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<ul><li>On November 13, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced financial results for the third quarter of 2024 and provided a business update.</li><li>The company announced positive topline results for the six-month Phase 2 trial of mavorixafor in patients with chronic neutropenia (CN).</li><li>The drug raised participants mean absolute neutrophil counts (ANC), and physicians were able to reduce G-CSF dosing in nine of 12 eligible patients, with three taken off G-CSF completely.</li></ul>

On November 13, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced financial results for the third quarter of 2024 and provided a business update.The company announced positive topline results for the six-month Phase 2 trial of mavorixafor in patients with chronic neutropenia (CN).The drug raised participants mean absolute neutrophil counts (ANC), and physicians were able to reduce G-CSF dosing in nine of 12 eligible patients, with three taken off G-CSF completely.

XFOR: Positive Results for Phase 2 Trial of Mavorixafor in Chronic Neutropenia 

<ul><li>On August 8, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced financial results for the second quarter of 2024 and provided a business update.</li><li>Following the commercial launch of XOLREMDI (mavorixafor) in April 2024 the company recorded the first commercial sales of the drug, which totaled approximately $0.6 million in the second quarter of 2024.</li><li>We anticipate the full data set from the Phase 2 trial of mavorixafor in chronic neutropenia (CN) to be presented in November 2024.</li></ul>

On August 8, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced financial results for the second quarter of 2024 and provided a business update.Following the commercial launch of XOLREMDI (mavorixafor) in April 2024 the company recorded the first commercial sales of the drug, which totaled approximately $0.6 million in the second quarter of 2024.We anticipate the full data set from the Phase 2 trial of mavorixafor in chronic neutropenia (CN) to be presented in November 2024.

XFOR: First Commercial Sales of XOLREMDI  

<ul><li>On June 27, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced positive interim results from the company's ongoing Phase 2 trial of mavorixafor in patients with chronic neutropenia (CN).</li><li>The data showed that treatment with mavorixafor, as either a monotherapy or in combination with granulocyte colony-stimulating factor (G-CSF) was generally well tolerated and led to durable increases in patients' absolute neutrophil counts.</li><li>The company also announced that it has begun screening patients for enrollment into the Phase 3 clinical trial of mavorixafor in people with congenital, acquired primary immune, or idiopathic CN who are experiencing recurrent and/or serious infections.</li></ul>

On June 27, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced positive interim results from the company's ongoing Phase 2 trial of mavorixafor in patients with chronic neutropenia (CN).The data showed that treatment with mavorixafor, as either a monotherapy or in combination with granulocyte colony-stimulating factor (G-CSF) was generally well tolerated and led to durable increases in patients' absolute neutrophil counts.The company also announced that it has begun screening patients for enrollment into the Phase 3 clinical trial of mavorixafor in people with congenital, acquired primary immune, or idiopathic CN who are experiencing recurrent and/or serious infections.

XFOR: Positive Interim Data from Ongoing Phase 2 Trial of Mavorixafor in Chronic Neutropenia Patients

<ul><li>On May 7, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced financial results for the first quarter of 2024.</li><li> The company will be hosting an investor event in June 2024 to present interim results from the ongoing Phase 2 trial of mavorixafor in chronic neutropenia (CN).</li><li>The company has initiated the launch of XOLREMDI (mavorixafor) for use in patients 12 years of age and older with WHIM syndrome.</li></ul>

On May 7, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced financial results for the first quarter of 2024. The company will be hosting an investor event in June 2024 to present interim results from the ongoing Phase 2 trial of mavorixafor in chronic neutropenia (CN).The company has initiated the launch of XOLREMDI (mavorixafor) for use in patients 12 years of age and older with WHIM syndrome.

XFOR: Data from Phase 2 CN Trial Expected in June 2024 

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Water Tower Research was founded by Wall Street veterans after witnessing a fundamental shift in the investment research industry with the result being a declining knowledge base among investors, a lack of awareness and a shrinking pool of fundamental research being conducted on companies across the small-cap and micro-cap spectrum. The same issues are now increasingly occurring in the mid-cap space. This sea change within the investment community is impacting not only the companies themselves but also current shareholders and potential investors. With significantly less active sell-side sponsorship by Wall Street, a substantial information gap has developed between companies and various constituents in the financial community. The net result is fewer channels for insight and information to flow between companies and investors.

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<ul>
<li>We review last week’s biopharma news for meaningful clinical data, regulatory updates, research innovation, and M&amp;A.</li>
<li>We recapped our IOC of Ainos, Inc. (AIMD) in a FlashCast Podcast. Those interested can listen to the episode on Apple Podcasts or Spotify.</li>
<li>Alnylam Pharmaceuticals, Inc. (ALNY) reported strong vutrisiran Phase 3 HELIOS-B results for ATTR-CM, showing a significant 28% reduction in the primary composite endpoint of mortality and CV events for all patients (HR=0.718, p=0.0118) and a 33% reduction for the mono subset (HR=0.672, p=0.0162) versus placebo. </li>
</ul>


We review last week’s biopharma news for meaningful clinical data, regulatory updates, research innovation, and M&amp;A.
We recapped our IOC of Ainos, Inc. (AIMD) in a FlashCast Podcast. Those interested can listen to the episode on Apple Podcasts or Spotify.
Alnylam Pharmaceuticals, Inc. (ALNY) reported strong vutrisiran Phase 3 HELIOS-B results for ATTR-CM, showing a significant 28% reduction in the primary composite endpoint of mortality and CV events for all patients (HR=0.718, p=0.0118) and a 33% reduction for the mono subset (HR=0.672, p=0.0162) versus placebo. 


United States

X4 Pharmaceuticals, Inc. operates as a clinical-stage biopharmaceutical company. The Company specializes in developing novel therapeutics designed to improve immune cell trafficking to treat rare diseases including primary immunodeficiencies and cancer. X4's oral small molecule drug candidates antagonize the CXCR4 pathway which plays a central role in immune surveillance.

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X4 Pharmaceuticals (XFOR US)

XFOR: Positive Results for Phase 2 Trial of Mavorixafor in Chronic Neutropenia

On November 13, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced financial results for the third quarter of 2024 and provided a business...

Zacks Small Cap Research

XFOR: First Commercial Sales of XOLREMDI

On August 8, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced financial results for the second quarter of 2024 and provided a business...

Zacks Small Cap Research

XFOR: Positive Interim Data from Ongoing Phase 2 Trial of Mavorixafor in Chronic Neutropenia Patients

On June 27, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced positive interim results from the company's ongoing Phase 2 trial of mavorixafor in...

Zacks Small Cap Research

XFOR: Data from Phase 2 CN Trial Expected in June 2024

On May 7, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced financial results for the first quarter of 2024. The company will be hosting an...

Zacks Small Cap Research

Biopharma Week in Review - July 1, 2024

We review last week’s biopharma news for meaningful clinical data, regulatory updates, research innovation, and M&A.

Water Tower Research

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