Amicus Therapeutics To Present At Upcoming Investor Conferences In March 2025

Madrigal Pharmaceuticals MDGL reported fourth-quarter 2024 loss of $2.71 per share, narrower than the Zacks Consensus Estimate of a loss of $4.12. In the year-ago quarter, the company had incurred a loss of $5.68 per share.

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During the quarter, the company generated total revenues of $103.3 million entirely from product sales of its metabolic dysfunction-associated steatohepatitis (MASH) drug Rezdiffra (resmetirom), which was approved last year. The metric beat the Zacks Consensus Estimate of $101.6 million. Since this is also the first marketed drug in Madrigal’s portfolio, launched in April 2024, management did not generate any sales in the year-ago period.

MDGL shares are gaining in the premarket hour, in response to the better-than-expected earnings results.

MDGL’s Q4 Results in Detail

In March 2024, the FDA granted accelerated approval to Rezdiffra, making it the first and currently the only approved therapy for MASH indication. The eligible patient population includes adults with noncirrhotic MASH with moderate to advanced liver fibrosis. The drug’s commercial launch is off to a strong start in the country, driven by early patient demand for the drug.

During the quarter, research and development expenses declined 64% to $25.6 million. The decrease can be attributed to reduced clinical study costs and change in accounting for inventory costs following FDA approval of Rezdiffra, partially offset by increases in headcount.

Selling, general and administrative expenses were $141.2 million in the reported quarter compared with $46.5 million in the year-ago period. This exponential rise was on account of increased commercial launch activities for Rezdiffra, including significant commercial headcount expansion and stock compensation expense.

Madrigal had cash, cash equivalents and marketable securities worth $0.93 billion as of Dec. 31, 2024, compared with $1 billion reported as of Sept. 30, 2024.

In the past three months, Madrigal shares have lost 6.6% compared with the industry’s 8% decline.

MDGL’s Full-Year Results

In 2024, Madrigal recorded total revenues of $180.1 million, entirely from the sales of Rezdiffra since launch, which beat the Zacks Consensus Estimate of $174.3 million. No revenues were recorded in 2023.

The company’s loss per share in 2024 was $21.9, narrower than the Zacks Consensus Estimate of a loss of $23.5. In 2023, MDGL recorded a loss per share of $20.

MDGL’s Pipeline & Other Updates

Madrigal has also submitted a regulatory filing in the EU seeking approval for Rezdiffra for MASH indication. A final decision is expected in mid-2025.

As the FDA approved Rezdiffra under the accelerated pathway, the continued approval will be based on promising long-term safety and efficacy data from the pivotal phase III MAESTRO-NASH biopsy study. This late-stage study, which provided the data for the drug's accelerated approval for MASH, is ongoing as an outcomes study. The goal is to generate confirmatory 54-month data that could verify the clinical benefits and support the full approval of the drug for the noncirrhotic MASH indication.

In addition to the study, a second phase III outcomes study (MAESTRO-NASH OUTCOMES) is underway, evaluating the progression to liver decompensation events in patients with compensated MASH cirrhosis treated with Rezdiffra compared with placebo. Topline data is expected in 2027. A positive outcome from this study is also expected to support the full approval of Rezdiffra for noncirrhotic MASH and expand the eligible patient population for Rezdiffra.

The open-label extension (OLE) arm of the MAESTRO-NAFLD-1 study is also currently evaluating the drug in patients with compensated MASH cirrhosis. In a separate press release, Madrigal reported positive two-year data from the OLE arm. Per the data readout, a statistically significant mean reduction in liver stiffness of 6.7 kPa was observed in MASH patients upon treatment with Rezdiffra. This figure is also the largest reduction reported in an F4c MASH patient population to date.

Additionally, 51% of patients achieved a ≥25% reduction in liver stiffness, a level linked to a lower risk of end-stage liver disease. Rezdiffra maintained a favorable safety and tolerability profile, with a low discontinuation rate due to adverse events. Detailed data, along with additional findings from the MAESTRO-NAFLD-1 OLE study, are expected to be presented at a future medical conference.

The results reinforce Rezdiffra’s potential benefit for patients with compensated MASH cirrhosis and support the potential success of the ongoing MAESTRO-NASH OUTCOMES study. These ongoing studies demonstrate Madrigal's commitment to establishing the drug as the standard-of-care treatment for MASH.

