Jazz Pharmaceuticals Plc

- Exploratory analysis from Phase 1b/2 breast cancer trial reinforces CD47 as a predictive biomarker for response, as previously observed in the ASPEN-06 trial in HER2-positive gastric cancer – - Full data set submitted for presentation at an upcoming scientific conference - SOUTH SAN FRANCISCO, Calif., Jan. 30, 2026 (GLOBE NEWSWIRE) -- ALX Oncology Holdings Inc. (“ALX Oncology,” Nasdaq: ALXO), a clinical-stage biotechnology company advancing a pipeline of novel therapies designed to treat cancer and extend patients’ lives, announced new data from a Phase 1b/2 clinical trial evaluating the company’s investigational CD47-inhibitor evorpacept in combination with Jazz Pharmaceuticals’ ZIIHERA® (zanidatamab-hrii) in heavily pretreated patients with metastatic breast cancer (mBC). The topline findings, from an exploratory analysis in this trial, indicate that among patients with confirmed HER2-positive mBC, CD47 expression is predictive of evorpacept activity. “These new findings support a CD47-dependent, HER2-driven biology for evorpacept,” said Barbara Klencke, M.D., Chief Medical Officer at ALX Oncology. “Going forward, we believe that a biomarker-driven approach incorporating CD47 expression may optimize patient selection for evorpacept combinations with HER2-targeted agents. Additionally, taken together, the data from this trial and the ASPEN-06 clinical trial reinforce our confidence in the ongoing ASPEN-09-Breast Phase 2 trial.” The Phase 1b/2 open-label, multi-center clinical trial (NCT05027139) evaluated the potential of evorpacept in combination with zanidatamab as a novel treatment for patients with previously treated, inoperable, locally advanced, or metastatic HER2-expressing breast cancer and other cancers. The primary trial results, presented at the 2024 San Antonio Breast Cancer Symposium (SABCS), demonstrated that the investigational combination generated promising anti-tumor activity and a manageable safety pro patients with heavily pretreated HER2-positive breast cancer (median of six prior therapies), including treatment with ENHERTU. Researchers previously reported a 56% (5/9) confirmed objective response rate (cORR) and a median progression-free survival (mPFS) of 7.4 months in the nine patients with centrally confirmed HER2-positive breast cancer who received the investigational combination. The additional exploratory analysis, conducted to identify biomarkers of response to the evorpacept/zanidatamab combination, shows that responses in this trial were largely restricted to patients with higher CD47 expression. This finding reinforces results from the ASPEN-06 clinical trial , which demonstrated that CD47 expression is a predictive biomarker for response and durable benefit from evorpacept among patients with advanced gastric cancer that has retained HER2 expression. The full biomarker analysis from the Phase 1b/2 clinical trial has been submitted to an upcoming scientific congress for presentation. About ALX Oncology ALX Oncology (Nasdaq: ALXO) is a clinical-stage biotechnology company advancing a pipeline of novel therapies designed to treat cancer and extend patients’ lives. ALX Oncology’s lead therapeutic candidate, evorpacept, has demonstrated potential to serve as a cornerstone therapy upon which the future of immuno-oncology can be built. Evorpacept is currently being evaluated across multiple ongoing clinical trials in a wide range of cancer indications. ALX Oncology’s second pipeline candidate, ALX2004, is a novel EGFR-targeted antibody-drug conjugate with a differentiated mechanism of action. A Phase 1, dose-escalation trial of ALX2004 is ongoing in patients with EGFR-expressing solid tumors. More information is available at and on LinkedIn @ ALX Oncology . Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. Forward-looking statements include statements regarding future results of operations and financial position, business strategy, product candidates, planned preclinical studies and clinical trials, results of clinical trials, research and development costs, regulatory approvals, timing and likelihood of success, plans and objects of management for future operations, as well as statements regarding industry trends. Such forward-looking statements are based on ALX Oncology’s beliefs and assumptions and on information currently available to it on the date of this press release. Forward-looking statements may involve known and unknown risks, uncertainties and other factors that may cause ALX Oncology’s actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. These and other risks are described more fully in ALX Oncology’s filings with the Securities and Exchange Commission (SEC), including ALX Oncology’s Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q and other documents ALX Oncology files with the SEC from time to time. Except to the extent required by law, ALX Oncology undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Investor Relations Contact: Elhan Webb, CFA, IR Consultant ewebb@alxoncology.com Media Contact: Michele Parisi, SparkPoint Healthcare Communications mparisi@sparkpointpr.com (925) 864-5028

