Rocket Pharmaceuticals Has A Lot More Upside

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I covered Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) 6 months ago, and besides took a position. I americium happy to study that nan banal much than doubled successful these fewer months, and I expect much upside.

Rocket Pharma is a cistron therapy developer for uncommon diseases. It has 4 programs successful objective tests - RP-L102 (Fanconi anemia), RP-L201 (leukocyte adhesion deficiency-I), RP-301 (pyruvate kinase deficiency) and RP-A501 (Danon Disease). The first 3 programs usage a lentiviral vector for their cistron therapy, while nan past 1 uses an AAV vector. The shape 2 FA programme and nan shape 1/2 LAD-I programme are successful registration-enabling studies.

In my previous RCKT article, I discussed information from 2018 for nan FA program, 2020 information from nan Danon program, 2022 LAD-I programme data, and a toxicity rumor successful nan Danon programme that first led to a objective hold, which was lifted, and past a clang successful nan banal aft nan institution removed a higher dose cohort owed to toxicity concerns. Two of these programs, FA and LAD-I, whitethorn person NDAs this year.

Here’s a speedy summary of Rocket’s pipeline:

Rocket's pipeline




Latest objective data



Leukocyte adhesion deficiency-I

Ex vivo ITGB2 cistron therapy, lentiviral vector

100% wide endurance astatine 12mth (9/9 pts) per ASGCT 2022

Filing owed H1 2023


Fanconi anaemia

Ex vivo Fanca cistron therapy, lentiviral vector

6 of 10 pts showed accrued MMC guidance astatine 12mth, Ash 2022

Filing owed H2 2023


Danon disease

In vivo Lamp2B cistron therapy, AAV9 vector

Increases successful Lamp2B look seen successful ph1

Ph2 pivotal study to commencement H1 2023


Pyruvate kinase deficiency

Ex vivo PKLR cistron therapy, lentiviral vector

2 big pts: Hb 13.2 & 14.7g/dl astatine 24 mth (from 7.4 & 7.0g/dl), Ash 2022

Ph2 pivotal study to commencement 2023

REN-001 (via Renovacor)

BAG3-associated dilated cardiomyopathy

BAG3 cistron therapy, AAV9 vector


Ph1 to commencement 2023

Source - Co presentations, done Evaluate.

In September, Rocket acquired bosom illness cistron therapy developer Renovacor successful an all-stock deal. REN-001, their astir precocious program, is an AAV-based cistron therapy targeting BAG3-associated dilated cardiomyopathy (DCM), a type of bosom failure.

In September, also, Rocket’s Danon Disease programme produced further information which showed that nan supplier was safe and tolerable and showed objective use successful 2 children and 4 adults successful a shape 1 trial. Other cardinal data include:

The institution said early pediatric efficacy information were accordant pinch first improvements seen successful big patients astatine a akin timeframe of up to 9 months follow-up and sustained objective use crossed biomarker, objective and functional parameters successful presently enrolled big patients astatine 24 to 36 months of follow-up.

In nan pediatric group, LAMP2B cistron look by immunohistochemistry was 21.1% successful nan first diligent (1008) astatine six months and 34.7% successful nan 2nd diligent (1009) astatine 3 months.

This is simply a coagulated achievement, because a number of different programs successful cardiac cistron therapy person met pinch nonaccomplishment because of: a) deficiency of knowledge of nan underlying mechanism; and b) unreliable macromolecule look successful bosom tissue. Rocket’s early information has impressed analysts because RP-501 has been capable to induce nan applicable macromolecule look successful bosom tissue. The institution plans to commencement a azygous arm, unfastened explanation pivotal proceedings successful H1 2023, and nan proceedings will person a earthy history comparator and a biomarker-based composite endpoint, nan erstwhile because of nan rarity of nan disease, and deficiency of immoderate different applicable molecule to comparison with. The molecule comes pinch an RMAT tag successful this indication.


RCKT has a marketplace headdress of $1.48bn and a rate reserve of $306mn. Research and improvement expenses were $43.4 cardinal for nan 3 months ended September 30, 2022, while wide and administrative expenses were $15.1 million. At that rate, nan institution has capable rate for 4-5 quarters. Also successful September, nan institution raised $108mn done a successful secondary offering. This increases its rate runway by different 2 quarters.

Rocket’s awesome weakness, immoderate analysts person said, is deficiency of near-term awesome catalysts. However, this is hardly true. In 2023, they will beryllium filing 2 NDAs and they will statesman a 3rd pivotal trial. By 2024, they should person 1 aliases 2 narcotics approved for these uncommon diseases, which will commencement a bully small gross stream. The institution has immoderate slick trading ideas present - lighting up gangly buildings pinch uncommon illness slogans and truthful on. I think, fixed time, and immoderate well-needed funds, Rocket is going to spell overmuch higher than wherever it is now. Many analysts person beardown ratings connected nan stock.

Bottom Line

Rocket Pharmaceuticals, Inc. is doing a batch of uncommon illness therapy improvement pinch a very beardown group of data. It has been comparatively successful some pinch its lentiviral programme aft bluebird’s earlier fiasco, and besides pinch nan cardiac cistron therapy abstraction which has seen galore failures. Rocket Pharmaceuticals banal is up a batch successful 6 months, which isn’t specified a bad point if you recognize nan imaginable of its programs. All successful all, I americium a purchaser connected immoderate weakness.

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Editor: Naga

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