On March 12, 2020 Selecta Biosciences, Inc. (NASDAQ: SELB), a clinical-stage biotechnology company focused on unlocking the full potential of biologic therapies based on its immune tolerance platform, ImmTOR, reported financial results for the fourth quarter and year ended December 31, 2019 (Press release, Selecta Biosciences, MAR 12, 2020, View Source [SID1234555462]).

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"2019 was a pivotal year for Selecta. We made substantial clinical advancements, as we completed enrollment in the head-to-head COMPARE trial for our lead product candidate, SEL-212, in chronic refractory gout, and advanced our gene therapy program through strategic partnerships with AskBio. We also strengthened our organization with the addition of several key members to the executive team and welcomed a new Chairman of our Board of Directors, and completed a $70 million financing," said Carsten Brunn, Ph.D., President and CEO of Selecta. "In the coming year, we look forward to reporting topline data from the COMPARE study, commencing the Phase 3 clinical program of SEL-212, and advancing our gene therapy program into the clinic in partnership with AskBio."

Recent Highlights and Anticipated Upcoming Milestones:

Topline Results from COMPARE Clinical Trial Expected in the Third Quarter of 2020: In December 2019, we announced the completion of enrollment in the head-to-head COMPARE study of Selecta’s lead product candidate, SEL-212 (ImmTOR + pegadricase), vs. pegloticase. Topline data from this trial is expected in the third quarter of 2020. The trial is evaluating a once-monthly dose of SEL-212 compared to biweekly doses of pegloticase, with the primary endpoint of the maintenance of serum uric acid (SUA) levels of <6mg/dL at three and six months.

Meeting with FDA Provides Clarity on Phase 3 Clinical Program of SEL-212: Selecta held a meeting with the U.S. Food and Drug Administration (FDA) in January 2020 to inform the design of the planned Phase 3 clinical program. Selecta plans to commence its Phase 3 clinical program of SEL-212 against placebo in the second half of 2020.

Gene Therapy Program in the Clinic by the End of 2020: In August 2019, Selecta announced a strategic partnership with Asklepios BioPharmaceutical, Inc. (AskBio), to jointly develop, manufacture, and commercialize a broad portfolio of next-generation AAV gene therapies. This

partnership will leverage the unique proprietary technology platforms of both companies with a human proof of concept trial to validate this portfolio of products and their potential for re-dosing in patients, which could represent a significant advancement in the gene therapy field. Selecta and AskBio anticipate entering the clinic by the end of 2020. Additionally, the Company intends to advance its proprietary program in ornithine transcarbamylase (OTC) deficiency.

Broadened Strategic Partnership with AskBio: In December 2019, Selecta and AskBio jointly announced that the companies entered into a license agreement under which AskBio exercised its option to exclusively license rights to develop and commercialize Selecta’s immune tolerance platform, ImmTOR, for use in adeno-associated virus (AAV) gene therapy for the treatment of Pompe disease. Affecting 5,000-10,000 people worldwide, Pompe disease is a rare, genetic, lysosomal storage disease characterized by the abnormal buildup of a sugar molecule called glycogen inside cells. Under the terms of the agreement, Selecta received upfront payments of $7 million and is eligible to receive milestone payments of $237 million plus royalties on product sales.

Raised $70 Million in a Private Placement: In December 2019, Selecta announced the closing of a transaction to sell securities in a private placement with institutional investors and certain members of the Company’s Board of Directors, resulting in gross proceeds of approximately $70 million.

Strengthened Board of Directors: In November 2019, Selecta announced the appointment of Carrie S. Cox to the position of Chairman of the Board of Directors. A renowned industry leader and successful biopharmaceutical executive, Ms. Cox has served on multiple Boards, and has held the position of Chair, for several biopharmaceutical companies.

Fourth Quarter and Full Year 2019 Financial Results:

Cash Position: Selecta had $91.6 million in cash, cash equivalents, and restricted cash as of December 31, 2019, which compares to cash, cash equivalents, restricted cash, and short-term investments of $35.9 million as of September 30, 2019. Selecta believes its available cash, cash equivalents, and restricted cash will be sufficient to meet its operating requirements into the first quarter of 2021.

Net cash used in operating activities was $12.9 million and $51.4 million for the fourth quarter and fiscal year 2019, respectively, as compared to $12.7 million and $59.2 million for the same periods in 2018.

