Larimar Therapeutics ( LRMR Quick Quote LRMR - Free Report) is a clinical-stage company making medicines for rare diseases. The company has a Zacks Rank #1 (Strong Buy).
Here we discuss some reasons why investing in LRMR stock now is a good idea.
Its lead pipeline candidate is CTI-1601, which is being developed as the first Friedreich's ataxia (FA) therapy designed to potentially address frataxin deficiency, which is the underlying cause of this rare, progressive and fatal disease. Currently, there are no treatment options that address the problem of frataxin deficiency in FA patients. Larimar’s pipeline is progressing rapidly with some key catalysts expected this year.
The CTI-1601 program was placed on clinical hold by the FDA in May 2021, following deaths in high-dose arms in a pre-clinical toxicology study that was designed to support extended dosing of patients with CTI-1601. In September 2022, the FDA lifted the full clinical hold placed on the CTI-1601 program and imposed a partial hold. With the lifting of the full clinical hold, Larimar began enrollment in the first cohort (25 mg cohort) of the phase II four-week dose exploration study in FA patients. It has completed enrollment in the 25 mg cohort data.
Larimar expects to provide an update that will outline the next steps for the study in the second quarter after the FDA and independent data monitoring committee (IDMC) review data from the first cohort. Initiation of the second cohort and/or other clinical studies is contingent upon FDA and IDMC’s review of data from the 25 mg cohort. Top-line data from both dose cohorts of the phase II study is expected in the second half of 2023.
Prior phase I results on CTI-1601 provided clinical proof-of-concept for the candidate by demonstrating its ability to increase frataxin levels in peripheral tissues.
CTI-1601 has been granted Rare Pediatric Disease designation, Fast Track designation and Orphan Drug designations by the FDA.
In September 2022, Larimar raised net proceeds of approximately $75.2 million through an underwritten offering of common stock. This should boost the company’s cash position. At the end of 2022, Larimar had $118.4 million in cash on hand, which it expects can fund its operations till the second half of 2024.
The stock of Larimar Therapeutics has risen 37.4% in the past year against a decrease of 16.6% for the
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Loss estimates for 2023 have narrowed from $2.07 per share to $1.08 per share over the past 60 days, while that for 2024 have narrowed from $2.28 per share to $1.15 per share over the same timeframe.
We remind investors that last month, the FDA approved
Reata Pharmaceuticals’ drug Skyclarys, or omaveloxolone, across a broad patient population of FA. The FDA approved Skyclarys for treating FA in adults and adolescents aged 16 years and older.
The recommended dosage of Skyclarys is 150 mg taken orally once daily. The FDA actually gave a broad label to Skyclarys. Its label does not include any safety-related warning like a boxed warning or a drug safety program like Risk Evaluation and Mitigation Strategy. Only patients’ bilirubin levels and lipid parameters should be monitored prior to the initiation of Skyclarys. The FDA has also not issued any post-approval requirements. Reata has priced Skyclarys at an annual cost of $370,000. Skyclarys is the first drug to be approved specifically for the treatment of FA and is also Reata’s first commercial product. There are no currently approved disease-modifying therapies for FA.
The FDA approval for a broad label of Skyclarys increased investors’ focus on Larimar Therapeutics and increased the probability of the approval of CTI-1601.
Other Stocks to Consider
Some other top-ranked small drugmakers are
Surrozen ( SRZN Quick Quote SRZN - Free Report) and KalVista Pharmaceuticals ( KALV Quick Quote KALV - Free Report) . While Surrozen has the same Zacks Rank as Larimar, KalVista Pharmaceuticals has a Zacks Rank of 2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Estimates for Surrozen’s 2023 loss per share have narrowed from $1.18 per share to $1.00 per share over the past 60 days while that for 2024 have narrowed from 68 cents per share to 52 cents per share over the same timeframe. Surrozen’s stock has declined 78.8% in the past year.
Surrozen beat earnings expectations in the trailing four quarters. The company delivered a four-quarter earnings surprise of 43.85%, on average.
Estimates for KalVista Pharmaceuticals’ 2023 loss per share have narrowed from $3.90 per share to $3.76 per share over the past 60 days while that for 2024 has narrowed from $3.38 per share to $3.07 per share over the same timeframe. KalVista Pharmaceuticals’ stock has declined 40.6% in the past year.
KalVista Pharmaceuticals beat earnings expectations in the trailing four quarters. The company delivered a four-quarter earnings surprise of 12.88%, on average.