine intoxication will be assessed at pre-determined time points after treatment. After randomization, blood samples will be drawn at specific time points to assess the pharmacokinetics of TNX-1300 and levels of cocaine and its metabolites in the plasma. The primary endpoint of the study is reduction of systolic blood pressure associated with acute cocaine intoxication identified at study baseline comparing TNX-1300 to placebo with standard of care after 60 minutes. A variety of secondary endpoints will be measured, including reduction of circulating cocaine and levels of its metabolites at multiple post-baseline timepoints.
For more information, see ClinicalTrials.gov Identifier: NCT06045793
About TNX-1300
TNX-1300 (T172R/G173Q double-mutant cocaine esterase 200 mg, i.v. solution) is being developed under an Investigational New Drug application (IND) for the treatment of cocaine intoxication. TNX-1300 is a recombinant protein enzyme produced through recombinant DNA technology in a non-disease-producing strain of E. coli bacteria. Cocaine esterase (CocE) was identified in bacteria (Rhodococcus) that uses cocaine as its sole source of carbon and nitrogen and that grows in soil surrounding coca plants.5 The gene encoding CocE was identified and the protein was extensively characterized.5–8 CocE catalyzes the breakdown of cocaine into metabolite ecgonine methyl ester and benzoic acid. Wild-type CocE is unstable at human body temperature, so targeted mutations were introduced in the CocE gene and resulted in the T172R/G173Q double-mutant CocE, which is active for approximately 6 hours at body temperature.8 In a Phase 2 laboratory-based study in volunteers who use cocaine, TNX-1300, at 100 mg or 200 mg i.v. doses, was well tolerated and rapidly reduced cocaine effects after cocaine 50 mg i.v. challenge.1
About Cocaine Intoxication andOverdose
Cocaine is an illicit recreational drug which is taken for its pleasurable effects and associated euphoria. In 2022, over 5 million individuals in the U.S. reported current cocaine use, almost 2% of the population.9 Pharmacologically, cocaine blocks the reuptake of the neurotransmitter dopamine from central nervous system synapses, resulting in the accumulation of dopamine within the synapse and an amplification of dopamine signaling and its capacity to produce euphoric mood states. With the continued use of cocaine, however, intense cocaine cravings occur resulting in a high potential for abuse and addiction (dependence), as well as the risk of acute cocaine intoxication. Cocaine intoxication refers to the deleterious effects on several body systems, especially those involving the cardiovascular system. Common symptoms of cocaine intoxication include tachyarrhythmias and elevated blood pressure, either of which can be life-threatening. As a result, individuals with known or suspected cocaine intoxication are sent immediately to the emergency department (ED), preferably by ambulance in case cardiac arrest occurs during transit. The standard of care for treating cocaine intoxication in the ED focuses on symptom management, preventing complications, and supporting cardiovascular, respiratory, and neurological function, e.g. benzodiazepines for agitation, seizures, and sympathetic overdrive; antihypertensives for extremely elevated blood pressure; aspirin and nitroglycerine for cardiac ischemia. There are approximately 505,000 emergency room visits for cocaine abuse each year in the U.S., of which 61,000 require detoxification services.3,4 According to the National Institute on Drug Abuse, in 2022 the number of overdose death involving cocaine reached 27,569 individuals.2 In 2019, Black Americans experienced the highest death rate for overdoses involving cocaine, at 10.7 per 100,000.10
References
1 Nasser et al. A randomized, double-blind, placebo-controlled trial of RBP-8000 in cocaine abusers: pharmacokinetic profile of rbp-8000 and cocaine and effects of RBP-8000 on cocaine-induced physiological effects. J Addict Dis. 2014;33(4):289-302.
3 Substance Mental Health Services Administration, Drug Abuse Warning Network, 2011: National Estimates of Drug- Related Emergency Department Visits. HHS Publication No. (SMA) 13-4760, DAWN Series D-39. Rockville, MD: Substance Abuse and Mental Health Services Administration, 2013.
4 Drug Abuse Warning Network, 2011: Selected Tables of National Estimates of Drug-Related Emergency Department Visits. Rockville, MD: Center for Behavioral Health Statistics and Quality, SAMHSA, 2013.
5 Bresler, et al. Gene cloning and nucleotide sequencing and properties of a cocaine esterase from Rhodococcus sp. strain MB1. Appl Environ Microbiol. 2000. 66(3):904-8.
6 Larsen, et al. Crystal structure of a bacterial cocaine esterase. Nat Struct Biol. 2002. 9(1):17-21.
7 Turner, et al. Biochemical characterization and structural analysis of a highly proficient cocaine esterase. Biochemistry. 2002. 41(41):12297-307.
8 Gao, et al. Thermostable variants of cocaine esterase for long-time protection against cocaine toxicity. Mol Pharmacol. 2009. 75(2):318-23.
10 Kariisa, et al. Drug overdose deaths involving cocaine and psychostimulants with abuse potential among racial and ethnic groups – United States, 2004-2019. Drug Alcohol Depend. 2021. 1;227:109001.
Tonix Pharmaceuticals Holding Corp.*
Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and modernizing solutions for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders, and its priority is to submit a New Drug Application (NDA) to the FDA in the second half of 2024 for TNX-102 SL, a product candidate for which two statistically significant Phase 3 studies have been completed for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has Breakthrough Therapy designation. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease, including TNX-2900 for Prader-Willi syndrome, and infectious disease, including a vaccine for mpox, TNX-801. Tonix recently announced the U.S. Department of Defense (DoD), Defense Threat Reduction Agency (DTRA) awarded it a contract for up to $34 million over five years in an Other Transaction Agreement (OTA) to develop TNX-4200, small molecule broad-spectrum antiviral agents targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, MD, instrumental in progressing this development. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.
*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.
Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners.
This press release and further information about Tonix can be found at www.tonixpharma.com.
Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; risks related to the failure to successfully market any of our products; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission (the “SEC”) on April 1, 2024, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.
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