On August 9, 2022 Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, reported financial results for the second quarter ended June 30, 2022 (Press release, Beam Therapeutics, AUG 9, 2022, View Source [SID1234617934]).

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"2022 is a critical year for Beam’s transition to becoming a multi-program clinical-stage company, as we prepare for the near-term initiation of patient enrollment in our BEACON Phase 1/2 trial, the first clinical trial evaluating BEAM-101 in patients with sickle cell disease," said John Evans, chief executive officer of Beam. "In June, we submitted our IND for BEAM-201 for CD7-positive T-cell malignancies and recently received notification from the FDA of a clinical hold on the IND. We look forward to receiving more detail from the FDA and working with them in an effort to advance BEAM-201 for these difficult-to-treat cancer indications. We are on track to further expand our portfolio with a steady cadence of clinical and preclinical milestones expected in the quarters ahead, including the IND submission for BEAM-102, our second program in sickle cell disease, and the initiation of IND-enabling studies for BEAM-301, our first liver-directed base editing program in glycogen storage disease, both targeted in the second half of this year."

Mr. Evans added, "As pioneers and leaders in the field of base editing, we’ve continued to extend the potential reach of our base editing technology and applications with the development of new base editors, as well as novel base editing-enabled therapeutic strategies, such as our work on non-genotoxic conditioning to improve transplant regimens. We’ve also continued to enhance our team, and I’m thrilled to welcome John Lo as chief commercial officer. John has a deep science background and an extensive track record in the strategic development and commercialization of novel medicines, including cell therapy products, at leading companies. I can’t wait to work with him to advance our portfolio and our vision of providing a new class of precision genetic medicines to patients."

Pipeline Updates & Anticipated Milestones
Ex Vivo HSC Programs

Beam remains on track to begin patient enrollment in its BEACON trial, an open-label, single-arm, multicenter, Phase 1/2 clinical trial evaluating the safety and efficacy of BEAM-101 in adult patients with severe sickle cell disease (SCD) in the second half of 2022.
BEAM-102 continues to progress, and the company plans to submit an investigational new drug (IND) application for BEAM-102 for the treatment of SCD in the second half of 2022.
Ex Vivo T Cell Programs

Beam submitted its IND for BEAM-201 to the U.S. Food and Drug Administration (FDA) in June 2022, and on July 29, 2022, was notified via e-mail that the IND was placed on clinical hold. The FDA indicated it will provide an official clinical hold letter to Beam within 30 days. Beam plans to provide additional updates pending interaction with the FDA.
Beyond BEAM-201, Beam is focused on identifying the collection of multiplex base edits required to make cells fully allogeneic, with internal and external data suggesting a higher number of edits will be required to meet this goal. As a result, Beam does not expect to nominate a second CAR-T development candidate in 2022 and anticipates providing further updates in 2023.
In Vivo LNP Liver-targeting Programs

Beam presented updated preclinical data from its BEAM-301 program at the American Society of Cell and Gene Therapy (ASGCT) (Free ASGCT Whitepaper) meeting, highlighting that BEAM-301 demonstrated high and durable editing efficiency in a mouse model of glycogen storage disease 1a (GSDIa) out to 35 weeks. BEAM-301, a liver-targeting lipid nanoparticle (LNP) formulation of base editing reagents designed to correct the R83C mutation, the most common disease-causing mutation of GSDIa, is on track for initiation of IND-enabling studies in the second half of 2022.
At ASGCT (Free ASGCT Whitepaper), Beam also presented new preclinical data from its base editing program targeting the treatment of alpha-1 antitrypsin deficiency, highlighting optimizations made to the editor and the guide RNA that have led to two-fold increases in observed editing potency in mice, leading to potentially clinically relevant increases in circulating alpha-1 antitrypsin at doses below 1 mg/kg.
Beam plans to present new in vivo preclinical data from its multiplex base editing program for the potential treatment of hepatitis B virus (HBV) in a poster titled, "Cytosine base editing inhibits Hepatitis B Virus replication and reduces HBsAg expression in vitro and in vivo," at the 2022 International HBV Meeting from Sept. 18-22. The data will build on initial in vitro data presented in September 2021, which showed that base editing can introduce permanent mutations in covalently closed circular DNA (cccDNA) and prevent HBV rebound in relevant models.
Beam continues to anticipate the nomination of a second liver-targeted development candidate in 2022.
Recent Research Highlights