- Exploratory analysis from Phase 1b/2 breast cancer trial reinforces CD47 as a predictive biomarker for response, as previously observed in the ASPEN-06 trial in HER2-positive gastric cancer – - Full data set submitted for presentation at an upcoming scientific conference - SOUTH SAN FRANCISCO, Calif., Jan. 30, 2026 (GLOBE NEWSWIRE) -- ALX Oncology Holdings Inc. (“ALX Oncology,” Nasdaq: ALXO), a clinical-stage biotechnology company advancing a pipeline of novel therapies designed to treat cancer and extend patients’ lives, announced new data from a Phase 1b/2 clinical trial evaluating the company’s investigational CD47-inhibitor evorpacept in combination with Jazz Pharmaceuticals’ ZIIHERA® (zanidatamab-hrii) in heavily pretreated patients with metastatic breast cancer (mBC). The topline findings, from an exploratory analysis in this trial, indicate that among patients with confirmed HER2-positive mBC, CD47 expression is predictive of evorpacept activity. “These new findings support a CD47-dependent, HER2-driven biology for evorpacept,” said Barbara Klencke, M.D., Chief Medical Officer at ALX Oncology. “Going forward, we believe that a biomarker-driven approach incorporating CD47 expression may optimize patient selection for evorpacept combinations with HER2-targeted agents. Additionally, taken together, the data from this trial and the ASPEN-06 clinical trial reinforce our confidence in the ongoing ASPEN-09-Breast Phase 2 trial.” The Phase 1b/2 open-label, multi-center clinical trial (NCT05027139) evaluated the potential of evorpacept in combination with zanidatamab as a novel treatment for patients with previously treated, inoperable, locally advanced, or metastatic HER2-expressing breast cancer and other cancers. The primary trial results, presented at the 2024 San Antonio Breast Cancer Symposium (SABCS), demonstrated that the investigational combination generated promising anti-tumor activity and a manageable safety profile in patients with heavily pretreated HER2-positive breast cancer (median of six prior therapies), including treatment with ENHERTU. Researchers previously reported a 56% (5/9) confirmed objective response rate (cORR) and a median progression-free survival (mPFS) of 7.4 months in the nine patients with centrally confirmed HER2-positive breast cancer who received the investigational combination. The additional exploratory analysis, conducted to identify biomarkers of response to the evorpacept/zanidatamab combination, shows that responses in this trial were largely restricted to patients with higher CD47 expression. This finding reinforces results from the ASPEN-06 clinical trial, which demonstrated that CD47 expression is a predictive biomarker for response and durable benefit from evorpacept among patients with advanced gastric cancer that has retained HER2 expression. The full biomarker analysis from the Phase 1b/2 clinical trial has been submitted to an upcoming scientific congress for presentation. About ALX Oncology ALX Oncology (Nasdaq: ALXO) is a clinical-stage biotechnology company advancing a pipeline of novel therapies designed to treat cancer and extend patients’ lives. ALX Oncology’s lead therapeutic candidate, evorpacept, has demonstrated potential to serve as a cornerstone therapy upon which the future of immuno-oncology can be built. Evorpacept is currently being evaluated across multiple ongoing clinical trials in a wide range of cancer indications. ALX Oncology’s second pipeline candidate, ALX2004, is a novel EGFR-targeted antibody-drug conjugate with a differentiated mechanism of action. A Phase 1, dose-escalation trial of ALX2004 is ongoing in patients with EGFR-expressing solid tumors. More information is available at www.alxoncology.com and on LinkedIn @ALX Oncology. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. Forward-looking statements include statements regarding future results of operations and financial position, business strategy, product candidates, planned preclinical studies and clinical trials, results of clinical trials, research and development costs, regulatory approvals, timing and likelihood of success, plans and objects of management for future operations, as well as statements regarding industry trends. Such forward-looking statements are based on ALX Oncology’s beliefs and assumptions and on information currently available to it on the date of this press release. Forward-looking statements may involve known and unknown risks, uncertainties and other factors that may cause ALX Oncology’s actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. These and other risks are described more fully in ALX Oncology’s filings with the Securities and Exchange Commission (SEC), including ALX Oncology’s Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q and other documents ALX Oncology files with the SEC from time to time. Except to the extent required by law, ALX Oncology undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Investor Relations Contact: Elhan Webb, CFA, IR Consultant ewebb@alxoncology.com Media Contact: Michele Parisi, SparkPoint Healthcare Communications mparisi@sparkpointpr.com (925) 864-5028

