On August 4, 2022 Arbutus Biopharma Corporation (Nasdaq: ABUS), a clinical-stage biopharmaceutical company leveraging its extensive virology expertise to develop novel therapeutics that target specific viral diseases, reported its second quarter 2022 financial results and provides corporate updates (Press release, Arbutus Biopharma, AUG 4, 2022, View Source [SID1234617474]).

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"This quarter we presented data at the EASL ILC which highlighted the advancements we have made in developing our lead RNAi therapeutic AB-729, capsid inhibitor AB-836, and PD-L1 inhibitor AB-101 to potentially provide a functional cure for patients with chronic hepatitis B virus (cHBV). For AB-729 we presented exciting data showing achievement of virologic control in five cHBV patients after discontinuing treatment with AB-729 and nucleos(t)ide analog (NA)-therapy. We continue to follow these patients, as well as others that have elected to discontinue treatment, and look forward to providing additional follow-up data later this year," said William Collier, Arbutus’ President and Chief Executive Officer. "In addition, we were impressed with the competitive and robust antiviral activity seen with AB-836 and we plan to initiate a Phase 1 study in healthy volunteers to better characterize the unexpected ALT increases seen in some patients. We also look forward to completing IND-enabling studies this year with AB-161, our RNAi destabilizer, and AB-101."

Mr. Collier continued, "We are continuing to advance our development efforts with our nsp5 main protease (Mpro) and nsp12 viral polymerase programs for SARS-CoV-2 and future coronavirus outbreaks. Finally, our financial position is strong with a projected cash runway into the second quarter of 2024."

Pipeline Updates:
AB-729 (RNAi Therapeutic)

Arbutus presented new on-treatment data as well as long-term off-treatment data for cHBV patients in the AB-729-001 clinical trial at the 2022 European Association for the Study of the Liver (EASL) International Liver Congress (ILC) on June 25th. Key findings included:
AB-729 provided robust and comparable HBsAg declines regardless of dose, dosing interval or baseline characteristics
50% of patients (16 out of 32) maintained HBsAg levels below 100 IU/mL 24 weeks after their last dose of AB-729
There was no evidence of virologic or clinical relapse in 8-24 weeks of follow-up in the first five patients who discontinued both AB-729 and NA therapy
AB-729 continues to restore HBV-specific T-cells and decrease exhausted T-cells
AB-729 remains generally safe and well-tolerated after completing dosing in 41 patients
Dosed first patient in the AB-729-202 Phase 2a clinical trial evaluating AB-729, in combination with VTP-300, Vaccitech plc’s (Vaccitech) therapeutic vaccine and nucleos(t)ide analogue therapy (NA), in cHBV patients.
Enrollment is continuing in the Phase 2a clinical trial evaluating AB-729 in combination with ongoing NA therapy and short courses of Peg-IFNα-2a (AB-729-201) in cHBV patients. The Company is on-track to report initial data in the second half of 2022.
Dosing is continuing in the Phase 2a clinical trial evaluating AB-729 in combination with vebicorvir (VBR), Assembly Biosciences, Inc.’s HBV core inhibitor (capsid inhibitor), and an NA in cHBV patients. Preliminary data are expected in the second half of 2022.
AB-836 (Oral Capsid Inhibitor)

In June 2022 Arbutus presented data at EASL from its AB-836-001 Phase 1a/1b clinical trial in which the Company is evaluating the safety and tolerability of multiple doses of AB-836 in patients with cHBV infection. AB-836 dosed at 100mg or 200mg once daily for 28 days achieved mean declines in HBV DNA of 3.04 and 3.55 log10 IU/mL, respectively. Two HBeAg+ patients in the 100mg dose cohort had transient Grade 3 ALT elevations that resolved with continued dosing and were not considered treatment emergent adverse events (TEAEs). Two patients in the 200mg cohort had Grade 3 and Grade 4 ALT elevations on the last day of dosing (Day 28) that returned to baseline during follow up which were reported as TEAEs. Based on these ALT findings, the Company plans to conduct an additional Phase 1 trial in healthy volunteers to determine whether or not these ALT elevations are beneficial or could be the result of liver toxicity. The Company will provide an update with respect to the status and timing of this clinical trial in the second half of 2022.
AB-101 (Oral PD-L1 Inhibitor)

In June 2022 Arbutus presented data at EASL showing that once daily oral administration of AB-101 resulted in T-cell activation in a preclinical model. In addition, AB-101 activates and reinvigorates HBV-specific T-cells in vitro. The company is on-track to complete IND-enabling studies for AB-101 in the second half of 2022.
AB-161 (Oral RNA Destabilizer)

Arbutus is conducting IND-enabling studies for AB-161, its oral RNA destabilizer. The Company intends to complete IND-enabling studies for AB-161 in the second half of 2022.
COVID-19 and Pan-Coronavirus Programs

We see an opportunity to pursue a combination therapy consisting of compounds that inhibit the SARS-CoV-2 nsp5 main protease and nsp12 viral polymerase, to achieve better patient treatment outcomes and use in prophylactic settings.
Arbutus plans to nominate a lead candidate that inhibits the SARS-CoV-2 nsp5 main protease (Mpro) in the second half of 2022 and then advance that compound into IND-enabling studies.
The Company is continuing lead optimization activities for an nsp12 viral polymerase candidate.
PD-L1 in Oncology

Preclinical data was selected for publication at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2022 showing that Arbutus’ oral small-molecule PD-L1 inhibitors in development, which possess a novel mechanism of action, have the ability to mediate T-cell activation in primary human immune cells. The anti-tumor efficacy seen in vivo was comparable to anti-PD-L1 antibodies. The data was published in the Journal of Clinical Oncology.
Financial Results

Cash, Cash Equivalents and Investments

As of June 30, 2022, the Company had cash, cash equivalents and investments in marketable securities of $200.6 million, as compared to $191.0 million as of December 31, 2021.

