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Mirum Pharmaceuticals to Showcase LIVMARLI Data from its ALGS and PFIC Programs at the NASPGHAN Annual Meeting

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FOSTER CITY, Calif.–(BUSINESS WIRE)–Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced its participation in the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition (NASPGHAN) annual meeting taking place November 6-9, 2024, in Hollywood, Florida. Data highlighting clinical benefit and real-world evidence for patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC), treated with LIVMARLI ® (maralixibat) oral solution, will be presented during the meeting.

Highlighted below are the titles that have been accepted for presentation during the meeting. The abstracts are available via the NASPGHAN Program Book. Full analyses will be available following their presentation within the Publications & Presentations section on Mirum’s website.

Abstract 77: Maralixibat improves growth in patients with progressive familial intrahepatic cholestasis: Data from the MARCH/MARCH-ON trials
Thursday, November 7 between 5:00-7:00 p.m. ET during Poster Session I
Presented by Dr. Amal Aqul

Abstract 361: Clinical benefits of maralixibat for patients with Alagille syndrome are durable through 7 years of treatment: Data from the MERGE study
Friday, November 8 between 12:00-2:00 p.m. ET during Poster Session II
Presented by Dr. Karen Murray

Abstract 637: Improvements in serum bile acids are associated with improvements in markers of liver health after maralixibat treatment in children with PFIC: Data from the MARCH/MARCH-ON trials
Saturday, November 9 between 12:00-2:00 p.m. ET during Poster Session III
Presented by Dr. Alexander Miethke

Abstract 638: Improvements in pruritus after maralixibat treatment are associated with improved health-related quality of life for patients with cholestatic liver disease
Saturday, November 9 between 12:00-2:00 p.m. ET during Poster Session III
Presented by Dr. Alexander Miethke

Abstract 664: Long-term maralixibat impact on concomitant medication use for the treatment of cholestatic pruritus in Alagille syndrome: Real-world experience in the United States
Saturday, November 9 between 12:00-2:00 p.m. ET during Poster Session III
Presented by Dr. Jolan Terner-Rosenthal

About LIVMARLI ® (maralixibat) oral solution

LIVMARLI ® (maralixibat) oral solution is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com.

LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website.

IMPORTANT SAFETY INFORMATION

Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein.

LIVMARLI can cause side effects, including:

Liver injury. Changes in certain liver tests are common in patients with Alagille syndrome and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal.

Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you.

A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects.

US Prescribing Information
EU SmPC
Canadian Product Monograph

About Mirum Pharmaceuticals (NASDAQ:), Inc.

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI ® (maralixibat) oral solution, CHOLBAM ® (cholic acid) capsules, and CHENODAL ® (chenodiol) tablets.

LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is also initiating the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms or liver disease. CHENODAL has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX).

Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Lastly, chenodiol, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023. Mirum has submitted a new drug application with the FDA for the approval of chenodiol to treat CTX in the U.S.

To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook (NASDAQ:), LinkedIn, Instagram and Twitter (X).

Forward-Looking Statements

This press release includes forward-looking statements pertaining to the Company’s planned participation at a scientific conference, including data presentation title and synopsis, which may include discussion of the Company’s clinical and research data relating to the therapeutic potential and/or commercial viability of LIVMARLI in various liver disease indications and in patient populations that are investigational only. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as will, could, would, potential and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general and the other risks described in Mirum’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. A further description of risks and uncertainties can be found in Mirum’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov.

Media Contact:
Erin Murphy
media@mirumpharma.com

Investor Contact:
Andrew McKibben
ir@mirumphama.com

Source: Mirum Pharmaceuticals, Inc.

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Truist cuts Editas Medicine target to $8, keeps buy rating

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On Monday, Truist Securities revised its price target for Editas Medicine (NASDAQ:), a company specializing in gene editing technology. The firm reduced the target to $8.00 from the previous $12.00 while retaining a Buy rating on the stock.

The adjustment follows the company’s third-quarter report, which did not present any unexpected results following recent announcements. The analyst indicated that Editas Medicine is expected to provide updated data on its reni-cel therapy at the upcoming American Society of Hematology (ASH) meeting, as well as updates on its in vivo program in the first quarter of 2025.

