On March 21, 2022 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development and commercialization of targeted drugs for the treatment of cancer, reported financial results for the year ended December 31, 2021 and provided a corporate update (Press release, Cellectar Biosciences, MAR 21, 2022, View Source [SID1234610481]).

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Fourth Quarter and Recent Corporate Highlights

·Presented data from 11 multiple myeloma patients from the company’s ongoing Phase 2 CLOVER-1 study of iopofosine I-131 (iopofosine) at the 63rd ASH (Free ASH Whitepaper) Annual Meeting and Exposition. The patients included in this report were triple class refractory had a median 7.2 prior lines of therapy, 50% classified as high risk, showed a mean overall response rate (ORR) of 45.5%, clinical benefit rate (CBR) of 72.7% and disease control rate (DCR) of 100% were observed. A subset of 5 quad/penta drug refractory patients had an ORR of 80%, a CBR and DCR of 100%. Median progression free survival (PFS) for all 11 patients was 3.4 months. Treatment emergent adverse events were mostly limited to bone marrow suppression in line with prior observations. No patients experienced a treatment emergent adverse event of neuropathy, arrhythmia, cardiovascular event, bleeding, ocular toxicities, renal function, alterations in liver enzymes, or infusion-site reactions.

·Announced initial responses from patients in the company’s Phase 1 study of iopofosine in children and adolescents with relapsed and refractory high-grade gliomas (HGGs) and soft tissue sarcomas, with patients exhibiting positive changes in various tumor parameters. Patients received doses up to 60 mCi/m2 and initial response and tumor uptake were confirmed by further therapeutic responses as evidenced by changes in tumor parameters. These include patients with relapsed HGGs experiencing over 5 months of PFS. The independent data monitoring committee advised that based upon the initial data, the study could initiate the 75 mCi/m2 dosing cohort.

"The data presented from our ongoing trials continue to be very encouraging, and we believe iopofosine has the potential to provide a meaningful therapeutic benefit for patients with Waldenstrom’s macroglobulinemia (WM) and other treatment refractory B-cell lymphomas. We continue to make significant strides in our clinical trials as we enroll patients in our ongoing pivotal trial in WM and our Phase 2 CLOVER-1 study," said James Caruso, president and CEO of Cellectar. "We look forward to sharing our planned interim data safety monitoring assessment from our WM pivotal trial. In the near-term our efforts are supported by a strong balance sheet that will fund our expected clinical and regulatory milestones into the second half of 2023."

2021 Financial Highlights

·Cash and Cash Equivalents: As of December 31,2021, the company had cash and cash equivalents of $35.7 million, compared to $57.2 million at December 31, 2020. Cash used in operating activities during the twelve months ended December 31, 2021 was approximately $22.6 million. The company believes its cash on hand is adequate to fund basic budgeted operations for at least 12 months from the filing of the 2021 financial statements.

·Research and Development Expense: R&D expense for the year ended December 31, 2021 was approximately $17.6 million, compared to approximately $10.1 million for year ended December 31, 2020. The overall increase in R&D expense of approximately $7.5 million was a result of an increase in planned clinical project costs primarily related to the company’s WM pivotal study. Manufacturing and related costs remained relatively consistent year over year.

·General and Administrative Expense: G&A expense for year ended December 31, 2021 was $6.6 million compared to approximately $5.2 million for the year ended December 31, 2020. The increase of $1.4 million in G&A costs was primarily a result of an increase in professional fees and insurance, personnel costs and stock-based compensation expense.

·Net Loss: The net loss attributable to common stockholders for the year ended December 31, 2021 was ($24.1) million, or ($0.43) per share, compared to ($15.1) million, or ($0.76) per share, in 2020.

On March 21, 2022 Aulos Bioscience, an immuno-oncology company working to revolutionize cancer care through the development of potentially best-in-class IL-2 therapeutics, reported that new preclinical data demonstrating solid tumor elimination when dosing AU-007, a monoclonal antibody computationally designed by Biolojic Design (Press release, Aulos Bioscience, MAR 21, 2022, View Source [SID1234610480]). Data were presented at a recent antibody biology and engineering conference.

