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Ultragenyx Pharmaceutical 株価情報 (NASDAQ: RARE)

最新ニュース Ultragenyx Pharmaceutical (NASDAQ:RARE) 株価情報

Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.

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Ultragenyx Pharmaceutical Inc

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Cantor Fitzgerald analyst Kristen Kluska reiterates Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) with a Overweight and maintains $118 price target.

News

Price Target

Reiteration

Analyst Ratings

RARE

Cantor Fitzgerald Reiterates Overweight on Ultragenyx Pharmaceutical, Maintains $118 Price Target

HC Wainwright &amp; Co. analyst Ed Arce reiterates Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) with a Buy and maintains $95 price target.

HC Wainwright & Co. Reiterates Buy on Ultragenyx Pharmaceutical, Maintains $95 Price Target

Ultragenyx Pharmaceutical Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) today announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted for review the Biologics License Application (BLA) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) action date of August 18, 2025. The FDA also informed the company that they are not currently planning to hold an advisory committee meeting to discuss this application.

Biotech

General

Ultragenyx Pharmaceutical Announces FDA Acceptance And Priority Review Of The BLA For UX111 AAV Gene Therapy To Treat Sanfilippo Syndrome Type A

Canaccord Genuity analyst Whitney Ijem maintains Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) with a Buy and raises the price target from $121 to $136.

Canaccord Genuity Maintains Buy on Ultragenyx Pharmaceutical, Raises Price Target to $136