The reassessment of the reni-cel program prompted the analyst to moderate the outlook, leading to the lowered price target. Despite this change, the firm continues to support a Buy rating for Editas Medicine shares, suggesting confidence in the company’s long-term potential.

Editas Medicine is actively engaged in the development of gene-editing therapies, with reni-cel being one of its key investigational treatments. The forthcoming data presentations are anticipated to shed more light on the progress and efficacy of these treatments.

The updated price target of $8.00 reflects a more conservative valuation of Editas Medicine by Truist Securities, while the maintained Buy rating indicates a positive view of the stock’s future performance despite the recent adjustments.

In other recent news, Editas Medicine has been the focus of several analyst adjustments. Wells Fargo reduced its price target for Editas from $27.00 to $9.00, while maintaining an Overweight rating. This adjustment followed Editas Medicine’s disclosure of preclinical data for its in vivo hematopoietic stem and progenitor cell (HSPC) editing program.

The company also announced its intention to partner or out-license its reni-cel therapy. Baird also lowered its target for Editas Medicine to $10 from $18, keeping an Outperform rating.

The company has made significant strides in gene editing treatments for sickle cell disease and beta-thalassemia. It reported high levels of editing in hematopoietic stem and progenitor cells. Additionally, Editas secured an upfront payment of $57 million from a financing agreement with DRI Healthcare Trust. Leerink Partners and Truist Securities maintained their Market Perform and Buy ratings respectively on Editas’ stock.

InvestingPro Insights

Recent financial data from InvestingPro provides additional context to Truist Securities’ revised outlook on Editas Medicine (NASDAQ:EDIT). The company’s market capitalization stands at $245.89 million, reflecting its current valuation in the biotech sector. Editas’ stock has experienced significant volatility, with a 41.8% price decline over the past three months and a 48.68% drop in the last six months, aligning with the analyst’s more cautious stance.

InvestingPro Tips highlight that Editas is quickly burning through cash and is not expected to be profitable this year, factors that likely influenced Truist’s decision to lower the price target. The company’s gross profit margin is weak, with a negative 165.65% for the last twelve months as of Q2 2024, underscoring the challenges in its developmental stage.

Despite these headwinds, Editas maintains a strong liquidity position. An InvestingPro Tip notes that the company’s liquid assets exceed short-term obligations, providing some financial flexibility as it advances its gene-editing therapies. This could be crucial as Editas prepares to present updated data on reni-cel and its in vivo program.

For investors seeking a more comprehensive analysis, InvestingPro offers 13 additional tips for Editas Medicine, providing deeper insights into the company’s financial health and market position.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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Cemex stock hits 52-week low at $5.17 amid market challenges

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Cemex SAB de CV (NYSE:), a leading materials company in the construction industry, has seen its stock price touch a 52-week low, dipping to $5.17. This price level reflects a significant downturn from its previous positions, marking a challenging period for the company. Over the past year, Cemex’s stock has experienced a notable decline, with a 1-year change showing a decrease of 22.42%. This downturn is indicative of the broader pressures facing the construction sector, including fluctuating demand and cost pressures, which have impacted the company’s market valuation and investor confidence.

In other recent news, CEMEX has reported a year of substantial growth and strategic optimization despite facing natural disasters and undergoing significant divestitures. The company announced divestitures amounting to $2.2 billion, concentrating on operations in the Dominican Republic, Guatemala, and the Philippines. In spite of severe weather conditions, including three major hurricanes in the U.S., CEMEX achieved a net income increase of over 200% year-over-year.

CEMEX’s growth strategy, initiated in 2019, has resulted in a 14% compound annual growth rate (CAGR) since 2020. The company also reported a 3% reduction in Scope 1 emissions and received a €157 million grant from the EU for a carbon capture project in Germany.

Analysts from Thompson Davis and Goldman Sachs questioned the company’s valuation strategies and the impact of Mexican residential demand on CEMEX, respectively. In response, CEMEX executives emphasized careful evaluation of operational changes and positive expectations for significant impact starting in 2025.

These are among the recent developments that underline CEMEX’s resilience and strategic focus, allowing it to navigate a challenging environment while maintaining a positive outlook for growth.

