On March 21, 2023 X4 Pharmaceuticals Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel small-molecule therapeutics to benefit people with diseases of the immune system, reported financial results for the fourth quarter and full year ended December 31, 2022 and highlighted key 2022 events and expected upcoming milestones (Press release, X4 Pharmaceuticals, MAR 21, 2023, View Source [SID1234629106]).

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"We could not be more pleased with the progress we made in 2022 advancing our investigational therapy, mavorixafor, towards commercialization," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "Our clinical trial data continue to speak for themselves, demonstrating mavorixafor’s ability to elevate circulating levels of neutrophils, lymphocytes, and monocytes in people with immune system dysfunction. Following the positive results from our 4WHIM clinical trial in late 2022, we continue to expect presentation of additional Phase 3 data in the second quarter of 2023 and the submission of our first New Drug Application (NDA) in the U.S. early in the second half of the year. We are now enrolling patients in our Phase 2 trial in people with certain chronic neutropenic (CN) disorders and anticipate announcing clinical data from this trial along with clarity on the potential regulatory path forward for mavorixafor in CN disorders in the second or third quarter of 2023."

2022 Highlights & Key Anticipated Upcoming Milestones

Positive Clinical Results: With a tightened focus on advancing mavorixafor in chronic neutropenia indications, X4 announced successful conclusion of and positive data from key clinical programs in 2022:
•Pivotal, Phase 3 4WHIM trial: In late November, the company announced that its Phase 3 clinical trial evaluating oral mavorixafor in people with WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome met its primary endpoint and first key secondary endpoint, with mavorixafor achieving statistically significant and clinically relevant longer times above threshold levels for both absolute neutrophil and absolute lymphocyte counts versus placebo and demonstrating good tolerability in the trial.
◦Additional data from the 4WHIM trial are expected to be presented at medical conferences in the second quarter of 2023.

Exhibit 99.1
◦The company is now preparing to meet with U.S. regulatory authorities to discuss next steps in advancing mavorixafor towards an NDA submission, which is still anticipated early in the second half of 2023.
◦To the best of the company’s knowledge, mavorixafor is the first and only oral therapy in development to demonstrate durable improvements in severe chronic neutropenia and lymphopenia, the hallmarks of WHIM syndrome, a rare primary immunodeficiency for which there are no approved treatments.
•Phase 1b Chronic Neutropenia Trial: In September 2022, X4 presented positive data from a Phase 1b clinical trial demonstrating the ability of one dose of oral mavorixafor to increase and normalize absolute neutrophil counts (ANC) in people with idiopathic, cyclic, or congenital chronic neutropenia as monotherapy or administered concurrently with injectable granulocyte colony-stimulating factor (G-CSF).
◦The company continues to believe that these results suggest an expanded future market opportunity for mavorixafor that could include up to 50,000 diagnosed patients in the U.S. with certain forms of chronic neutropenia.
◦The Phase 1b trial has now been amended and expanded into a Phase 2 clinical trial to assess the long-term durability, safety, and tolerability of oral mavorixafor in a larger population with idiopathic, cyclic, or congenital chronic neutropenia.
◦Participants are currently being enrolled in the Phase 2 CN trial and X4 anticipates announcing clinical data and clarity on the scope and timing of the expected Phase 3 clinical program for mavorixafor in chronic neutropenia in the second or third quarter of 2023.

Greater Insight into Mavorixafor Market Potential: Throughout 2022, X4 presented results from both its clinical and research programs, further supporting the commercial potential of mavorixafor in a variety of chronic neutropenia indications, including WHIM syndrome, at multiple, prominent medical conferences.
•Conferences included: the American Academy of Allergy Asthma & Immunology (AAAAI) Annual Meeting, the Clinical Immunology Society (CIS) Annual Meeting, the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress, the North American Immuno-Hematology Clinical Education & Research (NICER) Symposium, the European Society for Immunodeficiencies (ESID), the National Organization for Rare Diseases (NORD) fall Summit, and the Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper).
•These presentations not only highlighted new insights into the genotype and phenotype of people with WHIM syndrome, helping to identify additional patients and educate treating physicians, but also spotlighted new understandings into and confirmation of mavorixafor’s ability to mobilize mature, functional white blood cells from the bone marrow into the blood, and helped elucidate the unmet medical needs and the size of the U.S. population living with severe chronic neutropenia.