At the Federation of American Societies for Experimental Biology (FASEB) Genome Engineering Conference in June, Beam presented the first research highlighting the company’s internal efforts to develop improved transplant conditioning regimens for patients with SCD undergoing hematopoietic stem cell transplantation (HSCT). With a goal of overcoming limitations of today’s conditioning regimens, Beam leveraged its base editing capabilities to develop a potentially non-genotoxic approach that combines antibody-based conditioning with multiplex gene-edited hematopoietic stem cells (HSCs) called ESCAPE, or Engineered Stem Cell Antibody Paired Evasion. These improved conditioning regimens could potentially be paired with BEAM-101 and BEAM-102, as well as other future programs.
At the CRISPR 2.0 conference in June, Beam highlighted research that led to the creation of an improved class of cytosine base editors (CBEs), leveraging a TadA enzyme-based CBE (CBE-T), that are capable of editing at levels comparable to traditional CBEs but with lower off-target editing potential. Further, Beam disclosed an additional editor, CABE-T, that can conduct both C-to-T and A-to-G edits with a single TadA deaminase.
Business Updates

Beam recently appointed John Lo, Ph.D., as chief commercial officer, where he will be responsible for commercial readiness, as well as leading product and portfolio strategy. Dr. Lo joins Beam after serving as an advisor to multiple private- and public-stage biotechnology companies, including Beam. Dr. Lo has held a number of global strategic and operating roles of increasing responsibility within the biopharmaceutical industry, including as senior vice president, worldwide hematology at Bristol Myers Squibb; head of global marketing and market access at Astra Zeneca; corporate vice president, hematology and oncology at Celgene; and multiple P&L roles at Novartis. While at these companies, Dr. Lo successfully helped launch numerous drugs in the U.S. and globally, including Tagrisso in lung cancer and two cell therapies. Dr. Lo has also helped build strategies and grow R&D pipelines as co-chair of the Development Committee at Celgene, leading to multiple pivotal study investments. Dr. Lo also spent several years as an associate principal at McKinsey & Company and holds a Ph.D. in molecular biology from MIT.
In July, Beam and Verve Therapeutics amended their collaboration and license agreement, originally executed in April 2019. The amended agreement returned two targets to Beam, while adding a new, third target toward an additional liver-mediated, cardiovascular disease target. Beam has the right to opt in on this target after Phase 1 to share 35% of worldwide costs and profits from the program. The two lead targets in the collaboration, PCSK9 and ANGPTL3, are unchanged. Verve also granted to Beam licenses and options to certain delivery technologies.
Second Quarter 2022 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $1.2 billion as of June 30, 2022, as compared to $965.6 million as of December 31, 2021.
Research & Development (R&D) Expenses: R&D expenses were $74.6 million for the second quarter of 2022, compared to $45.6 million for the second quarter of 2021.
General & Administrative (G&A) Expenses: G&A expenses were $24.1 million for the second quarter of 2022, compared to $13.4 million for the second quarter of 2021.
Net Loss: Net loss was $72.0 million for the second quarter of 2022, or $1.02 per share, compared to $76.3 million for the second quarter of 2021, or $1.23 per share.

On August 9, 2022 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a precision oncology company targeting virus-associated malignancies, reported financial results for the second quarter of 2022 and provided an update on recent corporate developments (Press release, Sunesis, AUG 9, 2022, View Source [SID1234617950]).

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"We are pleased with our corporate progress in the first half of 2022, having advanced both of our Nana-val clinical trials towards important milestones anticipated later this year, while maintaining a strong cash position that provides a projected runway into the middle of 2024," said Ivor Royston, M.D., President and Chief Executive Officer of Viracta. "The Phase 1b/2 trial in EBV-positive solid tumors is now enrolling patients in the second dose escalation cohort, and we are on track to report preliminary data in the fourth quarter. For the pivotal NAVAL-1 trial in EBV-positive lymphoma, we incorporated an important amendment into our clinical trial protocol, which allows for the evaluation of Nana-val as a second-line therapy in patients with EBV-positive peripheral T-cell lymphoma. Now, both T-cell lymphoma subtypes in NAVAL-1, extranodal NK/T-cell and peripheral T-cell, are enrolling second-line patients. We believe this is an acknowledgment of the high unmet medical need in T-cell lymphoma and the strength of our data to date."

