Biomarin Pharmaceutical 株価情報 (NASDAQ: BMRN)

最新ニュース Biomarin Pharmaceutical (NASDAQ:BMRN) 株価情報

BIOMARIN PHARMACEUTICAL INC

Biomarin Pharmaceutical

BioMarin is a global biotechnology company focused on developing and commercializing therapies for rare genetic diseases. BioMarin specializes in enzyme replacement therapies, gene therapies, and other advanced treatments aimed at addressing complex genetic disorders. The company has eight approved therapies for conditions including achondroplasia, phenylketonuria (PKU), hemophilia, and mucopolysaccharidosis. BioMarin has an expanding, yet relatively early-stage, pipeline of treatments in development.

BioMarin Pharmaceutical Inc. - Common Stock

Biomarin Pharmaceutical Inc

BioMarin Pharmaceutical Inc.&nbsp;(NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) released topline data from the Phase 3 PEGASUS trial evaluating Palynziq&nbsp;(pegvaliase-pqpz) compared to diet alone in 55 adolescents aged <a target="_blank" href="https://www.benzinga.com/general/health-care" rel="noreferrer noopener">12-17 with phenylketonuria (PKU)</a>.
PKU is&nbsp;a rare, inherited metabolic disorder that prevents the body from breaking down the amino acid phenylalanine. Untreated phenylketonuria can lead to brain damage, intellectual disabilities, <a target="_blank" href="https://www.benzinga.com/pressreleases/25/04/n44606179/biomarin-announces-positive-pivotal-data-for-palynziq-pegvaliase-pqpz-in-adolescents-with-phenylke" rel="noreferrer noopener">behavioral symptoms or seizures</a>.
The primary endpoints are the changes in blood Phe concentration and characterization of the safety profile in adolescents. Secondary endpoints include changes in total dietary protein intake and pharmacokinetics.&nbsp;
Also Read: <a target="_blank" href="https://www.benzinga.com/general/biotech/25/02/43849658/why-is-biomarin-stock-trading-higher-on-thursday" rel="noreferrer noopener">Why Is BioMarin Stock Trading Higher On Thursday?</a>
The study met its primary efficacy endpoint, demonstrating a statistically significant lowering in blood Phe levels in adolescents aged 12-17 with phenylketonuria (PKU) compared to diet alone.
Safety results were consistent with the known profile of the medicine.
Palynziq is the first and only enzyme therapy approved to treat adults with PKU.
Detailed results from the PEGASUS study will be presented at an upcoming medical meeting and submitted to global health authorities later this year to request a label expansion for Palynziq to include adolescents.
Palynziq substitutes the deficient phenylalanine hydroxylase (PAH) enzyme in PKU with a PEGylated version of the enzyme phenylalanine ammonia lyase to break down Phe.&nbsp;Palynziq is administered using a dosing regimen designed to facilitate tolerability.
Palynziq is approved to reduce blood Phe concentrations for adults in the U.S., for people 16 and older in the EU,&nbsp;Canada, and&nbsp;Brazil, and for people 15 and older in&nbsp;Japan&nbsp;with PKU who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management.
Price Action: BMRN stock is up 0.29% at $68.14 at the last check on Wednesday.
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BMRN

BioMarin Seeks Label Expansion For Palynziq After Positive Phase 3 Results In Adolescent Patients

New data also to be presented for PALYNZIQ&#xAE; (pegvaliaise-pqpz), reinforcing its value in sustaining blood Phe level reduction and improving health-related quality of life for adults with phenylketonuria
SAN RAFAEL, Calif., March 20, 2025 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) today announced positive new data from studies of VOXZOGO&#xAE;&nbsp;(vosoritide) in children with achondroplasia and in ongoing clinical trials investigating other skeletal conditions, as well as PALYNZIQ&#xAE;&nbsp;(pegvaliase-pqpz) in adults with phenylketonuria (PKU). The latest findings further validate the long-term benefit and established safety profiles of both medicines, which continue to play a pivotal role for people living with these genetically defined conditions.
The data will be presented at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Los Angeles.
Data Highlight VOXZOGO Treatment Adherence and Favorable Safety Profile in Infants
New data from a study in Japan showed strong treatment adherence with VOXZOGO in children with achondroplasia under the age of 3, with no reported treatment-related adverse events nor any dose interruptions among 63 children followed for up to 23.7 months. These real-world findings validate VOXZOGO's established safety profile and reinforce the therapeutic benefit seen in clinical studies. The study's safety results, including in infants as young as 1 month old, add to the growing body of evidence supporting early treatment initiation.

BioMarin Presents New Data Demonstrating Favorable Safety And Strong Adherence In Real-World Clinical Practice With VOXZOGO In Children Under 3 With Achondroplasia At 2025 ACMG Annual Clinical Genetics Meeting

Oppenheimer analyst Leland Gershell upgrades Biomarin Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) from Perform to Outperform and announces $98 price target.

