On May 13, 2021 Affimed N.V. (Nasdaq: AFMD), a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer, reported the publication of preclinical in vitro and in vivo research of its lead innate cell engager (ICE), AFM13 (CD16A/CD30), combined with healthy donor-derived NK cells in Clinical Cancer Research (Press release, Affimed, MAY 13, 2021, View Source [SID1234580037]). The preclinical data demonstrated that AFM13 strongly binds to NK cells, including cytokine-activated or cord blood-derived NK (cbNK) cells, resulting in enhanced tumor recognition and antibody-dependent cellular cytotoxicity (ADCC). The research was generated through a collaboration with The University of Texas MD Anderson Cancer Center and Washington University School of Medicine and supports use of AFM13 combined with NK cells as a promising therapy for CD30-positive hematological malignancies.

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"Our ROCK platform forms the basis to generate ICE molecules which have the ability to strongly and durably bind to CD16A on NK cells, resulting in unique antitumor properties," said Arndt Schottelius, M.D., Ph.D., Chief Scientific Officer of Affimed. "As demonstrated by the recent presentation of initial Phase 1 data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021 – which showed an emerging profile that appears to have the potential to provide meaningful benefit with a safety profile consistent with previous AFM13 data – pre-complexing AFM13 with NK cells presents an innovative and promising therapeutic approach for patients with impaired NK cell activity."

"This study provided new insights into how ICE molecules such as AFM13 may be impacted by NK cell receptor ligand alterations using multidimensional mass cytometry," said Todd Fehniger, M.D., Ph.D., Professor of Medicine, Oncology Division, at Washington University. "Further, blood NK cells primed and differentiated into memory NK cells exhibit potent responses to CD30+ cancer cells when directed by AFM13, providing further evidence for their powerful CD16-triggered cytokine production and killing."

Additional key findings from the research are outlined below:

AFM13 combined with donor NK cells, including conventional NK cells from healthy donors, cytokine-induced memory-like NK cells from peripheral blood and preactivated and expanded cbNK cells, enhanced tumor cell killing compared to NK cells alone.
When combined with AFM13, Hodgkin lymphoma patient-derived NK cells do not reach the same level of cytotoxicity compared to healthy donor-derived NK cells in vitro.
AFM13-directed tumor cell killing was enhanced when combined with cytokine (IL-12, IL-15 and IL-18) preactivated cbNK cells compared to non-cytokine preactivated cbNK cells.
Cytokine preactivated cbNK cells express different markers when compared to noncytokine preactivated cbNK cells, potentially accounting for superior cytotoxicity which is further enhanced with AFM13.
"This preclinical study confirmed the synergy between the cbNK cell platform developed at MD Anderson and AFM13 as a precomplexed product and provided the rationale to test this novel NK cell-based adoptive immunotherapy strategy for patients with relapsed/refractory CD30+ malignancies," said Katy Rezvani, M.D., Ph.D., Professor of Stem Cell Transplantation and Cellular Therapy at MD Anderson.

The preclinical data published in Clinical Cancer Research supported the Investigational New Drug (IND) application for the ongoing Phase I clinical study of AFM13 precomplexed with cytokine-preactivated cbNK cells followed by AFM13 monotherapy in patients with CD30-positivemalignancies. Results of the Phase 1 study as of March 31, 2021, demonstrated an objective response rate of 100% (ORR=4/4; PR=2/4; CR=2/4) among the first patients enrolled who were all heavily pretreated. There were no observed events of cytokine release syndrome, neurotoxicity, or graft-versus-host disease. The study will progress to the higher dose cohorts with additional updates expected throughout this year.

About AFM13
AFM13 is a first-in-class innate cell engager (ICE) that uniquely activates the innate immune system to destroy CD30-positive hematologic tumors. AFM13 induces specific and selective killing of CD30-positive tumor cells, leveraging the power of the innate immune system by engaging and activating natural killer (NK) cells and macrophages. AFM13 is Affimed’s most advanced ICE clinical program and is currently being evaluated as a monotherapy in a registration-directed trial in patients with relapsed/refractory peripheral T-cell lymphoma or transformed mycosis fungoides (REDIRECT). The study is actively recruiting, and additional details can be found at www.clinicaltrials.gov (NCT04101331).

