On May 11, 2021 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported that Walter Klemp, President and Chief Executive Officer of Moleculin, will present at the Q2 Virtual Investor Summit on Monday, May 17th at 12:30 PM ET (Press release, Moleculin, MAY 11, 2021, View Source [SID1234579690]).

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Moleculin Biotech, Inc. is a clinical stage pharmaceutical company focused on the development of a broad portfolio of oncology drug candidates for the treatment of highly resistant tumors. (PRNewsfoto/Moleculin Biotech, Inc.)

In addition to the presentation, management will be available to participate in virtual one-on-one meetings with qualified members of the investor community who are registered to attend the conference. For more information about the conference, please visit the conference website.

A live video webcast will be accessible on the Events page in the Investors section of the Company’s website (www.moleculin.com) and archived for 90 days following the event.

On May 11, 2021 Prelude Therapeutics Inc. (Nasdaq: PRLD), a clinical-stage precision oncology company, reported its financial results for the first quarter ended March 31, 2021 and provided an update on recent clinical and development pipeline progress (Press release, Prelude Therapeutics, MAY 11, 2021, View Source [SID1234579689]).

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"With several novel, internally discovered therapeutics now advancing in clinical trials, our pipeline products are off to a strong start in 2021 and we expect this momentum to carry us through multiple key inflection points," said Kris Vaddi, PhD, Chief Executive Officer. "We initiated numerous expansion cohorts for PRT543, our novel PRMT5 inhibitor, in the ongoing Phase 1 trial in patients with advanced solid tumors and hematologic malignancies. We expect to present initial clinical data for both PRT543 and PRT811, our brain penetrant PRMT5 inhibitor, in the second half of the year."

Dr. Vaddi added, "We continue to enroll the dose escalation portion of our Phase 1 trial of oral PRT1419, our MCL1 inhibitor, and plan to initiate dose expansion and combination cohorts in the second half of the year. Beyond our clinical pipeline, we remain focused on the advancement of our preclinical programs, and expect to submit an IND application for PRT2527, our CDK9 inhibitor, by year end. We are steadfast in our mission to develop novel therapeutic options for several cancers with high unmet need, and we look forward to providing updates on our continued progress as these programs mature."

Recent Highlights and Upcoming Milestones

PRT543

Enrollment Ongoing in Phase 1 Dose Expansion Cohorts. Patient enrollment is underway in additional solid tumor and hematologic malignancy expansion cohorts of the Company’s Phase 1 trial of PRT543, which is designed to be a potent and selective inhibitor of PRMT5. As previously announced, preliminary data from the dose escalation portion of the trial demonstrated early signs of clinical activity and tolerability. The Company anticipates presenting initial clinical data from the Phase 1 trial at medical meetings in the second half of 2021.

Preclinical Data Featured at the 2021 AACR (Free AACR Whitepaper) Annual Meeting. Three preclinical presentations on PRT543 were featured at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2021. A copy of each poster is available in the Publications section of the Prelude Therapeutics website.
PRT811

Phase 1 Dose Expansion Cohorts Remain on Track to Begin in Mid-2021. The dose escalation portion of the Company’s Phase 1 trial of its second clinical product candidate, PRT811, which is designed to be a potent, selective, and brain penetrant PRMT5 inhibitor, remains ongoing in patients with advanced solid tumors, including gliomas. As previously reported, the trial has demonstrated early signs of clinical activity and tolerability. Prelude remains on track to establish a recommended expansion dose and commence the dose expansion portion of the trial in mid-2021 in patients with central nervous system cancers, including glioblastoma multiforme (GBM), with initial clinical data expected by the end of 2021.
PRT1419

Oral Formulation: Planned Dose Expansion and Combination Cohorts Expected to be Added to Ongoing Phase 1 Trial in the Second Half of 2021. The dose escalation portion of the Company’s first-in-human Phase 1 study of oral PRT1419 in patients with relapsed/refractory hematologic malignancies, including acute myeloid leukemia and high-risk myelodysplastic syndromes, is ongoing. PRT1419, which is the Company’s third clinical candidate, is designed to be an orally available, potent, and selective MCL1 inhibitor. The Company expects to add dose expansion and combination cohorts to the Phase 1 clinical trial in the second half of 2021.