In the latest quarter, 7 analysts provided ratings for Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>), showcasing a mix of bullish and bearish perspectives.
The table below offers a condensed view of their recent ratings, showcasing the changing sentiments over the past 30 days and comparing them to the preceding 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">4</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>Last 30D</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</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">1</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">1</td>
<td align="right">2</td>
<td align="right">1</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 $98.0, a high estimate of $140.00, and a low estimate of $48.00. Witnessing a positive shift, the current average has risen by 2.54% from the previous average price target of $95.57.
<img src="https://www.benzinga.com/files/images/story/2025/1739541641_0.png" alt="price target chart" />
<h2>Understanding Analyst Ratings: A Comprehensive Breakdown</h2>
The perception of Ultragenyx Pharmaceutical by financial experts is analyzed through recent analyst actions. The following summary presents 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>Ed Arce</td>
<td>HC Wainwright &amp; Co.</td>
<td>Maintains</td>
<td>Buy</td>
<td>$95.00</td>
<td>$95.00</td>
</tr>
<tr>
<td>Kristen Kluska</td>
<td>Cantor Fitzgerald</td>
<td>Maintains</td>
<td>Overweight</td>
<td>$118.00</td>
<td>$118.00</td>
</tr>
<tr>
<td>Christopher Raymond</td>
<td>Piper Sandler</td>
<td>Raises</td>
<td>Overweight</td>
<td>$140.00</td>
<td>$135.00</td>
</tr>
<tr>
<td>Laura Chico</td>
<td>Wedbush</td>
<td>Maintains</td>
<td>Neutral</td>
<td>$48.00</td>
<td>$46.00</td>
</tr>
<tr>
<td>Ed Arce</td>
<td>HC Wainwright &amp; Co.</td>
<td>Maintains</td>
<td>Buy</td>
<td>$95.00</td>
<td>$95.00</td>
</tr>
<tr>
<td>Tiago Fauth</td>
<td>Wells Fargo</td>
<td>Raises</td>
<td>Overweight</td>
<td>$88.00</td>
<td>$75.00</td>
</tr>
<tr>
<td>Anupam Rama</td>
<td>JP Morgan</td>
<td>Lowers</td>
<td>Overweight</td>
<td>$102.00</td>
<td>$105.00</td>
</tr>
</tbody>
</table>
<h3>Key Insights:</h3>
<ul>
<li>Action Taken: In response to dynamic market conditions and company performance, analysts update their recommendations. Whether they 'Maintain', 'Raise', or 'Lower' their stance, it signifies their reaction to recent developments related to Ultragenyx Pharmaceutical. This insight gives a snapshot of analysts' perspectives on the current state of the company.</li>
<li>Rating: Offering insights into predictions, analysts assign qualitative values, from 'Outperform' to 'Underperform'. These ratings convey expectations for the relative performance of Ultragenyx Pharmaceutical compared to the broader market.</li>
<li>Price Targets: Gaining insights, analysts provide estimates for the future value of Ultragenyx Pharmaceutical's stock. This comparison reveals trends in analysts' expectations over time.</li>
</ul>
Navigating through these analyst evaluations alongside other financial indicators can contribute to a holistic understanding of Ultragenyx Pharmaceutical's market standing. Stay informed and make data-driven decisions with our Ratings Table.
<a href="https://www.benzinga.com/quote/RARE/analyst-ratings?utm_source=Benzinga Insights">Stay up to date on Ultragenyx Pharmaceutical analyst ratings.</a>
<h2>All You Need to Know About Ultragenyx Pharmaceutical</h2>
Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi, and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.
<h3>Ultragenyx Pharmaceutical: Financial Performance Dissected</h3>
Market Capitalization Analysis: Reflecting a smaller scale, the company's market capitalization is positioned below industry averages. This could be attributed to factors such as growth expectations or operational capacity.
Revenue Growth: Ultragenyx Pharmaceutical's remarkable performance in 3 months is evident. As of 30 September, 2024, the company achieved an impressive revenue growth rate of 42.27%. This signifies a substantial increase in the company's top-line earnings. When compared to others in the Health Care sector, the company faces challenges, achieving a growth rate lower than the average among peers.
Net Margin: The company's net margin is a standout performer, exceeding industry averages. With an impressive net margin of -95.71%, the company showcases strong profitability and effective cost control.
Return on Equity (ROE): Ultragenyx Pharmaceutical's ROE is below industry averages, indicating potential challenges in efficiently utilizing equity capital. With an ROE of -34.27%, the company may face hurdles in achieving optimal financial returns.
Return on Assets (ROA): Ultragenyx Pharmaceutical's ROA is below industry standards, pointing towards difficulties in efficiently utilizing assets. With an ROA of -8.46%, the company may encounter challenges in delivering satisfactory returns from its assets.
Debt Management: Ultragenyx Pharmaceutical's debt-to-equity ratio is below industry norms, indicating a sound financial structure with a ratio of 0.1.
<h2>Analyst Ratings: Simplified</h2>
Analyst ratings serve as essential indicators of stock performance, provided by experts in banking and financial systems. These specialists diligently analyze company financial statements, participate in conference calls, and engage with insiders to generate quarterly ratings for individual stocks.
Some analysts also offer predictions for helpful metrics such as earnings, revenue, and growth estimates to provide further guidance as to what to do with certain tickers. It is important to keep in mind that while stock and sector analysts are specialists, they are also human and can only forecast their beliefs to traders.
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Breaking Down Ultragenyx Pharmaceutical: 7 Analysts Share Their Views

Guidance

Ultragenyx Pharmaceutical Sees FY25 Revenue $640M-$670M Vs $652.946M Est.

Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) reported quarterly losses of $(1.39) per share which missed the analyst consensus estimate of $(1.29) by 7.75 percent. This is a 8.55 percent increase over losses of $(1.52) per share from the same period last year. The company reported quarterly sales of $164.877 which missed the analyst consensus estimate of $155.393 million by 100.00 percent. This is a 100.00 percent decrease over sales of $127.392 million the same period last year.

Earnings

Earnings Misses

Ultragenyx Pharmaceutical Q4 2024 GAAP EPS $(1.39) Misses $(1.29) Estimate, Sales $164.877 Miss $155.393M Estimate

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Hannon Armstrong Sustainable Infrastructure Capital

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Earnings Scheduled For February 13, 2025

Ultragenyx Announces New Data Demonstrating Treatment With UX111 AAV Gene Therapy Improved Clinical Function Across Multiple Developmental Domains In Children With Sanfilippo Syndrome Type A