In addition, The University of Texas MD Anderson Cancer Center is studying AFM13 in an investigator-sponsored Phase 1 trial in combination with cord blood-derived allogeneic NK cells in patients with recurrent or refractory CD30-positive lymphomas. The study is a dose-escalation trial of precomplexed NK cells, with patients receiving 1×106 NK cells/kg in Cohort 1, 1×107 NK cells/kg in Cohort 2, and 1×108 NK cells/kg in Cohort 3. The trial is designed to explore safety and activity and determine the recommended Phase 2 dose. In each cohort, the dose of the precomplexed NK cells with AFM13 is to be followed by weekly doses of 200 mg AFM13 monotherapy for three weeks, with each patient evaluated for dose-limiting toxicities and responses on day 28.

MD Anderson has an institutional financial conflict of interest with Affimed related to this research and has therefore implemented an Institutional Conflict of Interest Management and Monitoring Plan.

On May 13, 2021 Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, reported financial results for the first quarter ended March 31, 2021 and provided a corporate update (Press release, Decibel Therapeutics, MAY 13, 2021, View Source [SID1234585162]).

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"The start of 2021 marked a pivotal time for Decibel as we successfully closed our upsized IPO. With this strong financial foundation, we are in a position to further advance our exciting pipeline of clinical, preclinical and discovery programs with the overarching goal of bringing the privileges of hearing and balance to people in need. In early 2021, we announced encouraging preclinical results on our lead gene therapy program, DB-OTO, for the treatment of people with otoferlin deficiency. We also established a critical cGMP manufacturing and development relationship with Catalent," said Laurence Reid, Ph.D., Chief Executive Officer of Decibel. "Beyond DB-OTO, we remain encouraged by the progress in our gene therapy programs for congenital, monogenic hearing loss and look forward to announcing the program target for our AAV.104 program in 2021."

Company Highlights

Closed Second Tranche of Series D Financing and Upsized Initial Public Offering: In February 2021, Decibel completed its Series D financing, closing the second tranche and receiving net proceeds of $27.4 million. Additionally, Decibel completed its Initial Public Offering (IPO) of 7,662,000 shares of common stock at a public offering price of $18.00 per share. Net proceeds from the offering, after deducting underwriting discounts and offering expenses, were approximately $124.8 million.
Pipeline Progress

Gene Therapies for Congenital, Monogenic Hearing Loss

Continued Development of Lead Gene Therapy Program, DB-OTO, for Otoferlin (OTOF): In February 2021, Decibel established manufacturing capabilities for its lead gene therapy product candidate, DB-OTO. Under the new agreement, Catalent will provide Decibel with cGMP manufacturing and process and analytical development of DB-OTO.
Presented New Preclinical Data on DB-OTO and Surgical Approach at the 44th Annual Association for Research in Otolaryngology Conference (ARO) and the Annual Scientific and Technology Conference of the American Auditory Society: In the first quarter of 2021, Decibel presented new preclinical findings to support the development of DB-OTO. Presentations highlighted data demonstrating that the Company’s proprietary dual vector technology and cell-selective promoter enabled expression of OTOF in hair cells and durably restored hearing in mice and drove highly selective expression of a reporter gene in hair cells of non-human primates across the cochlear length.
On Track for DB-OTO Key Milestones in 2022: Decibel expects to submit an investigational new drug application (IND) with the U.S. Food and Drug Administration (FDA) and/or a Clinical Trials Application (CTA) in Europe and initiate a Phase 1/2 clinical trial for DB-OTO in pediatric patients with congenital hearing loss in 2022.
Preclinical Pipeline Expansion Continues: Decibel expects to announce the program target for its AAV.104 discovery program in patients with autosomal recessive hearing disorders in 2021.
Gene Therapies for Hair Cell Regeneration

