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Sector Spotlight: Orphan Drug Developers With Significant Upside

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–News Direct–

The biotech sector is finally beginning to recover following a two year stretch of underperformance that was largely driven by rising interest rates and other macro factors. Biotech has a track record of strong recoveries, with the sector typically seeing double-digit gains after a downturn. This is something investors shouldnt ignore.

This recent turnaround can be credited to a robust increase in fundraising efforts and an uptick in IPOs, reaching levels not seen since the peak of the mid-pandemic market boom.

According to the Financial Times, drug developers raised $6 billion in equity capital markets in January, the largest total since February 2021 a period when biotech stocks hit their all-time high. Moreover, the SPDR S&P Biotech ETF (XBI) returned 28.7% over the past six months alone, clearly illustrating investors' rising confidence in the biotech rebound.

Identifying biotech stocks with the potential to deliver superior returns is no easy feat. There are specific milestones that companies can achieve to signal positive momentum to investors. For example, smaller biotech companies may have received the nod from regulatory authorities such as the FDA, or new breakthroughs from clinical trials, to offer a compelling investment opportunity.

With that in mind, two stocks that investors should consider looking at are Cardiol Therapeutics (NASDAQ:CRDL) (TSX:CRDL) and Kiniksa Pharmaceuticals (NASDAQ:KNSA).

Cardiol Therapeutics lead product is CardiolRx, a small molecule therapy that appears on track to bring major disruption to the pericarditis market.

CardiolRx received a vote of confidence as it was granted Orphan Drug Designation (ODD) by the FDA for the treatment of pericarditis back in February, illustrating the potential of the drug. Apart from providing a seven-year term of market exclusivity upon final FDA approval, the ODD also positions Cardiol Therapeutics (NASDAQ:CRDL) (TSX:CRDL) to be able to leverage a wide range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of expensive FDA user fees, and certain tax credits.

The U.S. Orphan Drug Act is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders, defined as one that affects fewer than 200,000 people in the U.S. There are approximately 160,000 cases of recurrent pericarditis in the U.S. annually, which includes 38,000 cases with a recurrence. Having secured FDA approval, Cardiol Therapeutics is an exemplary company likely to attract biotech investors seeking assurance in smaller firms.

While the stock had a significant rally on the backdrop of this news and continues to show strength, we believe that there is still potential for further upside.

Cardiol Therapeutics (NASDAQ:CRDL) (TSX:CRDL) is expected to release topline data in June for its Phase 2 MAvERIC-Pilot clinical trial, which if positive, could unlock significant shareholder value. Several analysts have already indicated that they are highly optimistic that the data could be a catalyst, with one analyst noting that CardiolRxs potential to be a safe, new approach to RP (recurrent pericarditis) treatment is underappreciated.

CardiolRx stands out when compared to the only FDA-approved therapy because it is administered orally and expected to be offered as a first-line therapy for pericarditis, opening it up to an even bigger opportunity as it could be prescribed at the first occurrence.

Orphan drugs have become lucrative business opportunities because these drugs command premium drug pricing, with the average price for an orphan drug at $32,000 per year per patient according to a 2021 study published in the journal Rare Diseases & Journal of Orphan Drugs.

That could explain why analysts who have been tracking CRDL are so bullish on the stock. For instance, Joe Gantoss of Chimera Research Group says he wont be surprised to see Cardiols price break past the 3-year high at $4.96, while analyst Vernon Bernardino of H.C. Wainwright & Co. reiterated their Buy rating and issued a $9.00 price target.

That would imply that Cardiol Therapeutics (NASDAQ:CRDL) (TSX:CRDL) has a potential upside of about 300% from its current share price.

Kiniksa Pharmaceuticals (NASDAQ:KNSA) offers some insight into the revenue potential of an orphan drug that treats recurrent pericarditis.