Second Quarter 2022 and Recent Highlights

Clinical

Amended the protocol for NAVAL-1, the pivotal trial of Nana-val in relapsed/refractory EBV+ lymphoma to enable enrollment of second-line peripheral T-cell lymphoma patients. The recently enacted protocol amendment is designed to enable the evaluation of Nana-val as an earlier therapeutic option, as the protocol previously required patients in this cohort be third-line or later. NAVAL-1 employs a Simon two-stage design with Stage 1 enrolling patients into six cohorts based on lymphoma subtype. If a pre-specified activity threshold is reached, additional patients will be enrolled in Stage 2. Lymphoma subtypes demonstrating promising activity in Stage 2 may be further expanded. If successful, Viracta believes NAVAL-1 could potentially support multiple new drug application filings across various EBV+ lymphoma subtypes. The trial has sites open globally, including in the U.S., Canada, Europe, and Asia. An update on the initial cohort(s) that may advance into Stage 2 of the trial is anticipated in the fourth quarter of 2022.
Advanced to the second dose escalation cohort within the Phase 1b part of the Phase 1b/2 trial of Nana-val in patients with EBV+ recurrent/metastatic nasopharyngeal carcinoma (R/M NPC) and other EBV+ solid tumors. Initiation of the second dose escalation cohort followed a favorable review of safety data from the first dose escalation cohort by the trial’s independent Safety Monitoring Committee, which noted no dose-limiting toxicities in the trial to date. Enrollment is ongoing in the second dose cohort of the trial’s Phase 1b portion, which is designed to evaluate safety and determine the recommended Phase 2 dose (RP2D) of Nana-val in patients with EBV+ R/M NPC. In Phase 2 of the trial, up to 60 patients with EBV+ R/M NPC will be randomized to receive Nana-val at the RP2D with or without pembrolizumab to evaluate safety and preliminary efficacy. The trial also includes a Phase 1b dose expansion cohort designed to evaluate Nana-val at the RP2D in patients with other EBV+ solid tumors. Preliminary safety and efficacy data from the Phase 1b dose escalation portion of the trial are anticipated in the fourth quarter of 2022.
Corporate and Thought Leadership

Dr. Royston honored with the Science History Institute’s Biotechnology Heritage Award. First bestowed in 1999, the Award honors extraordinary individuals whose work in biotechnology is helping to heal, fuel, and feed the world through discovery, innovation, commercialization, or public understanding. It was presented to Dr. Royston by the Science History Institute alongside the Biotechnology Innovation Organization (BIO), which represents more than 1,200 biotech companies, academic institutions, state biotechnology centers, and related organizations across the United States and in more than 30 other nations.
Hosted key opinion leader (KOL) webinar on Nana-val for the treatment of advanced EBV+ solid tumors. During the webinar, KOL Ezra Cohen, M.D., FRCPSC, FASCO (University of California, San Diego) and members of the Viracta management team discussed the current treatment landscape and unmet medical need in NPC, the design of the Phase 1b/2 trial of Nana-val in advanced EBV+ solid tumors, and preclinical data supporting the trial. A replay of the webinar is available here.
Scientific Presentations

Highlighted the design of the Phase 1b/2 trial of Nana-val in advanced EBV+ solid tumors in a poster at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The poster was presented by A. Dimitrios Colevas, M.D. (Stanford Cancer Institute), during a session titled, "Head and Neck Cancer." A copy of the poster is available here.
Presented an overview of the Company’s novel "Kick and Kill" approach to targeting EBV-associated malignancies at the 20th International Symposium on EBV and Associated Diseases. The presentation was delivered by Viracta’s Chief Scientific Officer, Dr. Ayman Elguindy, and included previously announced data demonstrating Nan-val’s mechanism of action and anti-cancer activity. The symposium was sponsored by the International Association for Research on Epstein-Barr Virus and Associated Diseases.
Anticipated 2022 Milestones

Provide preliminary Phase 1b safety and efficacy data from the Phase 1b/2 trial in advanced EBV+ solid tumors: Q4 2022
Update on NAVAL-1 cohort(s) that may progress from Stage 1 to Stage 2: Q4 2022
Second Quarter 2022 Financial Results