During the six months ended June 30, 2022, the Company received a $40.0 million (net of withholding taxes) upfront payment from Qilu Pharmaceutical Co., Ltd. ("Qilu") related to a technology transfer and license agreement for AB-729 in greater China, $15.0 million of gross proceeds from Qilu’s equity investment in the Company and $0.3 million of net proceeds from the issuance of common shares under Arbutus’s "at-the-market" offering program. These cash inflows were partially offset by $43.7 million of cash used in operations. The Company expects a net cash burn between $90 to $95 million in 2022, not including the $55 million of proceeds received from Qilu, and believes its cash runway will be sufficient to fund operations into the second quarter of 2024.

Revenue

Revenues were $14.2 million for the three months ended June 30, 2022 compared to $2.3 million for the same period in 2021. The increase of $11.9 million was due primarily to $11.0 million of revenue recognition from the Company’s license agreement with Qilu based on employee labor hours expended by the Company during the three months ended June 30, 2022 to perform its manufacturing obligations under the license agreement.

Operating Expenses

Research and development expenses were $22.9 million for the three months ended June 30, 2022, compared to $15.8 million for the same period in 2021. The increase of $7.1 million was due primarily to an increase in expenses related to the Company’s multiple, ongoing AB-729 Phase 2a clinical trials, including its collaborations with Assembly and Vaccitech, and an increase in expenses for its early-stage development programs, including AB-101 and AB-161. General and administrative expenses were $5.2 million for the three months ended June 30, 2022, compared to $4.5 million for the same period in 2021. This increase was due primarily to increases in employee compensation and non-cash stock-based compensation expense.

Net Loss

For the three months ended June 30, 2022, the Company’s net loss attributable to common shares was $14.2 million, or a loss of $0.10 per basic and diluted common share, as compared to a net loss attributable to common shares of $22.7 million, or a loss of $0.23 per basic and diluted common share, for the three months ended June 30, 2021. Net loss attributable to common shares for the three months ended June 30, 2021 included $3.3 million of non-cash expense for the accrual coupon on the Company’s convertible preferred shares, which converted into 22.8 million common shares in October 2021.

Outstanding Shares

As of June 30, 2022, the Company had approximately 148.8 million common shares issued and outstanding, as well as approximately 15.9 million stock options outstanding. Roivant Sciences Ltd. owned approximately 26% of the Company’s outstanding common shares as of June 30, 2022.

COVID-19 Impact

The COVID-19 pandemic has resulted in and will likely continue to result in significant disruptions to businesses. Measures implemented around the world in attempts to slow the spread of COVID-19 have had, and will likely continue to have, a major impact on clinical development, at least in the near-term, including shortages and delays in the supply chain and prohibitions in certain countries on enrolling subjects and patients in new clinical trials. While the Company has been able to progress with its clinical and pre-clinical activities to date, it is not possible to predict if the COVID-19 pandemic will materially impact the Company’s plans and timelines in the future.

Conference Call and Webcast Today

Arbutus will hold a conference call and webcast today, Thursday, August 4, 2022, at 8:45 AM Eastern Time to provide a corporate update. You can access a live webcast of the call through the Investors section of Arbutus’ website at www.arbutusbio.com. Alternatively, you can dial (800) 715-9871 or (646) 307-1963 and reference conference ID: 5109143.

An archived webcast will be available on the Arbutus website after the event. Alternatively, you may access a replay of the conference call by calling (800) 770-2030 or (609) 800-9909, and reference conference ID: 5109143

About AB-729

AB-729 is an RNA interference (RNAi) therapeutic specifically designed to reduce all HBV viral proteins and antigens, including hepatitis B surface antigen, which is thought to be a key prerequisite to enable reawakening of a patient’s immune system to respond to the virus. AB-729 targets hepatocytes using Arbutus’ novel covalently conjugated N-Acetylgalactosamine (GalNAc) delivery technology that enables subcutaneous delivery. Clinical data generated thus far has shown single- and multi-doses of AB-729 to be generally safe and well-tolerated while providing meaningful reductions in hepatitis B surface antigen and hepatitis B DNA. AB-729 is currently in multiple Phase 2a clinical trials.

About AB-836

AB-836 is a next generation oral hepatitis B virus (HBV) capsid inhibitor that interacts with HBV core protein, which in turn is required for viral replication. The current standard-of-care therapy for HBV is primarily nucleos(t)ide analogues that inhibit the viral polymerase and significantly reduce, but do not eliminate viral replication. AB-836 in combination with nucleos(t)ide analogues is designed to completely eliminate viral replication in infected cells by preventing the assembly of functional viral capsids. In addition, AB-836 has been shown to inhibit the replenishment of covalently closed circular DNA (cccDNA), the viral genetic reservoir which the virus needs to replicate itself.

About AB-101

Immune checkpoints such as PD-1/PD-L1 play an important role in the induction and maintenance of immune tolerance and in T-cell activation. We have identified a class of small molecule oral PD-L1 inhibitors that we believe will allow for controlled checkpoint blockade, enable oral dosing, and mitigate systemic safety issues typically seen with checkpoint antibody therapies. Our lead oral PD-L1 inhibitor candidate, AB-101, is currently in IND-enabling studies. We believe AB-101 has the potential to be used in combination with other approved and investigational agents for our mission to achieve a functional cure for HBV chronically infected patients. We are also exploring oncology applications for our internal PD-L1 portfolio.