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"These preclinical data strongly support AU-007’s unique mechanism of action as an antibody that can promote anti-cancer activity, including complete tumor elimination when used with checkpoint inhibitors in a murine model of cancer," said Aron Knickerbocker, Aulos Bioscience’s chief executive officer. "AU-007’s ability to redirect IL-2 toward immune activation and away from immune suppression and vascular endothelium is a novel approach to long-standing obstacles in immunotherapy. The new data underscore the potential we see in AU-007 as we prepare to initiate a Phase 1/2, first-in-human clinical trial of AU-007 in solid tumor indications."

To address the challenges of existing interleukin-2 (IL-2) treatments, Biolojic Design used its artificial intelligence platform to computationally design AU-007, a human antibody that precisely blocks IL-2 from binding to CD25 in trimeric receptors while preserving IL-2’s binding to dimeric CD122/CD132 receptors. Through this differentiated mechanism of action, AU-007 could promote immune effector activation while preventing T regulatory (Treg) cell expansion and vascular leak syndrome driven by IL-2. AU-007 has also been shown to break the autoinhibitory loop caused by endogenous IL-2 secreted from activated CD4 T helper cells and CD8 T effector cells that would otherwise lead to Treg expansion and immune suppression.

The new data demonstrate strong anti-cancer activity in murine models when AU-007 is dosed with a single dose of human IL-2 (hIL-2), as well as in combination with a single dose of hIL-2 and checkpoint inhibitors. In a recent murine preclinical study, AU-007 with a single loading dose of hIL-2 and PD-1 antibody demonstrated MC38 colorectal tumor regressions, with complete tumor elimination achieved in five of 10 cases. Additionally, AU-007 with a single loading dose of hIL-2 and PD-L1 antibody similarly demonstrated tumor regressions, with complete tumor elimination achieved in five of nine cases. AU-007 has been shown to be safe and well tolerated in primate toxicology studies (data not presented), does not cross-react with human tissues, and is on track to become the first computationally designed human antibody to enter clinical development.

In February, Aulos Bioscience announced it had received approval from the Monash Health Human Research Ethics Committee (HREC) to initiate a Phase 1/2, first-in-human trial of AU-007 in Australia. Aulos anticipates initiating enrollment and dosing in the first half of 2022.

The poster presentation is available on the Aulos Bioscience website.

About AU-007

AU-007 is a computationally designed, human IgG1 monoclonal antibody that is highly selective to the CD25-binding portion of IL-2. With a mechanism of action unlike any other IL-2 therapeutic in development, AU-007 leverages IL-2 to reinforce anti-tumor immune effects. This is achieved by preventing IL-2, either exogenous or secreted by T effector cells, from binding to trimeric receptors on T regulatory cells while still allowing IL-2 to bind and expand T effector and NK cells. This prevents the negative feedback loop caused by other IL-2-based treatments and biases the immune system toward activation over suppression. AU-007 also prevents IL-2 from binding to trimeric receptors on vasculature and pulmonary endothelium, which may significantly reduce the vascular leak syndrome and pulmonary edema associated with high-dose IL-2 therapy.

On March 21, 2022 Qu Biologics Inc., a private clinical stage biopharmaceutical company developing Site Specific Immunomodulators (SSIs), a novel platform of immunotherapies designed to restore innate immune function, reported the closing of a USD$12M equity financing to enable expansion of Qu’s team, initiation of an additional three Phase 2 clinical studies, and the acceleration of Qu’s novel immunomodulation platform (Press release, Qu Biologics, MAR 21, 2022, View Source [SID1234610479]).
Hal Gunn, MD, CEO of Qu Biologics, stated, "Our team is grateful for this new equity funding that will allow us to expand our dedicated team and play an essential role in initiating three new Phase 2 randomized placebo-controlled clinical studies. Two of these important trials are in critical indications for which there are no effective treatments – innate immune decline in the elderly and post-operative innate immune suppression."

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"We are highly motivated and excited to be in the position to strategically move forward with the expansion of our science, manufacturing, and clinical teams to accelerate the development of Qu’s first-in-class multifaceted immunotherapy platform, which is designed to safely restore and mobilize the essential effector functions of the innate immune system through a process that involves innate immune training and tissue-specific targeting of activated immune cells. In contrast to other immunotherapies that try to target one cell or receptor, SSI therapy harnesses and directs the multiple components of innate immunity required by the body to overcome pathology and heal," said Dr. Shirin Kalyan (PhD), Qu’s VP Scientific Innovation. Dr. Gunn added, "Given the broad application of our SSI platform, this funding brings Qu Biologics one step closer to providing critical, game-changing treatments for people living with cancer, infections, and chronic inflammatory diseases."
For more information about Qu Biologics and the science behind SSIs, please visit www.qubiologics.com.

