On January 24 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 2022 corporate objectives and provided a financial update (Press release, Arbutus Biopharma, JAN 24, 2022, View Source [SID1234606726]).

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William Collier, President and CEO, stated, "This year is an important one for Arbutus and the HBV patient community based on the many accomplishments we achieved in 2021. With five clinical trials on-going in HBV that include either AB-729 or AB-836, we are now poised for several key data readouts throughout this year that we anticipate will support Phase 2b clinical development. In addition, we intend to advance AB-101, our oral PD-L1 inhibitor compound for HBV which is designed to re-awaken the immune system, through IND-enabling studies this year. Similarly, this year we intend to complete IND-enabling studies for our RNA destabilizer, AB-161."

Mr. Collier continued, "As the COVID-19 pandemic lingers, we are expediting our efforts to advance novel oral pan-coronavirus compounds against viral protease and polymerase targets. We intend to advance an Mpro candidate into IND-enabling studies for the treatment of SARS-CoV-2 this year.

"Lastly, we are entering 2022 from a position of financial strength, with a cash runway expected to extend into the second quarter of 2024. I am thrilled with the company’s accomplishments and execution in 2021 and look forward to providing further updates over the course of this coming year."

Summary of 2022 Anticipated Key Milestones:

HBV Franchise:

Announce preliminary AB-729 data in the second half of 2022 from the following three on-going Phase 2a proof-of-concept clinical trials in patients with chronic hepatitis B (HBV) infection:
AB-729 in combination with ongoing standard-of-care nucleos(t)ide analogues ("NA") therapy and short courses of PEG-IFNα-2a.
AB-729 plus vebicorvir ("VBR"), Assembly Bio’s lead investigational HBV core inhibitor (capsid inhibitor) candidate, and a NA.
AB-729 plus ATI-2173, Antios’ proprietary Active Site Polymerase Inhibitor Nucleotide (ASPIN), and Viread (tenofovir disoproxil fumarate).
Initiate a triple combination Phase 2a proof-of-concept clinical trial in the first half of 2022 to evaluate AB-729, combined with VTP-300, Vaccitech’s therapeutic vaccine, and a NA.
Present at a medical conference the long-term on- and off-treatment follow-up data from our Phase 1a/1b clinical trial evaluating multiple doses and dosing schedules of AB-729.
Announce data from our Phase 1a/1b clinical trial evaluating AB-836 in patients with chronic HBV in the first half of 2022.
Complete IND-enabling studies with our oral PD-L1 inhibitor compound, AB-101, in the second half of 2022.
Complete IND-enabling studies for AB-161, our next-generation oral HBV specific RNA destabilizer, in the second half of 2022. Arbutus has conducted extensive non-clinical safety evaluations with AB-161 that gives us confidence in this molecule’s ability to circumvent the peripheral neuropathy findings seen in non-clinical safety studies with the Company’s first-generation oral RNA destabilizer, AB-452.
COVID-19 & Pan-Coronavirus Franchise

Nominate a candidate that inhibits the SARS-CoV-2 nsp5 main protease (Mpro) in the first half of 2022 and advance into IND-enabling studies.
Continue lead optimization activities for an Nsp12 viral polymerase candidate.
Other Opportunities

Explore potential oncology applications for internal PD-L1 portfolio.
Financial Update:

We had cash, cash equivalents and investments in marketable securities totaling $191 million as of December 31, 2021. This amount does not include a $40 million upfront payment and $15 million of proceeds resulting from the sale of common stock to Qilu Pharmaceutical as part of the exclusive licensing agreement and strategic partnership to develop and commercialize AB-729 in mainland China, Hong Kong, Macau and Taiwan. These amounts were received in January 2022.
For the full year of 2021 we received $135 million of net proceeds from the issuance of common shares under Arbutus’s "at-the-market" offering program. As of December 31, 2021, we had approximately 145 million common shares issued and outstanding, and approximately 11.4 million stock options outstanding.
We expect our net cash burn to range from $90 to $95 million in 2022. We believe our cash, cash equivalents and investments in marketable securities of $191 million as of December 31, 2021, plus the amounts received from Qilu Pharmaceutical in January 2022 are sufficient to fund the Company’s operations into the second quarter of 2024.
The preliminary cash, cash equivalents and investments, the amount received from the issuance of common shares under Arbutus’s "at-the-market" offering program and the common shares and stock options outstanding as of December 31, 2021 were calculated prior to the completion of an audit by Arbutus’ independent registered public accounting firm and are therefore subject to adjustment.
COVID-19 Impact