Analysts' ratings for Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) over the last quarter vary from bullish to bearish, as provided by 11 analysts.
The following table encapsulates their recent ratings, offering a glimpse into the evolving sentiments over the past 30 days and comparing them to the preceding 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">4</td>
<td align="right">6</td>
<td align="right">1</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>Last 30D</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>
<tr>
<td>1M Ago</td>
<td align="right">1</td>
<td align="right">1</td>
<td align="right">1</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">1</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">3</td>
<td align="right">3</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 $101.91, a high estimate of $140.00, and a low estimate of $48.00. This upward trend is apparent, with the current average reflecting a 3.89% increase from the previous average price target of $98.09.
<img src="https://www.benzinga.com/files/images/story/2025/1737572415_0.png" alt="price target chart" />
<h2>Investigating Analyst Ratings: An Elaborate Study</h2>
An in-depth analysis of recent analyst actions unveils how financial experts perceive Ultragenyx Pharmaceutical. The following summary outlines 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>Kristen Kluska</td>
<td>Cantor Fitzgerald</td>
<td>Maintains</td>
<td>Overweight</td>
<td>$118.00</td>
<td>$118.00</td>
</tr>
<tr>
<td>Christopher Raymond</td>
<td>Piper Sandler</td>
<td>Raises</td>
<td>Overweight</td>
<td>$140.00</td>
<td>$135.00</td>
</tr>
<tr>
<td>Laura Chico</td>
<td>Wedbush</td>
<td>Maintains</td>
<td>Neutral</td>
<td>$48.00</td>
<td>$46.00</td>
</tr>
<tr>
<td>Ed Arce</td>
<td>HC Wainwright &amp; Co.</td>
<td>Maintains</td>
<td>Buy</td>
<td>$95.00</td>
<td>$95.00</td>
</tr>
<tr>
<td>Tiago Fauth</td>
<td>Wells Fargo</td>
<td>Raises</td>
<td>Overweight</td>
<td>$88.00</td>
<td>$75.00</td>
</tr>
<tr>
<td>Anupam Rama</td>
<td>JP Morgan</td>
<td>Lowers</td>
<td>Overweight</td>
<td>$102.00</td>
<td>$105.00</td>
</tr>
<tr>
<td>Whitney Ijem</td>
<td>Canaccord Genuity</td>
<td>Maintains</td>
<td>Buy</td>
<td>$121.00</td>
<td>$121.00</td>
</tr>
<tr>
<td>Whitney Ijem</td>
<td>Canaccord Genuity</td>
<td>Raises</td>
<td>Buy</td>
<td>$121.00</td>
<td>$109.00</td>
</tr>
<tr>
<td>Luca Issi</td>
<td>RBC Capital</td>
<td>Maintains</td>
<td>Outperform</td>
<td>$77.00</td>
<td>$77.00</td>
</tr>
<tr>
<td>Kristen Kluska</td>
<td>Cantor Fitzgerald</td>
<td>Maintains</td>
<td>Overweight</td>
<td>$116.00</td>
<td>$116.00</td>
</tr>
<tr>
<td>Ed Arce</td>
<td>HC Wainwright &amp; Co.</td>
<td>Raises</td>
<td>Buy</td>
<td>$95.00</td>
<td>$82.00</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 Ultragenyx Pharmaceutical. This information offers a snapshot of how analysts perceive the current state of the company.</li>
<li>Rating: Offering insights into predictions, analysts assign qualitative values, from 'Outperform' to 'Underperform'. These ratings convey expectations for the relative performance of Ultragenyx Pharmaceutical compared to the broader market.</li>
<li>Price Targets: Analysts provide insights into price targets, offering estimates for the future value of Ultragenyx Pharmaceutical's stock. This comparison reveals trends in analysts' expectations over time.</li>
</ul>
Capture valuable insights into Ultragenyx 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/RARE/analyst-ratings?utm_source=Benzinga Insights">Stay up to date on Ultragenyx Pharmaceutical analyst ratings.</a>
<h2>Discovering Ultragenyx Pharmaceutical: A Closer Look</h2>
Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi, and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.
<h3>Understanding the Numbers: Ultragenyx Pharmaceutical's Finances</h3>
Market Capitalization Analysis: Positioned below industry benchmarks, the company's market capitalization faces constraints in size. This could be influenced by factors such as growth expectations or operational capacity.
Revenue Growth: Ultragenyx Pharmaceutical displayed positive results in 3 months. As of 30 September, 2024, the company achieved a solid revenue growth rate of approximately 42.27%. This indicates a notable increase in the company's top-line earnings. When compared to others in the Health Care sector, the company faces challenges, achieving a growth rate lower than the average among peers.
Net Margin: Ultragenyx Pharmaceutical's financial strength is reflected in its exceptional net margin, which exceeds industry averages. With a remarkable net margin of -95.71%, the company showcases strong profitability and effective cost management.
Return on Equity (ROE): Ultragenyx Pharmaceutical's ROE falls below industry averages, indicating challenges in efficiently using equity capital. With an ROE of -34.27%, the company may face hurdles in generating optimal returns for shareholders.
Return on Assets (ROA): The company's ROA is below industry benchmarks, signaling potential difficulties in efficiently utilizing assets. With an ROA of -8.46%, the company may need to address challenges in generating satisfactory returns from its assets.
Debt Management: With a below-average debt-to-equity ratio of 0.1, Ultragenyx Pharmaceutical adopts a prudent financial strategy, indicating a balanced approach to debt management.
<h2>Analyst Ratings: What Are They?</h2>
Analyst ratings serve as essential indicators of stock performance, provided by experts in banking and financial systems. These specialists diligently analyze company financial statements, participate in conference calls, and engage with insiders to generate quarterly ratings for individual stocks.
Some analysts also offer predictions for helpful metrics such as earnings, revenue, and growth estimates to provide further guidance as to what to do with certain tickers. It is important to keep in mind that while stock and sector analysts are specialists, they are also human and can only forecast their beliefs to traders.
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This article was generated by Benzinga's automated content engine and reviewed by an editor.