Upgrades

Price Target

Analyst Ratings

Oppenheimer Upgrades Biomarin Pharmaceutical to Outperform, Announces $98 Price Target

In the last three months, 8 analysts have published ratings on Biomarin Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>), offering a diverse range of perspectives from bullish to bearish.
In the table below, you'll find a summary of their recent ratings, revealing the shifting sentiments over the past 30 days and comparing them to the previous months.
<table>
<thead>
<tr>
<th></th>
<th align="right">Bullish</th>
<th align="right">Somewhat Bullish</th>
<th align="right">Indifferent</th>
<th align="right">Somewhat Bearish</th>
<th align="right">Bearish</th>
</tr>
</thead>
<tbody>
<tr>
<td>Total Ratings</td>
<td align="right">2</td>
<td align="right">3</td>
<td align="right">3</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>Last 30D</td>
<td align="right">2</td>
<td align="right">2</td>
<td align="right">3</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>1M Ago</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>2M Ago</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>3M Ago</td>
<td align="right">0</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
</tbody>
</table>
Analysts provide deeper insights through their assessments of 12-month price targets, revealing an average target of $94.25, a high estimate of $126.00, and a low estimate of $70.00. This current average has increased by 2.02% from the previous average price target of $92.38. 
<img src="https://www.benzinga.com/files/images/story/2025/1740088999_0.png" alt="price target chart" />
<h2>Breaking Down Analyst Ratings: A Detailed Examination</h2>
A comprehensive examination of how financial experts perceive Biomarin Pharmaceutical is derived from recent analyst actions. The following is a detailed summary of key analysts, their recent evaluations, and adjustments to ratings and price targets.
<table>
<thead>
<tr>
<th>Analyst</th>
<th>Analyst Firm</th>
<th>Action Taken</th>
<th>Rating</th>
<th>Current Price Target</th>
<th>Prior Price Target</th>
</tr>
</thead>
<tbody>
<tr>
<td>George Farmer</td>
<td>Scotiabank</td>
<td>Raises</td>
<td>Sector Perform</td>
<td>$80.00</td>
<td>$78.00</td>
</tr>
<tr>
<td>David Lebovitz</td>
<td>Citigroup</td>
<td>Raises</td>
<td>Neutral</td>
<td>$82.00</td>
<td>$81.00</td>
</tr>
<tr>
<td>Luca Issi</td>
<td>RBC Capital</td>
<td>Maintains</td>
<td>Sector Perform</td>
<td>$70.00</td>
<td>$70.00</td>
</tr>
<tr>
<td>Olivia Brayer</td>
<td>Cantor Fitzgerald</td>
<td>Maintains</td>
<td>Overweight</td>
<td>$90.00</td>
<td>$90.00</td>
</tr>
<tr>
<td>Eliana Merle</td>
<td>UBS</td>
<td>Raises</td>
<td>Buy</td>
<td>$113.00</td>
<td>$109.00</td>
</tr>
<tr>
<td>Christopher Raymond</td>
<td>Piper Sandler</td>
<td>Raises</td>
<td>Overweight</td>
<td>$126.00</td>
<td>$122.00</td>
</tr>
<tr>
<td>Geoff Meacham</td>
<td>B of A Securities</td>
<td>Raises</td>
<td>Buy</td>
<td>$103.00</td>
<td>$99.00</td>
</tr>
<tr>
<td>Olivia Brayer</td>
<td>Cantor Fitzgerald</td>
<td>Maintains</td>
<td>Overweight</td>
<td>$90.00</td>
<td>$90.00</td>
</tr>
</tbody>
</table>
<h3>Key Insights:</h3>
<ul>
<li>Action Taken: Analysts respond to changes in market conditions and company performance, frequently updating their recommendations. Whether they 'Maintain', 'Raise' or 'Lower' their stance, it reflects their reaction to recent developments related to Biomarin Pharmaceutical. This information offers a snapshot of how analysts perceive the current state of the company.</li>
<li>Rating: Analysts assign qualitative assessments to stocks, ranging from 'Outperform' to 'Underperform'. These ratings convey the analysts' expectations for the relative performance of Biomarin Pharmaceutical compared to the broader market.</li>
<li>Price Targets: Analysts explore the dynamics of price targets, providing estimates for the future value of Biomarin Pharmaceutical's stock. This examination reveals shifts in analysts' expectations over time.</li>
</ul>
Capture valuable insights into Biomarin Pharmaceutical's market standing by understanding these analyst evaluations alongside pertinent financial indicators. Stay informed and make strategic decisions with our Ratings Table.
<a href="https://www.benzinga.com/quote/BMRN/analyst-ratings?utm_source=benzinga-insights&amp;adType=benzinga-insights&amp;ad=analyst-ratings&amp;campaign=wallstreetadvantage">Stay up to date on Biomarin Pharmaceutical analyst ratings.</a>
<h2>Get to Know Biomarin Pharmaceutical Better</h2>
BioMarin's focus is on rare-disease therapies. Genzyme markets Aldurazyme through its joint venture with BioMarin, and BioMarin markets Naglazyme, Vimizim, and Brineura independently. BioMarin also markets Kuvan and Palynziq to treat the rare metabolic disorder PKU. Voxzogo (vosoritide) was approved in achondroplasia in 2021. BioMarin's Roctavian (hemophilia A gene therapy) was approved in Europe in 2022 and in the US in 2023.
<h3>Financial Insights: Biomarin Pharmaceutical</h3>
Market Capitalization Analysis: Falling below industry benchmarks, the company's market capitalization reflects a reduced size compared to peers. This positioning may be influenced by factors such as growth expectations or operational capacity.
Positive Revenue Trend: Examining Biomarin Pharmaceutical's financials over 3 months reveals a positive narrative. The company achieved a noteworthy revenue growth rate of 28.28% as of 30 September, 2024, showcasing a substantial increase in top-line earnings. As compared to competitors, the company surpassed expectations with a growth rate higher than the average among peers in the Health Care sector.
Net Margin: Biomarin Pharmaceutical's net margin lags behind industry averages, suggesting challenges in maintaining strong profitability. With a net margin of 14.22%, the company may face hurdles in effective cost management.
Return on Equity (ROE): The company's ROE is a standout performer, exceeding industry averages. With an impressive ROE of 1.98%, the company showcases effective utilization of equity capital.
Return on Assets (ROA): Biomarin Pharmaceutical's financial strength is reflected in its exceptional ROA, which exceeds industry averages. With a remarkable ROA of 1.52%, the company showcases efficient use of assets and strong financial health.
Debt Management: Biomarin Pharmaceutical's debt-to-equity ratio is below industry norms, indicating a sound financial structure with a ratio of 0.11.
<h2>Analyst Ratings: Simplified</h2>
Analysts work in banking and financial systems and typically specialize in reporting for stocks or defined sectors. Analysts may attend company conference calls and meetings, research company financial statements, and communicate with insiders to publish "analyst ratings" for stocks. Analysts typically rate each stock once per quarter.
Some analysts publish their predictions for metrics such as growth estimates, earnings, and revenue to provide additional guidance with their ratings. When using analyst ratings, it is important to keep in mind that stock and sector analysts are also human and are only offering their opinions to investors.
If you want to keep track of which analysts are outperforming others, you can view updated analyst ratings along withanalyst success scores in <a href="https://www.benzinga.com/pro/">Benzinga Pro</a>.
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Demystifying Biomarin Pharmaceutical: Insights From 8 Analyst Reviews