IND Application Cleared. In March 2021, the Company announced that the U.S. Food and Drug Administration (FDA) cleared its Investigational New Drug (IND) application for an intravenous (IV) formulation of PRT1419. A Phase 1 trial of the IV formulation, which leverages the optimized physicochemical properties of PRT1419, is expected to commence in the coming months in patients with solid tumors.

Data on Preclinical Characterization Featured at the 2021 AACR (Free AACR Whitepaper) Annual Meeting. Data on the preclinical characterization of PRT1419 was featured at the AACR (Free AACR Whitepaper) Annual Meeting in April 2021. A copy of the poster is available in the Publications section of the Prelude Therapeutics website.
Discovery Programs

Advancement of Earlier-Stage Candidates Expected in 2021. The Company remains on track to submit an IND application in 2021 for PRT2527, which is designed to be a potent and selective CDK9 inhibitor. Prelude also continues to expect to initiate IND-enabling studies for PRT-SCA2, which is designed to be a SMARCA2 protein degrader, in 2021.

Preclinical Data on SMARCA2 Protein Degradation Featured at the 2021 AACR (Free AACR Whitepaper) Annual Meeting. A poster presentation on the Company’s SMARCA2 protein degradation program was featured at the AACR (Free AACR Whitepaper) Annual Meeting in April 2021. A copy of the poster is available in the Publications section of the Prelude Therapeutics website.
First Quarter 2021 Financial Results

Cash and Cash Equivalents: Cash and cash equivalents as of March 31, 2021 were $363.0 million.

Research and Development (R&D) Expenses: For the first quarter of 2021, R&D expense increased by $7.9 million to $16.5 million. The increase was mainly due to increased clinical research costs for the PRT543 and PRT811 clinical trials and increased costs associated with the initiation of the clinical trial for oral PRT1419, as well as preparation for the anticipated initiation of the Phase 1 trial for IV PRT1419 in the first half of 2021. We also incurred an increase in chemistry, manufacturing, and other costs for those trials.

General and Administrative (G&A) Expenses: For the first quarter of 2021, G&A expense increased by $4.3 million to $5.5 million. The increase was primarily due to an increase in personnel related expense due to increases in employee headcount and an increase in the Company’s professional fees as it expanded operations to support R&D efforts and incurred additional costs to operate as a public company.

Net Loss: For the first quarter of 2021, net loss was $21.3 million, or $0.47 per share, compared with a net loss of $9.5 million, or $5.12 per share, for the same period in 2020.

Financial Guidance: The Company believes that its current cash and cash equivalents will be sufficient to fund operating expenses and capital expenditure requirements into 2023.

On May 11, 2021 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in cell therapy to treat cancer, reported preclinical data from its HiT targeting mesothelin, being co-developed with Astellas, during a poster presentation at the American Society for Cell and Gene Therapy (ASGCT) (Free ASGCT Whitepaper) meeting today (Press release, Adaptimmune, MAY 11, 2021, View Source [SID1234579688]).

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"These data are very exciting because our HLA-independent TCR targeting mesothelin induced complete regression in a mouse tumor xenograft model and also outperformed a TRuC construct that we developed in-house," said Karen Miller, SVP, Pipeline Research. "The natural signaling machinery of the TCR, as well as our proprietary TCR engineering and affinity optimization expertise, offer distinct advantages over cell therapies that rely on antibody moieties to engage antigen. We are evaluating further HiT targets, as we deliver against our 2-2-5-2 strategic plan to bring new cell therapy products forward for clinical development and launch."

Adaptimmune has developed a T-cell receptor (TCR) that can directly bind to the cell surface protein mesothelin, independent of HLA, using the Company’s proprietary naïve phage display libraries and affinity optimization techniques. The HLA-independent TCR or HiT platform enables the Company to target extracellular proteins, such as those utilized by CAR or TRuC T-cell therapies. Data from the ASGCT (Free ASGCT Whitepaper) presentation are summarized below.