About HBV

Hepatitis B is a potentially life-threatening liver infection caused by the hepatitis B virus (HBV). HBV can cause chronic infection which leads to a higher risk of death from cirrhosis and liver cancer. Chronic HBV infection represents a significant unmet medical need. The World Health Organization estimates that over 290 million people worldwide suffer from chronic HBV infection, while other estimates indicate that approximately 2.4 million people in the United States suffer from chronic HBV infection. Approximately 820,000 people die every year from complications related to chronic HBV infection despite the availability of effective vaccines and current treatment options.

On August 4, 2022 Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company pioneering precision immune tolerance with its clinically validated ImmTOR platform to develop tolerogenic therapies for autoimmune diseases, unlock the potential of gene therapies and amplify the efficacy of biologic therapies, reported financial results for the second quarter ended June 30, 2022 and provided a business update (Press release, Selecta Biosciences, AUG 4, 2022, View Source [SID1234617473]).

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"We continued to make steady progress in the second quarter of 2022, highlighted by the completion of enrollment in DISSOLVE II, triggering a $10 million milestone payment obligation from Sobi, and the completion of an underwritten equity offering raising gross proceeds of $38.7 million," said Carsten Brunn, Ph.D., president and chief executive officer of Selecta. "Building on this momentum, we enter the second half of 2022 with a focused portfolio of proprietary programs and an expected financial runway into mid-2024. We remain on track clinically, with joint topline data from DISSOLVE I and II anticipated in Q1 2023 and the initiation of a Phase 1 trial of SEL-302, our wholly owned gene therapy in combination with ImmTOR for the treatment of MMA, anticipated in Q4 2022. We continue preclinical development across all three pillars of our pipeline: In partnership with Cyrus Bioscience we are progressing in identifying a proprietary IL-2 to combine with ImmTOR; we have progressed to IND-enabling studies and manufacturing scale-up work for Xork, our proprietary IgG protease as a pre-treatment to enable AAV gene therapies; and our collaborations toward the identification of a next-generation IgA protease for the treatment of IgA Nephropathy continue apace with candidate selection anticipated by year-end. We believe that collectively, these advancements bring us one step closer to our mission of re-imagining immunotherapy for autoimmune disease, unlocking the potential of AAV gene therapy and amplifying the efficacy of biologics."

Recent Program Highlights and Anticipated Upcoming Milestones:

Tolerogenic Therapies for Autoimmune Disease:

ImmTOR with proprietary IL-2 protein agonist (ImmTOR-ILTM): Preclinically, Selecta has observed synergistic activity when ImmTOR is combined with engineered IL-2 molecules that are selective for Tregs. Furthermore, when ImmTOR-IL was co-administered with an antigen of interest, the resulting data suggested that ImmTOR-IL may have profound synergistic effects in expanding antigen-specific Tregs when compared to ImmTOR alone, positioning ImmTOR-IL as a potential first-in-class antigen-specific therapy for the treatment of autoimmune disease.
Selecta is working with its partner, Cyrus Biotechnology, to develop a next generation IL-2 molecule to combine with ImmTOR and anticipates selecting an IL-2 candidate by year end 2022.
Selecta continues internal work on identifying additional target indications in autoimmune disease. Selecta plans to adopt a staged development approach, starting first with diseases driven by a single pathogenic antigen, such as Primary Biliary Cholangitis (PBC), then accelerating the development of across related indications.

Primary biliary cholangitis (PBC): Selecta intends to co-administer ImmTOR-IL with PDC-E2, the autoantigen implicated in PBC and continues IND-enabling work for this combination.
Gene Therapies:

SEL-302 for MMA: Selecta expects to initiate a Phase 1 clinical trial of SEL-302, an AAV gene therapy combined with ImmTOR for the treatment of MMA, in the fourth quarter of 2022.

SEL-018 IgG Protease (Xork): In collaboration with Genovis, Selecta continues to advance Xork, a next-generation IgG protease, to help address disease in patients who are ineligible for gene therapies due to pre-existing anti-AAV antibodies. Selecta believes the novel combination of Xork and ImmTOR has the potential to address two of the key hurdles in gene therapy today: pre-existing immunity and the inability to re-dose AAV gene therapies due to the immune response to AAV capsids.

IND-enabling studies and manufacturing scale-up activities are ongoing.
ImmTOR-IL in Gene Therapy: Building on our pre-clinical studies of ImmTOR-IL in inhibiting the formation of neutralizing antibodies to AAV gene therapies, we are pleased to announce that we continue to see mitigating effects in mice at gene therapy doses of 10x our prior studies.
Biologic Therapies:

SEL-212 for chronic refractory gout: Selecta continues to advance DISSOLVE, the Phase 3 development program of SEL-212, which has been licensed to Sobi.
Selecta completed enrollment for DISSOLVE II in June 2022, with 153 study participants, triggering a $10 million milestone payment obligation from Sobi which has been received in Q3 2022.
DISSOLVE I & II trials are on track for completion in Q4 2022 with joint topline readout expected in Q1 2023.

ImmTOR with IgA1 protease for IgA nephropathy: Selecta is working with both Ginkgo Bioworks and IGAN Biosciences to identify and develop a next generation IgA protease to combine with ImmTOR.