Scotiabank analyst George Farmer maintains Biomarin Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) with a Sector Perform and raises the price target from $78 to $80.

Scotiabank Maintains Sector Perform on Biomarin Pharmaceutical, Raises Price Target to $80

Citigroup analyst David Lebovitz maintains Biomarin Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) with a Neutral and raises the price target from $81 to $82.

Citigroup Maintains Neutral on Biomarin Pharmaceutical, Raises Price Target to $82

RBC Capital analyst Luca Issi reiterates Biomarin Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) with a Sector Perform and maintains $70 price target.

Reiteration

RBC Capital Reiterates Sector Perform on Biomarin Pharmaceutical, Maintains $70 Price Target

Cantor Fitzgerald analyst Olivia Brayer reiterates Biomarin Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) with a Overweight and maintains $90 price target.

Cantor Fitzgerald Reiterates Overweight on Biomarin Pharmaceutical, Maintains $90 Price Target

On Wednesday, BioMarin Pharmaceutical Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) reported fourth-quarter adjusted EPS of 92 cents, up from 49 cents reported a year ago, beating the <a href="https://www.benzinga.com/earnings" target="_blank" rel="noreferrer noopener">consensus of 53 cents</a>.
The company reported sales of $747.31 million, beating the consensus of $713.44 million. Total Revenues increased 16% year over year, driven by Voxzogo contributions from new <a target="_blank" href="https://www.benzinga.com/pressreleases/25/02/n43829778/biomarin-reports-fourth-quarter-and-full-year-2024-results-sets-full-year-2025-guidance" rel="noreferrer noopener">patient starts in all regions</a>.
In the quarter, revenues from BioMarin's Enzyme Therapies increased 9%, driven by increased patient demand in all regions and the timing of large government orders in certain regions outside the U.S.
Also Read: <a target="_blank" href="https://www.benzinga.com/general/biotech/25/02/43708807/why-is-rare-disease-focused-ascendis-pharma-stock-trading-higher-on-thursday" rel="noreferrer noopener">Why Is Rare Disease Focused Ascendis Pharma Stock Trading Higher On Thursday?</a>
Guidance: BioMarin Pharmaceutical expects fiscal year 2025 adjusted EPS of $4.20-$4.40 versus consensus of $3.04. The company expects 2025 sales of $3.1 billion-$3.2 billion versus a consensus of $3.11 billion.
"We expect 2025 to be another year of strong execution and growth in our Skeletal Conditions business unit, led by the continued global expansion of VOXZOGO for achondroplasia&#8230;In summary, we expect continued high performance as we benefit from BioMarin's revamped corporate strategy and operating model in 2025 and beyond," said Alexander Hardy, President and CEO of BioMarin.
In 2025, the company will continue to implement additional components of the $500 million cost transformation program announced in September of 2024, with full realization of benefits expected in 2026.
This ongoing integration of efficiencies in 2025 will enable BioMarin to realize a 40% adjusted operating margin in 2026.
William Blair notes that while BioMarin achieved its financial targets in 2024, its current valuation already accounts for expected revenue growth over the next 12–18 months.
Analyst Sami Corwin sees the limited expansion of Voxzogo's market until 2030–2031, when potential approvals for idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency could broaden its reach. A smaller boost may come in 2027 if its label expands to hypochondroplasia (HCH).
Corwin also flags Ascendis Pharma A/S' (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ASND#NASDAQ">ASND</a>) navepegritide as a potential challenge for Voxzogo, with a near-term stock overhang as Ascendis moves toward a possible NDA submission in early 2025.
