Inovio (INO)
Inovio Pharmaceuticals (NASDAQ:INO) is a biotech company that is making waves with its innovative DNA-based drugs. Its flagship product, INO-3107, has just received FDA clearance as a potential breakthrough for the treatment of recurrent respiratory papillomatosis (RRP), a rare disease caused by HPV.
This breakthrough therapy designation paves the way for Inovio to file a Biologics License Application (BLA) more quickly than anticipated, skipping the previously planned Phase 3 trial.
What is truly exciting is that INO-3107 could be the first DNA drug to reach the U.S. market, promising a new frontier in healthcare. The green light from the FDA and the possibility of suspending the Phase 3 trial have accelerated Inovio’s commercialization plans, putting it on the map for potential investors.
With $167.5 million and a strategy focused on INO-3107, they look poised to tap the potential of DNA-based drugs.
But at Inovio, it’s not all science and trials. They also recognize the value of their team and recently granted stock options to new employees. This move underscores Inovio’s commitment to attracting and retaining top talent, in line with its vision of a future where its innovative medicines make a difference.
Arcturus (ARCT)
Arcturus Therapeutics (NASDAQ:ARCT) is a global biotechnology company specializing in advanced mRNA medicines, particularly vaccines for infectious diseases and rare conditions affecting the liver and respiratory system. Its revenue sources include licensing fees and collaboration payments from partnerships with pharmaceutical and biotechnology companies.
In the second quarter of 2023, they recorded revenues of $10.5 million, with a notable decline due to the termination of agreements, offset by gains from collaborations with CSL Seqirus and BARDA. Overall, revenues for the first half of 2023 increased by $58.5 million, largely attributed to strategic collaborations initiated in late 2022.
The company’s dedication to innovative treatments is evidenced by its expanded collaboration with the Cystic Fibrosis Foundation, which secures a $25 million commitment to advance ARCT-032, a promising mRNA therapeutic candidate using Arcturus’ LUNAR delivery technology. This expanded agreement builds on a successful research program initiated in 2017, demonstrating Arcturus’ commitment to addressing critical medical needs.
In a significant development, ARCALIS, Arcturus’ manufacturing joint venture in Japan, received additional financial support from Japan’s Ministry of Economy, Trade and Industry. This funding of $165 million is to build a state-of-the-art DNA template manufacturing facility. ARCALIS, focused on GMP-integrated vaccine manufacturing, is set to play a key role in accelerating vaccine production, with the ability to deliver vaccines within 100 days of identifying a target viral strain.
Madrigal (MDGL)
Madrigal Pharmaceuticals (NASDAQ:MDGL) is on a mission to combat non-alcoholic steatohepatitis (NASH), a serious liver disease associated with obesity and metabolic disorders. They are making waves with their lead candidate, resmetirom, an oral therapy designed to get to the root causes of NASH. Think of it as a potential booster for those dealing with liver health issues.
Recently, the U.S. Food and Drug Administration (FDA) raised an eyebrow, initiating a priority review of resmetirom’s new drug application (NDA) for the treatment of NASH. This indicates that the FDA sees some promise in what Madrigal is bringing to the table. Bill Sibold, the new CEO as of September 2023, is steering the ship through these critical waters backed by a solid $500 million in funding. Money talks, especially in the world of drug development.
Results from the Phase 3 MAESTRO-NASH trial, presented at the American Association for the Study of Liver Diseases (AASLD) Liver Meeting, paint a hopeful picture. Resmetirom is not just a one-trick pony, it is the only candidate to show positive results in both fibrosis improvement and NASH resolution in a Phase 3 trial.
Beyond NASH, resmetirom flexes its muscles by positively influencing LDL cholesterol and other heart-related lipids. It’s like killing two birds with one stone, or in this case, addressing liver health and heart disease risks simultaneously. With the FDA’s approval of the NDA with priority review, Madrigal is rushing to offer the first approved treatment for NASH with liver fibrosis.
As of this writing, Gabriel Osorio-Mazzilli did not hold (either directly or indirectly) any positions in the securities mentioned in this article. The opinions expressed in this article are those of the writer, subject to the InvestorPlace.com Publishing Guidelines.
Gabriel Osorio is a former Goldman Sachs and Citigroup employee. He possesses discipline in bottom-up value investing and volatility-based long/short equities trading.