Stock Markets
Mirum Pharmaceuticals to Showcase LIVMARLI Data from its ALGS and PFIC Programs at the NASPGHAN Annual Meeting
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
ImaginAb, Inc. Innovative Biologics Technology platform acquired by Telix to enable Next-Generation Therapeutic Assets discovery
INGLEWOOD, Calif., Jan. 22, 2025 /PRNewswire/ — ImaginAb, Inc., announces that it has entered into an agreement to sell a pipeline of next-generation therapeutic candidates, proprietary novel biologics technology platform, and a protein engineering and discovery research facility to Telix Pharmaceuticals Limited (ASX: TLX; Nasdaq: TXL).
Following the closing of this transaction, ImaginAb Inc., will focus on developing its lead imaging candidate, CD8 ImmunoPET, which is currently in Phase 2 clinical trials and has been licensed by numerous pharmaceutical and biotech companies for use in imaging within immunotherapy clinical trials, primarily in oncology. In addition, ImaginAb will continue to partner in advancing the pivotal prostate cancer imaging agent, which is currently being evaluated in Phase 2 clinical trials and as a surgical resection tool.
Dr. Anna Wu, Founder of ImaginAb, commented, “We are very pleased that Telix recognizes the potential of our novel biological technology platform including enabling Telix to explore new disease areas with state-of-the-art radiotherapeutic technology. These radiopharmaceutical agents represent the culmination of significant effort and resources by our scientific team. I extend my congratulations to everyone at ImaginAb for reaching this significant milestone. This transaction further validates our novel minibody platform.”
Dr. Wu continued, “With the sale of our radiopharmaceutical platform, ImaginAb will continue the development of its CD8 platform. We are encouraged that numerous pharmaceutical and biotech companies have incorporated our technology in their immuno-oncology clinical trials.”
Jefferies LLC and Stifel, Nicolaus & Company, Incorporated served as financial advisors to ImaginAb on the transaction.
About ImaginAb, Inc.
ImaginAb, Inc. is a clinical stage, revenue-generating global biotechnology company developing the next generation of radiopharmaceutical and imaging agent products. These patented products contain engineered antibodies that maintain the specificity of full-length antibodies while remaining biologically inert in the body. Used with widely available positron emission tomography (PET) and optical imaging technology, these novel targeting agents are able to bind specifically to cell surface targets.
The company is backed by top tier venture capital firms and strategic corporate firms including, Adage Capital, The Cycad Group, Norgine Ventures, Innoviva, Jim Pallotta of the Raptor Group, The Parker Institute for Cancer Immunotherapy, and Merck (NSE:) (MSD) Pharma. For more information about ImaginAb’s pipeline and technology, visit www.imaginab.com.
About CD8 ImmunoPET
The 89Zr CD8 ImmunoPET technology (zirconium Zr 89 crefmirlimab berdoxam) is a [89Zr]-labelled minibody that binds the CD8 receptor on human T cells and is used for quantitative, non-invasive PET imaging of CD8+ cells in patients. CD8+ cells are the main effector cells involved in the immune response against tumor cells induced by immunotherapies and they also play a key role in multiple autoimmune diseases. As such, quantitative imaging of CD8+ cells can be used to diagnose the immune status of a patient, to measure the efficacy of immunotherapies and predict patient outcomes.
About Optical PSMA
The Optical PSMA Imaging Agent (IR-800 IAB2 Minibody) is a fluorescent labelled minibody that binds the PSMA receptor present on cancer cells including prostate cancer and is used for quantitative, non-invasive PET imaging of PSMA+ cells in patients undergoing surgery to remove cancerous tissue . As such, imaging of PSMA + cells may be used to guide clinicians during surgery to identify cancerous tissue and aid tissue resection.
Stock Markets
Trump escalates campaign against diversity, threatens private sector probes
By Daniel Trotta and Bianca Flowers
(Reuters) -U.S. President Donald Trump escalated his campaign against diversity programs on Tuesday by pressuring the private sector to join the initiative and telling government employees in offices administering such programs they would be placed on paid leave.
On his first day in office Trump issued a series of executive orders to end diversity, equity and inclusion programs, which attempt to promote opportunities for women, ethnic minorities, LGBTQ+ people and other traditionally underrepresented groups.
Civil rights advocates have argued such programs are necessary to address longstanding inequities and structural racism.
In an executive order issued on Tuesday, Trump revoked executive orders dating as far back as 1965 on environmental actions, equal employment opportunities and encouragement to federal contractors to achieve workforce balancing on race, gender and religion.
The 1965 order that was revoked was signed by then-President Lyndon Johnson to protect the rights of workers employed by federal contractors and ensure they remained free from discrimination on the basis of race, color, religion, sex, sexual orientation, gender identity or national origin, according to the Labor Department.
The Trump executive order seeks to dissuade private companies that receive government contracts from using DEI programs and hiring on the basis of race and sex – what the order called “illegal DEI discrimination and preferences” – and asked government agencies to identify private companies that might be subject to civil investigation.
“As a part of this plan, each agency shall identify up to nine potential civil compliance investigations of publicly traded corporations, large non-profit corporations or associations, foundations with assets of 500 million dollars or more, State and local bar and medical associations, and institutions of higher education with endowments over 1 billion dollars,” the order said.
Full details on how the Trump administration would enforce “civil compliance investigations” were not immediately available.
The order issued on Tuesday stipulates that federal and private-sector employment preferences for military veterans could continue.
The executive order was celebrated by conservative activists and Republican leaders. It was also met with swift condemnation from civil rights leaders.
Rev. Al Sharpton, founder and president of the National Action (WA:) Network, announced on Wednesday the organization and its partners plan to identify two companies in the next 90 days that will be boycotted for abandoning DEI pledges.