Breaking Down Ultragenyx Pharmaceutical: 11 Analysts Share Their Views

In conjunction with the 2025 J.P. Morgan Healthcare Conference, the analyst hosted an investor lunch with Ultragenyx Pharmaceutical Inc (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>).
The analyst <a href="https://www.benzinga.com/quote/RARE/analyst-ratings" target="_blank" rel="noreferrer noopener">keeps an Overweight rating</a> on Ultragenyx with a price target of $102 and says the company is uniquely positioned in the long term with an expansive and diverse pipeline of orphan disease assets and highly regarded management. JP Morgan sees multiple value-creating catalysts over the next six to 18 months.
<ul>
<li>Ultragenyx&#8217;s recent meeting focused on setrusumab and the ongoing Phase 3 ORBIT trial. The analyst noted that the company remains confident in the trial&#8217;s success, with a second interim analysis expected by mid-2025 and final results by the end of 2025.</li>
<li>At current share levels, the analyst noted the market is not fully valuing the setrusumab program and anticipates positive trial data could push Ultragenyx stock price to a ~$7-handle.</li>
<li>Ultragenyx emphasized that moving forward to the second interim analysis does not change the study&#8217;s likelihood of success. Instead, the additional time allows for clearer differentiation between the setrusumab and placebo groups, with the minimum follow-up increasing from 7 months in the first interim analysis to 12 months in the second.</li>
<li>On the commercial front, Ultragenyx plans to leverage its existing Crysvita infrastructure in the U.S. for setrusumab, given overlapping call points.</li>
<li>Approval could also make the company eligible for a Priority Review Voucher, providing a potential source of non-dilutive capital. In Europe, where Mereo owns the rights, Ultragenyx will lead the regulatory filing process and anticipates submitting around 2-3 months after the U.S. filing.</li>
</ul>
On Sunday, Ultragenyx reported preliminary 2024 revenue of $555 million—$560 million, compared to the consensus of $543.02 million.
Crysvita&#8217;s revenue for 2024 is estimated to be $405 million—$410 million, and Dojolvi&#8217;s revenue is estimated to be $87 million—$89 million.
The company expects 2025 sales of $640 million—$670 million versus a <a target="_blank" href="https://www.benzinga.com/pressreleases/25/01/g42935224/ultragenyx-reports-preliminary-2024-revenue-financial-guidance-for-2025-pipeline-updates-and-2025-" rel="noreferrer noopener">consensus of $648.54 million</a>.
Price Action: RARE stock is up 4.31% at $41.75 at last check Wednesday.
Read Next:
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<li><a href="https://www.benzinga.com/media/25/01/43003727/microsoft-to-cut-costs-pause-hiring-in-us-consulting-tighten-spending" target="_blank" rel="noreferrer noopener">Microsoft To Cut Costs, Pause Hiring In US Consulting, Tighten Spending</a></li>
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Photo via Shutterstock.