So far, KNSA has had a great run, gaining about 36% over the past year and trading at about $18.00 per share despite challenges in the sector. It is currently valued at $1.3 billion. Kiniksas portfolio of assets includes ARCALYST, the first and only FDA-approved therapy for recurrent pericarditis.

For some context, the FDA granted Breakthrough Therapy designation to ARCALYST for recurrent pericarditis in 2019; the FDA granted Orphan Drug exclusivity to ARCALYST in March 2021 for the treatment of recurrent pericarditis and a reduction in the risk of recurrence in adults and pediatric patients 12 years of age and older. The European Commission granted Orphan Drug Designation to ARCALYST for the treatment of idiopathic pericarditis in 2021.

Sales of ARCALYST were $38.5 million in 2021; $122.5 million in 2022; $233.2 million in 2023 further reaffirming the massive revenue potential for treating recurrent pericarditis. For the first quarter of 2024, ARCALYST sales were $78.9 million representing 85% year-over-year growth.

Furthermore, since its launch in April 2021, approximately 2,000 prescribers have written ARCALYST prescriptions for recurrent pericarditis, illustrating the massive demand for effective recurrent pericarditis therapies.

Going forward, Kiniksa anticipates that it will bring in between $370 million and $390 million in 2024, up from the earlier guidance of $360 million to $380 million. The revised outlook represents roughly 63% year-over-year growth at the midpoint.

Disclaimers: RazorPitch Inc. "RazorPitch" is not operated by a licensed broker, a dealer, or a registered investment adviser. This content is for informational purposes only and is not intended to be investment advice. The Private Securities Litigation Reform Act of 1995 provides investors a safe harbor in regard to forward-looking statements. Any statements that express or involve discussions with respect to predictions, expectations, beliefs, plans, projections, objectives, goals, assumptions, or future events or performance are not statements of historical fact may be forward looking statements. Forward looking statements are based on expectations, estimates, and projections at the time the statements are made that involve a number of risks and uncertainties which could cause actual results or events to differ materially from those presently anticipated. Forward looking statements in this action may be identified through use of words such as projects, foresee, expects, will, anticipates, estimates, believes, understands, or that by statements indicating certain actions & quote; may, could, or might occur. Understand there is no guarantee past performance will be indicative of future results. Investing in micro-cap and growth securities is highly speculative and carries an extremely high degree of risk. It is possible that an investors investment may be lost or impaired due to the speculative nature of the companies profiled. RazorPitch has been retained and compensated by Cardiol Therapeutics to assist in the production and distribution of content related to CRDL. RazorPitch is responsible for the production and distribution of this content. It should be expressly understood that under no circumstances does any information published herein represent a recommendation to buy or sell a security. This content is for informational purposes only, you should not construe any such information or other material as legal, tax, investment, financial, or other advice. Nothing contained in this article constitutes a solicitation, recommendation, endorsement, or offer by RazorPitch or any third party service provider to buy or sell any securities or other financial instruments. All content in this article is information of a general nature and does not address the circumstances of any particular individual or entity. Nothing in this article constitutes professional and/or financial advice, nor does any information in the article constitute a comprehensive or complete statement of the matters discussed or the law relating thereto. RazorPitch is not a fiduciary by virtue of any persons use of or access to this content.

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View source version on newsdirect.com: https://newsdirect.com/news/sector-spotlight-orphan-drug-developers-with-significant-upside-473277125

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Press Release

Cambridge Research Reveals the Centralization of Bitcoin Leveraged Computing Power XBIT Exchange Reveals Hegemony or Risk?

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The latest research from the Cambridge Center for Alternative Finance (CCAF) shows that the United States currently controls 75.4% of the hash power of the global Bitcoin network, which is more concentrated than the period when China dominated in 2021 (65-75%). This data comes from a survey of 49 leading mining companies, whose combined computing power accounts for nearly 50% of the entire Bitcoin network. XBIT said that as the United States has become the world’s largest mining center, the controversy over the possibility that computing power monopoly may threaten the principle of network decentralization continues to heat up.