Selecta anticipates enzyme candidate selection by year end 2022.
Further Corporate and Partnership Updates:

Sarepta extended its Research License and Option Agreement for ImmTOR in Duchenne Muscular Dystrophy (DMD) and certain Limb-Girdle Muscular Dystrophies (LGMD) by nine months.
Additionally, in June 2022 Selecta was informed by Sarepta of the achievement of certain pre-clinical milestones.
Selecta expects to receive a $2 million payment for extending Sarepta’s option periods under the agreement to Q1 2023, and an additional $4 million payment for achievement of the pre-clinical milestone. Receipt of both payments is expected in Q3 2022.
At the 25th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Selecta showcased six presentations, including three joint presentations with its partner Asklepios BioPharmaceutical, Inc. (AskBio). These presentations highlighted the immunogenic potential of empty AAV and the potential of ImmTOR and ImmTOR-IL in addressing key efficacy and safety challenges in gene therapy.
Our CSO, Kei Kishimoto, was awarded an ‘Outstanding Poster Presentation Award’ for the abstract titled: Combination of ImmTOR Tolerogenic Nanoparticles and IL-2 Mutein Synergistically Inhibits the Formation of Anti-AAV Antibodies.
Second Quarter 2022 Financial Results:

Cash Position: Selecta had $143.4 million in cash, cash equivalents, marketable securities, and restricted cash as of June 30, 2022, as compared to cash, cash equivalents, marketable securities, and restricted cash of $129.4 million as of December 31, 2021. The increase in cash was primarily due to proceeds from the completion of an equity offering during the second quarter, raising gross proceeds of $38.7 million. Net cash used in operating activities was $24.1 million for the six months ended June 30, 2022, as compared to $18.2 million of cash used in operating activities for the same period in 2021. Selecta believes its available cash, cash equivalents, restricted cash, and marketable securities will be sufficient to meet its operating requirements into mid-2024.

Collaboration and License Revenue: Collaboration and license revenue for the second quarter of 2022 was $39.3 million, as compared to $19.7 million for the same period in 2021. Revenue was primarily driven by the shipment of clinical supply and the reimbursement of costs incurred for the Phase 3 DISSOLVE clinical program under the license agreement with Sobi and the shipment of manufactured supply under the Sarepta Agreement.

Research and Development Expenses: Research and development expenses for the second quarter of 2022 were $19.2 million, as compared to $14.5 million for the same period in 2021. The increase in cost was primarily the result of expenses incurred for the SEL-212 clinical program, stock compensation, and salaries.

General and Administrative Expenses: General and administrative expenses for the second quarter of 2022 were $6.2 million, as compared to $4.7 million for the same period in 2021. The increase in costs was primarily the result of expenses incurred for issuance costs for the 2022 equity offering and stock compensation.

Net Income (loss): For the second quarter of 2022, Selecta reported net income of $8.6 million, or basic net income per share of $0.06, compared to net income of $4.6 million, or $0.04 basic net income per share, for the same period in 2021.

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 second quarter 2022 financial results. Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or +1 (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or +1 (412) 317-0088 (international) and using confirmation code 10157873. 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 August 4, 2022 X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel CXCR4-targeted small-molecule therapeutics to benefit people with diseases of the immune system, reported financial results for the second quarter ended June 30, 2022 (Press release, X4 Pharmaceuticals, AUG 4, 2022, View Source [SID1234617472]). In addition, the company highlighted important upcoming milestones related to its lead clinical candidate, mavorixafor, including the presentation of data from its ongoing Phase 1b clinical trial in chronic neutropenic disorders and from the global, pivotal Phase 3 clinical trial of mavorixafor in WHIM (Warts, Hypogammaglobulinemia, Infections, & Myelokathexis) syndrome. The company also provided an update on its clinical oncology program following its recent strategic announcement prioritizing its use of resources to advance mavorixafor solely in chronic neutropenic disorders.

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"With the completion of our recent financing and the sharpening of our strategic focus on chronic neutropenic disorders, we believe that we are well positioned to deliver significant future value to both patients and shareholders as we approach key milestones in the second half of this year," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "While the data from our cancer programs continue to show promise, we are now pivoting our efforts towards unlocking the full value of our oncology portfolio through potential strategic partnerships, enabling us to focus on making the largest possible impact on the treatment of chronic neutropenic disorders, including WHIM syndrome. In late September, we expect to deliver on our next milestone – data from our fully enrolled proof-of-concept study in chronic neutropenic disorders; we expect these results to inform the regulatory path forward for mavorixafor across a broad range of chronic neutropenic patient populations. Finally, and notably, the unveiling of data from our pivotal Phase 3 4WHIM trial is on track for the fourth quarter; these results are expected to support our first regulatory filing in the U.S. early in the second half of 2023 and to bring us one large step closer to improving the lives of patients with chronic neutropenic disorders."