Analyst Color

Equities

Health Care

Trading Ideas

Ultragenyx's Multiple Commercial Assets And Candidates Impress Analyst; Sees Solid Upside

Piper Sandler analyst Christopher Raymond maintains Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) with a Overweight and raises the price target from $135 to $140.

Piper Sandler Maintains Overweight on Ultragenyx Pharmaceutical, Raises Price Target to $140

Wedbush analyst Laura Chico reiterates Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) with a Neutral and maintains $48 price target.

Wedbush Reiterates Neutral on Ultragenyx Pharmaceutical, Maintains $48 Price Target

Ultragenyx Reported Sunday Preliminary 2024 Total Revenue Of $555M To $560M, Exceeding Top End Of Guidance; 2025 Expected Total Revenue Guidance Of $640M To $670M

William Blair has initiated coverage on Denali Therapeutics Inc (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/DNLI#NASDAQ">DNLI</a>), a company focused on developing drugs for <a target="_blank" href="https://www.benzinga.com/quote/DNLI" rel="noreferrer noopener">neurodegenerative diseases</a>.
The analyst highlights that Denali is developing a specialized transport vehicle (TV) platform to enhance the delivery of treatments across the blood-brain barrier.
It stands out from competitors with its promising potential for top-tier performance and scalability across different therapies and conditions.
The company&#8217;s lead program Tividenofusp alfa, an enzyme-replacement therapy for Hunter syndrome, shows promise as a best-in-class treatment, the analyst highlights.
William Blair has initiated with an Outperform rating and a <a target="_blank" href="https://www.benzinga.com/quote/DNLI/analyst-ratings" rel="noreferrer noopener">fair value of $35 per share</a>.
The company has reached an agreement with the FDA on the requirements for accelerated approval and plans to submit a biologics license application <a target="_blank" href="https://www.benzinga.com/pressreleases/24/09/g40684612/denali-therapeutics-announces-successful-meeting-with-the-fda-and-plans-to-file-for-accelerated-ap" rel="noreferrer noopener">in early 2025</a>.
Insights gained from tividenofusp alfa may also simplify the development and approval process for future treatments targeting other lysosomal storage diseases, such as DNL126 for Sanfilippo syndrome type A.
Most recently, Denali Therapeutics initiated dosing in a global BEACON Phase 2a trial of BIIB122 (DNL151) for LRRK2-associated <a target="_blank" href="https://www.benzinga.com/pressreleases/24/12/g42336987/denali-therapeutics-announces-first-participant-dosed-in-phase-2a-study-of-lrrk2-inhibitor-biib122" rel="noreferrer noopener">Parkinson&#8217;s disease</a>.
Denali&#8217;s market cap stands at $2.93 billion, with an enterprise value of $2.26 billion.
Compared to its peers in neuroscience, rare diseases, and platform biotech, its valuation is lower than some but higher than others, reflecting the strength of its pipeline and technology.
William Blair notes the potential for significant growth as Denali advances its assets, including tividenofusp, which might receive accelerated approval and priority review by late 2025.
If additional clinical data reduces risks associated with its TV-enabled approach, Denali could achieve valuations similar to companies like Avidity Biosciences Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RNA#NASDAQ">RNA</a>), Ultragenyx Pharmaceutical Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>), Biogen Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BIIB#NASDAQ">BIIB</a>), Alnylam Pharmaceuticals, Inc.(NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ALNY#NASDAQ">ALNY</a>), and Ionis Pharmaceuticals, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/IONS#NASDAQ">IONS</a>).
Price Action: DNLI stock is up 3.17% at $21.50 at the last check on Friday.
Read Next:
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<li><a href="https://www.benzinga.com/news/large-cap/25/01/42788070/hedge-fund-giant-dan-loebs-pge-bet-beats-market" target="_blank" rel="noreferrer noopener">Hedge Fund Giant Dan Loeb&#8217;s PG&amp;E Bet Beats Market</a></li>
</ul>