Research and Development Expenses: Research and development expenses for the fourth quarter and fiscal year 2019 were $15.2 million and $42.7 million, respectively, which compares with $10.3 million and $47.7 million for the same periods in 2018. The quarterly increase reflects additional costs incurred specific to our Phase 2 head-to-head (COMPARE) clinical trial of SEL-212, for which we completed enrollment in December 2019. The decrease year over year reflects reduced costs in 2019 due to the completion of prior programs in 2018, combined with reduced salaries and benefits resulting from the headcount reduction in early 2019. The cost reductions were offset by an overall increase in costs incurred on our lead product candidate, SEL-212.

General and Administrative Expenses: General and administrative expenses for the fourth quarter and fiscal year 2019 were $4.1 million and $16.4 million, respectively, which compares with $5.1 million and $18.2 million for the same periods in 2018. The decrease is the result of lower salaries and stock compensation expense resulting from reduced headcount at the end of 2018, combined with reduced patent and professional fees.

Net Loss: For the fourth quarter and fiscal year 2019, Selecta reported a net loss of $14.9 million, or $0.28 per share and $55.4 million, or $1.22 per share, compared to a net loss of $14.7 million, or $0.65 per share, and $65.3 million, or $2.92 per share, for the same periods in 2018.

Conference Call and Webcast Reminder:
Selecta management will host a conference call at 8:30 AM ET today to provide a corporate update and review the company’s fourth quarter 2019 financial results. Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10138603. Investors and the public can access the live and archived webcast of this call and a copy of the presentation via the Investors & Media section of the company’s website, www.selectabio.com.

On March 12, 2020 Nurix Therapeutics, Inc., a company developing targeted protein modulation drugs, reported it has closed an oversubscribed $120 million financing (Press release, Nurix Therapeutics, MAR 12, 2020, View Source [SID1234555461]). The round was led by Foresite Capital with participation from Bain Capital Life Sciences, Boxer Capital (Tavistock Group), EcoR1 Capital, Redmile Group, Wellington Management Company and an undisclosed investor, as well as Nurix’s founding investors The Column Group and Third Rock Ventures.

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"With the funds raised in this financing, Nurix is well positioned to bring its targeted protein modulation therapeutics into the clinic," said Arthur Sands, M.D., Ph.D., chief executive officer of Nurix Therapeutics. "We will also continue to use our powerful DELigase platform to discover new therapies aimed at previously undruggable targets of high therapeutic potential."

Proceeds from the financing will enable the company to advance Nurix’s wholly owned development candidates into clinical development. The leading edge of Nurix’s pipeline includes an orally delivered BTK chimeric targeting molecule (CTM) for B cell malignancies for which the Company expects to file an IND application with the FDA by the end of the year. The second molecule in Nurix’s preclinical pipeline is an orally delivered inhibitor of the CBL-B ligase for stimulation of T cell activation and IL-2 secretion as a novel immuno-oncology agent. Both development candidates were derived from Nurix’s DELigaseTM targeted protein modulation platform, which combines the use of DNA-encoded libraries (DEL) with an expanding set of E3 ligases to achieve targeted protein modulation. Nurix’s scientific approach enables either the harnessing of E3 ligases to degrade specific target proteins, as in the case of the BTK CTM, or the inhibition of specific ligases to raise substrate protein levels, as in the case of the CBL-B inhibitor.

"Nurix’s CTM and DELigaseTM platform technologies have led to the discovery of novel BTK degraders with the potential to transform the treatment landscape for hematological indications," said Michael Rome, Ph.D., partner at Foresite Capital. "Their differentiated platform and novel approach have resulted in strategic corporate partnerships and we believe position the company as a leader in the emerging protein modulation field."

In addition to advancing its wholly owned pipeline, Nurix recently formed two new strategic collaborations with Sanofi and Gilead to develop novel protein degradation therapies in multiple therapeutic areas. Together, these collaborations provided Nurix with $100 million in upfront payments and the potential for over $4.5 billion in milestones with additional royalties and certain co-development options.

On March 12, 2020 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported that the Company will release its fourth quarter and full-year 2019 financial results on Thursday, March 19, 2020, after the close of US markets. Management will host a conference call on the same day at 4:30 p.m. ET (Press release, Curis, MAR 12, 2020, View Source [SID1234555460]).

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To access the live conference call, please dial (888) 346-6389 from the United States or (412) 317-5252 from other locations, shortly before 4:30 p.m. ET. The conference call can also be accessed on the Curis website at www.curis.com in the ‘Investors’ section. A replay of the financial results conference call will be available on the Curis website shortly after completion of the call.

On March 12, 2020 Idera Pharmaceuticals, Inc. ("Idera") (NASDAQ: IDRA) reported on March 11, 2020 its financial and operational results for the fourth quarter and year ended December 31, 2019 (Press release, Idera Pharmaceuticals, MAR 12, 2020, View Source [SID1234555459]).

