Precigen (PGEN)
Precigen Inc (NASDAQ:PGEN) is a company dedicated to creating innovative gene and cell therapies to improve the lives of patients. In their latest updates, they have shared financial information and progress on PRGN-2012, an innovative treatment for recurrent respiratory papillomatosis (RRP).
Financially, they have reduced research and development expenses, as well as general and administrative expenses. However, total revenues experienced a significant decline, mainly due to lower revenues from collaborations and licensing. The positive note was the increase in total other income, thanks to lower interest expenses related to the redemption of convertible bonds.
Regarding PRGN-2012, the U.S. Food and Drug Administration (FDA) confirmed that the ongoing Phase 1/2 study for the treatment of PRR would be pivotal for accelerated approval. This means that additional time-consuming trials are not necessary, thus streamlining the regulatory process. The study’s primary endpoint, the complete response rate, was accepted by the FDA for accelerated approval.
PRGN-2012, which utilizes Precigen’s gorilla adenovector technology, has shown promise in inducing immune responses against HPV 6 or HPV 11. The breakthrough therapy and orphan drug designation by the FDA underscores its potential. Positive results from the Phase 1 study, in which 50% of patients did not undergo surgery after treatment, contributed to FDA designation as a breakthrough therapy. Following the completion of the Phase 2 study, ongoing patient follow-up is expected to yield data in the second quarter of 2024.
Iovance (IOVA)
Iovance Biotherapeutics (NASDAQ:IOVA) is like the biotech superhero, focusing on creating new treatments for cancer using their innovative TIL therapies. They have $427.8 million in their pockets as of September 2023, proving they have the financial muscles to keep their superhero research going until at least 2025. They recently suffered a net loss of $113.8 million in Q3 2023 but have a plan with anticipated revenue from lifileucel and Proleukin in 2024 to keep the lights on.
The big headline is that the FDA has given them the go-ahead for a priority review of their Biologics License Application (BLA) for lifileucel in advanced melanoma, with a target date of February 24, 2023. They also have their sights set on Europe and Canada for filing in 2024. It is as if they are preparing for take-off with incorporation completed at Authorized Treatment Centers (ATCs), getting ready to launch lifileucel as the first approved TIL therapy.
Recently, at the 38th Annual Meeting of the Society for Cancer Immunotherapy, they dropped some knowledge bombs with data showing that lifileucel is not just a one-hit wonder. In patients with advanced melanoma refractory to anti-PD-1, the four-year analysis revealed an overall survival rate of 22.2%. That’s like hitting the bull’s eye in a tough game and boasting the record of 59.9 months of longest positive response.
There’s more! At the European Society for Medical Oncology (ESMO) Congress 2023, results from a subgroup of 12 patients with difficult-to-treat mucosal melanoma were released. The data showed a 50% response rate, shouting to the world that lifileucel is not just another player in the immunotherapy game; it is a game changer.
Verastem (VSTM)
Verastem Inc (NASDAQ:VSTM) is a company passionate about developing innovative cancer treatments, with a particular focus on low-grade serous ovarian cancer (LGSOC) and non-small cell lung cancer (NSCLC) with specific mutations, such as KRAS G12C.
Although they experienced an increase in operating expenses in Q3 2023 due to an upfront payment to GenFleet and higher research costs, the company has $165.7 million in cash. Despite a reported net loss of $20.0 million, a non-GAAP adjusted net loss of $19.0 million stands out, showing solid financial management.
Verastem has exciting plans for the future, with the expected filing of an accelerated approval application for avutometinib and defactinib at LGSOC in the first half of 2024. In addition, initiation of the Phase 3 RAMP 301 trial is expected in late 2023. The company is also collaborating with GenFleet Therapeutics to advance new cancer programs driven by the RAS pathway, demonstrating a strategic vision and commitment to innovation.
In a humane and empathetic gesture, in November 2023, they shared the results of the first LGSOC Patient Impact Survey, revealing the unique challenges faced by people with this type of cancer. This patient-centered approach demonstrates the company’s concern for understanding and addressing the real needs of those battling the disease.
In addition, in October 2023, they presented promising data on the efficacy of the combination of Avutometinib and sotorasib in patients with NSCLC, showing positive responses in a variety of clinical settings.
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 (no position)
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.