Recent Highlights & Anticipated Upcoming Milestones

Completed a private investment in public equity (PIPE) financing, raising gross proceeds of approximately $56 million; the financing included participation from new and existing investors.
Entered into an amendment to the company’s loan and security agreement with Hercules Capital to extend the interest-only period of its loan facility by up to twelve months (into 2024), subject to achieving certain financial and business milestones.
Announced a strategic re-prioritization of resources towards advancing mavorixafor in chronic neutropenic disorder indications, including WHIM syndrome, while progressing oncology programs only upon completion of strategic partnership(s); the announced strategic update was also inclusive of cost-cutting initiatives estimated to support extension of X4’s cash runway into the third quarter of 2023.
Presented research data at the June 2022 meeting of the European Hematology Association (EHA) (Free EHA Whitepaper) further supporting mechanism and market potential of mavorixafor across of range of clinical indications.
Planned late September 2022 presentation of data from the fully enrolled Phase 1b trial evaluating mavorixafor for the treatment of chronic neutropenic disorders, including congenital, idiopathic, and cyclic neutropenia.
Planned fourth quarter 2022 presentation of results from the global, pivotal Phase 3 clinical trial of mavorixafor for the treatment of patients with WHIM syndrome; data anticipated to support a submission for U.S. regulatory approval early in the second half of 2023.
Positive Data Update from Phase 1b Trial in Waldenström’s Macroglobulinemia (WM)

The Phase 1b, open-label, multicenter, single-arm study is evaluating the safety and efficacy of mavorixafor in combination with the BTK inhibitor ibrutinib in adult patients (either treatment naïve or relapsed/refractory) with the rare B-cell lymphoma Waldenström’s macroglobulinemia (WM) and confirmed MYD88 and CXCR4 genetic mutations.
A total of 16 patients were enrolled in the study; all were dosed with oral, once-daily doses of ibrutinib (420 mg) and escalating doses (200 mg, 400 mg, 600 mg) of oral mavorixafor, also once daily.
As of June 2022, 10 of 12 evaluable patients (83%) achieved a major response (MR) to therapy, or a greater than 50% reduction in serum IgM from baseline:
In relapsed/refractory patients, 8 of 9 (89%) achieved a MR
In treatment-naïve patients, MR was seen in all patients escalated to >200 mg of mavorixafor
Adding mavorixafor to ibrutinib was associated with a higher MR rate at 9, 12, and 24 months compared to previously reported MR rates achieved with ibrutinib monotherapy.
In addition, patients achieved elevations in absolute neutrophil count (ANC), with no neutropenic events reported; patients also experienced fewer infections over time with chronic dosing.
No major safety signals had been identified in the trial as of the data cut-off; mavorixafor in combination with ibrutinib showed a safety profile similar to ibrutinib monotherapy (N=16, including 8 patients escalated to the 600 mg dose of mavorixafor).
In June, mavorixafor was granted Orphan Drug Designation by the U.S. Food & Drug Administration for the treatment of patients with WM, regardless of CXCR4 mutation status.
The Phase 1b clinical trial is expected to be completed in the fourth quarter of 2022; further clinical studies in WM will now be subject to completing a strategic partnership.
Second Quarter 2022 Financial Results

Cash, Cash Equivalents & Restricted Cash: X4 had $48.7 million in cash, cash equivalents, and restricted cash as of June 30, 2022. On June 30, 2022, the company announced a private equity financing (PIPE) of approximately $56 million before deducting offering costs. The financing closed on July 6, 2022, and proceeds are not reflected in the cash on hand as of June 30, 2022. Including funds raised in this offering, the company’s recently announced cost-cutting measures, and recent amendments to its loan and security agreement with Hercules, X4 believes that it has sufficient funds to support company operations into the third quarter of 2023.
Research and Development (R&D) Expenses were $13.8 million for the second quarter of 2022 as compared to $13.2 million for the comparable period in 2021. R&D expenses include $0.7 million and $0.8 million of certain non-cash expenses for the second quarter of 2022 and 2021, respectively.
Selling, General, and Administrative Expenses (SG&A) were $6.7 million for the second quarter of 2022 as compared to $5.8 million for the comparable period in 2021. SG&A expenses include $0.8 million and $1.0 million of certain non-cash expenses for the second quarter of 2022 and 2021, respectively.
Net Loss: X4 reported a net loss of $21.2 million for the second quarter of 2022, as compared to $19.6 million for the comparable period in 2021. Net losses include $1.5 million and $1.8 million of certain non-cash expenses for the second quarter of 2022 and 2021, respectively.
Conference Call and Webcast
X4 will host a conference call and webcast today at 8:30 am EDT to discuss financial results and business highlights. The conference call can be accessed by dialing (855) 327-6837 from the United States or (631) 891-4304 internationally, followed by the conference ID: 10019589. 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 website.

On August 4, 2022 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global biotechnology company developing and commercializing innovative and affordable oncology medicines to improve treatment outcomes and access for far more patients worldwide, reported financial results for the second quarter of 2022, recent business highlights, and anticipated upcoming milestones (Press release, BeiGene, AUG 4, 2022, View Source [SID1234617471]).

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"BeiGene is well positioned for growth with momentum across our commercial portfolio and geographies and a strong capital position. We are continuing to execute with discipline and realize the benefits of our strategic investments in research and commercial capabilities"

"We made significant progress in our mission to reach far more cancer patients with innovative and affordable medicines, with growth in product revenues across our portfolio, driven primarily by our internally developed medicines, BRUKINSA and tislelizumab. We have now expanded our approvals to more than 50 markets globally, and BRUKINSA global revenue more than tripled on a year-over-year basis," said John V. Oyler, Co-Founder, Chairman and Chief Executive Officer of BeiGene. "We continue to unlock opportunity driven by our research and development engine and, during the second half of this year, expect to share final analysis data for our global Phase 3 ALPINE trial of BRUKINSA, including progression-free survival in chronic lymphocytic leukemia as well as topline data for tislelizumab as a first-line treatment for patients with unresectable hepatocellular cancer."

"BeiGene is well positioned for growth with momentum across our commercial portfolio and geographies and a strong capital position. We are continuing to execute with discipline and realize the benefits of our strategic investments in research and commercial capabilities," said Julia Wang, Chief Financial Officer, BeiGene.