Adaptimmune’s HiTs are CD8 and HLA-independent and kill mesothelin expressing human tumor cells

Human T-cells expressing the mesothelin-targeted HiT kill human tumor cell lines and primary human lung tumor cells expressing mesothelin
Data demonstrate that the mesothelin HiT is not dependent on CD8 to kill target cells, and CD4 cells expressing the HiT are cytotoxic towards tumor cells
A TRuC construct was developed by Adaptimmune as a comparator for these experiments, which also killed mesothelin target cells independently of CD8
Adaptimmune’s HiTs are not neutralized by free mesothelin, but the TRuC construct is

Data from additional in vitro experiments clearly show that Adaptimmune’s HiT is not neutralized by soluble mesothelin, unlike the TRuC construct
It has been described that CAR-T and similar therapies, which rely on high affinity antibody moieties to engage antigen, can be neutralized by soluble target protein, inhibiting their function1
This is particularly important for many tumor targets that are both cell-surface bound and secreted, as is the case with mesothelin
Adaptimmune’s HiT T-cells targeting mesothelin induce complete tumor regression in a mouse xenograft tumor model and outperform a comparator TRuC construct

Immunodeficient mice were implanted with human pancreatic Capan-2 mesothelin-expressing tumor cells
Mice with pre-established tumors received HiT T-cells or TRuC comparator T-cells by a single IV injection
Data showed a strong, dose-dependent, and persistent tumor regression with HiT T-cells
TRuC T-cells only inhibited tumor growth at comparative doses to those used with the HiT T-cells

On May 11, 2021 Phio Pharmaceuticals Corp. (Nasdaq: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (INTASYL) therapeutic platform, reported positive new in vivo data showing that PH-762 significantly enhanced the antitumor efficacy of HER2-targeted CAR-T cells (HER2CART) in solid tumors (Press release, Phio Pharmaceuticals, MAY 11, 2021, View Source [SID1234579687]). Compared to untreated HER2CART cells, HER2CART cells treated with PH-762 showed a statistically significant and durable inhibition of tumor growth. These data, using a HER2-targeted CAR-T cell product against a HER2-expressing ovarian cancer xenograft model, provide proof-of-concept for the application of PD-1 checkpoint silencing with INTASYL in CAR-T cells prior to adoptive cell therapy to enhance the therapeutic efficacy of CAR-T cell therapy in solid tumors.

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"The in vivo data announced today further support advancing the development of PH-762 into the clinic as a viable approach to improve various forms of T cell based immunotherapy. These data are especially impressive considering that to date CAR T-cell therapy effectiveness in solid tumors has been disappointing and the use of additional genetic engineering to address these issues has proven challenging and costly. Indeed, in a recent clinical study it was shown that CRISPR-Cas9 mediated PD-1 disruption resulted in low efficiency, namely an editing efficiency of less than 6% and a median disruption of PD-1 expression of less than 50% in the edited T cells. This compares sharply with our results where we show that our INTASYL compound, PH-762, achieves PD-1 silencing efficiency of ~90% in nearly 100% of the HER2CART cells used in this study," stated Dr. Simon Fricker, Phio’s VP of Research. "In addition, these results are achieved by merely adding PH-762 to the HER2CART cell culture media, without the need for cell delivery vehicles or vectors, and without negative impact on cell growth/survival. In a prior presentation we also showed how PH-762 can improve the tumor cell killing activity of another adoptive cell therapy platform, namely tumor infiltrating lymphocytes. Taken together, you can start to see the full picture of the potential advancement that INTASYL could provide in adoptive cell therapy."

In this study the Company assessed the potential of PH-762, a PD-1 targeting INTASYL compound, to enhance the therapeutic efficacy of HER2CART cells in the treatment of a subcutaneous HER2-expressing SKOV3 model of human ovarian cancer in mice. On-target silencing of PD-1 in vitro was demonstrated in a dose associated manner in activated HER2CART cells, without significant impact on viability, and resulted in an enrichment of CD8+ and CD25+ cells. Analyses of PH-762-treated HER2CART cells isolated from tumors suggest that PH-762 enhances CAR-T function through multiple mechanisms including enhanced efficiency, degranulation, decreased suppressive potential, and promotion of memory/stem populations.

These data were presented today during the 24th Annual Meeting of the ASGCT (Free ASGCT Whitepaper) in a poster titled "INTASYL PH-762 Self-Delivering RNAi Targeting PD-1 Enhances the Therapeutic Efficacy of Systemically Administered HER2-Targeted CAR-T Cells in a SKOV3 Model of Human Ovarian Adenocarcinoma in NCG Mice". An archived version of the poster presentation will be made available on the "Investors – Events and Presentations" section of the Company’s website (click here).

On May 11, 2021 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported recent business highlights and pipeline updates, as well as first quarter 2021 financial results (Press release, Beam Therapeutics, MAY 11, 2021, View Source [SID1234579686]).