Beyond Voxzogo, BioMarin's enzyme therapy unit provides stable but modest revenue growth, though upcoming competition for Palynziq in 2025 could affect its compound annual growth rate.
Analyst Reaction:
<ul>
<li>BofA Securities maintains BioMarin with a Buy, raising the price target from $99 to $103.</li>
<li>Piper Sandler maintains BioMarin with an Overweight rating while raising the price target from $122 to $126.</li>
</ul>
Price Action: BMRN stock is up 3.19% at $67.70&nbsp;at the last check on Thursday.
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</ul>

Analyst Color

Earnings

Guidance

Movers

Trading Ideas

ASND

Why Is BioMarin Stock Trading Higher On Thursday?

Piper Sandler analyst Christopher Raymond maintains Biomarin Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) with a Overweight and raises the price target from $122 to $126.

Piper Sandler Maintains Overweight on Biomarin Pharmaceutical, Raises Price Target to $126

B of A Securities analyst Geoff Meacham maintains Biomarin Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) with a Buy and raises the price target from $99 to $103.

B of A Securities Maintains Buy on Biomarin Pharmaceutical, Raises Price Target to $103

WIIM

BioMarin Pharma shares are trading higher after the company reported better-than-expected Q4 financial results.

2025 Full-Year Financial Guidance (in millions, except % and EPS amounts)
<figure class="table"> 
<table>
<tbody>
<tr>
<td colspan="1" rowspan="1">Item</td>
<td colspan="1" rowspan="1"> &nbsp;</td>
<td colspan="5" rowspan="1">2025 Guidance</td>
</tr>
<tr>
<td colspan="1" rowspan="1">Total Revenues (1)</td>
<td colspan="1" rowspan="1"> &nbsp;</td>
<td colspan="1" rowspan="1">$3,100</td>
<td colspan="1" rowspan="1"> &nbsp;</td>
<td colspan="1" rowspan="1">to</td>
<td colspan="1" rowspan="1"> &nbsp;</td>
<td colspan="1" rowspan="1">$3,200</td>
</tr>
<tr>
<td colspan="1" rowspan="1">Non-GAAP Operating Margin % (2)</td>
<td colspan="1" rowspan="1"> &nbsp;</td>
<td colspan="1" rowspan="1">32&nbsp;%</td>
<td colspan="1" rowspan="1"> &nbsp;</td>
<td colspan="1" rowspan="1">to</td>
<td colspan="1" rowspan="1"> &nbsp;</td>
<td colspan="1" rowspan="1">33&nbsp;%</td>
</tr>
<tr>
<td colspan="1" rowspan="1">Non-GAAP Diluted EPS (2)(3)</td>
<td colspan="1" rowspan="1"> &nbsp;</td>
<td colspan="1" rowspan="1">$4.20</td>
<td colspan="1" rowspan="1"> &nbsp;</td>
<td colspan="1" rowspan="1">to</td>
<td colspan="1" rowspan="1"> &nbsp;</td>
<td colspan="1" rowspan="1">$4.40</td>
</tr>
</tbody>
</table>
</figure><figure class="table"> 
<table>
<tbody>
<tr>
<td colspan="1" rowspan="1"> &nbsp;</td>
<td colspan="1" rowspan="1"> &nbsp;</td>
</tr>
<tr>
<td colspan="1" rowspan="1">(1)</td>
<td colspan="1" rowspan="1">VOXZOGO contribution to full-year 2025 Total Revenues expected to be in the range of $900 million to $950 million.</td>
</tr>
<tr>
<td colspan="1" rowspan="1">(2)</td>
<td colspan="1" rowspan="1">Refer to Non-GAAP Information beginning on page 10&nbsp;of this press release for definitions of Non-GAAP Operating Margin and Non-GAAP Diluted EPS.</td>
</tr>
<tr>
<td colspan="1" rowspan="1">(3)</td>
<td colspan="1" rowspan="1">Non-GAAP Diluted EPS guidance assumes approximately 200 million Weighted-Average Diluted Shares Outstanding.</td>
</tr>
</tbody>
</table>
</figure>
BioMarin will host a conference call and webcast to discuss fourth quarter and full-year 2024 financial results today, Wednesday, February&nbsp;19, 2025, at 4:30 p.m. ET. This event can be accessed through this link or on the investor section of the BioMarin website at&nbsp;www.biomarin.com.