On March 21, 2022 AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company developing first-in-class antibody product candidates focused on emerging immune control mechanisms applicable to inflammation and immuno-oncology indications, reported the appointment of Daniel Faga as interim president and chief executive officer (CEO), effective immediately (Press release, AnaptysBio, MAR 21, 2022, View Source [SID1234610477]). Mr. Faga currently serves on the company’s Board of Directors and will succeed Hamza Suria, who has stepped down from his role as president and CEO and as a board director. Mr. Suria will continue to support the Company in an advisory capacity.

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"We are highly confident in Dan’s leadership, who has extensive corporate strategy and public company experience in leading and advising world-class biopharmaceutical companies," said Jamie Topper, M.D., Ph. D., chairman of AnaptysBio’s Board of Directors. "On behalf of the entire Board, I want to thank Hamza for his contributions to AnaptysBio, including over 10 years of service as our CEO, resulting in a strong scientifically-oriented and financially sound organization that is developing a wholly-owned multi-asset clinical portfolio directed towards inflammatory diseases."

AnaptysBio is continuing to advance the development of its three wholly-owned pipeline programs in the following indications:

Imsidolimab (anti-IL-36R Ab) has demonstrated efficacy and safety in the GALLOP Phase 2 trial in generalized pustular psoriasis (GPP) and enrollment of the GEMINI-1 GPP Phase 3 trial is ongoing. Top-line data from the imsidolimab HARP Phase 2 trial in moderate-to-severe hidradenitis suppurativa is anticipated during the second half of 2022
Rosnilimab (PD-1 agonist Ab) enrollment of the AZURE Phase 2 trial in moderate-to-severe alopecia areata is ongoing
ANB032 (BTLA modulator Ab) healthy volunteer Phase 1 top-line data is anticipated in the second quarter of 2022
"Looking ahead, I am confident in the future of imsidolimab and our pipeline of innovative antibody-based therapies and pleased to help lead AnaptysBio into this next chapter. We look forward to evaluating further clinical data across all three of our clinical stage programs throughout 2022," said Mr. Faga. "In parallel, our Board of Directors will undergo a strategic portfolio review. This will include defining the clinical path forward across a breadth of potential inflammation-focused indications that could be pursued for each of our clinical and preclinical therapeutic antibody programs as well as the optimal deployment of our approximately $615 million in cash as of the end 2021."

Mr. Faga has been a member of AnaptysBio’s board of directors since December 2021. He is a seasoned executive with more than 20 years of industry and advisory experience in the life sciences industry. Most recently, he was the chief operating officer at Mirati Therapeutics responsible for leading the company’s strategy, corporate finance, legal and other business operations. Prior to Mirati, Dan was chief business officer at Spark Therapeutics until its acquisition by Roche. Previously, Dan was a managing director and founding member of Centerview Partner’s healthcare advisory practice. Dan’s earlier experience includes healthcare investment banking at Merrill Lynch and management consulting in the life sciences practice at PRTM. Dan has earned a B.S. in engineering from Cornell University and an M.B.A. in healthcare management from the Wharton School of the University of Pennsylvania.

On March 21, 2022 Nuance Pharma ("the Company") reported that it has entered into an exclusive license and supply agreement with Bavarian Nordic, a Copenhagen-based fully integrated vaccines company focused on the development, manufacturing and commercialization of life-saving vaccines, on the development and commercialization of MVA-BN RSV against respiratory syncytial virus (RSV) in adults in Chinese Mainland, Hong Kong, Macau, Taiwan, South Korea and certain Southeast Asian countries[1] (Press release, Bavarian Nordic, MAR 21, 2022, View Source [SID1234610475]). The agreement entails clinical development, including a Phase 3 trial to support regulatory approval of MVA-BN RSV in China, which will be conducted separately from Bavarian Nordic’s own Phase 3 trial planned for initiation later in the first half of 2022 to support a U.S. Biologics License Application.