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"In 2019, we made great progress with ILLUMINATE-301, our registrational trial of tilsotolimod in combination with ipilimumab in anti-PD-1 refractory patients with advanced melanoma. This was the result of outstanding focus and dedication across our organization and enabled us to finish enrollment in the first quarter of 2020, earlier than anticipated. It also positions us to have initial data from the trial early next year," stated Vincent Milano, Idera’s Chief Executive Officer. "2019 was also notable for our progress in taking tilsotolimod beyond melanoma through both our collaboration in squamous cell carcinoma of the head and neck and through our ILLUMINATE-206 trial, which we initiated in the microsatellite stable colorectal cancer cohort."

ILLUMINATE (tilsotolimod) Clinical Development Update

ILLUMINATE-301: Randomized phase 3 trial of tilsotolimod in combination with ipilimumab versus ipilimumab alone in patients with anti-PD-1 refractory advanced melanoma:

·Primary endpoint family of ORR per RECIST v1.1 and overall survival (OS);
·Trial initiated in March 2018;
·Enrollment completed in March 2020; and
·ORR and other preliminary data expected in Q1 2021.

ILLUMINATE-206: Phase 2, open-label, multicohort, multicenter study to test the safety and effectiveness of tilsotolimod in combination with ipilimumab and nivolumab for the treatment of solid tumors:

·Trial initiated in September 2019 with the microsatellite stable colorectal cancer (MSS-CRC) cohort;

·Initial safety run-in cohort of 10 patients with MSS-CRC fully enrolled; and
·Preliminary data from this cohort expected in Q2 2020.

ILLUMINATE-204: Phase 1/2 trial of tilsotolimod in combination with ipilimumab or pembrolizumab in patients with anti-PD-1 refractory advanced melanoma:

·Enrollment completed in February 2019 at tilsotolimod 8 mg with ipilimumab; and
·Final top-line results from the ILLUMINATE-204 trial, to include ORR, median OS, duration of response (DOR), and safety, are expected to be released in Q2 2020.

Corporate Updates

Since September 30, 2019, the following corporate updates were announced:

·Received new U.S. Patent for tilsotolimod, providing exclusivity through September 2037 when tilsotolimod is used with certain checkpoint inhibitors.
·Entered into private placement financing of up to $97.7 million, with $10.1 million received in December 2019 from initial proceeds and option fees.
·Dr. Jonathan Yingling departed Idera as its Chief Scientific Officer effective January 31, 2020.

Financial Results

Fourth Quarter Results

Revenue in the fourth quarter of 2019 and 2018 was nominal. Research and development expenses for the three months ended December 31, 2019 totaled $8.4 million compared to $8.9 million for the same period in 2018. General and administrative expense for the three months ended December 31, 2019, totaled $3.4 million compared to $3.6 million for the same period in 2018.

Additionally, during the fourth quarter of 2019 the Company recorded $0.6 million and $11.0 million of non-cash warrant revaluation expense and non-cash future tranche right revaluation expense, respectively. These expenses were related to a private placement transaction consummated pursuant to a Securities Purchase Agreement, dated as of December 23, 2019, by and between us and certain institutional investors, whereby the Company sold shares of Series B1 convertible preferred stock and warrants to purchase common stock for $3.9 million and received an upfront option fee of approximately $6.2 million related to certain rights provided to such institutional investors to purchase shares of Series B2, Series B3, and Series B4 convertible preferred stock and warrants to purchase common stock in future tranches, subject to shareholder approval to increase authorized shares. The 2019 period also included non-cash deemed dividends of approximately $28.0 million, increasing net loss attributable to common stockholders, related to the transaction.

As a result of the factors above, net loss applicable to common stockholders for the three months ended December 31, 2019, was $51.3 million, or $1.76 per basic and diluted share, compared to net loss applicable to common stockholders of $12.2 million, or $0.45 per basic and diluted share, for the same period in 2018. Net loss applicable to common stockholders excluding non-cash expense of approximately $11.6 million and deemed dividends of approximately $28.0 million related to the Securities Purchase Agreement for the three months ended December 31, 2019 was $11.7 million, or $0.40 per basic and diluted share.

Full Year Results

Revenue for the year ended December 31, 2019, was $1.5 million compared to revenue of $0.7 million for the same period in 2018. Research and development expenses for the year ended December 31, 2019, totaled $34.9 million compared to $41.8 million for the same period in 2018. General and administrative expenses for the year ended December 31, 2019, totaled $12.5 million compared to $15.4 million for the same period in 2018. Merger-related costs, net for the year ended December 31, 2018, totaled $1.2 million. No such costs were incurred during 2019. Restructuring costs for the year ended December 31, 2019, totaled $0.2 million compared to $3.1 million for the same period in 2018 and related to our decision in July 2018 to wind-down our discovery operations.