Preclinical Pipeline Expansion Continues: Decibel continues to advance DB-ATO and AAV.201, its gene therapy programs for regeneration of hair cells in the vestibule for the treatment of bilateral vestibulopathy, and its gene therapy program to regenerate hair cells in the cochlea for the treatment of sensorineural hearing loss. Based on findings from recently completed behavioral studies of DB-ATO, the Company did not see sufficient functional recovery to continue to move DB-ATO to development candidate in 2021. The Company plans to announce the program target for AAV.201 in 2022.
Otoprotection Therapeutic

Updated Timeline to Report Interim Results from Phase 1b Proof-of-Concept Trial of DB-020 for the Treatment of Cisplatin-Induced Hearing Loss: The reporting of interim results from the ongoing Phase 1b clinical trial of DB-020 in patients with cisplatin-induced hearing loss is now expected in the first half of 2022 due to continued impact of the COVID-19 pandemic on the pace of patient recruitment in the United States. Due to COVID-19 restrictions, sites in the United States have been delayed in recruiting, but are now open and actively recruiting for the trial, along with the active sites in Australia.
First Quarter 2021 Financial Results:

Cash Position: As of March 31, 2021, cash, cash equivalents and available-for-sale securities were $191.1 million, compared to $54.3 million as of December 31, 2020. The increase in cash, cash equivalents and available-for-sale securities was due to the sale of the Company’s Series D convertible preferred stock and common stock in the Company’s IPO completed in February 2021.
Research and Development Expenses: Research and development expenses were $6.0 million for the first quarter of 2021, compared to $7.4 million for the first quarter of 2020. The decrease in research and development expenses for the first quarter of 2021 was primarily due to $1.8 million decrease in personnel-related costs due to reduced headcount, driven primarily by a reduction-in-force conducted in January 2020 and a $0.8 million decrease in expenses incurred for our DB-020 program driven by decreased activity as a result of delays due to the COVID-19 pandemic, partially offset by an increase of $1.2 million in other indirect research and development expenses.
General and Administrative Expenses: General and administrative expenses were $4.9 million for the first quarter of 2021, compared to $4.2 million for the same period in 2020. The increase in general and administrative expenses for the first quarter of 2021 was primarily attributable to $1.0 million increase in professional fees, driven primarily by expenses related to consulting, accounting advisory and audit services incurred as a result of becoming a public company in February 2021.
Financial Guidance:

Based on its current operating and development plans, Decibel believes that its existing cash, cash equivalents and available-for-sale securities will fund its pipeline programs and operating expenses into 2024.

On May 13, 2021 Y-mAbs Therapeutics, Inc. (the "Company" or "Y-mAbs") (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported that Nai-Kong V. Cheung, MD, PhD, Enid A. Haupt Endowed Chair, Pediatric Oncology, Memorial Sloan Kettering Cancer Center ("MSK") will present a research update on the Company’s SADA technology platform at PEGS Boston Virtual Conference on May 13, 2021 at 1:20 p.m. Eastern Time (Press release, Y-mAbs Therapeutics, MAY 13, 2021, View Source [SID1234579902]). The SADA technology was licensed by the Company from MSK and the Massachusetts Institute of Technology ("MIT").

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Dr. Cheung’s presentation will focus on cancer therapeutics failing in development because of dose-limiting toxicities or subtherapeutic dosing as a consequence of insufficient therapeutic index ("TI"). The two step SADA technology uses unique pharmacokinetics to potentially improve the TI. When applied to pre-targeted radioimmunotherapy ("PRIT"), the bispecific SADA antibodies visualize tumors with high precision using PET (diagnostic approach) and has been shown to ablate aggressive solid tumors using both beta and alpha emitters such as 177Lu or 225Ac (therapeutic approach), potentially without significant toxicity to bone marrow, liver, kidney, or CNS. Furthermore, the modularity of the SADA platform might allow easy adaptation to different tumor targets and a variety of payloads.