BioMarin Pharmaceutical Sees FY25 Adj. EPS $4.20-$4.40 Vs $3.04 Est.; Revenue $3.1B-$3.2B Vs $3.105B Est.

Biomarin Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) reported quarterly earnings of $0.92 per share which beat the analyst consensus estimate of $0.53 by 73.58 percent. This is a 87.76 percent increase over earnings of $0.49 per share from the same period last year. The company reported quarterly sales of $747.31 million which beat the analyst consensus estimate of $713.44 million by 4.75 percent. This is a 15.65 percent increase over sales of $646.21 million the same period last year.

Biomarin Pharmaceutical Q4 Adj $0.92 Beats $0.53 Estimate, Sales $747.31M Beat $713.44M Estimate

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Earnings Scheduled For February 19, 2025

On Wednesday, Ascendis Pharma (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ASND#NASDAQ">ASND</a>) reported a fourth-quarter EPS loss of 64 cents, beating the <a target="_blank" href="https://www.benzinga.com/calendars/earnings" rel="noreferrer noopener">consensus loss of 1.07 euros</a>.
Total revenue for the fourth quarter of 2024 was 173.9 million euros, beating the consensus of 111.178 million euros compared to 137.7 million <a target="_blank" href="https://www.benzinga.com/pressreleases/25/02/g43674382/ascendis-pharma-reports-fourth-quarter-and-full-year-2024-financial-results" rel="noreferrer noopener">euros reported a year ago</a>.
The increase was primarily due to Novo Nordisk A/S&#8217;s (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/NVO#NYSE">NVO</a>) $100 million upfront fee and the EU launch of Yorvipath.
TransCon hGH (lonapegsomatropin, marketed as Skytrofa) sales reached 58.5 million euros.
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TransCon PTH (palopegteriparatide, marketed as Yorvipath) revenue for the fourth quarter of 2024 totaled €13.6 million.
The company reported a strong start to the U.S. Yorvipath launch, with 908 prescriptions as of February 7, 2025, and 539 unique prescribing health care providers.
As of December 31, 2024, cash and cash equivalents totaled 559.5 million euros. Including the $100 million upfront payment, cash at the end of 2024 would have totaled 655 million euros.
Concurrently, Ascendis Pharma's board authorized the repurchase of up <a target="_blank" href="https://www.benzinga.com/pressreleases/25/02/g43674574/ascendis-pharma-as-announces-share-repurchase-program-net-settlement-of-certain-rsus" rel="noreferrer noopener">to $18.25 million shares</a>.
Goldman Sachs analyst writes that Yorvipath has only been available in the U.S. for a few months, but early prescription trends have been strong and widespread. However, insurance coverage and reimbursement challenges may slow revenue growth in the short term.
Looking ahead, analyst Paul Choi writes investors will likely focus on the company's progress toward achieving breakeven cash flow and key milestones, including:
<ul>
<li>The pace of Yorvipath's rollout in the U.S. and EU</li>
<li>Data from the COACH study on achondroplasia, expected in Q2 2025</li>
<li>Regulatory discussions on Transcon CNP</li>
<li>Competitive updates from BioMarin Plc (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) in achondroplasia and AstraZeneca Plc (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/AZN#NASDAQ">AZN</a>) in hypoparathyroidism.</li>
</ul>
With Yorvipath showing potential for a strong launch in 2025, Goldman Sachs maintains the Buy rating and raises the price target <a target="_blank" href="https://www.benzinga.com/quote/CVS/analyst-ratings" rel="noreferrer noopener">to $225 from $200</a>.
Price Action: ASND stock is up 15.8% at $146.08 at last check Thursday.
Read Next:
<ul>
<li><a href="https://www.benzinga.com/general/health-care/25/02/43697905/zoetis-drops-as-2025-guidance-falls-short-of-estimates" target="_blank" rel="noreferrer noopener">Zoetis Drops As 2025 Guidance Falls Short Of Estimates</a></li>
</ul>

Why Is Rare Disease Focused Ascendis Pharma Stock Trading Higher On Thursday?