Second Quarter 2022 Financial Results

Cash, Cash Equivalents, Restricted Cash, and Short-Term Investments were $5.7 billion as of June 30, 2022 and $6.6 billion as of December 31, 2021.

In the three months ended June 30, 2022, cash used in operating activities was $380.0 million, primarily due to our net loss of $571.4 million, offset by a decrease in our net operating assets and liabilities of $96.3 million, and by non-cash charges of $95.2 million. Net loss for the three months ended June 30, 2022 includes $129.6 million of other losses due primarily to the strengthening of the U.S. dollar and the related revaluation of foreign currencies held by U.S. functional currency subsidiaries. Capital expenditures were $50.3 million and cash used in financing activities was $17.6 million. In addition, the impact of foreign currency deposits being translated into the U.S. dollar negatively impacted ending cash by $80.2 million in the three months ended June 30, 2022 compared to a positive impact of $9.3 million in the prior year period.
Revenue for the three months ended June 30, 2022 was $341.6 million, compared to $150.0 million in the same period of 2021.

Product revenue totaled $304.5 million for the three months ended June 30, 2022, compared to $138.6 million in the same period of 2021, including:
Global sales of BRUKINSA of $128.7 million for the second quarter of 2022, compared to $42.4 million in the prior year period;
Sales of tislelizumab in China of $104.9 million for the second quarter of 2022, compared to $74.9 million in the prior year period;
Sales of Amgen in-licensed products in China of $29.5 million for the second quarter of 2022, compared to $3.3 million in the prior year period. Prior year period sales do not include sales of BLINCYTO and KYPROLIS, which were launched in China in August 2021 and January 2022, respectively; and
Sales of BMS in-licensed products in China of $23.4 million for the second quarter of 2022, compared to $13.4 million in the prior year period.
Collaboration revenue for the three months ended June 30, 2022 was $37.1 million, resulting from partial recognition of the upfront payments from Novartis of $650.0 million related to the tislelizumab agreement and $300.0 million related to the ociperlimab agreement, which were entered into in the first quarter and fourth quarter of 2021, respectively. Collaboration revenue for the three months ended June 30, 2021 was $11.4 million, resulting from the partial recognition of revenue related to the tislelizumab agreement.
Expenses for the three months ended June 30, 2022 were $781.0 million, compared to $624.8 million in the same period of 2021.

Cost of Sales for the three months ended June 30, 2022 were $71.2 million, compared to $36.3 million in the same period of 2021. Cost of sales increased primarily due to increased product sales of tislelizumab and BRUKINSA, as well as BLINCYTO, which commenced in August 2021, and KYPROLIS and POBEVCY, which commenced in January 2022.
R&D Expenses for the three months ended June 30, 2022 were $378.2 million, compared to $356.1 million in the same period of 2021. The increase in R&D expenses was primarily attributable to increases in headcount and costs related to investment in our discovery and development activities, including our continued efforts to internalize research and clinical development activities, partially offset by decreased expense related to upfront fees for in-process R&D. Upfront fees related to in-process R&D for in-licensed assets totaled nil and $45.0 million in the second quarters of 2022 and 2021, respectively. Employee share-based compensation expense also contributed to the overall increase in R&D expenses and was $37.1 million for the three months ended June 30, 2022, compared to $30.2 million for the same period of 2021.
SG&A Expenses for the three months ended June 30, 2022 were $331.4 million, compared to $232.3 million in the same period of 2021. The increase in SG&A expenses was primarily attributable to increased headcount, largely related to the expansion of our commercial teams, higher professional service fees and higher external commercial expenses, including selling and marketing, market access studies and promotional activities. The overall increase in SG&A expenses was also attributable to higher SG&A-related share-based compensation expense, which was $44.2 million and $34.6 million for the second quarters of 2022 and 2021, respectively.
Operating Loss for the three months ended June 30, 2022 decreased by $35.4 million, or 7.5% to $439.4 million, compared to $474.8 million in the same period of 2021. The decrease in operating loss for the quarter was driven by increased gross profit on product sales, which exceeded the growth in operating expenses.
Net Loss for the quarter ended June 30, 2022 was $571.4 million, or $0.43 per share, and $5.56 per American Depositary Share (ADS), compared to $480.3 million, or $0.40 per share, and $5.23 per ADS in the same period of 2021. Net loss for the quarter was negatively impacted by other non-operating expenses of $129.6 million, primarily related to foreign exchange losses resulting from the strengthening of the U.S. dollar and the revaluation impact of foreign currencies held in U.S. functional currency subsidiaries.
Recent Business Highlights

Commercial Operations

Product sales increased 120% in the second quarter of 2022 compared to the prior year period, primarily due to increased sales of our internally developed products, BRUKINSA and tislelizumab, as well as increased sales of in-licensed products from Amgen;
Global sales of BRUKINSA totaled $128.7 million in the second quarter, representing a 203% increase compared to the prior year period. U.S. sales of BRUKINSA totaled $88.4 million in the second quarter, representing growth of 456% compared to the prior year period, as the U.S. prescribing base continued to grow and as clinician use increased within approved indications — mantle cell lymphoma (MCL), Waldenström’s macroglobulinemia (WM) and marginal zone lymphoma (MZL). BRUKINSA sales in China totaled $36.7 million in the second quarter, representing growth of 39% compared to the prior year period, driven by a continued increase in all approved indications; and
Sales of tislelizumab in China totaled $104.9 million in the second quarter, representing a 40% increase compared to the prior year period. In the second quarter, we saw increased market penetration and market share for tislelizumab across nine approved indications, resulting from new patient demand from broader reimbursement in additional National Reimbursement Drug List.
Development Programs

BRUKINSA (zanubrutinib), a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) designed to maximize BTK occupancy and minimize off-target effects, approved in more than 50 markets including the U.S., China, European Union (EU), Great Britain, Canada, Australia, South Korea and Switzerland in selected indications and under development for additional approvals globally. The global BRUKINSA development program includes more than 4,500 subjects enrolled to-date in more than 25 countries and regions.

