Stock Markets

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

Published

3 weeks ago

November 1, 2024

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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20241031636363/en/

Media Contact:
Erin Murphy
media@mirumpharma.com

Investor Contact:
Andrew McKibben
ir@mirumphama.com

Source: Mirum Pharmaceuticals, Inc.

Stock Markets

Needham initiates coverage on On Holding with buy rating

Published

16 mins ago

November 23, 2024

Investing.com — Needham on Friday initiated its coverage on On Holding AG (NYSE:) with a “buy” rating and a target price of $64.

Brokerage said On has shown industry-leading growth, with impressive revenue increases and healthy margin expansion. The company is likely to keep growing as it increases brand awareness and gains space with top sneaker retailers worldwide.

“We believe the company has a continued runway for strong growth, as they increase brand awareness and gain shelf space with the biggest and best sneaker retailers in the world,” analyst Tom Nikic wrote in the note.

Needham analyst noted that Roger Federer-backed On was valued at 5 times its expected 2025 revenues, which make stock may seem expensive but strong fundamentals could support continued stock momentum.

“Although valuation metrics are lofty, we believe the shares can continue to exhibit momentum as long as fundamentals”

ON is the fastest growing company in Needham’s coverage, with expected 32% revenue growth in 2024. Its Direct-to-Consumer (DTC) growing 43% year-to-date, compared to 24% growth for wholesale sales.

Brokerage highlighted despite this growth, the brand’s awareness is still relatively low. In major markets like the U.S., U.K., France, and Australia, awareness was under 10% a year ago. However, it’s increasing rapidly, with U.S. awareness doubling to around 20%, and tripling in France.

Stock Markets

Toll Brothers Announces Final Opportunity at Verona Estates Community in Chatsworth, California

Published

16 mins ago

November 23, 2024

CHATSWORTH, Calif., Nov. 22, 2024 (GLOBE NEWSWIRE) — Toll Brothers , Inc. (NYSE:), the nation’s leading builder of luxury homes, today announced the final opportunity to own a new home at Verona Estates, an exclusive gated community in Chatsworth, California. Only a few homes remain available for sale in this prestigious community, including the professionally decorated Siena Modern Farmhouse model home.

The intimate gated enclave of Verona Estates is a rare find showcasing award-winning architecture and innovative home designs. Nestled in an established Chatsworth neighborhood south of the Santa Susana Mountains and adjacent to the Vineyards at Porter Ranch, this exceptional community offers a serene and relaxed atmosphere with the convenience of nearby shopping and easy access to freeways, entertainment, and recreation.

Toll Brothers residents in Verona Estates will enjoy distinctive architecture, quality craftsmanship, luxurious home designs with open floor plans, expansive home sites, and proximity to the future 50-acre Porter Ranch community park. Verona Estates offers generous two-story home designs ranging from 4,700 to 6,000+ square feet, with 5 to 6 bedrooms, 4.5 to 6.5 bathrooms, and 3-car garages. The homes also feature popular floor plan options including prep kitchens, guest suites, floating staircases, indoor and outdoor fireplaces, and more. Move-in ready homes in the community are priced from $1,979,995.

We are thrilled to offer the final opportunity to own a home in the exclusive Verona Estates community, said Nick Norvilas, Division President of Toll Brothers in Los Angeles. The Siena model home is a showcase of luxury and design, and we encourage interested home buyers to visit and experience this exceptional home along with the final few quick move-in homes remaining in the community firsthand.

The Siena Modern Farmhouse model home features designer upgrades throughout, including fully landscaped and furnished interiors, offering an unparalleled living experience. The professionally decorated model home is priced at $2,999,995.

For more information, call 844-700-8655 or visit TollBrothers.com/LA. The Sales Center for Verona Estates is located at 20508 Edgewood Court in Chatsworth and is open by appointment only.

About Toll Brothers

Toll Brothers, Inc., a Fortune 500 Company, is the nation’s leading builder of luxury homes. The Company was founded 57 years ago in 1967 and became a public company in 1986. Its common stock is listed on the New York Stock Exchange under the symbol TOL. The Company serves first-time, move-up, empty-nester, active-adult, and second-home buyers, as well as urban and suburban renters. Toll Brothers builds in over 60 markets in 24 states: Arizona, California, Colorado, Connecticut, Delaware, Florida, Georgia, Idaho, Indiana, Maryland, Massachusetts, Michigan, Nevada, New Jersey, New York, North Carolina, Oregon, Pennsylvania, South Carolina, Tennessee, Texas, Utah, Virginia, and Washington, as well as in the District of Columbia. The Company operates its own architectural, engineering, mortgage, title, land development, smart home technology, and landscape subsidiaries. The Company also develops master-planned and golf course communities as well as operates its own lumber distribution, house component assembly, and manufacturing operations.