This whale alert can help traders discover the next big trading opportunities.
Whales are entities with large sums of money and we track their transactions here at Benzinga on our options activity scanner.
Traders often look for circumstances when the market estimation of an option diverges away from its normal worth. Abnormal amounts of trading activity could push option prices to hyperbolic or underperforming levels. 
Here's the list of options activity happening in today's session: 
<table>
<thead>
<tr>
<th>Symbol</th>
<th>PUT/CALL</th>
<th>Trade Type</th>
<th>Sentiment</th>
<th>Exp. Date</th>
<th>Strike Price</th>
<th>Total Trade Price</th>
<th>Open Interest</th>
<th>Volume</th>
</tr>
</thead>
<tbody>
<tr>
<td>PFE</td>
<td>CALL</td>
<td>SWEEP</td>
<td>BULLISH</td>
<td>03/21/25</td>
<td>$27.50</td>
<td>$48.6K</td>
<td>44.4K</td>
<td>7.2K</td>
</tr>
<tr>
<td>ACAD</td>
<td>CALL</td>
<td>SWEEP</td>
<td>BULLISH</td>
<td>06/20/25</td>
<td>$20.00</td>
<td>$157.6K</td>
<td>109</td>
<td>1.9K</td>
</tr>
<tr>
<td>TEM</td>
<td>CALL</td>
<td>TRADE</td>
<td>BEARISH</td>
<td>04/17/25</td>
<td>$65.00</td>
<td>$28.5K</td>
<td>2.8K</td>
<td>840</td>
</tr>
<tr>
<td>ZTS</td>
<td>CALL</td>
<td>SWEEP</td>
<td>BEARISH</td>
<td>02/21/25</td>
<td>$180.00</td>
<td>$43.0K</td>
<td>1.4K</td>
<td>316</td>
</tr>
<tr>
<td>EBS</td>
<td>CALL</td>
<td>TRADE</td>
<td>BEARISH</td>
<td>09/19/25</td>
<td>$11.00</td>
<td>$43.8K</td>
<td>4</td>
<td>200</td>
</tr>
<tr>
<td>BMRN</td>
<td>CALL</td>
<td>TRADE</td>
<td>BULLISH</td>
<td>02/21/25</td>
<td>$65.00</td>
<td>$29.2K</td>
<td>1.3K</td>
<td>159</td>
</tr>
<tr>
<td>HUM</td>
<td>CALL</td>
<td>TRADE</td>
<td>BEARISH</td>
<td>04/17/25</td>
<td>$245.00</td>
<td>$191.7K</td>
<td>141</td>
<td>125</td>
</tr>
<tr>
<td>HIMS</td>
<td>CALL</td>
<td>TRADE</td>
<td>BULLISH</td>
<td>02/21/25</td>
<td>$37.00</td>
<td>$69.7K</td>
<td>1.0K</td>
<td>87</td>
</tr>
<tr>
<td>EXEL</td>
<td>CALL</td>
<td>TRADE</td>
<td>NEUTRAL</td>
<td>02/21/25</td>
<td>$17.00</td>
<td>$29.5K</td>
<td>98</td>
<td>50</td>
</tr>
<tr>
<td>LLY</td>
<td>CALL</td>
<td>TRADE</td>
<td>BEARISH</td>
<td>02/21/25</td>
<td>$800.00</td>
<td>$37.5K</td>
<td>843</td>
<td>31</td>
</tr>
</tbody>
</table>

