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Adaptimmune Reports Second-Quarter Financial Results and Business Update

On August 4, 2022 Adaptimmune Therapeutics plc (Nasdaq: ADAP), a leader in cell therapy to treat cancer, reported financial results for the second quarter ended June 30, 2022 and provided a business update (Press release, Adaptimmune, AUG 4, 2022, View Source [SID1234617476]). For the three months ended June 30, 2022, Revenue was $5.5 million, Total Operating Expenses (Research and Development and General and Administrative) were $49.3 million, and Net Loss was $44.5 million.

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"We continue to demonstrate progress with our four focus areas for 2022: submitting our BLA for afami-cel, building a MAGE-A4 franchise, scaling up manufacturing capabilities, and advancing our allogeneic platform, pipeline and collaborations," said Adrian Rawcliffe, Adaptimmune’s Chief Executive Officer. "The data presented at ASCO (Free ASCO Whitepaper) further confirm the potential of afami-cel for the initial indication of synovial sarcoma with the BLA submission on-track for Q4 this year. To support commercialization of afami-cel and other products both near- and longer-term, we are investing in focused but scalable operational infrastructure to set us up for success. At the same time, we are carefully monitoring market conditions. We will continue to prudently manage expenses and stop or delay non-core activities."

Adaptimmune’s first potential commercial product, afami-cel, supported by positive data readouts

Biologics License Application (BLA) submission for afami-cel on track for Q4 2022

Adaptimmune is preparing its BLA and continues to target submission to the U.S. Food and Drug Administration (FDA) in the fourth-quarter 2022 for the treatment of synovial sarcoma. This BLA is supported by data from Cohort 1 of the pivotal trial SPEARHEAD-1, which met its primary endpoint for efficacy. In addition, the Company has the following progress updates:

●Miltenyi Biotec vector facility released for cGMP manufacture
●Vector and T-cell product characterization nearing completion
●Method validation for commercial T-cell lot release assays (including potency assays) in progress
●Vector process performance qualification (PPQ) initiated
●Pre-market approval (PMA) for the companion diagnostic is on-track to be submitted simultaneously with BLA

Afami-cel presentation at ASCO (Free ASCO Whitepaper) 2022 – responses reported across all patient subgroups

As reported in June, data based on pooled analyses of characteristics associated with clinical responses (per investigator assessment) from both Cohort 1 of the SPEARHEAD-1 trial, as well as the Phase 1 trial of afami-cel in patients with advanced synovial sarcoma or myxoid/round cell liposarcoma (MRCLS), were presented at the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper)

●Overall response rate was 36% in heavily pre-treated patients across both types of sarcomas (41% in synovial sarcoma and 10% for MRCLS), with a median duration of response of 52 weeks
●Patients who responded to afami-cel had longer progression-free survival (median 58 weeks) compared to non-responders (median 12 weeks)

●Responses occurred across all clinical subgroups, with greater response rates associated with lower baseline tumor burden, fewer prior lines of therapy, and higher MAGE-A4 expression
●Benefit:risk profile of afami-cel has been favorable, to date

Potential of next-gen MAGE-A4 targeted cell therapy in multiple solid tumors

Phase 1 signal-finding SURPASS trial update at ESMO (Free ESMO Whitepaper) in September

●The purpose of the next-generation ADP-A2M4CD8 program is to improve the potency of Adaptimmune’s first-generation product targeting MAGE-A4, afami-cel, to achieve meaningful clinical responses beyond sarcoma
●ADP-A2M4CD8 is being investigated in the SURPASS family of trials
●Last year, at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2021 Congress, Adaptimmune reported data from 22 evaluable patients with confirmed responses in ovarian, head and neck, esophagogastric junction, and bladder cancers from the Phase 1 signal-finding SURPASS trial, with this next-generation cell therapy
●Based on positive signals in gastroesophageal cancers in the Phase 1 SURPASS trial, Adaptimmune initiated a Phase 2 SURPASS-2 trial last year
●The Company plans to initiate an additional Phase 2 trial, SURPASS-3, this year for people with ovarian cancer, based on positive signals from the SURPASS Phase 1 trial
●On September 10, 2022 at 15:45 CET, Dr. David Hong from the MD Anderson Cancer Center, will present a data update from the Phase 1 signal-finding SURPASS trial at the ESMO (Free ESMO Whitepaper) 2022 Congress
●Data at ESMO (Free ESMO Whitepaper) will include 44 patients who received ADP-A2M4CD8 and 43 patients will be evaluable for efficacy (as of the data cut-off date of August 01, 2022)
●On September 9th, the Company will host a live virtual event from 8 a.m. to 9 a.m. EDT to discuss the ESMO (Free ESMO Whitepaper) data and its SURPASS family of trials