Researchers at MSK, including Dr. Cheung, developed the SADA technology for radioimmunotherapy, which is exclusively licensed by MSK to Y-mAbs. Dr. Cheung has intellectual property rights and interests in the technology, and as a result of this licensing arrangement, MSK has institutional financial interests in the technology and Y-mAbs.

On May 13, 2021 Alpine Immune Sciences, Inc. (NASDAQ: ALPN), a leading clinical-stage immunotherapy company focused on developing innovative treatments for cancer and autoimmune/inflammatory diseases, reported financial results for the first quarter ended March 31, 2021 (Press release, Alpine Immune Sciences, MAY 13, 2021, View Source [SID1234579918]).

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"The first quarter of this year has been highly productive as we continue to make strong progress across our pipeline. We look forward to sharing the first clinical data update on NEON-1, a Phase 1 dose escalation and expansion study of ALPN-202 monotherapy, at the upcoming ASCO (Free ASCO Whitepaper) Annual Meeting," said Mitchell H. Gold, M.D., Executive Chairman and Chief Executive Officer of Alpine. "In addition, we continue our focus on the imminent global Phase 2 study of ALPN-101 in systemic lupus erythematosus, targeted to initiate around the middle of the year."

First Quarter 2021 and Recent Updates

ALPN-202: Conditional CD28 costimulator and dual checkpoint inhibitor
In April 2021, at the 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting I, Dr. Mark Voskoboynik from Nucleus Network and The Alfred Hospital in Melbourne, Australia, presented a Trials in Progress poster describing the ongoing first-in-human, Phase 1 clinical trial involving monotherapy with ALPN-202, the company’s lead oncology program.
In April 2021, Alpine announced an upcoming clinical data presentation on NEON-1 at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting scheduled to take place June 4, 2021.
Research
In April 2021, at the 2021 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting I, Alpine researchers demonstrated the use of directed evolution to engineer novel variant immunoglobulin domain (vIgD) Fc fusion proteins that enable tumor antigen-dependent CD28 costimulation.
General Corporate
Promotion of Remy Durand, Ph.D. to Chief Business Officer: Dr. Durand joined Alpine Immune Sciences in 2018 as Vice President, Business Development and Corporate Strategy, and has played a leading role in building the company’s partnerships with AbbVie and Adaptimmune, and has represented the company at investor meetings and conferences.
Appointed Pamela Holland, Ph.D. as Senior Vice President, Research: Dr. Holland is an experienced cancer biologist with a proven track record of successfully discovering and progressing multiple preclinical therapeutics into clinical development, most recently at Surface Oncology and Amgen.
First Quarter 2021 Financial Results

As of March 31, 2021, we had cash, cash equivalents, restricted cash, and investments totaling $115.4 million. Net cash used in operating activities for the quarter ended March 31, 2021 was $16.0 million compared to net cash used in operating activities of $9.7 million for the quarter ended March 31, 2020. We recorded net losses of $10.6 million and $5.5 million for the quarters ended March 31, 2021 and 2020, respectively.

Collaboration revenue for the quarter ended March 31, 2021 was $3.2 million and related to our agreement with AbbVie, compared to $1.1 million related to our agreement with Adaptimmune for the quarter ended March 31, 2020.

Research and development expenses for the quarter ended March 31, 2021 were $10.4 million compared to $4.9 million for the quarter ended March 31, 2020. The increase was primarily attributable to increases in contract manufacturing and process development of our product candidates, clinical trial activities, and personnel-related expenses.

General and administrative expenses for the quarter ended March 31, 2021 were $3.3 million compared to $1.8 million for the quarter ended March 31, 2020. The increase was primarily attributable to increases in professional and legal services, personnel-related expenses, and insurance and facility costs to support the growth and expansion of our business.

Alpine expects that its current cash resources, combined with the potential $75 million in pre-option exercise milestones payable under its option and license agreement with AbbVie, for the development and commercialization of ALPN-101, are sufficient to fund Alpine’s planned operations through 2023.