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"As Beam prepares to enter the clinic, we also continue to extend our leadership position in the field of base editing by expanding our platform and delivery capabilities," said John Evans, chief executive officer of Beam. "The data presented at ASGCT (Free ASGCT Whitepaper) on our proprietary lipid nanoparticle (LNP) formulation show efficient in vivo base editing in hepatocytes of non-human primates (NHPs), while our novel analytical assays confirm specificity and efficiency of base editing to directly correct disease-causing mutations. As we look to the rest of 2021, we are on-track with our plans to submit our first Investigational New Drug (IND) application for BEAM-101 and initiate IND-enabling studies for BEAM-102 and BEAM-201, as well as nominate our first development candidate from our liver portfolio. We are well positioned today to advance our base editing platform and pipeline and remain focused on achieving our vision of providing life-long cures for patients suffering from serious diseases."

"We are pleased to share our development of a proprietary LNP formulation for non-viral in vivo delivery to the liver at ASGCT (Free ASGCT Whitepaper)," said Giuseppe Ciaramella, Ph.D., president and chief scientific officer of Beam. "These data demonstrate that our LNPs are well tolerated in NHPs and can achieve levels of editing of hepatocytes that we expect would be therapeutic for many genetic diseases. Initial data also demonstrated the stability of our LNPs at -20 degrees Celsius after 8 weeks, which is important for enabling broad clinical and commercial development. We are continuing to work on optimizing our LNP formulations and are on track to nominate our first development candidate from our liver portfolio in the second half of 2021."

Base Editing Progress

Data Demonstrating Optimization of LNP-mRNA Formulation for Liver Editing Presented at the 24th American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting: Beam’s approach to developing a novel lipid nanoparticle (LNP) formulation for in vivo liver editing will be presented during a poster session titled, "Using Base Editing and LNP Delivery to Correct Disease-Causing Mutations Underlying Genetic Liver Diseases" at ASGCT (Free ASGCT Whitepaper). Using an mRNA-encoding adenine base editor (ABE) and guide RNA to target the ALAS1 gene, a surrogate payload for genetic liver diseases, Beam evaluated various LNP formulations and mRNA production processes to improve in vivo editing in the livers of NHPs from less than 10% initially to 52% at a total RNA dose of 1.5 mg/kg. These formulations were also well tolerated by NHPs at 1.5 mg/kg with mild and transient liver enzyme elevations, and showed promising interim stability, maintaining potency after 8 weeks at -20⁰C.
Data Highlighting Beam’s Approach to Confirming Precision Correction Using Novel Analytical Assays Presented at ASGCT (Free ASGCT Whitepaper): Base editing is emerging as a powerful next-generation editing technology; however, developing adequate analytical assays to confirm precise base editing at the protein level is critical and has proven historically challenging. In an oral presentation at ASGCT (Free ASGCT Whitepaper) titled, "LC-MS Confirmation of Single Amino Acid Correction by Base Editing," Beam will describe its approach to using liquid chromatography mass spectrometry (LC-MS) for multiple analytical assays to confirm the precise correction of disease-causing mutations at the amino acid level, providing a unique solution for confirmation and quantitation of single amino acid corrections after base editing.
Data Highlighting Ability to Rationally Design Base Editors for Precise Editing Published in The CRISPR Journal: In April 2021, work describing Beam’s approach to developing inlaid base editors (IBEs) was published in The CRISPR Journal. IBEs are architectural variants of base editors that demonstrate enhanced specificity and altered activity windows relative to foundational base editors. The work highlights Beam’s application of its IBEs for BEAM-102, one of its base editing programs in development for the treatment of sickle cell disease.
First Quarter 2021 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $503.5 million as of March 31, 2021, compared to $253.4 million as of March 31, 2020.
Research & Development (R&D) Expenses: R&D expenses were $190.1 million for the first quarter of 2021, compared to $21.5 million for the first quarter of 2020. R&D expenses for the first quarter of 2021 includes $155.0 million of expense related to in-process research and development acquired from of Guide Therapeutics, Inc.
General & Administrative (G&A) Expenses: G&A expenses were $10.3 million for the first quarter of 2021, compared to $6.8 million for the first quarter of 2020.
Net Loss: Net loss attributable to common stockholders was $201.6 million, or $3.35 per share, for the first quarter of 2021, compared to $31.7 million, or $1.03 per share, for the first quarter of 2020.