The 2019 period also included $0.6 million and $11.0 million of non-cash warrant revaluation expense and non-cash future tranche right revaluation expense, respectively, as well as non-cash deemed dividends of approximately $28.0 million, increasing net loss attributable to common stockholders, as further discussed above under fourth quarter results.

As a result of the factors above, net loss applicable to common stockholders for the year ended December 31, 2019, was $84.6 million or $2.96 per basic and diluted share, compared to net loss applicable to common stockholders of $59.9 million, or $2.25 per basic and diluted share, for the same period in 2018. Net loss applicable to common stockholders excluding non-cash expense of approximately $11.6 million and deemed dividends of approximately $28.0 million related to the Securities Purchase Agreement for the three months ended December 31, 2019 was $45.0 million, or $1.57 per basic and diluted share.

As of December 31, 2019, our cash, cash equivalents, and short-term investments totaled $42.8 million, which includes a $6.2 million contingently refundable option fee received in connection with the December 2019 private placement transaction, discussed above. We currently anticipate that, based on our current operating plan, our current cash, cash equivalents, and short-term investments, excluding the $6.2 million contingently refundable option fee, will fund our operations into the first quarter of 2021.

On March 12, 2020 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, provided a corporate update and reported financial results for the fourth quarter and full year ended December 31, 2019 (Press release, X4 Pharmaceuticals, MAR 12, 2020, View Source [SID1234555458]).

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"2019 was a remarkable year for X4, with significant achievements across the entire organization," said Paula Ragan, Ph.D., President and Chief Executive Officer of the Company. "We made important progress advancing our lead therapeutic candidate mavorixafor into pivotal Phase 3 development for patients with WHIM syndrome, while also initiating two proof-of-concept clinical trials in SCN and Waldenström’s, and strengthening both our leadership team and Board of Directors. We’re now well positioned for the years ahead as we focus on near-term clinical trial execution and prepare for key value creation events in our quest to bring new transformative therapies to patients with rare diseases."

Key 2019 Program Achievements and Upcoming Milestones

•Initiated Pivotal Phase 3 Clinical Trial of Mavorixafor for the Treatment of WHIM Syndrome – June 2019: The 4WHIM trial is a pivotal Phase 3 global clinical trial of mavorixafor for the treatment of WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease.
◦Top-line data from the trial are expected in the second half of 2021.
◦Company to hold Analyst Day, which will also be webcast, on April 7, 2020 to discuss strategic focus on WHIM.
◦Phase 2 open-label extension study data update expected in mid-2020.
•Received Orphan Drug Designation from the European Commission for Mavorixafor for the Treatment of WHIM Syndrome – July 2019
◦Received Scientific Advice from the European Medicines Agency to align the Phase 3 registration trial globally.
•Announced Positive Data from Phase 2a Trial of Mavorixafor in Clear Cell Renal Cell Carcinoma (ccRCC) Patients – September 2019: Data presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) demonstrated that mavorixafor, in combination with axitinib yielded clinically meaningful improvements in median progression-free survival (mPFS) in a heavily pretreated advanced ccRCC patient population, and was generally well tolerated.

•Initiated Phase 1b Clinical Trial of Mavorixafor in Patients with Severe Congenital Neutropenia (SCN) – November 2019: The Phase 1b trial is a 14-day, proof-of-concept trial designed to assess the safety and tolerability of daily, oral mavorixafor in up to 45 patients with SCN and other selected congenital neutropenia disorders. The trial will evaluate the neutrophil response and genetic profiles in this patient population as an independent agent or in combination with granulocyte-colony stimulating factor (G-CSF).
◦Initial data from this trial are expected in the second half of 2020.
•Received Breakthrough Therapy Designation from U.S. FDA for Mavorixafor for the Treatment of WHIM Syndrome – November 2019: Highlighting the severity of the disease and the relevance of mavorixafor’s Phase 2 clinical trial data to support the drug’s role as a potential disease-modifying therapeutic option to this underserved patient population, Breakthrough Therapy Designation could expedite the development and regulatory review of mavorixafor.
•Initiated Phase 1b Clinical Trial of Mavorixafor in Combination with Ibrutinib in Patients with Waldenström’s Macroglobulinemia – December 2019: The Phase 1b multi-center, open-label, dose-escalation clinical trial is designed to assess the safety and tolerability of mavorixafor in combination with ibrutinib as well as to obtain certain efficacy signals in patients with Waldenström’s, a rare form of non-Hodgkin’s lymphoma, who have acquired a "gain of function" mutation in CXCR4 in addition to the MYD88 mutation, which is a hallmark of Waldenström’s.
◦Initial data from this trial are expected in the second half of 2020.
•Granted New Composition of Matter Patent by U.S. PTO for Mavorixafor – February 2020: Patent expected to provide exclusivity through 2038.

