On August 3, 2021 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage company developing highly differentiated agents that target the underlying mechanisms of cancer, reported financial results for the three months ended June 30, 2021 and provided a corporate update (Press release, Aptose Biosciences, AUG 3, 2021, View Source [SID1234585668]).

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The net loss for the quarter ended June 30, 2021 was $13.5 million ($0.15 per share) compared with $15.8 million ($0.21 per share) for the quarter ended June 30, 2020. The net loss for the six months ended June 30, 2021 was $29.7 million ($0.33 per share), compared with $27.3 million ($0.36 per share) for the six months ended June 30, 2020. Total cash and cash equivalents and investments as of June 30, 2021 were $103.3 million. Based on current operations, Aptose expects that cash on hand and available capital provide the Company with sufficient resources to fund all planned Company operations including research and development into the first half of 2023.

"The progress we are seeing in our Phase 1 a/b clinical trials with luxeptinib in very challenging patient populations with AML and B-cell malignancies is especially encouraging," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. "As we have begun to observe dose-dependent anti-tumor activity, we plan to continue dose escalation for extended duration to treat as many patients as possible on these higher dose levels and tackle these increasingly refractory patient populations."

Key Corporate Highlights

Luxeptinib Phase 1 a/b Clinical Study in AML – Luxeptinib is currently being evaluated in a Phase 1 a/b dose escalation clinical study in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML). In concurrence with participation at the EHA (Free EHA Whitepaper)2021 Virtual Congress (EHA) (Free EHA Whitepaper) in June, Aptose reported encouraging anti-leukemic activity in multiple patients, including a durable MRD-negative complete response in a FLT3-ITD AML patient who had relapsed after two allogeneic stem cell transplants, multiple lines of chemotherapy, and prior FLT3 inhibitor therapy. Aptose has completed the 450 and 600 mg dose levels, with some patients remaining on treatment at those levels, and has fully enrolled the 750 mg dose cohort. More information is available at www.clinicaltrials.gov (NCT04477291).

Luxeptinib Phase 1 a/b Clinical Study in B-cell Malignancies – In parallel with the trial in AML patients, luxeptinib is being evaluated in a Phase 1 a/b dose escalation clinical study in patients with B-cell malignancies, including chronic lymphocytic leukemia (CLL) and non-Hodgkin’s lymphomas (NHL), who have failed or are intolerant to two or more lines of established therapies, including drugs such as ibrutinib, rituximab and venetoclax or for whom no other treatment options are available. Thus far, luxeptinib has been well-tolerated in patients treated at 150mg, 300mg, 450mg and 600mg BID over multiple cycles. Of the evaluable patients at these dose levels, two-thirds of them experienced various reductions of lesion size or IgM measurements compared to baseline, demonstrating measurable anti-tumor activity. Patients now are being treated at the fifth dose level of 750mg BID, which is fully enrolled. More information is available at www.clinicaltrials.gov (NCT03893682).

APTO-253 Phase 1 a/b Clinical Study in AML and MDS – As a direct inhibitor of MYC transcription, APTO-253 represents a novel approach for targeting MYC, an oncogene estimated to contribute to the majority of all human cancers, including hematologic malignancies. Given this mode of action, Aptose is expanding the target patient population to include those with relapsed or refractory B-cell malignancies characterized by chromosomal translocations involving the MYC locus. These include Burkitt’s lymphoma, double- or triple-hit diffuse large B-cell lymphomas, and other aggressive lymphomas with MYC translocations.

In the ongoing Phase 1a/b study in patients with relapsed or refractory AML and high-risk myelodysplastic syndrome (MDS), APTO-253 has been well-tolerated in five dose cohorts ranging from 20 – 150 mg/m2 over multiple cycles. Thus far, there is no evidence of drug-related adverse events, including no myelosuppression. Aptose currently is treating patients in the sixth dose cohort at 210 mg/m2 and several subsequent dose escalations are anticipated. More information is available at www.clinicaltrials.gov (NCT02267863).
RESULTS OF OPERATIONS

A summary of the results of operations for the three-month and six-month periods ended June 30, 2021 and 2020 is presented below:

The net loss for the three-month period ended June 30, 2021 decreased by $2.3 million to $13.5 million as compared with $15.8 million for the comparable period in 2020. The net loss for the six-month period ended June 30, 2021 increased by $2.4 million to $29.7 million as compared with $27.3 million for the comparable period in 2020. Components of the net loss are presented below:

Research and Development
The research and development expenses for the three-month and six-month periods ended June 30, 2021 and 2020 were as follows:

Research and development expenses increased by $3.0 million to $9.8 million for the three-month period ended June 30, 2021, as compared with $6.9 million for the comparative period in 2020. Changes to the components of our research and development expenses presented in the table above are primarily as a result of the following events:

Program costs for luxeptinib increased by approximately $1.97 million, mostly as a result of higher manufacturing costs, including costs to scale up manufacturing and research costs associated with optimizing the formulation and higher costs related to the luxeptinib AML trial, for which we received an IND allowance in June 2020.