In 2024, Toll Brothers marked 10 years in a row being named to the Fortune World’s Most Admired Companies™ list and the Company’s Chairman and CEO Douglas C. Yearley, Jr. was named one of 25 Top CEOs by Barron’s magazine. Toll Brothers has also been named Builder of the Year by Builder magazine and is the first two-time recipient of Builder of the Year from Professional Builder magazine. For more information visit TollBrothers.com.

Contact: Andrea Meck | Toll Brothers, Director, Public Relations & Social Media | 215-938-8169 | ameck@tollbrothers.com

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/cbb8cf4a-a018-4df0-955e-3cf4ab63edeb

Sent by Toll Brothers via Regional Globe Newswire (TOLL-REG)

Verona Estates by Toll Brothers

Toll Brothers announced the final opportunity to own a new home at Verona Estates, including the designer-decorated Siena model home, in Chatsworth, California.

Source: Toll Brothers, Inc.

Stock Markets

Northvolt crisis may be make or break for Europe’s EV battery ambitions

Published

16 mins ago

November 23, 2024

By Marie Mannes, Alessandro Parodi and Stine Jacobsen

STOCKHOLM/GDANSK (Reuters) – Northvolt’s financial collapse deals a blow to Europe’s plan to set up its own battery industry to power electric cars, stirring a debate about whether it needs to do more to attract investment as startups struggle to catch up with Chinese rivals.

Europe’s biggest hope for an electric vehicle battery champion filed for U.S. Chapter 11 bankruptcy protection on Thursday after talks with investors and creditors including Volkswagen (ETR:) and Goldman Sachs for funding failed.

The Swedish company, whose motto is “make oil history”, has received more than $10 billion in equity, debt and public financing since its 2016 start-up. Volkswagen and Goldman Sachs each own about one fifth of its shares.

Northvolt said on Friday it needed $1.0-$1.2 billion in new funds under the restructuring process, which it hopes will end by the end of March.

In recent months, it has shrunk the business and cut jobs in a bid to shore up its finances. But it has struggled to produce sufficient volumes of high-quality batteries, and lost a 2 billion euro ($2.1 billion) contract from BMW (ETR:) in June.

That has left Europe’s ambitions to build its own battery industry looking a distant dream.

In recent years, Northvolt led a wave of European startups investing tens of billions of dollars to serve the continent’s automakers as they switch from internal combustion engines to electric vehicles.

But growth in EV demand is moving at a slower pace than many in the industry projected, and China has taken a huge lead in powering EVs, controlling 85% of global battery cell production, International Energy Agency data shows.

Making batteries and cells, the units that store and convert chemical energy into electricity, is a delicate process and doing so at scale is a challenge for any battery maker.

Northvolt has missed some in-house targets and curtailed production at its battery cells plant in northern Sweden, underscoring the difficulties, Reuters reported on Monday.

“The biggest issue is that batteries are not easy to make and Northvolt haven’t satisfied the supply demands of their customers – that is a management issue,” said Andy Palmer, founder of consultancy Palmer Automotive said.

“The Chinese are technologically 10 years ahead of the West in batteries. That’s a fact,” he said.

At least eight companies have postponed or abandoned EV battery projects in Europe this year, including China’s Svolt and joint venture ACC (NS:), led by Stellantis (NYSE:) and Mercedes-Benz (OTC:).

In 2024, Europe’s battery pipeline capacity out to 2030 has fallen by 176 gigawatt-hours, according to data firm Benchmark Minerals. That’s equivalent to almost all the current installed capacity in Europe, according to Reuters calculations.

RETHINK

Some executives say Europe should do more to attract and support home-grown projects so they can compete with Chinese rivals such as CATL and BYD (SZ:).

“Europe needs to rethink how it supports a nascent sector before China eats up the entire value chain, which is due to smart planning,” said James Frith, European head of Volta Energy Technologies, which specialises in battery and energy storage technology.

Among its $5.8 billion in debts, Northvolt owes the European Investment Bank (EIB) some $313 million.

EIB vice president Thomas Östros said it had been a constructive partner to Northvolt, but it needed to safeguard the EIB and EU’s interests.

“It remains the case that Europe has a strategic interest in a European battery industry for electric cars and we will follow developments very closely. But it is much to early to say what the outcome will be,” he said.

The Swedish government has repeatedly said it does not plan to take a stake in Northvolt.

On Friday, Northvolt’s outgoing CEO and co-founder Peter Carlsson said he was a “little worried” Europe is giving up on its dream of competing with China.

He said Europe would regret it in 20 years time if it retreated.

“It’s not a straight journey and right now, we’re all in a bit of a down in that journey where there’s more hesitations, there’s more questions on the speed of the transition from the carmakers, from policymakers, from the investor community,” he told reporters in a call.