Initiation

BIIB

DNLI

IONS

Denali Therapeutics Neurodegenerative Disease Platform Sparks Analyst Optimistic

In the last three months, 11 analysts have published ratings on Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</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">4</td>
<td align="right">7</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
<tr>
<td>Last 30D</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>
<tr>
<td>1M 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>
<tr>
<td>2M Ago</td>
<td align="right">4</td>
<td align="right">3</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">2</td>
<td align="right">0</td>
<td align="right">0</td>
<td align="right">0</td>
</tr>
</tbody>
</table>
The 12-month price targets assessed by analysts reveal further insights, featuring an average target of $100.18, a high estimate of $121.00, and a low estimate of $73.00. This current average has increased by 4.45% from the previous average price target of $95.91. 
<img src="https://www.benzinga.com/files/images/story/2024/1734703209_0.png" alt="price target chart" />
<h2>Diving into Analyst Ratings: An In-Depth Exploration</h2>
A clear picture of Ultragenyx Pharmaceutical's perception among financial experts is painted with a thorough analysis of recent analyst actions. The summary below outlines 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>Tiago Fauth</td>
<td>Wells Fargo</td>
<td>Raises</td>
<td>Overweight</td>
<td>$88.00</td>
<td>$75.00</td>
</tr>
<tr>
<td>Anupam Rama</td>
<td>JP Morgan</td>
<td>Lowers</td>
<td>Overweight</td>
<td>$102.00</td>
<td>$105.00</td>
</tr>
<tr>
<td>Whitney Ijem</td>
<td>Canaccord Genuity</td>
<td>Maintains</td>
<td>Buy</td>
<td>$121.00</td>
<td>$121.00</td>
</tr>
<tr>
<td>Whitney Ijem</td>
<td>Canaccord Genuity</td>
<td>Raises</td>
<td>Buy</td>
<td>$121.00</td>
<td>$109.00</td>
</tr>
<tr>
<td>Luca Issi</td>
<td>RBC Capital</td>
<td>Maintains</td>
<td>Outperform</td>
<td>$77.00</td>
<td>$77.00</td>
</tr>
<tr>
<td>Kristen Kluska</td>
<td>Cantor Fitzgerald</td>
<td>Maintains</td>
<td>Overweight</td>
<td>$116.00</td>
<td>$116.00</td>
</tr>
<tr>
<td>Ed Arce</td>
<td>HC Wainwright &amp; Co.</td>
<td>Raises</td>
<td>Buy</td>
<td>$95.00</td>
<td>$82.00</td>
</tr>
<tr>
<td>Kristen Kluska</td>
<td>Cantor Fitzgerald</td>
<td>Maintains</td>
<td>Overweight</td>
<td>$116.00</td>
<td>$116.00</td>
</tr>
<tr>
<td>Yaron Werber</td>
<td>TD Cowen</td>
<td>Raises</td>
<td>Buy</td>
<td>$73.00</td>
<td>$61.00</td>
</tr>
<tr>
<td>Kristen Kluska</td>
<td>Cantor Fitzgerald</td>
<td>Maintains</td>
<td>Overweight</td>
<td>$116.00</td>
<td>$116.00</td>
</tr>
<tr>
<td>Luca Issi</td>
<td>RBC Capital</td>
<td>Maintains</td>
<td>Outperform</td>
<td>$77.00</td>
<td>$77.00</td>
</tr>
</tbody>
</table>
<h3>Key Insights:</h3>
<ul>
<li>Action Taken: Analysts adapt their recommendations to changing market conditions and company performance. Whether they 'Maintain', 'Raise' or 'Lower' their stance, it reflects their response to recent developments related to Ultragenyx Pharmaceutical. This information provides 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 Ultragenyx Pharmaceutical compared to the broader market.</li>
<li>Price Targets: Analysts gauge the dynamics of price targets, providing estimates for the future value of Ultragenyx Pharmaceutical's stock. This comparison reveals trends in analysts' expectations over time.</li>
</ul>
To gain a panoramic view of Ultragenyx Pharmaceutical's market performance, explore these analyst evaluations alongside essential financial indicators. Stay informed and make judicious decisions using our Ratings Table.
<a href="https://www.benzinga.com/quote/RARE/analyst-ratings?utm_source=Benzinga Insights">Stay up to date on Ultragenyx Pharmaceutical analyst ratings.</a>