Program costs for APTO-253 increased by approximately $263 thousand, mostly as a result of higher manufacturing costs.

Personnel-related expenses increased by $668 thousand, mostly related to new positions hired to support our clinical trials and manufacturing activities.

Stock-based compensation increased by approximately $65 thousand in the three months ended June 30, 2021, compared with the three months ended June 30, 2020, mostly related to higher total compensation expense in the current period on options issued in the first half of 2021.
Research and development expenses increased by $5.3 million to $18.1 million for the six-month period ended June 30, 2021, as compared with $12.8 million for the comparative period in 2020. Changes to the components of our research and development expenses presented in the table above are primarily as a result of the following events:

Program costs for luxeptinib increased by approximately $3.0 million, mostly as a result of higher manufacturing costs, including costs to scale up manufacturing and research costs associated with optimizing the formulation and higher costs related to the luxeptinib AML trial, for which we received an IND allowance in June 2020.

Program costs for APTO-253 increased by approximately $474 thousand, mostly as a result of higher manufacturing costs.

Personnel-related expenses increased by $1.2 million, mostly related to new positions hired to support our clinical trials and manufacturing activities.

Stock-based compensation increased by approximately $643 thousand in the six months ended June 30, 2021, compared with the six months ended June 30, 2020, mostly related to higher total compensation expense in the current period on options issued in the first half of 2021.
General and Administrative
The general and administrative expenses for the three-month and six-month periods ended June 30, 2021 and 2020 were as follows:

General and administrative expenses for the three-month period ended June 30, 2021 were $3.7 million, as compared with $9.0 million for the comparative period in 2020, a decrease of approximately $5.4 million. The decrease was primarily as a result of the following:

General and administrative expenses, other than stock-based compensation and depreciation of equipment, increased by approximately $242 thousand in the three months ended June 30, 2020, primarily as a result of higher insurance costs, professional costs and investor relations advisory costs offset by lower personnel related costs and lower office administrative costs.

Stock-based compensation decreased by approximately $5.6 million in the three months ended June 30, 2021 as compared with the three months ended June 30, 2020, mostly as a result of a lower number of options granted in the six month period ended June 30, 2021 as compared with the six month period ended June 30, 2020, that those options granted in the current period had a lower grant date fair value, and that in the comparative period the Company had issued restricted share units (RSUs) that had fully vested by the end of the comparative period. No RSUs were granted in the current period.
General and administrative expenses for the six-month period ended June 30, 2021 were $11.7 million as compared with $14.9 million for the comparative period, a decrease of approximately $3.2 million. The decrease was primarily a result of the following:

General and administrative expenses, other than share-based compensation and depreciation of equipment, increased by approximately $702 thousand in the six months ended June 30, 2021, primarily as a result of higher insurance costs, higher professional costs, higher investor relations advisory costs offset by lower office administrative costs and lower travel expenses.

Stock-based compensation decreased by approximately $3.9 million in the six months ended June 30, 2021, compared with the six months ended June 30, 2020. Stock-based compensation decreased by approximately $5.6 million mostly as a result of a lower number of options granted in the six-month period ended June 30, 2021 as compared with the six month period ended June 30, 2020, that those options granted in the current period had a lower grant date fair value, and that in the comparative period the Company had issued RSUs that had fully vested by the end of the comparative period. This decrease was offset by increased compensation of approximately $1.7 million, mostly related to the modification of option agreements of one officer as part of a separation and release agreement. Vested options of 1,679,169 with exercise prices ranging from $1.03 to $7.44 were allowed to continue to be exercisable for an additional twelve-month period, and also 504,833 options that would have expired unvested, were allowed to continue to vest for a twelve-month period. As there was no service requirement, the Company recorded $945 thousand and $663 thousand additional compensation in the current period related to these modifications for the vested and unvested options, respectively.
Conference Call and Webcast

Aptose will host a conference call to discuss results for the quarter ended June 30, 2021 today, Tuesday, August 3, 2021 at 5:00 PM ET. Participants can access the conference call by dialing 1-844-882-7834 (North American toll-free number) and 1-574-990-9707 (international/toll number) and using conference ID # 7272387. The conference call can be accessed here and will also be available through a link on the Investor Relations section of Aptose’s website at View Source An archived version of the webcast along with a transcript will be available on the Company’s website for 30 days. An audio replay of the webcast will be available approximately two hours after the conclusion of the call for seven days by dialing 1-855-859-2056 (toll free number) and 1-404-537-3406 (international/toll number), using the conference ID # 7272387.