iStock, Moor Studio The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding for the Immigration and Customs Enforcement’s large-scale crackdown in Minnesota and other states. The delay of reauthorizing the rare disease priority review voucher program has become an unexpected headache for biotech leaders, who have been lobbying the government to get the program moving again. “We think it’s absolutely critical to support innovative research, to support pediatric, rare disease medicines coming forward,” Ionis CEO Brett Monia told BioSpace at the J.P. Morgan Healthcare Conference earlier this month. The Senate failed on Thursday to pass a massive spending bill that included the rare pediatric PRV program. It also included funding for the Department of Homeland Security (DHS), under which the Immigration and Customs Enforcement (ICE) is executing its large-scale crackdown in Minnesota and other states. The vote was 55–45 against the bill. While President Donald Trump and Senate Democrats reached a new deal on Thursday that separates the DHS funding from that of other departments, House Speaker Mike Johnson said his chamber is unlikely to return to vote on the deal before Monday, Bloomberg reported Friday , almost assuring a partial government shutdown over the weekend. Rare diseases Rare Disease Biotechs Stand To Lose $4B if Priority Voucher Program Not Reinstated: Report Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs. January 26, 2026 · 7 min read · Heather McKenzie Read More Meanwhile, biopharma companies with PRV-eligible programs are preparing for either eventuality. Monia said Ionis was already racing to get zilganersen submitted for FDA review as fast as possible to help patients with Alexander disease who have few options. But the prospect of losing out on a potential priority review voucher that could support the company’s next rare disease treatment added another flame to the fire. Under a temporary extension, rare disease drugs approved by Sept. 30, 2026, will still qualify for a PRV. “Getting the drug to patients, of course, is number one. But not losing out on a potential PRV is one of our other objectives,” Monia explained. “Our team has worked through the holidays to get the NDA submitted as quickly as possible to meet that deadline.” PRVs can be sold, with most recent figures valuing them at around $150 million. Earlier this month, Jazz Pharmaceuticals revealed it had sold a voucher for $200 million to an unnamed buyer. Sometimes companies keep them—Ionis has numerous programs in development that a PRV could be applied to. But many smaller companies often choose to sell them and reinvest the money. Rare diseases Rare Disease Vouchers Caught in Political Abyss. It Didn’t Have To Be This Way The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since. January 23, 2026 · 3 min read · Heather McKenzie Read More The fate of the PRV program will also have an effect on mid-sized companies with broader R&D pipelines. “The PRV directly impacts how many of our rare-disease shots on goal we can fund and move into the clinic,” Justin To, CEO of skeletal dysplasia programs at BridgeBio, told BioSpace earlier this month. Catherine Owen Adams, CEO of Acadia Pharmaceuticals, has been lobbying the government on the PRV issue as well. She credits the program with accelerating investment in the rare disease space. “Since that’s come into being, we’ve seen an acceleration of rare drug approvals because the innovation has been worthwhile in terms of the large investment for small patient populations,” Owen Adams said. Monia was clear to state that this is an issue that Congress has to solve, not the FDA. Both he and Owen Adams said that FDA leadership has signaled nothing but support for the program. Monia met with FDA Commissioner Marty Makary in San Diego last year when the chief regulator was touring the country. Monia said PRVs came up in the meeting, and that Makary indicated that the program has his support. Owen Adams said the holdup has mainly been about finding the right legislation to pin the PRV reauthorization to. “There does seem to be widespread understanding and support that this is a good thing for rare disease patients, as well as American innovation,” she said, speaking to her experience lobbying on Capitol Hill to Republicans and Democrats alike. “I feel vaguely positive that it is going to be renewed. But, you know, stranger things have happened.” With a government shutdown looming over the omnibus bill, rare disease biotechs may have to wait even longer for certainty. Rare diseases Rare Disease Biotechs Left in a Lurch as Congress Fails To Renew Priority Review Program Congress did not reauthorize the rare pediatric disease priority review program at the end of 2024. Advocates say the ripple effect is already being felt across biopharma. March 5, 2025 · 9 min read · Annalee Armstrong Read More