Received approval in Mexico for BRUKINSA for the treatment of adult patients with previously treated MCL;
Received U.S. FDA fast track designation to investigate zanubrutinib in combination with obinutuzumab, for the treatment of adults with relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy;
Received acceptance by Health Canada for supplemental new drug submission (sNDS) for zanubrutinib in chronic lymphocytic leukemia (CLL);
Presented primary analysis of the global Phase 2 ROSEWOOD trial (NCT03332017) of zanubrutinib plus obinutuzumab versus obinutuzumab monotherapy in patients with R/R FL in an oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Congress as well as at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The ROSEWOOD trial met its primary endpoints of overall response rate (ORR) and was generally well-tolerated, with safety results consistent with previous studies of both medicines; and
Presented long-term follow-up safety and efficacy results from the Phase 3 ASPEN trial (NCT03053440) of zanubrutinib versus ibrutinib in patients with WM at the 2022 EHA (Free EHA Whitepaper) Congress and ASCO (Free ASCO Whitepaper) Annual Meetings, which showed that, at a median follow up of 43 months, zanubrutinib continued to demonstrate clinically meaningful efficacy and a tolerable safety profile in patients with WM.
Tislelizumab, a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages; approved in China in nine indications and under development for additional approvals globally. The global tislelizumab clinical development program includes more than 11,000 subjects enrolled to-date in 30 countries and regions.

Received approval from the China National Medical Products Administration (NMPA) for tislelizumab in combination with chemotherapy as a first-line (1L) treatment for patients with recurrent or metastatic nasopharyngeal cancer (NPC);
Announced acceptance by the Center for Drug Evaluation (CDE) of the NMPA for a supplemental biologics application (sBLA) for tislelizumab in combination with chemotherapy as a 1L treatment for patients with advanced or metastatic gastric or gastroesophageal junction adenocarcinoma whose tumors express PD-L1;
Notification of successful dossier validated from Australia’s Therapeutic Goods Administration (TGA) for the new drug application of tislelizumab in 1L and second-line (2L) non-small cell lung cancer (NSCLC) and 2L esophageal squamous cell carcinoma (ESCC);
In collaboration with Novartis, the United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) has validated the tislelizumab submission for review as a treatment for 1L and 2L NSCLC and 2L ESCC in Great Britain;
Presented a late-breaking oral presentation at the 2022 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) World Congress on Gastrointestinal Cancer on new data from RATIONALE 306 (NCT03783442), a global Phase 3 trial which showed overall survival benefit for tislelizumab plus chemotherapy versus chemotherapy alone in 1L advanced or metastatic ESCC; and
Presented updated results in an oral plenary session from the global Phase 3 RATIONALE 309 trial (NCT03924986) of tislelizumab in 1L patients with NPC, and in a poster session reviewed clinical outcomes associated with tislelizumab in patients with advanced hepatocellular carcinoma (HCC) who have previously been treated with sorafenib or lenvatinib in RATIONALE 208 (NCT03419897) at the 2022 ASCO (Free ASCO Whitepaper) Annual Meeting.
Early-Stage Programs

Presented two posters from dose escalation studies of BGB-11417 (NCT04277637 and NCT04771130), a highly selective investigational BCL2 inhibitor in CLL, non-Hodgkin’s lymphoma and acute myeloid leukemia (AML) at the EHA (Free EHA Whitepaper) 2022 Congress;
Initiated tumor-specific dose expansion cohorts in Phase 1 trial (NCT04215978) in patients with solid tumors of BGB-A445, an investigational non-ligand competing OX40 monoclonal antibody, as monotherapy;
Initiated patient dosing in the Phase 1 trial (NCT05381909) in patients with advanced or metastatic solid tumors for BGB-24714, an investigational second mitochondrial-derived activator of caspase (SMAC) mimetic;
Continued to advance our early-stage clinical pipeline of internally developed product candidates at dose escalation stage, including:
BGB-15025: an investigational hematopoietic progenitor kinase 1 (HPK1) inhibitor as monotherapy or in combination with tislelizumab in solid tumors;
BGB-10188: an investigational PI3Kδ inhibitor as monotherapy or in combination with BRUKINSA in hematology malignancies, or in combination with tislelizumab in solid tumors; and
BGB-23339: a potent, allosteric investigational tyrosine kinase 2 (TYK2) inhibitor.
Amgen Milestones

In collaboration with Amgen, launched BLINCYTO (blinatumomab) for injection for the treatment of pediatric patients with R/R CD19-positive B-cell precursor acute lymphoblastic leukemia (ALL); and
Entered into a clinical trial collaboration and supply agreement with Amgen, in which Amgen provides KYPROLIS for a combination study conducted by BeiGene of BGB-11417 plus dexamethasone and carfilzomib in R/R multiple myeloma.
Zymeworks Milestones