In December 2019 an outbreak of a novel strain of coronavirus (COVID-19) was identified in Wuhan, China. This virus has been declared a pandemic by the World Health Organization and has spread to nearly every country in the world. The impact of this pandemic has been, and will likely continue to be, extensive in many aspects of society. The pandemic has resulted in and will likely continue to result in significant disruptions to businesses. A number of countries and other jurisdictions around the world have implemented extreme measures to try and slow the spread of the virus. These measures include the closing of businesses and requiring people to stay in their homes, the latter of which raises uncertainty regarding the ability to travel to hospitals in order to participate in clinical trials. Additional measures that have had, and will likely continue to have, a major impact on clinical development, at least in the near-term, include shortages and delays in the supply chain, and prohibitions in certain countries on enrolling subjects and patients in new clinical trials. While we have been able to progress with our clinical and pre-clinical activities to date, it is not possible to predict if the COVID-19 pandemic will materially impact our plans and timelines in the future.

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. Preliminary data from an on-going Phase 1a/1b clinical trial has shown that AB-836 is generally safe and well-tolerated and provides robust antiviral activity.

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 250 million people worldwide suffer from chronic HBV infection, while other estimates indicate that approximately 2 million people in the United States suffer from chronic HBV infection. Approximately 900,000 people die every year from complications related to chronic HBV infection despite the availability of effective vaccines and current treatment options.

On January 24, 2022 HCW Biologics Inc. (the "Company") (NASDAQ: HCWB), a biopharmaceutical company focused on discovering and developing novel immunotherapies to lengthen health span by disrupting the link between inflammation and age-related diseases, reported that the Masonic Cancer Center, University of Minnesota was cleared by the U.S. Food and Drug Administration (FDA) to proceed to evaluate the Company’s lead drug candidate, HCW9218, in a Phase 1 clinical trial in patients with advanced solid tumors with progressive disease after prior chemotherapies (Press release, HCW Biologics, JAN 24, 2022, View Source [SID1234606725]). HCW9218 is an injectable, bifunctional fusion protein complex designed to drive anti-tumor activity by activating desired immune responses to attack cancer cells while simultaneously blocking unwanted immunosuppressive activities.

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The Principal Investigator of the clinical trial is Melissa A. Geller, M.D., M.S., Professor and Division Director of Gynecologic Oncology in the Department of Obstetrics, Gynecology and Women’s Health at the University of Minnesota. Dr. Geller stated, "Because the immunosuppressive tumor microenvironment limits the success of current therapies, there is a critical need for novel immune based therapies for advanced solid tumors. The estimated percentages of patients who are eligible for and who respond to cancer treatments such as checkpoint inhibitor drugs remain modest. We are excited to be able to sponsor a clinical study for treatment-resistant solid tumors using HCW9218. This immunotherapeutic might be the answer we need to augment anti-tumor activities of existing standards of care chemotherapies."

"HCW9218 is a unique combination of a TGF-ß receptor that neutralizes a highly immunosuppressive cytokine secreted by tumors, and IL-15, a potent cytokine that stimulates the natural killer and CD8+-T cell cytotoxicity. As a result, this bifunctional immunotherapeutic has the potential to drive significant anti-tumor activity," noted Jeffrey A. Miller, M.D., the Deputy Director of the Masonic Cancer Center, and co-Principal Investigator on this study. Dr. Miller continued, "HCW9218 also may define a new category of cancer treatment through modifying factors related to drug resistance and disease recurrence. We plan to conduct correlative studies to evaluate this potential in our current clinical trial."

Hing C. Wong, Ph.D., Founder and CEO of HCW Biologics, stated, "HCW Biologics is honored to have the Masonic Cancer Center as the sponsor for the second clinical trial to evaluate HCW9218 in difficult-to-treat cancers. This is an important milestone for HCW Biologics and our efforts to advance the development of potentially groundbreaking immunotherapeutic candidates for cancer and other age-related diseases. We have demonstrated that HCW9218 can augment the anti-tumor activities and lessen the side effects of chemotherapies by reducing the therapy-induced senescent cells in preclinical animal models."