Twitter : @XBITDEX

The CCAF report pointed out that the current computing power scale of the United States is 600 EH/s (accounting for 75.4% of the global total of 796 EH/s), far exceeding other regions. The formation of this pattern is closely related to policy orientation-the Trump administration regards Bitcoin as “digital gold” and simplifies the energy approval process for mines through the “Acceleration Plan” of the Ministry of Commerce, attracting a large number of mining companies to move in. However, the trend of centralization has caused XBIT (dex Exchange) analysts to worry: If the US government adjusts its position in the future, will it be possible to use the computing power advantage to implement regulatory intervention?

History provides a warning case. After China banned mining in 2021, computing power was dispersed around the world in the short term, but it was eventually concentrated in the United States. Although there were no network abuse incidents during the period of China’s dominance, the current US computing power monopoly may give the federal government greater intervention capabilities. For example, through sanctions or executive orders to review transactions, or even require miners to implement specific block screening rules. XBIT (dex Exchange) researcher admitted: “The concentration of computing power may put Bitcoin at risk of ‘politicization’, which runs counter to the anti-censorship vision designed by Satoshi Nakamoto.”

US Secretary of Commerce Howard Lutnick’s recent statement highlights policy tendencies. He defined Bitcoin as a “commodity with a fixed supply” and promoted the reduction of mining costs through off-grid power generation facilities. “Imagine that your data center is next to a power plant – this will completely change the combination of energy and computing power.” His remarks reflect the federal government’s strategic intention to attract computing power investment.

Twitter : @XBITDEX

However, the checks and balances of the federal system may form a natural barrier. Officials in major mining states such as Texas have publicly opposed excessive intervention, believing that “damaging the value of Bitcoin will shake investor confidence.” In addition, the weakening trend of the US monetary sanctions system (such as shifting to tariffs rather than financial blockades) may reduce the government’s motivation to directly control the Bitcoin network. However, analysts at XBIT (dex Exchange) pointed out: “The risk has not been eliminated. If the concentration of computing power is superimposed on policy shifts, the struggle for network governance rights may trigger a chain reaction.”

The Bitcoin community’s experience in dealing with the concentration of computing power may be the key. The Chinese ban in 2021 caused the computing power to plummet by 50%, but miners migrated to North America, Central Asia and other places, ultimately driving the network computing power to rebound by 130% at the end of the year. This history shows that the distribution of computing power is dynamically adaptable, but under the current US-dominated pattern, the difficulty of decentralization has increased significantly.

Even if the current US computing power share is reduced to 50%, it is still far beyond the historical warning line. XBIT (dex Exchange) analysts pointed out: “The centralization of computing power is not irreversible, but it requires systematic efforts. Global miners need to find a balance between compliance and censorship resistance. XBIT (dex Exchange) crypto asset custody is not only an asset protection tool for high net worth investors, but also a core service that allows them to focus on strategic investment and optimize asset allocation.”

Twitter : @XBITDEX

The industry is facing a critical choice: to rely on US energy and policy dividends to maintain growth, or to accelerate the diversification of computing power in terms of geography and technology? The answer may lie in a combination of the two – through legislation to protect miners’ rights, develop anti-censorship mining protocols, and establish a cross-border computing power alliance, a more resilient network ecosystem may be built. As an early advocate of Bitcoin said: “The real enemy of decentralization is not centralization, but the habit of centralization.”

Disclaimer: The information provided in this press release is not a solicitation for investment, nor is it intended as investment advice, financial advice, or trading advice. It is strongly recommended you practice due diligence, including consultation with a professional financial advisor, before investing in or trading cryptocurrency and securities.

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Foundation Capital Announces Strategic Investment in Arkon to Advance CeDeFi Innovation

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PALO ALTO, CA — April 30, 2025 — Foundation Capital, a leading Silicon Valley venture capital firm, today announced a strategic investment in Arkon, a next-generation CeDeFi incubation platform. This partnership underscores Foundation Capital’s long-term commitment to supporting pioneering blockchain innovations and visionary entrepreneurs, jointly building a sustainable future for decentralized finance and accelerating global adoption.