Strong Balance Sheet to Support Drive to Commercialization: Despite continued challenging market conditions, during 2022, X4 successfully completed two large financings, raising aggregate gross proceeds of approximately $120 million and has subsequently re-negotiated its loan facility agreement:
•In July 2022, X4 completed a private investment in public equity (PIPE) financing, receiving aggregate gross proceeds of approximately $55 million.
•In December 2022, the company completed a public offering that yielded gross proceeds of approximately $65.0 million before deducting underwriting discounts and estimated offering expenses and any proceeds from the exercise of the warrants to be issued in the offering.

Exhibit 99.1
•On January 6, 2023, X4 entered into a Second Amended and Restated Loan and Security Agreement with Hercules Capital that amended and restated all previous loan and security agreements, and that included the extension of the interest-only payment period through the third quarter of 2024 and an agreement to further extend the interest-only period to the first quarter of 2026 if certain milestones are achieved.
•In addition, the covenant under the Hercules agreement requiring that the company maintain cash in an aggregate amount greater than $30.0 million was lowered to $20.0 million, subject to certain terms and conditions.

Fourth Quarter and Full Year 2022 Financial Results
•Cash, Cash Equivalents & Restricted Cash: X4 had $123.0 million in cash, cash equivalents, and restricted cash as of December 31, 2022. X4 believes that it has sufficient funds to support company operations into the second quarter of 2024.
•Research and Development (R&D) Expenses were $19.0 million and $61.1 million for the fourth quarter and full year ended December 31, 2022, respectively, as compared to $12.2 million and $50.6 million for the comparable periods in 2021. R&D expenses included $0.5 million and $2.5 million of certain non-cash expenses for the fourth quarter and full year ended December 31, 2022, respectively.
•Selling, General and Administrative Expenses (SG&A) were $6.6 million and $27.0 million for the fourth quarter and full year ended December 31, 2022, respectively, as compared to $7.1 million and $24.7 million for the comparable periods in 2021. SG&A expenses included $0.6 million and $2.7 million of certain non-cash expenses for the fourth quarter and full year ended December 31, 2022, respectively.
•Net Loss: X4 reported a net loss of $29.1 million and $93.9 million for the fourth quarter and full year ended December 31, 2022, respectively, as compared to $30.2 million and $88.7 million for the comparable periods in 2021. Net loss included $1.1 million and $5.2 million of stock-based compensation expenses for the fourth quarter and full year ended December 31, 2022, respectively. Net loss included $1.6 million and $6.2 million of stock-based compensation expenses for the fourth quarter and full year ended December 31, 2021, respectively. Net loss for the fourth quarter and full year ended December 31, 2021 included a non-cash goodwill impairment charge of $9.8 million. There was no goodwill impairment charge in 2022.
Conference Call and Webcast
X4 will host a conference call and webcast today at 9:00 am ET to discuss these financial results and business highlights. The conference call can be accessed by dialing 1-855-327-6837 within the United States or 1-631-576-4098 internationally, followed by the conference ID: 10020997. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the company website.

On March 21, 2023 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, reported the grant of non-qualified stock options to purchase an aggregate of 7,130 shares of common stock of the company and 5,885 restricted stock units of the company’s common stock to ten newly hired non-executive officers of the company (Press release, Ultragenyx Pharmaceutical, MAR 21, 2023, View Source [SID1234629105]). The awards were approved by the compensation committee of the company’s board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of March 16, 2023, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4).

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The restricted stock units vest over four years, with 25% of the underlying shares vesting on each anniversary of the grant date, subject to the employee being continuously employed by the company as of such vesting dates. The stock options vest over four years, with 25% of the shares underlying the option vesting on the first anniversary of the grant date and the remainder vesting with respect to 1/48th of the shares underlying the options on each monthly anniversary thereafter, subject to the employee being continuously employed by the company as of such vesting dates. The stock options have a ten-year term and an exercise price of $40.22 per share, equal to the per share closing price of Ultragenyx’s common stock on March 16, 2023.