<h2>Explanation</h2>
These bullet-by-bullet explanations have been constructed using the accompanying table. 
• For PFE (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/PFE#NYSE">PFE</a>), we notice a call option sweep that happens to be bullish, expiring in 37 day(s) on March 21, 2025. This event was a transfer of 2561 contract(s) at a $27.50 strike. This particular call needed to be split into 21 different trades to become filled. The total cost received by the writing party (or parties) was $48.6K, with a price of $19.0 per contract. There were 44426 open contracts at this strike prior to today, and today 7236 contract(s) were bought and sold. 
• For ACAD (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ACAD#NASDAQ">ACAD</a>), we notice a call option sweep that happens to be bullish, expiring in 128 day(s) on June 20, 2025. This event was a transfer of 901 contract(s) at a $20.00 strike. This particular call needed to be split into 8 different trades to become filled. The total cost received by the writing party (or parties) was $157.6K, with a price of $175.0 per contract. There were 109 open contracts at this strike prior to today, and today 1920 contract(s) were bought and sold. 
• For TEM (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/TEM#NASDAQ">TEM</a>), we notice a call option trade that happens to be bearish, expiring in 64 day(s) on April 17, 2025. This event was a transfer of 20 contract(s) at a $65.00 strike. The total cost received by the writing party (or parties) was $28.5K, with a price of $1425.0 per contract. There were 2849 open contracts at this strike prior to today, and today 840 contract(s) were bought and sold. 
• For ZTS (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/ZTS#NYSE">ZTS</a>), we notice a call option sweep that happens to be bearish, expiring in 9 day(s) on February 21, 2025. This event was a transfer of 200 contract(s) at a $180.00 strike. This particular call needed to be split into 6 different trades to become filled. The total cost received by the writing party (or parties) was $43.0K, with a price of $215.0 per contract. There were 1473 open contracts at this strike prior to today, and today 316 contract(s) were bought and sold. 
• Regarding EBS (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/EBS#NYSE">EBS</a>), we observe a call option trade with bearish sentiment. It expires in 219 day(s) on September 19, 2025. Parties traded 200 contract(s) at a $11.00 strike. The total cost received by the writing party (or parties) was $43.8K, with a price of $219.0 per contract. There were 4 open contracts at this strike prior to today, and today 200 contract(s) were bought and sold. 
• Regarding BMRN (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>), we observe a call option trade with bullish sentiment. It expires in 9 day(s) on February 21, 2025. Parties traded 150 contract(s) at a $65.00 strike. The total cost received by the writing party (or parties) was $29.2K, with a price of $195.0 per contract. There were 1333 open contracts at this strike prior to today, and today 159 contract(s) were bought and sold. 
• For HUM (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/HUM#NYSE">HUM</a>), we notice a call option trade that happens to be bearish, expiring in 64 day(s) on April 17, 2025. This event was a transfer of 65 contract(s) at a $245.00 strike. The total cost received by the writing party (or parties) was $191.7K, with a price of $2950.0 per contract. There were 141 open contracts at this strike prior to today, and today 125 contract(s) were bought and sold. 
• For HIMS (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/HIMS#NYSE">HIMS</a>), we notice a call option trade that happens to be bullish, expiring in 9 day(s) on February 21, 2025. This event was a transfer of 81 contract(s) at a $37.00 strike. The total cost received by the writing party (or parties) was $69.7K, with a price of $861.0 per contract. There were 1026 open contracts at this strike prior to today, and today 87 contract(s) were bought and sold. 
• For EXEL (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/EXEL#NASDAQ">EXEL</a>), we notice a call option trade that happens to be neutral, expiring in 9 day(s) on February 21, 2025. This event was a transfer of 18 contract(s) at a $17.00 strike. The total cost received by the writing party (or parties) was $29.5K, with a price of $1640.0 per contract. There were 98 open contracts at this strike prior to today, and today 50 contract(s) were bought and sold. 
• Regarding LLY (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/LLY#NYSE">LLY</a>), we observe a call option trade with bearish sentiment. It expires in 9 day(s) on February 21, 2025. Parties traded 5 contract(s) at a $800.00 strike. The total cost received by the writing party (or parties) was $37.5K, with a price of $7518.0 per contract. There were 843 open contracts at this strike prior to today, and today 31 contract(s) were bought and sold. 
Options Alert Terminology 
 - Call Contracts: The right to buy shares as indicated in the contract. 
 - Put Contracts: The right to sell shares as indicated in the contract. 
 - Expiration Date: When the contract expires. One must act on the contract by this date if one wants to use it. 
 - Premium/Option Price: The price of the contract. 
For more information, visit our <a href="https://pro.benzinga.com/blog/how-to-understand-option-alerts/">Guide to Understanding Options Alerts</a> or read <a href="https://www.benzinga.com/markets/options">more about unusual options activity</a>. 
This article was generated by Benzinga's automated content engine and reviewed by an editor.