Basil Smikle Jr., a political strategist and policy adviser, said he was troubled by the Trump administration’s assertion that diversity programs were “diminishing the importance of individual merit, aptitude, hard work, and determination” because it suggested women and people of color lacked merit or qualifications.
“There’s this clear effort to hinder, if not erode, the political and economic power of people of color and women,” Smikle said.
“What it does is opens up the door for more cronyism,” he said.
The White House did not immediately respond to a request from Reuters to address criticism from civil rights advocates.
Separately, the Trump administration instructed U.S. federal government departments and agencies to dismantle all DEI programs, advising employees of such programs that they would be immediately placed on paid leave.
The government should by the end of business on Wednesday inform employees of any government offices or units focused exclusively on DEI that their programs will be shut down and employees placed on leave, the Office of Personnel Management said in a memorandum.
Trump also signed a memorandum on Tuesday that ends a Biden administration initiative to promote diversity in the Federal Aviation Administration (FAA), ordering the FAA administrator to immediately stop DEI hiring programs, the White House said.
Trump ordered the FAA to conduct a safety review that would replace any employees who fail to demonstrate their competence.
“President Trump is immediately terminating this illegal and dangerous program and requiring that all FAA hiring be based solely on ensuring the safety of airline passengers and overall job excellence,” the White House said in a fact sheet.
Stock Markets
Trump US energy emergency order should withstand court challenges
(Reuters) – U.S. President Donald Trump’s declaration of a national energy emergency to boost drilling and speed up pipeline construction should withstand court challenges but will not allow oil and gas producers to skirt all environmental laws, according to legal experts.
Trump, a Republican who campaigned on a promise to “drill baby drill,” has said the declaration will speed permitting and approval of energy projects to fix what he has called an inadequate and unaffordable U.S. energy supply.
The U.S. is the world’s largest oil producer and the world’s largest exporter of liquefied , according to U.S. Energy Information Administration data.
Trump’s energy declaration, among the executive orders he signed his first day in office, invokes a federal law giving the president broad discretion to declare emergencies and unlock special powers. Legal experts say challenging the declaration itself in court would likely be futile because courts rarely question the president’s judgment in using the National Emergencies Act.
“The law doesn’t define what an emergency is, and so far no court has been willing to overturn a finding that there is an emergency,” said University of California, Berkeley Law School professor Dan Farber.
The National Emergencies Act can unlock presidential powers in 150 different statutes but has limited reach into environmental laws and regulations.
The true legal tests will likely arise in implementation of the order, which directs federal agencies to scour their books for laws and regulations that could be used to speed along approval and permitting for projects like drilling, refining and pipeline construction.
The order cites laws including the Clean Water Act, Endangered Species Act and Mammal Protection Act, which impose review and permitting requirements on energy projects.
“It could expedite energy projects but also harm water standards, endangered species protections, fill in the blank,” said Emory University School of Law professor Mark Nevitt.
“There’s a reason those emergency regulations aren’t tapped on a day-to-day basis.”
Erik Schlenker-Goodrich, Executive Director of the Western Environmental Law Center, said he expects most of the legal fighting to arise over what federal agencies actually do, rather than the declaration itself.
“We anticipate that political appointees will work to implement Trump’s agenda through secretarial orders and specific agency actions, whether regulatory rollbacks, new lease sales, drilling permits, pipeline approvals, etc. That’s where the fight will prove most intensive,” Schlenker-Goodrich said.
The emergency declaration could be a useful tool for defending those agency decisions in court, providing a national security rationale that judges would be unlikely to question, some experts said.
The order includes a prominent role for the president’s National Security Advisor, who could sign off on reports concluding that certain regulatory rollbacks are necessary to protect vital national interests.
“Once you have that badge of approval from the National Security Council, you can flash it to every federal judge that tries to stand in the way, because courts consistently defer to national security claims,” said Tyson Slocum of the consumer advocacy group Public Citizen.
Environmental groups have condemned the energy emergency order, saying climate change driven by fossil fuels consumption is the true emergency.
But some have said they do not expect to file lawsuits until they see what the administration actually does.
“It’s hard to challenge an executive order in general,” said Brett Hartl of the Center for Biological Diversity. “If they start doing things that are egregious and use the executive order as a rationale, we would be prepared to sue,” Hartl added.
David Doniger, a senior attorney with the Natural Resources Defense Council, said in a statement that the emergency declaration does not override other laws and that any regulatory rollbacks outlined in executive orders will have to be done through proper legal channels.
“We certainly will challenge rollbacks that lack legal and scientific support.”
While Trump can encourage new drilling by rolling back regulations and pushing for more fossil fuel output in places like Alaska, the cadence at which oil and gas production increases will ultimately be decided by energy companies and market forces.
Many energy firms have restrained growth in recent years to focus on shareholder returns and buybacks after investors soured on the sector. Meanwhile, natural gas producers are looking to a boom in new U.S. LNG facilities to boost demand after cutting output in 2024 as prices fell to the lowest in decades. (This story has been refiled to change the date to Jan 22, not Jan 21, in the dateline)
- Forex3 years ago
Forex Today: the dollar is gaining strength amid gloomy sentiment at the start of the Fed’s week
- Forex2 years ago
How is the Australian dollar doing today?
- Forex2 years ago
Unbiased review of Pocket Option broker
- Forex2 years ago
Dollar to pound sterling exchange rate today: Pound plummeted to its lowest since 1985
- Cryptocurrency2 years ago
What happened in the crypto market – current events today
- World2 years ago
Why are modern video games an art form?
- Commodities2 years ago
Copper continues to fall in price on expectations of lower demand in China
- Forex2 years ago
The dollar is down again against major world currencies