Leading Financial Innovation and Empowering Web3 Ecosystem

Arkon positions itself at the cutting edge of financial technology, uniquely integrating the compliance strengths of centralized finance (CeFi) with the autonomy and innovation of decentralized finance (DeFi). The platform aims to become an essential resource hub, providing blockchain startups comprehensive tools—from multi-chain asset management, regulatory-compliant governance frameworks, technical incubation, to strategic market access—earning its reputation as the indispensable “Swiss Army knife” for Web3 entrepreneurs globally.

With this strategic investment, Foundation Capital will further empower Arkon by bolstering its technical capabilities, attracting top talent, enhancing brand presence, and expanding the global ecosystem, enabling startups to scale rapidly and sustainably.

Rodolfo Gonzalez, Head of Crypto Investments at Foundation Capital, stated:“Arkon exemplifies the type of innovative integration and market potential Foundation Capital seeks. By effectively bridging CeFi and DeFi, Arkon fosters industry innovation while maintaining robust global regulatory compliance, laying a solid foundation for sustainable growth in decentralized finance.”

With Rodolfo Gonzalez, Eric, Anand, Kumar, and Carolyn at Foundation Capital’s Palo Alto office in January 2025.

Building a Compliant and Robust Global Financial Ecosystem

This strategic partnership represents a shared vision between Foundation Capital and Arkon: to establish a technologically advanced, regulatory-compliant, and user-centric decentralized financial ecosystem on a global scale. Leveraging Arkon’s advanced cross-chain interoperability and unified account systems, users and institutions worldwide will benefit from seamless, secure, and efficient digital asset management across diverse blockchain environments.

Additionally, both parties will collaboratively explore innovative blockchain governance models, balancing decentralized community autonomy with stringent global regulatory requirements, ensuring transparent and efficient on-chain governance.

Foundation Capital will utilize its extensive global resources across North America, Europe, and Asia, enabling Arkon to cultivate a broader international partnership network and accelerate market penetration for incubated projects.

Exploring Future Opportunities to Accelerate Mainstream Adoption

Looking ahead, Foundation Capital and Arkon will work closely to identify and nurture the most promising Web3 innovations, driving healthy ecosystem growth and global adoption. This collaboration will further bridge innovation with regulatory compliance, strengthening Arkon’s global market presence and accelerating the mainstream acceptance of blockchain technology and decentralized finance solutions.

This strategic alliance heralds the advent of a transformative era in digital finance, positioning Arkon as a pivotal force leading the evolution and adoption of future financial technology.

About Foundation Capital

Founded in 1995, Foundation Capital has a longstanding history of investing in groundbreaking technologies. With over $6 billion in assets under management, the firm has backed more than 400 companies, including notable names like Netflix, Solana, and OpenSea. Foundation Capital continues to support early-stage ventures that are poised to make significant impacts across various industries.​

About Arkon

Arkon is a pioneering CeDeFi incubation platform that offers end-to-end support for blockchain startups. By combining the regulatory compliance of CeFi with the innovative spirit of DeFi, Arkon provides a unique environment for the development and growth of decentralized financial applications.​

For more information, please visit Foundation Capital and Arkon.

Disclaimer: The information provided in this press release is not a solicitation for investment, nor is it intended as investment advice, financial advice, or trading advice. It is strongly recommended you practice due diligence, including consultation with a professional financial advisor, before investing in or trading cryptocurrency and securities.

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Press Release

CCAI Coin Surges 180% on Hotcoin Exchange Debut, Highlighting AI-Blockchain Potential

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Hotcoin Daily Update (April 29, 2025): CCAI surged by 180.53%, ranking among top-performing tokens.