Madrigal Pharmaceuticals, Inc. Price, Consensus and EPS Surprise

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

MDGL’s Zacks Rank & Stocks to Consider

Madrigal currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks from the sector are Beam Therapeutics BEAM, BioMarin Pharmaceutical BMRN and Amicus Therapeutics FOLD, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 (Strong Buy) Rank stocks here.

In the past 30 days, Beam Therapeutics’ loss estimates for 2025 have narrowed from $4.29 to $4.27 per share. During the same timeframe, the loss per share for 2026 has improved from $5.16 to $4.54. In the past three months, shares of Beam Therapeutics have gained 19.1%.

BMRN’s earnings beat estimates in three of the trailing four quarters and missed the mark once, delivering an average surprise of 3.92%.

In the past 30 days, estimates for BioMarin Pharmaceutical’s 2025 earnings per share have increased from $4.01 to $4.19. Estimates for 2026 earnings per share have decreased from $5.21 to $5.20 during the same timeframe. In the past three months, BioMarin Pharmaceutical shares have lost 0.9%.

BMRN’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 32.36%.

In the past 30 days, the estimate for Amicus Therapeutics’ 2025 earnings per share has remained constant at 43 cents. The estimate for 2026 earnings per share has deteriorated from 72 cents to 71 cents. In the past three months, shares of Amicus Therapeutics have lost 1.9%.

FOLD’s earnings beat estimates in three of the trailing four quarters and missed once, delivering an average surprise of 45.42%.

This article originally published on Zacks Investment Research (zacks.com).

Zacks Investment Research

Amicus Therapeutics FOLD reported fourth-quarter 2024 adjusted earnings of 9 cents per share, which missed the Zacks Consensus Estimate of 10 cents. The company had reported earnings of 1 cent per share in the year-ago quarter.

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Revenues in the fourth quarter totaled $149.7 million, up 30% year over year on a reported basis and a constant-currency (cc) basis. The figure marginally beat the Zacks Consensus Estimate of $149 million. The top line comprised sales of Galafold (migalastat), which is approved for Fabry disease and the newly approved combo drug, Pombiliti + Opfolda.

The FDA approved Pombiliti + Opfolda, a two-component therapy for treating late-onset Pompe disease, in September 2023.

The top-line increased year over year owing to higher revenues from Galafold sales and incremental revenues from the sale of Pombiliti + Opfolda. Both Fabry disease and Pompe disease are rare diseases. Amicus is currently advancing ongoing studies to broaden labels in Fabry and Pompe diseases.

In the past year, shares of Amicus have plunged 29.5% compared with the industry’s decline of 8.1%.

More on FOLD's Q4 Results

In the fourth quarter, Galafold sales were $127.5 million, up 20% year over year at cc, driven by continued strong demand. However, Galafold sales missed the Zacks Consensus Estimate of $141 million and also our model estimate of $127.7 million.

Weaker-than-expected Galafold sales could be the reason for the stock price to decline 2.9% on Feb. 19.

Net product sales of Pombiliti + Opfolda were $22.2 million, reflecting sequential growth of 5.2%. The figure narrowly missed the Zacks Consensus Estimate of $22.3 million and was in-line with our model estimate.

Per Amicus, as of the end of 2024, almost 220 patients were treated or are scheduled to be treated with the combo drug in five markets (the United States, Germany, the United Kingdom, Spain and Austria).

As of Dec. 31, 2024, Amicus had cash, cash equivalents and marketable securities worth $249.9 million compared with $249.8 million as of Sept. 30, 2024.

FOLD's Full-Year Results

For 2024, Amicus generated revenues of $528.3 million, reflecting a 33% increase year over year at cc.

For the same period, the company reported adjusted earnings of 24 cents per share against a loss of 13 cents per share reported in the year-ago period.

FOLD's 2025 Guidance

The company reiterated its total revenue guidance for 2025, which it had provided last month.

For full-year 2025, management expects total revenues to grow in the range of 17%-24% on a constant exchange rate (CER).

Revenues from Galafold are expected to grow in the range of 10%-15% at CER in 2025.

The company expects Pombiliti + Opfolda revenue growth of 65%-85% at CER.

Total adjusted operating expenses are expected in the band of $350-$370 million.