InvestingPro Insights

Cemex’s recent stock performance aligns with several key insights from InvestingPro. The company is currently trading near its 52-week low, as reflected in the article, with InvestingPro data showing the stock price at $5.18 at the previous close. This represents just 56.02% of its 52-week high, underscoring the significant downturn mentioned.

Despite the challenging market conditions, InvestingPro Tips highlight that Cemex remains a prominent player in the Construction Materials industry. The company’s Price to Book ratio of 0.63 suggests it may be undervalued relative to its assets, potentially offering value for investors looking beyond current market sentiment.

Importantly, an InvestingPro Tip indicates that net income is expected to grow this year, which could provide a positive catalyst for the stock. This growth expectation, combined with the company’s low valuation multiples, may present an opportunity for investors willing to weather the current downturn.

For those seeking a more comprehensive analysis, InvestingPro offers 7 additional tips that could provide further insights into Cemex’s financial health and future prospects.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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Jefferies cuts Booz Allen stock rating to Hold, sees slowdown in the long-term

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On Monday, Jefferies adjusted its stance on shares of Booz Allen Hamilton (NYSE:), downgrading the stock from Buy to Hold, though the firm increased the price target to $190 from $180. The revision comes despite acknowledging the company’s strong management and share price performance.

The analyst from Jefferies noted that while Booz Allen Hamilton has shown stellar performance, a pause on the stock is suggested as earnings per share (EPS) revisions through the fiscal year 2025 (ending in March 2025) may be limited.

The limited potential for EPS revisions is attributed to margins being range-bound, with the Defense sector—accounting for 48% of sales and approximately 10% margins—outperforming the Civil sector, which makes up 33% of sales and has a 13% margin. The analyst further pointed out that there is an anticipated slowdown in organic growth excluding items, from 11% in the fiscal year 2025 to 8% in fiscal years 2026 to 2027 estimates.

The new price target of $190 is based on a 30% market premium or twice the three-year average, reflecting the analyst’s valuation of the stock. This price target suggests a modest upside from the previous target, indicating a positive outlook on the company’s value despite the rating downgrade.

The downgrade to Hold reflects a cautious approach towards Booz Allen Hamilton’s stock, considering the expected limitations in earnings growth and margin expansion in the upcoming years. The firm’s analysis suggests that while the company has been performing well, future gains might not be as robust as in the previous periods.

In other recent news, Booz Allen Hamilton reported a robust second quarter for fiscal year 2025, with major revenue hikes in its civil, defense, and intelligence sectors.

The company’s VOLT growth strategy, a record $41 billion backlog, a $115 million insurance recovery, and a $200 million boost from payroll modernization were significant contributors to this performance. Adjusted EBITDA reached $364 million, a 25% year-over-year increase, and net income surged by 129% to $390 million.

Despite the loss of the Advana contract and a Department of Veterans Affairs contract to Deloitte, Booz Allen maintains a strong demand environment with a qualified pipeline of over $20 billion.

The firm’s operating model allows for quick adaptation to client needs amid shifting priorities, and recruitment and retention trends remain strong, making Booz Allen an attractive destination for tech talent. These recent developments emphasize Booz Allen’s strong market presence and potential for continued growth.

InvestingPro Insights

While Jefferies has downgraded Booz Allen Hamilton (NYSE:BAH) to Hold, recent data from InvestingPro paints a nuanced picture of the company’s financial health and market performance. BAH’s revenue growth of 13.94% over the last twelve months and a strong 18.01% quarterly growth align with the company’s solid performance noted in the article.

The stock’s P/E ratio of 28.58 and an adjusted P/E ratio of 31.27 for the last twelve months as of Q2 2025 suggest that investors are willing to pay a premium for BAH’s earnings, which could be justified by its consistent growth. This valuation is further supported by the company’s robust EBITDA growth of 30.89% over the same period.

InvestingPro Tips highlight that BAH has raised its dividend for 9 consecutive years and maintained payments for 13 years, indicating a commitment to shareholder returns. This is particularly relevant given the article’s focus on the company’s financial outlook. Additionally, the tip that BAH operates with a moderate level of debt provides context to the company’s financial stability, which could be a factor in its ability to navigate potential growth slowdowns mentioned in the analyst’s report.

For investors seeking a more comprehensive analysis, InvestingPro offers 11 additional tips that could provide further insights into BAH’s market position and future prospects.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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