Key 2019 Corporate Highlights

•Shares of X4 Pharmaceuticals Began Trading on the Nasdaq Capital Market Under the Symbol "XFOR" — March 2019
•Completed Two Public Stock Offerings that Raised Gross Proceeds of $150.8 Million: The first offering was completed in April and the second offering was completed in November.
•Announced Multiple Development and Collaboration Agreements:
◦Announced Collaboration with The Leukemia & Lymphoma Society (LLS) – May 2019: Mavorixafor selected for LLS’ Therapy Acceleration Program (TAP), a strategic initiative creating an alliance to develop mavorixafor for patients with Waldenström’s macroglobulinemia.
◦Announced Partnership with Invitae to Provide No-Cost Genetic Testing to Patients – June 2019: The collaborative PATH4WARD program provides greater access to faster and earlier diagnosis for individuals who may carry genetic mutations known to be associated with WHIM syndrome and SCN.
◦Entered into Oncology Development and Commercialization Agreement with Abbisko for Mavorixafor in Greater China – July 2019: Provided Abbisko Therapeutics with the exclusive rights in China, Taiwan, Hong Kong, and Macau to develop and commercialize mavorixafor in combination with checkpoint inhibitors or other agents in solid tumor oncology indications.
•Strengthened Leadership Team and Board of Directors Throughout the Year: Appointed E. Lynne Kelley, M.D., as Chief Medical Officer and Murray W. Stewart, M.D., to Board of Directors in April 2019; appointed Renato Skerlj, Ph.D., as Senior Vice President of Research and Development and William E. Aliski to Board of Directors in September 2019; appointed Derek Meisner, J.D., as General Counsel in November 2019.

Financial Results

•Cash, Cash Equivalents & Restricted Cash: X4 had $128.1 million in cash, cash equivalents and restricted cash, as of December 31, 2019. We expect that our cash and cash equivalents will fund our operations into early 2022.
•Research and Development Expenses were $7.1 million for the fourth quarter of 2019, and $30.2 million for the year ended December 31, 2019, as compared to $4.7 million and $20.3 million for the comparable periods in 2018, respectively.
•General and Administrative Expenses were $3.9 million for the fourth quarter of 2019, and $17.6 million for the year ended December 31, 2019, as compared to $3.4 million and $8.7 million for the comparable periods in 2018, respectively.
•Net Loss: X4 reported a net loss of $10.8 million for the fourth quarter of 2019, and a net loss of $52.8 million for the year ended December 31, 2019, as compared to a net loss of $11.3 million and a net loss of $33.3 million for the comparable periods in 2018, respectively. Net loss of $52.8 million for the year ended December 31, 2019 includes $3.9 million of non-cash losses related to the sale of in-process research and development intangible assets.

Conference Call and Webcast
The Company will host a webcast and conference call to discuss its fourth quarter and full year 2019 results and provide an update on recent corporate activities today at 8:30 a.m. Eastern Time. The webcast will be accessible under "Events & Presentations" in the Investors page of the Company’s website at www.x4pharma.com. Individuals can participate in the conference call by dialing (866) 721-7655 (domestic) or (409) 216-0009 (international), followed by the conference ID: 3816258.

About Mavorixafor
Mavorixafor is an investigational, oral, targeted therapy that antagonizes the chemokine receptor CXCR4 via allosteric inhibition. The CXCR4 receptor plays a key role in enabling the trafficking of immune cells and effective immunosurveillance. Over-stimulation of CXCR4 results in various immune-system deficiencies. X4 Pharmaceuticals is currently investigating mavorixafor as a once-daily treatment in a global Phase 3 pivotal trial in patients with WHIM syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. The FDA has granted Breakthrough Therapy Designation to mavorixafor for the treatment of WHIM syndrome. Mavorixafor is being developed in two proof-of-concept Phase 1b clinical trials – as monotherapy in patients with Severe Congenital Neutropenia (SCN), a group of rare blood disorders characterized by abnormally low levels of white blood cells, and in combination with ibrutinib in patients with Waldenström’s macroglobulinemia, a form of non-Hodgkin’s lymphoma, who have acquired mutations in the CXCR4 receptor.