The press release, the financial statements and the management’s discussion and analysis for the quarter ended June 30, 2021 will be available on SEDAR at www.sedar.com and EDGAR at www.sec.gov/edgar.shtml.

On August 3, 2021 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases of the immune system resulting from dysfunction of the CXCR4 pathway, reported financial results for the second quarter and six months ended June 30, 2021 (Press release, X4 Pharmaceuticals, AUG 3, 2021, View Source [SID1234585702]). The company also announced key enrollment milestone achievements for its lead product candidate, mavorixafor, a novel, oral small molecule currently being evaluated in a Phase 3 clinical trial (4WHIM) for patients with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome and in two Phase 1b clinical trials for patients with Waldenström’s macroglobulinemia and Severe Congenital Neutropenia (SCN) and chronic neutropenia disorders, respectively.

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"We are very encouraged by the strong interest from both the physicians and patients participating in our mavorixafor clinical programs," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "With enrollment nearly complete in the 52-week placebo-controlled Phase 3 trial in WHIM syndrome, our initial indication for mavorixafor, and continued supportive data coming from our ongoing open-label Phase 2 trial in WHIM, we are starting to ramp up our pre-commercial planning, as we now look forward to Phase 3 top-line data in the fourth quarter of 2022. In addition, we are making strong progress in our ongoing Phase 1b clinical trial in Waldenström’s and intend to announce preliminary high-dose data along with certain response measures from this trial in the fourth quarter of 2021 that we believe will build on the low- and mid-dose data we presented at EHA (Free EHA Whitepaper) this past June. Lastly, as enrollment continues in our ongoing Phase 1b trial in SCN, from which we expect the first data in the fourth quarter of 2021, we are exploring the potential broader use of mavorixafor across the larger chronic neutropenia landscape. We look forward to reporting on our continued progress with mavorixafor, presenting additional clinical, pre-clinical and prevalence data, and providing a variety of company updates later this year."

Mavorixafor Clinical Trial Updates

Phase 3 Trial in WHIM Syndrome (4WHIM):
The company reported that it has surpassed the 18-patient minimum enrollment needed for primary endpoint analyses, determination of clinical benefit, and U.S. regulatory filing (if supported by the Phase 3 data), having enrolled 23 patients to date in its ongoing Phase 3 trial in WHIM syndrome. Enrollment will be completed in the third quarter of 2021, allowing the remaining identified patients to complete screening and potential enrollment. Top-line data are expected to be announced in the fourth quarter of 2022.
The 4WHIM Phase 3 trial is a global, randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the safety and efficacy of mavorixafor in 18-28 genetically confirmed WHIM patients over the course of a 52-week study with open-label extension. The primary endpoint for the trial will compare the level of circulating neutrophils relative to a clinically meaningful threshold in response to treatment with mavorixafor versus placebo over 24-hour periods. Secondary endpoints will assess infection rates, wart burden, markers of immune system function, and quality of life, among others.
The company is planning to announce new data from the open-label extension of its ongoing Phase 2 clinical trial, as well as an update on patient prevalence, and new data from research into the genetics of WHIM that will detail new insights into genotype/phenotype correlations and the identification of a new WHIM variant.
Phase 1b Trial in Waldenström’s Macroglobulinemia (WM):
The company also announced today that it has surpassed enrollment of the minimum 12 patients (Cohorts A and B) required to determine optimal dosing of mavorixafor in combination with ibrutinib in the ongoing Phase 1b clinical trial. The company is continuing enrollment in the optional Cohort C (up to an additional 6 patients).
This ongoing Phase 1b, open-label, multicenter, single-arm study examines intra-patient dose escalation, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of mavorixafor (200 mg, 400 mg, and 600 mg) in combination with ibrutinib (420 mg), both delivered orally once daily, in patients with Waldenström’s macroglobulinemia and confirmed MYD88 and CXCR4 mutations. Patients are followed for adverse events and change from baseline in IgM and hemoglobin, PK, and PD (including peripheral white blood cell counts), in addition to clinical response.
The company remains on track to announce additional dosing, efficacy, safety, and clinical response data from the ongoing trial in the fourth quarter of 2021, including patient data at the highest planned mavorixafor dose of 600 mg.
Phase 1b Trial in Severe Congenital Neutropenia (SCN):
Enrollment continues in this clinical trial, with initial data anticipated in the fourth quarter of 2021. The company expects that the initial data from this trial, in combination with additional data emerging from prior and ongoing studies that show chronic, sustained white blood cell increases across a number of patient groups treated with mavorixafor, will support the company’s exploration of opportunities for mavorixafor use across larger chronic neutropenic populations and more broadly in cellular immunodeficiencies.
Second Quarter Highlights and Upcoming Events