On April 28 at 20:00 (UTC+8), the CCAI token launched on the Hotcoin exchange, demonstrating strong market performance and receiving enthusiastic investor attention. According to official Hotcoin data, CCAI rapidly climbed the exchange’s trending token list, briefly reaching over 6.3 USDT—a remarkable 180.53% increase from its initial listing price. This impressive growth positioned CCAI as one of the standout newly-listed assets for the day. In celebration of the successful launch, the CCAI project initiated global events, providing a reward pool of 50,000 CCAI tokens, further fueling community engagement.

Long-term Potential in AI and Blockchain Integration

The significant attention garnered by CCAI is linked closely to the ongoing surge in integrating Artificial Intelligence (AI) with blockchain (Web3). Recent rapid advancements and widespread application of AI, combined with blockchain’s decentralization and trust mechanisms, have unlocked new real-world scenarios for AI implementation. Industry reports indicate that as of Q2 2025, five of the top 20 crypto market narratives involve AI, attracting interest from over 35.7% of investors. Furthermore, 87% of cryptocurrency users express willingness to allow AI to manage portions of their investment portfolios, highlighting the market’s confidence in AI-driven crypto projects as high-potential growth areas. Consequently, projects merging AI and blockchain technologies are expected to continually benefit from these dual technological advances and are projected by industry insiders to show strong long-term growth.

CCAI is strategically positioned in this burgeoning sector. According to Hotcoin’s official announcement, CCAI serves as a core token for an AI-powered quantitative trading ecosystem, empowering various blockchain applications like GameFi, SocialFi, and DeFi. In essence, CCAI leverages AI algorithms to enhance gaming, social, and decentralized financial applications, facilitating value transmission and incentive mechanisms through blockchain technology. This strategic alignment with two major trending sectors—AI and Web3—has been affirmed by CCAI’s robust debut performance. Analysts suggest projects that integrate AI technology with blockchain attributes are likely to distinguish themselves, driving sustained growth through technological innovation and practical applications.

Global “CCA MILES TOUR” Initiative Launched

Building upon its early technical and market achievements, the CCAI project is actively expanding its global community presence. Official sources confirm that the CCAI team has launched the “CCA MILES TOUR,” a worldwide AI-blockchain summit tour. Over the next year, this initiative aims to visit 100 cities globally, directly engaging developers, investors, and community members. The tour will focus on innovations at the intersection of AI and blockchain, present CCAI’s technological vision and ecosystem roadmap, and gather local community feedback. Through these extensive global roadshows, CCAI aims to strengthen international community participation, fostering consensus and laying groundwork for global growth. Such large-scale in-person exchanges are relatively rare in the blockchain sector, underscoring the project’s emphasis on community building and sustainable development.

CCAI has outlined a clear expansion roadmap. Firstly, regarding trading channels, the team plans to progressively list on more mainstream cryptocurrency exchanges to enhance CCAI’s global liquidity and accessibility. Following the successful Hotcoin debut, CCAI is expected to appear on additional leading exchanges, reaching a broader investor base. Secondly, regarding its ecosystem applications, CCAI intends to expand its global ecosystem through collaboration with international developers and partners. This strategy aims to enrich practical applications across sectors including GameFi, SocialFi, quantitative trading, and DeFi, encouraging more innovative projects to integrate into its AI ecosystem. Ultimately, CCAI aspires to build a decentralized, AI-empowered economic system, deeply integrating AI technologies with blockchain economies to create new value networks and business models. Whether CCAI can sustain growth within the dynamic crypto landscape remains a focal point of industry interest.

Disclaimer: The information provided in this press release is not a solicitation for investment, nor is it intended as investment advice, financial advice, or trading advice. It is strongly recommended you practice due diligence, including consultation with a professional financial advisor, before investing in or trading cryptocurrency and securities.

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Disclaimer: The views, suggestions, and opinions expressed here are the sole responsibility of the experts. No Digi Observer journalist was involved in the writing and production of this article.

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