On January 24, 2022 Lantern Pharma (NASDAQ: LTRN), a clinical stage biopharmaceutical company using its proprietary RADR artificial intelligence ("A.I.") platform to transform the cost, pace, and timeline of oncology drug discovery and development reported that the U.S. Food and Drug Administration (FDA) has granted both Rare Pediatric Disease Designation and Orphan Drug Designation for the company’s drug candidate LP-184 for the treatment of pediatric patients with ATRT (Press release, Lantern Pharma, JAN 24, 2022, View Source [SID1234606724]). LP-184 is being pursued as a potential new therapy across a range of genetically defined solid tumors, including pancreatic cancer, GBM (Glioblastoma Multiforme) and ATRT (Atypical Teratoid Rhabdoid Tumor).

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"Historical approaches to treating pediatric ATRT such as surgery, radiation, and chemotherapy have had largely unfavorable long term outcomes for children, and new options are urgently needed," said Kishor Bhatia, Ph.D., chief scientific officer of Lantern Pharma. "The gene SMARCB1 was included among several genes whose expression negatively correlated with LP-184 sensitivity in tumors. This in silico correlation was convincingly confirmed by in vitro and in vivo assessments of LP-184 in ATRT. The highest potency of LP-184 in-vivo has been seen in ATRT xenografts." Dr. Bhatia continued, "Based on both the in-silico and in-vivo observations, LP-184 has the potential to become a critical part of the armamentarium of approved treatment options specifically for these patients. Receiving Rare Pediatric Disease Designation from the FDA underscores the critical value of our growing focus on pediatric oncology indications at Lantern and represents another significant milestone for the LP-184 program."

The National Cancer Institute (NCI) classifies ATRT as Grade IV tumors, meaning they are malignant (cancerous), aggressive and fast-growing. ATRTs are very aggressive childhood malignancies of the central nervous system. The underlying genetic cause are inactivating bi-allelic mutations in SMARCB1 (also called INI1) or in SMARCA4. Nearly 90 percent of pediatric ATRTs are caused by changes in the gene known as SMARCB1.

The FDA grants rare pediatric disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to receive a priority review voucher following approval of a product with rare pediatric disease designation if the marketing application submitted for the product satisfies certain conditions, including approval prior to September 30, 2026 unless changed by legislation. If issued, a sponsor may redeem a priority review voucher for priority review of a subsequent marketing application for a different product candidate, or the priority review voucher could be sold or transferred to another sponsor.

Orphan drug designation is granted by the FDA Office of Orphan Products Development to investigational treatments that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the United States. The program was developed to encourage the development of medicines for rare diseases, and benefits include tax credits and application fee waivers designed to offset some development costs, as well as eligibility for market exclusivity for seven years post approval.

LP-184 is being developed for multiple targeted oncology indications. Lantern Pharma intends to further advance LP-184 as a new, potent treatment option in genetically defined subsets of patient populations in areas of high unmet clinical need, including pancreatic cancers, GBM, and other cancers that are DNA damage repair deficient. The U.S. Food and Drug Administration (FDA) has previously granted LP-184 Orphan Drug Designation for the treatment of pancreatic cancer, and for the treatment of malignant glioma, including GBM.

On January 24, 2022 Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for eltanexor, a novel oral, Selective Inhibitor of Nuclear Export (SINE) compound, for the treatment of myelodysplastic syndromes (MDS) (Press release, Karyopharm, JAN 24, 2022, View Source [SID1234606723]). MDS are a group of diseases characterized by ineffective production of the components of the blood due to poor bone marrow function with a risk of progression to acute myeloid leukemia.

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Karyopharm is currently investigating eltanexor in an ongoing open-label Phase 1/2 study as a single-agent or in combination with approved and investigational agents in patients with several types of hematologic and solid tumor cancers (KCP-8602-801; NCT02649790). Previously, Karyopharm reported positive data from an investigator-sponsored Phase 1 study evaluating single-agent eltanexor in patients with hypomethylating agent (HMA)-refractory MDS, where eltanexor demonstrated a 53% overall response rate and median overall survival of 9.9 months. This compares favorably to historical survival of four to six months for HMA-refractory MDS patients.

Approximately 15,000 people are diagnosed with intermediate-to-high risk MDS each year in the U.S.1 HMAs are the current standard of care for newly diagnosed, higher-risk MDS patients. However, only 40-60% of patients respond, with these responses typically lasting less than two years.2 The prognosis in HMA-refractory disease is poor, with a median overall survival of four to six months.3,4 There are currently no approved therapies for HMA- refractory MDS.