EHA 2021: In June, the company announced the presentation of positive data from its ongoing Phase 1b clinical trial of mavorixafor in combination with ibrutinib in Waldenström’s macroglobulinemia. Data showed robust decreases in serum IgM at low- and mid-doses of mavorixafor, suggesting best-in-class potential for this combination treatment; meaningful increases in hemoglobin levels suggested reduction in cancer burden in the bone marrow; and at 6 months, patients achieved median IgM level reductions of 60%-75%, with one patient achieving normal IgM; two of four patients (50%) had >50% reduction in serum IgM from baseline. The poster is available here; slides from the company’s associated analyst event are available here.
X4 management will be participating in the following upcoming investor conferences:
Canaccord Genuity Growth Conference – taking place virtually August 10-12, 2021
Citi Annual BioPharma Conference– taking place virtually September 8-10, 2021
Oppenheimer Fall Healthcare Life Science & Med Tech Summit – taking place virtually September 20-23, 2021
Cantor Fitzgerald Healthcare Conference – taking place virtually September 27-30, 2021
Second Quarter 2021 Financial Results

Cash, Cash Equivalents & Restricted Cash: X4 had $96.5 million in cash, cash equivalents, and restricted cash as of June 30, 2021. The company expects that its cash and cash equivalents will fund company operations into the fourth quarter of 2022.
Research and Development Expenses were $13.2 million for the second quarter ended June 30, 2021, as compared to $9.3 million for the comparable period in 2020. R&D expenses include $0.8 million and $0.5 million of certain non-cash expenses for the quarters ended June 30, 2021 and 2020, respectively.
General and Administrative Expenses were $5.8 million for the second quarter ended June 30, 2021, as compared to $5.3 million for the comparable period in 2020. G&A expenses include $1.0 million and $0.7 million of certain non-cash expenses for the quarters ended June 30, 2021 and 2020, respectively.
Net Loss: X4 reported a net loss of $19.6 million for the quarter ended June 30, 2021, as compared to a net loss of $15.1 million for the comparable period in 2020. Net losses include $1.8 million and $1.2 million of certain non-cash expenses for the quarters ended June 30, 2021 and 2020, respectively.
Conference Call and Webcast
X4 will host a conference call and webcast today at 8:30 a.m. ET to discuss these financial results and business highlights. The conference call can be accessed by dialing (866) 721-7655 from the United States or (409) 216-0009 internationally, followed by the conference ID: 2236266. 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 August 3, 2021 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported that it will release second quarter 2021 financial results and recent highlights before the market opens on Thursday, August 12, 2021 (Press release, Hookipa Pharma, AUG 3, 2021, View Source [SID1234585575]).

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The Company will not be conducting a conference call in conjunction with this earnings release.

On August 3, 2021 MannKind Corporation (Nasdaq: MNKD), a company focused on the development and commercialization of inhaled therapeutic products for patients with endocrine and orphan lung diseases, reported that its Chief Executive Officer, Michael Castagna, PharmD, will participate in a Fireside Chat in the BTIG Virtual Biotechnology Conference 2021 on Tuesday, August 10, 2021 at 12:30 PM (ET) (Press release, Mannkind, AUG 3, 2021, View Source [SID1234585597]).

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BTIG hosted events are intended for prospective and existing BTIG clients only. To listen to the live event, please contact your BTIG representative with interest.

On August 3, 2021 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported that it will report financial results and highlights for the quarter ended June 30, 2021 on Tuesday, August 10, 2021, after the U.S. financial markets close.

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The Company will host a conference call and live audio webcast on Tuesday, August 10, 2021 at 5:00 p.m. Eastern Daylight Time to report its second quarter 2021 financial results and provide a corporate update. To access the call, please dial (877) 257-8599 (domestic) or (970) 315-0459 (international) and refer to conference ID 2825907. To access the telephone replay of the call, dial (855) 859-2056 (domestic) or (404) 537-3406 (international), passcode ID 2825907. The webcast and telephone replay will be archived on the Company’s website at www.regulusrx.com following the call.

Wedbush PacGrow Healthcare Conference

Additionally, the Company announced that Jay Hagan, President and Chief Executive Officer, will participate in a panel at the 2021 Wedbush PacGrow Healthcare Conference on Wednesday, August 11, 2021, at 4:05 p.m. E.T.

A live webcast of the panel will be available on the investor relations section of the Company’s website at www.regulusrx.com. A replay of the webcast will be archived for 30 days following the presentation date.