Press Release: Rocket Pharmaceuticals To Present Updated Data From The Phase 1 Clinical Trial Of Rp-A501 For Danon Disease At The American Heart Association's 2024 Late-Breaking Science Sessions

Rocket Pharmaceuticals, Inc. RCKT incurred a loss of 71 cents per share in the third quarter of 2024, narrower than the Zacks Consensus Estimate of a loss of 78 cents. In the year-ago quarter, the company had reported a loss of 75 cents per share.

In the absence of any marketed product in its portfolio, Rocket Pharmaceuticals did not record any revenues during the third quarter.

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More on RCKT's Q3 Results

In the reported quarter, general and administrative (G&A) expenses rose 45.7% year over year to $27.1 million. The increase was due to a rise in the company’s commercial preparation expenses, which included commercial strategy, medical affairs, legal and other expenses.

Research and development (R&D) expenses were $42.3 million, down 9.6% from the year-ago quarter’s figure. The downside was due to reduced manufacturing and development costs.

As of Sept. 30, 2024, Rocket Pharmaceuticals had cash, cash equivalents and investments of $235.7 million compared with $278.8 million on June 30, 2024. Management expects this cash balance to fund operations and meet capital expenditure requirements into 2026.

Shares of RCKT have plunged 41.8% so far this year compared with the industry’s 0.9% fall.

RCKT's Recent Pipeline Updates

Kresladi is being developed to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder. The FDA issued a complete response letter (CRL) to the biologics license application (BLA) seeking approval for Kresladi to treat LAD-I in June 2024.

Per the CRL, the FDA sought "limited" additional information on the Chemistry Manufacturing and Controls (CMC), which is part of the BLA submission. The regulatory body is currently reviewing the limited additional CMC information for Kresladi.

RCKT expects approval for Kresladi in 2025.

Rocket Pharmaceuticals is evaluating two other gene therapies, namely RP-L102 and RP-L301, for the treatment of Fanconi anemia (“FA”) and pyruvate kinase deficiency (“PKD”), respectively.

Along with the earnings release, the company announced that it has initiated a rolling BLA for RP-L102 for treating FA. The candidate is also currently under review in the European Union for the same indication.

RP-L301 is currently being evaluated in a pivotal phase II study for treating PKD.

Rocket Pharmaceuticals is developing adeno-associated virus (AAV) gene therapies to target genetic CV indications caused by mutations or defects in different genes.

In September 2024, the company completed patient enrollment in a pivotal phase II study evaluating its investigational gene therapy candidate, RP-A501, for treating male patients with Danon disease. Dosing in the study is currently underway.

Per management, RP-A501 is the first gene therapy product to demonstrate safety and efficacy in clinical studies for a cardiovascular condition.

Another AAV-based gene therapy candidate is RP-A601, which is in early-stage development for arrhythmogenic cardiomyopathy. The company completed patient enrollment in the low-dose cohort of the study during the third quarter of 2024. Preliminary data from the phase I study is expected in the first half of 2025.

Rocket Pharmaceuticals, Inc. Price, Consensus and EPS Surprise

Rocket Pharmaceuticals, Inc. price-consensus-eps-surprise-chart | Rocket Pharmaceuticals, Inc. Quote

RCKT's Zacks Rank & Key Picks

Rocket Pharmaceuticals currently carries a Zacks Rank #4 (Sell).

Some better-ranked stocks from the biotech sector are Immunocore Holdings plc IMCR, Amicus Therapeutics, Inc. FOLD and Castle Biosciences, Inc. CSTL, each carrying a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for Immunocore’s 2024 loss per share have narrowed from $1.67 to $1.11. Loss per share estimates for 2025 have narrowed from $2.35 to $1.70 during the same time. Year to date, shares of IMCR have declined 49.2%.

IMCR’s earnings beat estimates in two of the trailing four quarters while missing the same on the remaining two occasions, the average surprise being 25.57%.

In the past 60 days, estimates for Amicus’ 2024 earnings per share have moved up from 20 cents to 22 cents. Earnings per share estimates for 2025 have improved from 49 cents to 53 cents during the same time. Year to date, shares of FOLD have declined 22.6%.

FOLD’s earnings beat estimates in three of the trailing four quarters while missing on the remaining occasion, the average surprise being 27.09%.

In the past 60 days, estimates for Castle Biosciences’ 2024 loss per share have narrowed from 58 cents to 8 cents. Loss per share estimates for 2025 have narrowed from $2.13 to $1.88 during the same time. Year to date, shares of CSTL have surged 54.2%.

CSTL’s earnings beat estimates in each of the trailing four quarters, the average surprise being 172.72%.

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