In collaboration with Zymeworks, presented preliminary results from two Phase 1b/2 studies at the ASCO (Free ASCO Whitepaper) 2022 Annual Meeting evaluating zanidatamab:
In combination with docetaxel as a 1L therapy for patients with advanced HER2-positive breast cancer; and
In combination with chemotherapy and tislelizumab as a 1L therapy for patients with HER-2 positive gastric/gastroesophageal junction adenocarcinoma.
Manufacturing Operations

Construction has begun on the U.S. flagship commercial-stage manufacturing and clinical R&D campus at the Princeton West Innovation Campus in Hopewell, N.J. The property has more than one million square feet of developable real estate for potential future expansion;
Continued construction on our new small molecule manufacturing campus in Suzhou, China. Phase 1 of construction is expected to bring more than 52,000 square meters and expand production capacity to 600 million tablets/capsules and be completed in 2023. Once completed, qualified, and approved, the total production capacity is expected to increase our small molecule manufacturing capability in China by up to a total of ten times capacity; and
Continued construction on our state-of-the-art biologics facility in Guangzhou, China, which currently is approved for 8,000 liters of biologics capacity, with an additional Phase of construction to bring total capacity to 64,000 liters expected to be completed and GMP-ready by the end of 2022.
Corporate Developments

Announced a strategic research collaboration with InnoRNA to jointly discover mRNA therapies, under which we will hold exclusive global development and commercialization rights; and
Appointed Chan Lee as General Counsel. Mr. Lee will serve on our Executive Committee and report directly to John V. Oyler.
Expected Milestones

BRUKINSA

Continue to support ongoing FDA review of the sNDA for CLL/small lymphocytic lymphoma, which has a PDUFA target action date of January 2023;
Continue to support the European Medicines Agency (EMA) review of new indication applications for CLL and MZL;
Continue to support Health Canada review of sNDA for CLL;
Announce final analysis data for the global Phase 3 ALPINE trial (NCT03734016) including progression-free survival in 2022; and
Continue to expand BRUKINSA’s registration program globally in new geographies and indications, including potential launches in 2022 in more than 10 markets.
Tislelizumab

Continue to support NMPA review of BLA application for tislelizumab in combination with chemotherapy as a 1L treatment for patients with advanced or metastatic gastric or gastroesophageal junction adenocarcinoma whose tumors express PD-L1;
Continue to support Australia’s TGA review of NDA for tislelizumab in 1L/2L NSCLC and 2L ESCC;
In collaboration with Novartis, continue to support UK MHRA review of tislelizumab for treatment of 1L/2L NSCLC and 2L ESCC in Great Britain;
In collaboration with Novartis, continue to support the EMA review of marketing authorization applications for tislelizumab in NSCLC and 2L ESCC;
In collaboration with Novartis, continue to support the ongoing FDA review of the BLA submission in 2L ESCC to facilitate scheduling the required inspections as soon as possible. In the FDA’s general advice letter communicating the deferral of action, the FDA cited only the inability to complete inspections due to restrictions on travel as the reason for the deferral and did not provide a new anticipated action date as they continue to monitor the public health situation and travel restrictions;
Continue to support planned regulatory submissions by Novartis for 1L gastric cancer, 1L and localized ESCC, and 1L HCC in the U.S. in 2023. No additional submissions planned in the U.S. in 2022;
Announce topline results from the global Phase 3 clinical trial (NCT03412773) of tislelizumab as a 1L treatment for patients with HCC in Q3 2022; and
Present clinical data at 2022 World Conference on Lung Cancer including final analysis of the global, Phase 3 RATIONALE 303 trial (NCT03358875) with tislelizumab monotherapy compared to chemotherapy in previously treated advanced NSCLC and Phase 1 data on tislelizumab in combination with ociperlimab in metastatic NSCLC (NCT04047862); with sitravatinib in PD-L1+, locally advanced/metastatic, non-squamous NSCLC (NCT03666143); and with sitravatinib in PD-L1+, locally advanced/metastatic, squamous NSCLC (NCT03666143).
Ociperlimab

Initiate additional pivotal clinical trials in 2022; and
Announce data from Phase 1 trial (NCT04047862) cohorts in various solid tumor types in 2022.
BGB-11417 (BCL-2)

Initiate pivotal trials in 2022; and
Present Phase 1 clinical data for non-Hodgkin’s lymphoma, CLL, AML and multiple myeloma (MM) (NCT04883957, NCT04277637, NCT04771130, and NCT04973605) at a medical congress in late 2022.
Early Stage Programs

In collaboration with Leads Biolabs, initiate patient dosing of LBL-007, a novel investigational antibody targeting the LAG-3 pathway, in combination with tislelizumab and surzebiclimab (TIM3) in 2022.
COVID-19 Impact and Response

We expect that the worldwide health crisis of COVID-19 will continue to have a negative impact on our operations, including commercial sales, regulatory interactions, inspections, filings, manufacturing, and clinical trial recruitment, participation, and data readouts. There remains uncertainty regarding the future impact of the pandemic both globally and specifically in China due to outbreaks and restrictions and potential impact on clinical, manufacturing and commercial operations. We are striving to minimize delays and disruptions, have put protocols and procedures in place, and continue to execute on our commercial, regulatory, manufacturing, and clinical development goals globally.

On August 4, 2022 IMV Inc. (Nasdaq: IMV; TSX: IMV) ("IMV" or "the Company"), a clinical-stage company developing a portfolio of immune-educating therapies based on its novel DPX platform to treat solid and hematologic cancers, reported that it will hold a conference call and webcast on Thursday, August 11, 2022, at 8:00 a.m. ET to discuss the company’s 2022 second quarter financial and operational results (Press release, IMV, AUG 4, 2022, View Source [SID1234617470]).

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