2023-05-11 14:34:03 ET
Summary
- Today, we peek back in on Rocket Pharmaceuticals, Inc. for the first time in 2023.
- The company is seeing some momentum from positive trial developments and new-found enthusiasm from the analyst community so far here in May.
- An investment analysis of Rocket Pharmaceuticals, Inc. follows in the paragraphs below.
I know not all that may be coming, but be it what it will, I'll go to it laughing .”? Herman Melville.
Today, we are going to revisit an intriguing developmental concern called Rocket Pharmaceuticals, Inc. ( RCKT ). The company has seen some positive trial developments so far this year ( last covered in November 2022) as well as a rash of analyst buy reiterations so far in the month of May. Therefore, it seems a good time to peak back in on Rocket and see how its pipeline is progressing. An analysis follows below.
Company Overview:
New Jersey-based Rocket Pharmaceuticals’ two viral vector platforms have spawned five clinical programs, one of which has been discontinued. RCKT stock currently trades right around $21.00 a share and sports an approximate market capitalization of $1.7 billion.
The company has a diverse pipeline of candidates it is currently advancing. The two most advanced are focused on Hematology.
The first of these is RP-L102 which is being developed to treat Fanconi Anemia or FA. This a rare DNA repair disorder characterized by bone marrow failure. This condition can lead to predisposition to cancer and unfortunately frequently death before the age of 30. Fanconi anemia occurs in 1 in 160,000 individuals worldwide. This condition is most common among people of Ashkenazi Jewish descent as well as the Roma population of Spain. Currently, Rocket is in the process of finding and enrolling a dozen patients into an open-label, global registrational clinical trial of RP-L102 for FA.
Rocket's candidate RP-L201 also is being developed for an ultra-rare condition called Leukocyte adhesion deficiency or LAD-I. This genetic immune disorder predisposes patients to recurrent and fatal infections.
LAD-1 often results in death within the first two years of life. A Phase 2 trial is currently in the process of enrolling nine patients for this study.
Recent Developments:
Rocket has made some progress advancing its pipeline since our last piece on this name in November of last year. In late February, the FDA granted the company regenerative medicine advanced therapy or RMAT designation to gene therapy RP-A501 to treat Danon Disease. The company has completed Phase 1 development and is proceeding to a potentially pivotal Phase 2 study. This has a bigger potential market as far as individuals affected than the company's other two late-stage efforts. Danon Disease is a metabolic disorder. More specifically it is an X-linked lysosomal and glycogen storage disorder associated with hypertrophic cardiomyopathy, skeletal muscle weakness, and intellectual disability.
Then on Tuesday of this week, the government agency also approved Rocket's Investigational New Drug or IND application for RP-A601. This is a gene therapy that is targeting an inherited heart disease called arrhythmogenic cardiomyopathy .
Analyst Commentary & Balance Sheet:
The analyst firm community has regained its enthusiasm around the company, as five analyst firms including Raymond James and Needham have reissued Buy/Outperform ratings on Rocket Pharmaceuticals, Inc. since last week. Price targets proffered range from $35 to $61 a share.
Approximately 13% of the outstanding shares in Rocket are currently held short. So far in 2023, a few insiders have sold just over $360,000 worth of shares collectively. The company ended the first quarter with approximately $400 million of cash and marketable securities on its balance sheet , which management has guided provides Rocket Pharmaceuticals operational "runway" through 2024.
Verdict:
As you can see above, Rocket Pharmaceuticals has a packed calendar of scheduled events for 2023 and 2024, including potentially its first marketing application submission late this year.
Now, any developmental firm focused on developing gene therapies for rare and devastating diseases is going to be a high risk/high reward endeavor. Rocket is well-funded for the moment. More importantly, it has multiple "shots on goal," a significant amount of potential catalysts over the next 12-18 months, is making some progress advancing its pipeline, as well as getting closer to potential commercialization, and enjoys solid support from the analyst community.
Therefore, Rocket Pharmaceuticals, Inc. merits at least a ' watch item' position while we continue to monitor this developing story. Given its pipeline and under $2 billion market capitalization, Rocket could also make a nice strategic " bolt on" acquisition for a large drug concern that wants to expand their footprint in gene therapies.
An adventure is only an inconvenience rightly considered. An inconvenience is only an adventure wrongly considered .”? G.K. Chesterton.
For further details see:
Rocket Pharmaceuticals: A Catalyst-Rich Year Ahead