Amicus expects to achieve positive GAAP net income in the second half of 2025. The company expects to exceed $1 billion in total sales in 2028.

FOLD Settles Galafold Patent Litigation

In October 2024, Amicus signed a licensing agreement with Teva Pharmaceuticals TEVA, resolving the patent lawsuit that it had filed earlier.

The litigation arose after Teva submitted an abbreviated new drug application seeking approval to sell a generic version of Amicus' Galafold 123 mg capsules before the related patents expired.

Per the settlement terms, TEVA will not be able to sell its generic version of Galafold in the United States until January 2037.

The agreement also ended all ongoing litigation between Amicus and Teva concerning Galafold patents in the U.S. District Court for Delaware.

Amicus Therapeutics, Inc. Price, Consensus and EPS Surprise

Amicus Therapeutics, Inc. price-consensus-eps-surprise-chart | Amicus Therapeutics, Inc. Quote

FOLD's Zacks Rank & Other Stocks to Consider

Amicus currently carries a Zacks Rank #2 (Buy).

Some other top-ranked stocks in the biotech sector are Harmony Biosciences Holdings, Inc. HRMY and Pacira BioSciences, Inc. PCRX, each sporting 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 Harmony Biosciences’ earnings per share have increased from $3.01 to $3.16 for 2025. In the past year, shares of HRMY have risen 6.9%.

HRMY’s earnings beat estimates in three of the trailing four quarters while missing the same on the remaining occasion, the average surprise being 147.24%.

In the past 60 days, estimates for Pacira BioSciences’ earnings per share have increased from $2.79 to $3.46 for 2025. In the past year, shares of PCRX have decreased 12.8%.

PCRX’s earnings beat estimates in two of the trailing four quarters, met the same once and missed the same on the remaining occasion, the average surprise being 7.13%.

Zacks Investment Research

Krystal Biotech KRYS reported fourth-quarter earnings per share of $1.52, which beat the Zacks Consensus Estimate of $1.29 as Vyjuvek gains traction in the U.S. market. In the year-ago quarter, the company reported earnings of 30 cents per share.

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However, revenues totaled $91.1 million, which missed the Zacks Consensus Estimate of $97 million. Revenues came in solely from the sales of Vyjuvek. In the year-ago quarter, revenues amounted to $42.1 million.

KRYS’ Q4 Results in Details

In 2023, the FDA approved Vyjuvek, the first-ever revocable gene therapy for treating patients aged six months or older with dystrophic epidermolysis bullosa (“DEB”). DEB is a rare and severe monogenic disease that affects the skin and mucosal tissues. It is caused by one or more mutations in a gene called COL7A1.

As of February 2025, KRYS secured more than 510 reimbursement approvals for Vyjuvek in the United States. It also achieved positive access determinations for 97% of lives covered under commercial and Medicaid plans. The gross margin in the reported quarter was 95%. Additionally, patient adherence to weekly treatment while on the drug remained high at 85% as of the end of 2024.

Despite the mixed nature of earnings results in the fourth quarter of 2024, Krystal Biotech’s shares gained 12.4% yesterday, likely due to record revenue growth from Vyjuvek sales.

In the past three months, shares of KRYS have lost 3.4% against the industry’s 0.8% growth.

Research and development expenses amounted to $13.5 million, up 19% year over year. Selling, general and administrative expenses totaled $31.3 million, up 26% from the year-ago level.

Cash, cash equivalents and investments totaled $749.6 million as of Dec. 31, 2024, compared with $694.2 million as of Sept. 30, 2024.

KRYS’ Full-Year 2024 Results

In 2024, Krystal Biotech recorded total revenues of $290.5 million which fell short of the Zacks Consensus Estimate of $292.1 million. In 2023, the company’s total revenues amounted to $50.7 million.

For the full year, KRYS recorded earnings per share of $3, which surpassed the Zacks Consensus Estimate of $2.9. In the previous year, the company recorded earnings of 39 cents per share.

KRYS Makes Good Pipeline Progress

Vyjuvek is referred to as B-VEC outside the country. The European Medicines Agency’s review of the Marketing Authorization Application (“MAA”) for B-VEC for the treatment of DEB is ongoing.

KRYS expects an opinion from the Committee for Medicinal Products for Human Use on the MAA in the first quarter of 2025.