Corporate and other news

●Adaptimmune has implemented rigorous cost containment measures to enable delivery against its four key focus areas: submitting the BLA for afami-cel, building its MAGE-A4 franchise, scaling up manufacturing capabilities, and progressing the allogeneic platform
●To prioritize near- and mid-term value creation from its MAGE-A4 franchise, including the BLA submission for afami-cel and the SURPASS family of trials, Adaptimmune will delay submission of an IND for its new next-generation cell therapy ADP-A2M4N7X19, which it is developing in collaboration with Noile-Immune. Core preclinical activities will continue.
●Construction is underway to increase the Company’s cGMP manufacturing space at its Navy Yard site in Philadelphia, PA to support current clinical trials and future commercial products
●A dedicated allogeneic manufacturing facility in the United Kingdom is nearing completion, which will support the IND planned for 2023 for Adaptimmune’s wholly owned allogeneic cell therapy targeting MAGE-A4

Financial Results for the three and six months ended June 30, 2022

●Cash / liquidity position: As of June 30, 2022, Adaptimmune had cash and cash equivalents of $97.8 million and Total Liquidity1 of $258.1 million, compared to $149.9 million and $369.6 million, respectively, as of December 31, 2021.
●Revenue: Revenue for the three and six months ended June 30, 2022 was $5.5 million and $9.1 million, respectively, compared to $3.1 million and $3.5 million for the same periods in 2021. Revenue has increased primarily due to an increase in development activities under our
1 Total liquidity is a non-GAAP financial measure, which is explained and reconciled to the most directly comparable financial measures prepared in accordance with GAAP below

collaboration arrangements, in particular due to development activities under the Genentech Strategic Collaboration and License Agreement, which become effective in October 2021.
●Research and development (R&D) expenses: R&D expenses for the three and six months ended June 30, 2022 were $34.7 million and $71.5 million, respectively, compared to $28.9 million and $53.4 million for the same periods in 2021. R&D expenses increased due to an increase in the average number of employees engaged in research and development, increases in subcontracted expenditures and increases in in-process research and development costs. These were offset by an increase in reimbursements receivable for research and development tax and expenditure credits.
●General and administrative (G&A) expenses: G&A expenses for the three and six months ended June 30, 2022 were $14.6 million and $31.4 million, respectively, compared to $13.5 million and $27.4 million for the same periods in 2021 due to increases in employee-related costs and other corporate costs.
●Net loss: Net loss attributable to holders of the Company’s ordinary shares for the three and six months ended June 30, 2022 was $44.5 million and $94.8 million, respectively ($(0.05) and $(0.10) per ordinary share), compared to $39.1 million and $76.8 million, respectively ($(0.04) and $(0.08) per ordinary share), for the same periods in 2021.

Financial Guidance

The Company believes that its existing cash, cash equivalents and marketable securities, together with the additional payments under the Strategic Collaboration and License Agreement with Genentech, will fund the Company’s current operations into early 2024, as further detailed in the Company’s Quarterly Report on Form 10-Q for the three and six months ended June 30, 2022, to be filed with the Securities and Exchange Commission following this earnings release.

Conference Call Information

The Company will host a live teleconference and webcast to provide additional details at 8:00 a.m. EDT (1:00 p.m. BST) today, August 4, 2022. A live webcast of the conference call and replay can be accessed at View Source Call in information is as follows: (800)-952-5114 (US or Canada) or +1 (416)-406-0743 (International and additional options available HERE). After placing the call, please ask to be joined into the Adaptimmune conference call and provide the confirmation code (5869059).

Purple Biotech Reports Second Quarter 2022 Financial Results

On August 4, 2022 Purple Biotech Ltd. ("Purple Biotech", or the "Company") (NASDAQ/TASE: PPBT), a clinical-stage company developing first-in-class, effective and durable therapies by harnessing the power of the tumor microenvironment to overcome tumor immune evasion and drug resistance, reported financial results for the second quarter ended June 30, 2022 (Press release, Purple Biotech, AUG 4, 2022, View Source [SID1234617475]).

"This is the first report of Purple Biotech financial results since my appointment as Chief Executive Officer," said Gil Efron, Chief Executive Officer of Purple Biotech. "It is an opportunity for me to emphasize our main objectives. Purple Biotech has two exciting and innovative lead assets in development. We are focused on performing robust studies aiming to achieve meaningful clinical data and are continuing to explore opportunities to expand our current clinical programs to additional indications, while maintaining our cash runway, currently through the end of 2024. We are seeking opportunities for additional assets and collaborations to increase our footprint, through accretive transactions. I believe that together with strong science and by leveraging on our capabilities we can achieve these objectives and solidify Purple Biotech as a significant player within the oncology field."