On March 21, 2023 Sensei Biotherapeutics, Inc. (Nasdaq: SNSE), an immuno-oncology company focused on the discovery and development of next-generation therapeutics for cancer patients, reported the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for a Phase 1/2 clinical trial of SNS-101, a conditionally active VISTA-blocking antibody, in patients with solid tumors (Press release, Sensei Biotherapeutics, MAR 21, 2023, View Source [SID1234629104]).

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SNS-101 is a conditionally active, human monoclonal IgG1 antibody designed to selectively block the VISTA checkpoint in the tumor microenvironment, which acts as a suppressor of T cells by binding the receptor PSGL-1. Preclinical studies have demonstrated SNS-101’s potential to inhibit tumor growth as monotherapy, significantly enhance the anti-tumor effects of PD-1 blockade, avoid poor pharmacokinetics from target-mediated drug disposition and lower the risk of cytokine release syndrome. Sensei plans to evaluate SNS-101 as a novel treatment for patients with solid cancers, as both a monotherapy and in combination with other therapies.

On March 21, 2023 Prestige Biopharma reported that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for PBP1510 (International Non-proprietary name: Ulenistamab), in the treatment of unresectable or metastatic pancreatic adenocarcinoma (PDAC) that has relapsed following and/or is refractory to at least one line of prior therapy (Press release, Prestige BioPharma, MAR 21, 2023, View Source [SID1234629103]).

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PBP1510 targets Pancreatic Adenocarcinoma Upregulated Factor (PAUF), a tumour-specific protein, found to be overexpressed in majority of pancreatic cancer cases. PAUF overexpression promotes key cellular functions, including proliferation, migration, invasion, and growth of pancreatic cancer cells, and contributes to the development of acquired resistance to chemotherapeutic agents. PBP1510 is designed to target these key biological mechanisms that results in limited effectiveness of current treatment options and rapid progression of pancreatic cancer

By effectively inhibiting the tumorigenic effects of PAUF overexpression in preclinical models, PBP1510 represents a promising therapeutic strategy for addressing the unmet medical needs of pancreatic cancer patients. A global Phase 1/2a clinical trial is currently underway in the United States, Europe, and Asia, with the aim of bringing this innovative therapy to the clinic

The first-in-human Phase 1/2a study is an open-label, multicentre, two-part study in patients with advanced/metastatic pancreatic cancer. Phase 1 is a dose-escalation phase, wherein PBP1510 will be administered, either as monotherapy or in combination with gemcitabine, in two separate dose-escalation cohorts. From Phase 1 part of the study a recommended Phase 2a dose (RP2D) will be determined based on the analysis of pharmacokinetics, safety, and efficacy data. Phase 2 is a dose-expansion phase, wherein PBP1510 at the RP2D in combination with gemcitabine will be administered to evaluate efficacy and safety of PBP1510

Overall, the Phase 1/2a study aims to collect important safety data on the use of PBP1510 as a monotherapy or in combination with gemcitabine and explore the efficacy of a combined PBP1510 and gemcitabine regimen. The study will substantiate the preclinical findings of PBP1510’s synergistic antitumour activity in combination therapy with gemcitabine without increased toxicity, as anticipated from their distinct mechanisms of action

With Fast Track designation from the FDA, PBP1510 represents a promising advancement in the treatment of pancreatic cancer. Prestige Biopharma intends to take full advantage of the benefits offered by the designation to provide faster access for patients in need.

On March 21, 2023 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported that it will present at the Stifel 2023 CNS Days at 12:30 p.m. Eastern Time on Tuesday, March 28, 2023 (Press release, Neurocrine Biosciences, MAR 21, 2023, View Source [SID1234629102]). Eiry Roberts, Chief Medical Officer, and Kyle Gano, Chief Business Development and Strategy Officer, will participate at the conference.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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(PRNewsfoto/Neurocrine Biosciences, Inc.)

The live presentation will be webcast and may be accessed on the Company’s website under Investors at www.neurocrine.com. A replay of the presentation will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month.