In October, Krystal Biotech filed a new drug application with Japan’s Pharmaceuticals and Medical Devices Agency. A decision on the same is expected in the second half of 2025.

KRYS is also advancing a robust preclinical and clinical pipeline of investigational genetic medicines in the fields of respiratory, oncology, dermatology, ophthalmology and aesthetics.

Krystal Biotech is evaluating KB407 for the treatment of cystic fibrosis. In December 2024, the company announced that dosing with the candidate was safe and well-tolerated by patients in both Cohort 1 and Cohort 2 of the ongoing KB407 phase I CORAL-1 study. Interim molecular data for Cohort 3 patients of the study is expected to be reported in mid-2025.

Another candidate, KB408, is being evaluated for the treatment of alpha-1 antitrypsin deficiency. During the reported quarter, Krystal Biotech reported successful SERPINA1 gene delivery and functional alpha-1 antitrypsin expression at therapeutic levels in Cohorts 1 and Cohorts 2 of its ongoing KB408 phase I SERPENTINE-1 study. Inhaled KB408 was safe and well-tolerated at both tested doses. Based on these findings, Cohort 2 was expanded and Cohort 3 was opened for further molecular assessments. Results from both cohorts are expected in the second half of 2025.

The company is also evaluating KB803 (B-VEC formulated as an eyedrop) for ocular complications of DEB. Patient enrollment has been completed in a natural history study to prospectively collect data on the frequency of corneal abrasions in patients with DEB. This will allow KRYS to screen eligible patients for its registrational phase III IOLITE study, which will evaluate the effect of KB803 on corneal abrasions of DEB. The late-stage study is expected to begin in the first half of 2025.

In December 2024, KRYS provided an initial clinical update from the phase I/II KYANITE-1 study, evaluating inhaled KB707 in patients with locally advanced or metastatic solid tumors of the lung. Enrollment in KYANITE-1 is ongoing.

Krystal Biotech has also resumed the development of KB105 for the treatment of lamellar ichthyosis. It expects to commence the phase II portion of the JADE-1 study in pediatric patients in 2026.

KRYS’ wholly-owned subsidiary, Jeune Aesthetics, previously announced positive interim safety and efficacy results from the PEARL-1 study of KB301 in the treatment of dynamic wrinkles of the décolleté. Based on such data, Jeune Aesthetics is currently gearing up to initiate a phase II study of the candidate for the same indication in the second half of 2025.

During the reported quarter, Jeune Aesthetics also began dosing patients in a phase I PEARL-2 study, evaluating its second clinical-stage investigational aesthetic product, KB304, for the treatment of wrinkles. Top-line data is expected in the second half of 2025.

Krystal Biotech, Inc. Price, Consensus and EPS Surprise

Krystal Biotech, Inc. price-consensus-eps-surprise-chart | Krystal Biotech, Inc. Quote

KRYS’ Zacks Rank and Stocks to Consider

Krystal carries a Zacks Rank #3 (Hold) at present.

Some better-ranked stocks from the sector are Castle Biosciences CSTL, BioMarin Pharmaceutical BMRN and Amicus Therapeutics FOLD, carrying a Zacks Rank #2 (Buy) each at present. You can see the complete list of today’s Zacks #1 (Strong Buy) Rank stocks here.

In the past 30 days, Castle Biosciences’ earnings estimates for 2024 have increased from 34 cents to 45 cents per share. During the same timeframe, the loss per share for 2025 has improved from $1.84 to $1.51. In the past three months, shares of Castle Biosciences have plunged 5.9%.

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

In the past 30 days, estimates for BioMarin Pharmaceutical’s 2024 earnings per share have increased from $3.28 to $3.29. Estimates for 2025 earnings per share have decreased from $4.02 to $4.01 during the same timeframe. In the past three months, BioMarin Pharmaceutical shares have gained 3.4%.

BMRN’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 28.7%.

In the past 30 days, the estimate for Amicus Therapeutics’ 2024 earnings per share has remained constant at 23 cents. The estimate for 2025 earnings per share has deteriorated from 44 cents to 43 cents. In the past three months, shares of Amicus Therapeutics have gained 0.6%.

FOLD’s earnings beat estimates in three of the trailing four quarters and missed once, delivering an average surprise of 27.09%.

Zacks Investment Research