<h2>About Ultragenyx Pharmaceutical</h2>
Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi, and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.
<h3>A Deep Dive into Ultragenyx Pharmaceutical's Financials</h3>
Market Capitalization Analysis: The company's market capitalization is above the industry average, indicating that it is relatively larger in size compared to peers. This may suggest a higher level of investor confidence and market recognition.
Revenue Growth: Ultragenyx Pharmaceutical's revenue growth over a period of 3 months has been noteworthy. As of 30 September, 2024, the company achieved a revenue growth rate of approximately 42.27%. This indicates a substantial increase in the company's top-line earnings. In comparison to its industry peers, the company trails behind with a growth rate lower than the average among peers in the Health Care sector.
Net Margin: The company's net margin is a standout performer, exceeding industry averages. With an impressive net margin of -95.71%, the company showcases strong profitability and effective cost control.
Return on Equity (ROE): Ultragenyx Pharmaceutical's ROE lags behind industry averages, suggesting challenges in maximizing returns on equity capital. With an ROE of -34.27%, the company may face hurdles in achieving optimal financial performance.
Return on Assets (ROA): Ultragenyx Pharmaceutical's ROA is below industry averages, indicating potential challenges in efficiently utilizing assets. With an ROA of -8.46%, the company may face hurdles in achieving optimal financial returns.
Debt Management: The company maintains a balanced debt approach with a debt-to-equity ratio below industry norms, standing at 0.1.
<h2>Analyst Ratings: What Are They?</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.
In addition to their assessments, some analysts extend their insights by offering predictions for key metrics such as earnings, revenue, and growth estimates. This supplementary information provides further guidance for traders. It is crucial to recognize that, despite their specialization, analysts are human and can only provide forecasts based on their beliefs.
Which Stocks Are Analysts Recommending Now?
Benzinga Edge gives you instant access to all major analyst upgrades, downgrades, and price targets. Sort by accuracy, upside potential, and more. <a href="https://www.benzinga.com/premium/ideas/benzinga-edge-checkout/?t=be8be9arja3be22bz21&amp;utm_source=benzinga-insights">Click here to stay ahead of the market</a>.
This article was generated by Benzinga's automated content engine and reviewed by an editor.

Expert Outlook: Ultragenyx Pharmaceutical Through The Eyes Of 11 Analysts

Wells Fargo analyst Tiago Fauth maintains Ultragenyx Pharmaceutical (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RARE#NASDAQ">RARE</a>) with a Overweight and raises the price target from $75 to $88.

Wells Fargo Maintains Overweight on Ultragenyx Pharmaceutical, Raises Price Target to $88

Press Releases

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

Ultragenyx to Present at the 43rd Annual J.P. Morgan Healthcare Conference

Ultragenyx Reports Preliminary 2024 Revenue, Financial Guidance for 2025, Pipeline Updates, and 2025 Milestones

Ultragenyx Announces New Data Demonstrating that Treatment with UX111 AAV Gene Therapy Significantly Improved Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome Type A (MPS IIIA) Correlated with Sustained Reductions in CSF-HS

Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2024 Financial Results and Corporate Update

Ultragenyx Reports Fourth Quarter and Full Year 2024 Financial Results and Corporate Update

Ultragenyx Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)

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