"We are pleased to receive the FDA’s orphan drug designation for eltanexor in MDS and believe it reinforces eltanexor’s potential to improve clinical outcomes for patients with HMA-refractory MDS," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "We are focused on advancing our ongoing clinical trials and remain steadfast in our commitment to bringing this new treatment option to patients and their families."

Orphan drug designation by the FDA is granted to promote the development of drugs that target conditions affecting 200,000 or fewer U.S. patients annually and are expected to provide a significant therapeutic advantage over existing treatments. Orphan designation qualifies a company for certain incentives that apply across all stages of drug development, including the potential for seven years of market exclusivity following marketing approval, tax credits on qualified U.S. clinical trials, eligibility for orphan drug grants, and exemption from certain administrative fees.

About Eltanexor

Eltanexor (KPT-8602) is an investigational novel SINE compound that, like selinexor, functions by binding with, and inhibiting, the nuclear export protein, XPO1, leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells.

In preclinical models, eltanexor has a broad therapeutic window with minimal penetration of the blood brain barrier and, therefore, has the potential to serve as another SINE compound for cancer indications. Following oral administration, animals treated with eltanexor show lower percentage of body weight loss and improved food consumption than animals similarly treated with selinexor. This allows more frequent dosing of eltanexor, enabling a longer period of exposure than is possible with selinexor.

Eltanexor is an investigational medicine and has not been approved by the United States Food and Drug Administration or any other regulatory agency.

On January 24, 2022 MaxCyte, Inc., (NASDAQ: MXCT; LSE: MXCT, MXCN), a leading commercial cell-engineering company focused on providing enabling platform technologies to advance innovative cell-based research as well as next-generation cell therapeutic discovery, development and commercialization, reported a preliminary update on revenue results for the fourth quarter and full year 2021 (Press release, MaxCyte, JAN 24, 2022, View Source [SID1234606722]).

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Preliminary Unaudited Fourth Quarter 2021 and Full Year Revenue

Management expects total revenue for the fourth quarter of 2021 to be more than $10.0 million, up from $8.5 million of total revenue in the fourth quarter of 2020, reflecting growth of at least 17% in total revenue and at least 37% in core business revenue.

MaxCyte’s revenue for the fourth quarter of 2021 was derived from its core business, which is defined as sales or leases of instruments, sales of single-use disposables, and sales of consumables (buffer) to the cell therapy and drug discovery markets.

MaxCyte also generates revenue under Strategic Platform License agreements (SPLs) with cell therapy developers, such as precommercial milestone payments. These revenues are categorized as program-related revenue and are excluded from core business revenue.

Preliminary revenue for the full year ended December 31, 2021 is expected to be more than $33.7 million, up from $26.2 million in full year 2020, reflecting growth of at least 28% in total revenue and at least 36% in core business revenue. Revenue for the full year ended December 31, 2021 includes $2.5 million of program-related revenue, compared to $3.3 million of program-related revenue in 2020.

MaxCyte ended the year with 15 SPLs, including 4 SPLs added during 2021: Nkarta, Inc., Myeloid Therapeutics, Celularity, Inc. and Sana Biotechnology, Inc.

Doug Doerfler, President and CEO of MaxCyte said: "We are proud of our performance in the fourth quarter as well as the full year, which has been a year of key achievements for the company. This includes raising $257.2 million in gross equity proceeds, the completion of an IPO in the United States and commencement of trading in our common stock on the Nasdaq, continuing significant organic growth in our core business, and our ongoing success in signing SPLs with innovative cell therapy developers. We are also excited to confirm that our ExPERT VLx instrument became available for sale at the end of December."

"We remain optimistic about the potential for our SPLs to generate meaningful revenue over the next 12 to 18 months and beyond. Our partners continue to achieve clinical success – particularly in moving their next-generation product candidates into pivotal trials. We also see the potential for several IND filings by our SPL customers for novel ex vivo engineered cell therapies this year. Finally, we continue to benefit from the ongoing investment in the ex vivo engineered cell therapy space. As a result, we believe our SPL pipeline remains as robust and diverse as ever. We look forward to a strong 2022."

MaxCyte’s fourth quarter and full year 2021 financial results presented in this release are preliminary and unaudited and are subject to revision based on the completion of MaxCyte’s normal quarter and year-end process and year-end audit. As a result, these preliminary results may be different from the actual results that will be reflected in MaxCyte’s consolidated financial statements for the quarter and year ended December 31, 2021, which are expected to be released by the end of March 2022.