We’ve narrowed down the list of penny stocks for which we need to perform due diligence by using TipRanks’ database to focus solely on those that have received positive feedback from the analyst community. Two were identified as having sufficient analyst support to receive the consensus recommendation of “strong buy.” Not to mention that each has tremendous upside potential; some analysts anticipate that they will each reach a share price of $15 in the near future.
- Capricor Therapeutics (CAPR)
The first stock well look at is Capricor Therapeutics, a clinical-stage biopharma company working on new treatments for Duchenne muscular dystrophy (DMD), a rare, genetically-based physical developmental disease that most commonly appears in boys around age 4. Symptoms are progressive , and patients may lose the ability to walk by age 12. Capricor is taking a cell and exosome-based approach to the development of therapeutic agents to treat, or even prevent, serious cases of DMD.
Capricor’s leading drug candidate is CAP-1002, a therapeutic agent based on cardiosphere-derived cells (CDCs), a proprietary technology of the company. The company’s CDCs have been undergoing a range of studies, including some early-stage human trials, since 2007, and some 200 DMD patients have participated. The drug candidate uses cardiac cell therapy for its demonstrated immunomodulatory activity, and CAP-1002’s trials are investigating its ability to encourage cellular regeneration.
In recent months, Capricor has presented important results from the HOPE-2 open label extension study of CAP-1002 in the treatment of patients with late-stage DMD. The results were considered positive, and resulted in statistically significant improvements in skeletal muscle function, observable by objective improvement in upper limb function. The company is currently enrolling patients in the HOPE-3 study, a Phase 3 pivotal trial on the double-blind placebo-controlled model. Capricor plans to release an interim data analysis of the HOPE-3 study in mid-2023.
Earlier this year, Capricor out-licensed the US rights for CAP-1002 to Nippon Shinyaku for a $30 million upfront fee. In addition, the company is entitled to up to $705 million in potential milestone payments, and a double-digit royalty of U.S. sales.
The company is also engaged in ongoing discussions with the FDA regarding completion and submission of a Biologics License Application (BLA) for CAP-1002. Acceptance of a BLA will be an important milestone toward later commercialization of the product.
- Vor Biopharma (VOR)
The second penny stock we’ll look at, Vor Biopharma, is another clinical-stage medical research company. Vor is focused on the development of new treatments for blood cancers, treatments that will ‘change the standard of care’ for these difficult-to-treat disease conditions. The company is using engineered hematopoietic stem cells to enable precision-targeted cancer therapies for post-transplant use. The company’s goal is to protect healthy cells while exposing cancer cells to medicinal agents.
Vor currently has two main drug candidates, VOR33 and VCAR33, in multiple research tracks, including 4 pre-clinical, 1 clinical-stage, and 1 in preparation for clinical trials. VOR33, the leading candidate, is an eHSC product, created by genetically modifying healthy donor HSCs to remove the CD33 surface target. The goal is to create HSCs that are protected from the therapeutic agents post-transplant, leaving the cancer cells exposed to the targeted therapy. This candidate is the subject of a Phase 1/2 clinical trial, with initial clinical data on track for release before the end of this year. The trial has nine sites currently active, and is continuing to recruit patients
VCAR33, the other leading candidate, is a CD33-directed chimeric antigen receptor T cell (CAR-T) therapeutic agent, under investigation as a treatment for adults with acute myeloid leukemia (AML). The drug candidate is planned as the target of a Phase 1/2 clinical trial next year, and the IND application is on track for 1H23.
In a review of Vor for JMP, analyst Silvan Tuerkcan writes: “VOR33 data are still expected in 4Q22… We remain confident in this readout based on preclinical work from several groups showing successful engraftment of edited CD33KO eHSCs and natural history data in humans with a spontaneous mutation leading to lack of CD33 in HSCs. We are also confident in the cell process, considering VOR’s CSO, Tirtha Chakraborty, Ph.D., was already involved in the successful translation of another engineered HSC that showed engraftment in humans (CTX001)… Successful engraftment would be very meaningful, in our view, validating a significant portion of the VOR approach.”
All of the above makes it clear why Tuerkcan is now standing with the bulls. The analyst rates VOR an Outperform (i.e. Buy), while his $15 price target implies an upside of ~242% for the year ahead