Recent Corporate Highlights

NT219

In June, Purple presented Phase 1 interim monotherapy data of the ongoing Phase 1/2 clinical trial of NT219 at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, demonstrating encouraging safety & efficacy profile including one confirmed partial response in gastroesophageal junction cancer patient and stable disease in 75% of patients with mutated-KRAS colorectal cancer.

CM24

The Company initiated the Phase 2 portion of its ongoing study of CM24, a first-in-class monoclonal antibody with the potential to treat multiple cancers. The Phase 2 is an open-label, multicenter study in patients with metastatic pancreatic cancer (PDAC) to evaluate the safety and tolerability of CM24 in combination with the PD-1 inhibitor Opdivo (nivolumab) and chemotherapy. The primary study endpoint is to evaluate preliminary efficacy in 2nd line PDAC.

This follows the recent favorable safety and efficacy data supporting the advancement of CM24 at the recommended phase 2 dose of 20 mg/Kg. The data from the Phase 1b study of CM24 in combination with Opdivo (nivolumab) was presented in a poster entitled "Interim Safety and Efficacy Results from a Phase 1b Study of CM24 in Combination with Nivolumab in Adults with Advanced Solid Tumors" at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) 2022 Annual Meeting in May.

"Based on this progress last quarter, starting from phase 2, we will be performing randomized studies, such as our CM24 study in PDAC, which we are now in the process of expanding accordingly," added Efron. "The recent clinical data for NT219 together with previously published preclinical results have shown the potential of NT219 on solid tumors harboring KRAS mutation and we are looking into the development of NT219 for treatment of mutated KRAS patients. In addition, Insulin Receptor Substrates 1/2 (IRS) was identified as one of the pathways for resistance mechanism to other treatments in this field, opening this opportunity for NT219, which is the only IRS inhibitor in clinical development to date."

"We are ending the quarter in a strong financial position with $38.7 million in cash, cash equivalent short term and long term deposits and a cash runway of two and a half years as we continue to control our costs while advancing our programs. I’m proud of the progress made in the last quarter and we look forward to continuing to advance our clinical stage programs on behalf of cancer patients."

Financial Results for the three Months Ended June 30, 2022

Research and Development Expenses were $2 million, same as to $2 million in the same period of 2021.

Selling, General and Administrative Expenses were $1.5 million, same as to $1.5 million in the same period of 2021.

Operating Loss was $3.6 million, same as the $3.6 million in the same period of 2021.

On a non-IFRS basis (as reconciled below), adjusted operating loss was $3 million, an increase of $0.1 million, compared to $2.9 million in the same period of 2021.

Net Loss for the three months ended June 30, 2022 was $3.6 million, or $0.20 per basic and diluted ADS, compared to a net loss of $3.6 million, or $0.21 per basic and diluted ADS, in the three months ended June 30, 2021. Adjusted net loss for the three months ended June 30, 2022 was $2.9 million, the same as in the three months ended June 30, 2021.

Financial Results for the Six Months Ended June 30, 2022

Research and Development Expenses were $8.0 million, an increase of $1.1 million, or 15.9%, compared to $6.9 million in the same period of 2021. The increase was mainly due to an increase of $500 thousand in CMC expenses in support of our clinical studies and $300 thousands in wages in support of our growing development activities.

Sales, General and Administrative Expenses were $2.9 million, compared to $3.1 million in the same period of 2021, a decrease of $0.2 million. The decrease was mainly due to a decrease in employee equity-based compensation (ESOP) costs.

Operating Loss from continuing operations was $10.9 million, an increase of $0.8 million, or 7.9%, compared to $10.1 million in the same period of 2021.

On a non-IFRS basis (as reconciled below), adjusted operating loss was $10 million, an increase of $1.3 million, compared to $8.7 million in the same period of 2021, mainly due to an increase in R&D expenses.

Net Loss for the first six months ended June 30, 2022 was $10.8 million, or $0.61 per basic and diluted ADS, compared to a net loss of $10.2 million, or $0.58 per basic and diluted ADS, in the same period of 2021. The increase in net loss was mainly due to an increase of $0.8 million in operating expenses. Adjusted net loss for the first six months ended June 30, 2022 was $10 million, an increase from $8.7 million in the first six months ended June 30, 2021.

During the six months ended June 30, 2022, the Company sold, under the Open Market Sale Agreementsm with Jefferies LLC, approximately 179 thousand ADSs, at an average price of $3.64 per ADS. Net proceeds to the Company, were approximately $0.57 million, net of issuance expenses.

Arbutus Reports Second Quarter 2022 Financial Results and Provides Corporate Update

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]).

"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.

Selecta Biosciences Reports Second Quarter 2022 Financial Results and Provides Business Update

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]).

"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.

X4 Pharmaceuticals Reports Second Quarter 2022 Financial Results and Provides Corporate Update

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.

"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.