On May 13, 2021 SELLAS Life Sciences Group, Inc. (Nasdaq: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on developing novel cancer immunotherapies for a broad range of indications, reported its financial results for the quarter ended March 31, 2021 and provided a business update (Press release, Sellas Life Sciences, MAY 13, 2021, View Source [SID1234579934]).

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"Under our exclusive license agreement with 3D Medicines for the development and commercialization of galinpepimut-S (GPS) in Greater China, we advanced the technology transfer to 3D Medicines during the first quarter of 2021. In February, our progress triggered a milestone payment of $1.0 million. This achieved milestone is the first of milestone payments that have the potential to total $194.5 million over the life of the agreement. In addition to the $1.0 million payment, we further strengthened our balance sheet during the first quarter with approximately $3.0 million in net proceeds from warrant exercises," said Angelos Stergiou, MD, ScD. h.c., President and Chief Executive Officer of SELLAS. "During the quarter, we continued to progress our GPS clinical program with the activation of additional clinical sites in the United States, and also preparations for opening clinical sites and enrolling patients in European countries, for our Phase 3 REGAL study of GPS in patients with acute myeloid leukemia (AML). Screening and enrollment of patients for the REGAL study is ongoing in the United States."

Pipeline Updates:

Galinpepimut-S (GPS)

During the first quarter of 2021, SELLAS triggered and received a milestone payment in the amount of $1.0 million related to the completion of a technology transfer plan under its license agreement with 3D Medicines.

In April 2021, the Company received National Ethics Committee approval in Greece for the Phase 3 REGAL study in patients with AML. The Company expects to activate sites and begin enrolling patients in France, Germany and Greece by the end of the second quarter of 2021.
Nelipepimut-S (NPS)

In January 2021, the data safety monitoring board for the ongoing investigator sponsored study of NPS plus trastuzumab in high risk HER2 3+ breast cancer patients recommended that, given the small size of the study and in order to preserve the statistical power of the study, the primary analysis of the study be completed upon the completion of three years of follow-up on every patient or until more events are collected. The Company expects the primary analysis in this study to be completed by the end of 2021.

In February 2021, the subgroup analysis of the cohort of patients with triple negative breast cancer (TNBC) from the Phase 2b investigator-sponsored study of NPS plus trastuzumab in HER2 low-expressing breast cancer patients was published in the peer-reviewed journal Clinical Immunology. As previously reported, the subset analysis identified significant improvement in 36-month disease-free survival between NPS (n=55) and placebo (n=44) in TNBC.
Corporate Highlights for the First Quarter 2021:

The Company received approximately $3.0 million in net proceeds from the exercise of common stock warrants.
Financial Results for the First Quarter 2021:

Licensing revenue: During the first quarter of 2021 the Company recorded $5.7 million of licensing revenue which consists of the recognition of revenue from the upfront license fee received from 3D Medicines in 2020 and the milestone payment received in the first quarter of 2021 from the achievement of a milestone under the Company’s license agreement with 3D Medicines. The Company did not record any licensing revenue for the first quarter of 2020.

R&D Expenses: Research and development expenses for the first quarter of 2021 were $4.3 million, as compared to $1.9 million for the first quarter of 2020. The increase was primarily due to a ramp up of the manufacture of clinical trial materials and registration batches of GPS, a technology transfer to a new contract manufacturer, and clinical drug supply purchase costs in the European Union in preparation for opening sites and enrolling patients in European Union countries.

G&A Expenses: General and administrative expenses for the first quarter of 2021 were $3.6 million, as compared to $2.2 million for the first quarter of 2020. The increase was primarily due to amortization expense associated with the capitalized contract acquisition costs of the 3D Medicines license agreement as well as an increase in legal fees.

Net Loss: Net loss attributable to common stockholders was $2.4 million for the first quarter of 2021, or a basic and diluted loss per share attributable to common stockholders of $0.16, as compared to a net loss attributable to common stockholders of $4.2 million for the first quarter of 2020, or a basic and diluted loss per share attributable to common stockholders of $0.66.

Cash Position: As of March 31, 2021, cash and cash equivalents totaled approximately $28 million.