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Under the terms of the agreement, Bavarian Nordic will receive an upfront payment of USD 12.5 million and is eligible to receive future milestone payments of up to USD 212.5 million that are triggered upon achievement of certain clinical, regulatory, and commercial milestones, in addition to tiered, double-digit royalties on future net sales.

Nuance Pharma will assume all costs and responsibility for the clinical development, regulatory filings, and commercialization of the vaccine in territories covered by the agreement. Subject to Chinese regulatory authority (National Medical Products Administration, NMPA) approval, Phase 1 and Phase 3 trials are planned for the vaccine approval in China.

The parties have also entered into a supply agreement by which Bavarian Nordic will assume future commercial manufacturing and supply of the vaccine.

"Nuance is thrilled to enter into this agreement with Bavarian Nordic on MVA-BN RSV, to set a new standard in the prevention of RSV, and to address the critical unmet medical needs of the highly vulnerable elderly patients. MVA-BN RSV represents an ideal fit for Nuance’s portfolio, given our focus in the respiratory space in Greater China and the Asian markets. This is an opportune moment for us to be developing and commercializing MVA-BN RSV in the Territory, riding the wave of appreciably heightened awareness to the benefits of vaccination in the COVID-era", commented Mark G. Lotter, CEO and Co-Founder of Nuance Pharma.

"We are pleased to partner with Nuance Pharma in our efforts to bring our late-stage RSV vaccine candidate to market. Their regional presence combined with a strong leadership and focus on respiratory diseases provides a unique first-to-market opportunity for MVA-BN RSV to fulfil the unmet need for a vaccine to prevent severe disease from RSV infections in the aging Chinese population. With our plans to initiate a self-funded Phase 3 trial of MVA-BN RSV in the USA and Germany later in the first half of 2022, we remain committed to the development and commercialization of the vaccine globally," said Paul Chaplin, President and CEO of Bavarian Nordic.

About Respiratory Syncytial Virus (RSV)

RSV is a common virus that usually causes mild, cold-like symptoms, but in serious cases can lead to severe lung infections, including bronchiolitis and pneumonia, which ultimately can lead to death. At-risk individuals typically include infants and older adults as well as immunocompromised individuals.

A prospective study in the U.S. has estimated the disease burden from RSV-induced infections and subsequent deaths to be similar to that of non-pandemic influenza in adults aged 65 years and older[2]. Accordingly, preventing RSV-induced infections is a top priority for governments and medical professionals globally. Currently there is no approved vaccine against RSV, thus representing a large and critical unmet medical need and a potential multi-billion-dollar vaccines market annually.

The Chinese target population for an RSV vaccine is estimated to be nearly 400 million people aged 60 years or older with an estimated annual growth rate of 3%.

About MVA-BN RSV

Bavarian Nordic’s vaccine candidate, MVA-BN RSV, is being developed for the prevention of respiratory syncytial virus (RSV) in older adults. The vaccine incorporates five distinct RSV antigens to stimulate a broad immune response against both RSV subtypes (A and B), thus mimicking the immune response observed following a natural response to an RSV infection. The vaccine candidate is based on Bavarian Nordic’s proven MVA-BN platform technology, which is employed in several approved vaccines.

In 2021, Bavarian Nordic reported results from a clinical Phase 2 double-blinded, placebo-controlled trial (n=61), which enrolled healthy adult volunteers, who were randomized to receive either a single vaccination of MVA-BN RSV or placebo. Volunteers were challenged intranasally with an RSV type A strain 28 days after vaccination. The study demonstrated a significant reduction in viral load in vaccinated subjects (n=30) versus placebo (n=31), thus meeting the primary endpoint. In the vaccinated subjects, clinical symptoms typically associated with RSV infections were significantly reduced. Further, the vaccine demonstrated an efficacy of up to 79% in preventing symptomatic RSV infections[3].

Bavarian Nordic has also previously reported strong results from a Phase 2 trial of MVA-BN RSV in 421 older adults, demonstrating that the vaccine was well-tolerated and induced both broad and durable antibody and T-cell responses against RSV, as well as mucosal immune responses that may be important for protection against RSV. The trial included a revaccination of subjects after one year, following which the immune responses were rapidly and significantly increased, notably in subjects with the weakest immunity prior to the booster vaccination[4].

MVA-BN RSV has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration for active immunization for prevention of lower respiratory tract disease caused by RSV in adults aged 60 years or older.