Options

ACAD

EXEL

Hims & Hers Health, Inc. Class A Common Stock

HIMS

10 Health Care Stocks With Whale Alerts In Today's Session

These eleven large-cap stocks were the worst performers in the last week. Are they in your portfolio?
<ol>
<li>FTAI Aviation Ltd. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/FTAI#NASDAQ">FTAI</a>) shares plummeted 35.42% after short seller firm Muddy Waters Research announced they <a href="https://www.benzinga.com//node/43040046" target="_blank" rel="noreferrer noopener">initiated a short position in the company</a>.</li>
<li>Moderna, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/MRNA#NASDAQ">MRNA</a>) stock plunged 19.38% after the company updated its 2025 expected revenue range to $1.5 billion to $2.5 billion, below <a href="https://www.benzinga.com//node/42946454" target="_blank" rel="noreferrer noopener">analyst estimates of $2.951 billion</a>. Morgan Stanley maintained an Equal-Weight rating and lowered the <a href="https://www.benzinga.com/quote/mrna/analyst-ratings" target="_blank" rel="noreferrer noopener">price forecast from $70 to $38</a>.</li>
<li>GameStop Corporation (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/GME#NYSE">GME</a>) stock declined 14.86%. In a recent X post, CNBC's Jim Cramer <a href="https://www.benzinga.com//node/43053808" target="_blank" rel="noreferrer noopener">steered investors toward</a> quantum stocks, suggesting they reinvest gains from GameStop into Rigetti Computing Inc (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RGTI#NASDAQ">RGTI</a>).</li>
<li>Snap Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/SNAP#NYSE">SNAP</a>) shares dipped 12.91% following a report suggesting that Trump is considering an executive order to save TikTok from a U.S. ban.</li>
<li>Rocket Lab USA, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RKLB#NASDAQ">RKLB</a>) shares were down 10.81%. The company disclosed that its second Pioneer spacecraft for Varda Space Industries, Inc. is<a href="https://www.benzinga.com//node/43028933" target="_blank" rel="noreferrer noopener"> successfully operating in orbit</a>.</li>
<li>Natera, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/NTRA#NASDAQ">NTRA</a>) stock lost 9.51% last week despite Guggenheim <a href="https://www.benzinga.com/quote/ntra/analyst-ratings" target="_blank" rel="noreferrer noopener">raising the price forecast</a> from $170 to $200. The company is due to provide a roadmap and outlook at the J.P. Morgan Healthcare Conference.</li>
<li>Eli Lilly and Company (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/LLY#NYSE">LLY</a>) stock decreased 9.27%  after the company issued FY24 revenue guidance <a href="https://www.benzinga.com//node/42976923" target="_blank" rel="noreferrer noopener">below the previous estimate</a>.</li>
<li>ICON plc (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ICLR#NASDAQ">ICLR</a>) stock fell 9.22% after the company issued FY25 guidance with <a href="https://www.benzinga.com//node/42971328" target="_blank" rel="noreferrer noopener">its midpoint below estimates</a>.</li>
<li>BioNTech SE (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BNTX#NASDAQ">BNTX</a>) shares declined 8.89% after Moderna issued 2025 revenue <a href="https://www.benzinga.com//node/42951363" target="_blank" rel="noreferrer noopener">guidance below analyst estimates</a>.</li>
<li>Novo Nordisk A/S (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/NVO#NYSE">NVO</a>) shares fell 8.89% after Ozempic and Wegovy were included in a list of 15 medications for <a href="https://www.benzinga.com//node/43062779" target="_blank" rel="noreferrer noopener">Medicare drug price negotiations</a>.</li>
<li>BioMarin Pharmaceutical Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) shares tumbled 8.68%. The company initiated a legal action against Ascendis Pharma A/S for infringement of European patent EP 3 175 863 B1 at the <a href="https://www.benzinga.com//node/42953403" target="_blank" rel="noreferrer noopener">Unified Patent Court (UPC) in Munich, Germany</a>.</li>
</ol>
Photos courtesy: Shutterstock

Equities

Short Sellers

BNTX

FTAI

MRNA

NTRA

RGTI

RKLB

SNAP

Moderna, GameStop And Snap Are Among Top Large-Cap Losers Last Week (Jan 13-Jan 17): Are The Others In Your Portfolio?

BioMarin Pharmaceutical Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BMRN#NASDAQ">BMRN</a>) today announced that the company has initiated a legal action against Ascendis Pharma A/S for infringement of European patent EP 3 175 863 B1 at the Unified Patent Court (UPC) in Munich, Germany. The patent covers long-acting variants of C-Type Natriuretic Peptide (CNP).
The legal action, which would result in a decision in the next 12-15 months, is based on BioMarin's belief that Ascendis' TransCon CNP investigational product and its development program in Germany and elsewhere in Europe infringe BioMarin's patent. The BioMarin patent was confirmed by the European Patent Office's Opposition Division in 2024.
The UPC, which formally started in June of 2023, has jurisdiction over European patents for 18 European member states that have ratified the Unitary Patents (UPs), including: Austria, Belgium, Bulgaria, Denmark, Estonia, Finland, France, Germany, Italy, Latvia, Lithuania, Luxembourg, Malta, Netherlands, Portugal, Romania, Slovenia and Sweden.

Legal

BioMarin Pharmaceutical Announces The Company Has Initiated A Legal Action Against Ascendis Pharma A/S For Infringement Of European Patent EP 3 175 863 B1 At The Unified Patent Court In Munich, Germany

Press Releases

BioMarin to Present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, at 9:00 am PT / 12:00 pm ET, in San Francisco, CA

BioMarin Announces Legal Action Against Ascendis Pharma A/S in European Unified Patent Court

BioMarin to Host Fourth Quarter and Full-Year 2024 Financial Results Conference Call and Webcast on Wednesday, February 19, 2025, at 4:30pm ET

BioMarin Reports Fourth Quarter and Full Year 2024 Results; Sets Full Year 2025 Guidance

BioMarin Announces Appointment of Timothy P. Walbert to Board of Directors

BioMarin to Present at the TD Cowen 45th Annual Healthcare Conference on Tuesday, March 4 at 6:50 AM PT / 9:50 AM ET in Boston, MA

Seaport Therapeutics Appoints Robert J. Hombach to its Board of Directors as Audit Committee Chair

PureTech Founded Entity Seaport Therapeutics Appoints Robert J. Hombach to its Board of Directors as Audit Committee Chair

BioMarin Presents New Data Demonstrating Favorable Safety and Strong Adherence in Real-World Clinical Practice with VOXZOGO® (vosoritide) in Children Under 3 with Achondroplasia at 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting

BioMarin Announces Positive Pivotal Data for PALYNZIQ® (pegvaliase-pqpz) in Adolescents with Phenylketonuria

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