Cash position – Cash, cash equivalents and short-term investments totaled approximately $83.1 million as of June 30, 2022, which Viracta expects will be sufficient to fund its operations into mid-2024, excluding any incremental borrowing under its previously announced $50.0 million credit facility from Silicon Valley Bank and Oxford Finance.
Research and development expenses – Research and development expenses were approximately $6.3 million and $12.4 million for the three and six months ended June 30, 2022, respectively, compared to approximately $5.4 million and $9.5 million for the same periods in 2021. The increase in research and development expenses was primarily due to increases in costs incurred to support the advancement and expansion of our clinical development programs, including incremental costs to support NAVAL-1, our pivotal trial in R/R EBV+ lymphoma, and the initiation of our Phase 1b/2 trial for the treatment of EBV+ solid tumors, as well as an increase in headcount and non-cash share-based compensation.
Acquired in-process research and development – The acquired in-process research and development for the six months ended June 30, 2021 included non-cash and non-recurring cost of $84.5 million associated with the estimated fair value of the in-process research and development projects acquired in the asset acquisition with no alternative future use, which was charged to expense upon the completion of the transaction in February 2021.
General and administrative expenses – General and administrative expenses were approximately $4.2 million and $8.5 million for the three and six months ended June 30, 2022, respectively, compared to approximately $3.9 million and $7.7 million for the same periods in 2021. The change was primarily driven by an increase in non-cash share-based compensation.
Gain on royalty purchase agreement – The gain on royalty purchase agreement for the six months ended June 30, 2021, was associated with upfront proceeds of $13.5 million recorded in connection with the multi-license milestone and royalty monetization transaction with XOMA (US) LLC.
Adjusted loss from operations – There was not a comparative adjustment to loss from operations for the quarters ended June 30, 2022, and 2021. There was not a comparative adjustment to loss from operations for the six months ended June 30, 2022. Adjusted loss from operations for the six months ended June 30, 2021, excluding the non-recurring and non-cash operating expenses associated with the write-off of in-process research and development acquired in the merger (a non-GAAP measure) was $3.7 million, compared to an unadjusted loss from operations of $88.2 million.
Net loss – Net loss was approximately $10.6 million, or $0.28 per share (basic and diluted) for the quarter ended June 30, 2022, compared to a net loss of $9.2 million or $0.25 per share (basic and diluted) for the same period in 2021. Net loss was approximately $21.1 million, or $0.56 per share (basic and diluted) for the six months ended June 30, 2022, compared to a net loss of $88.4 million or $3.37 per share (basic and diluted) for the same period in 2021.
About Nana-val (Nanatinostat and Valganciclovir)

Nanatinostat is an orally available histone deacetylase (HDAC) inhibitor being developed by Viracta. Nanatinostat is selective for specific isoforms of Class I HDACs, which is key to inducing viral genes that are epigenetically silenced in Epstein-Barr virus (EBV)-associated malignancies. Nanatinostat is currently being investigated in combination with the antiviral agent valganciclovir as an all-oral combination therapy, Nana-val, in various subtypes of EBV-associated malignancies. Ongoing trials include a pivotal, global, multicenter, open-label Phase 2 basket trial in multiple subtypes of relapsed/refractory EBV+ lymphoma (NAVAL-1) as well as a multinational Phase 1b/2 trial in patients with EBV+ recurrent or metastatic nasopharyngeal carcinoma and other EBV+ solid tumors.

About EBV-Associated Cancers

Approximately 90% of the world’s adult population is infected with Epstein-Barr virus (EBV). Infections are commonly asymptomatic or associated with mononucleosis. Following infection, the virus remains latent in a small subset of lymphatic cells for the duration of the patient’s life. Cells containing latent virus are increasingly susceptible to malignant transformation. Patients who are immunocompromised are at an increased risk of developing EBV+ lymphomas. EBV is estimated to be associated with approximately 2% of the global cancer burden including lymphoma, nasopharyngeal carcinoma and gastric cancer.

On August 9, 2022 Catalent, Inc. (NYSE: CTLT), the global leader in enabling biopharma, cell, gene, and consumer health partners to optimize development, launch, and supply of better patient treatments across multiple modalities, reported that it has reached an agreement to acquire Metrics Contract Services (Metrics), a full-service specialty Contract Development and Manufacturing Organization (CDMO) with a facility in Greenville, North Carolina, for $475 million from Mayne Pharma Group Limited (ASX: MYX) (Mayne Pharma) (Press release, Catalent, AUG 9, 2022, View Source [SID1234617965]). Upon completion, the acquisition will strengthen Catalent’s capabilities in integrated oral solid formulation development, manufacturing, and packaging to help customers simplify and accelerate their programs, while also expanding Catalent’s capacity to handle highly potent compounds.

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The 333,000 square-foot Greenville facility features comprehensive capabilities to accelerate and de-risk customer programs from early development through commercial launch through a streamlined one-site solution. Over the past five years, the facility has seen more than $100 million in capital improvements and now includes 16 manufacturing suites, with 11 designed to handle highly potent compounds, as well as two packaging lines that can support a large variety of development and commercial supply programs. The facility’s estimated annual production capacity exceeds one billion oral solid dose units.

"This acquisition will further expand Catalent’s ability to meet our customers’ expectations in fast-growing areas of the business and patient need. The experienced team and consistently improved, state-of-the-art facility in Greenville will provide Catalent’s customers with immediate, fit-for-scale capacity for in-demand highly potent drugs and other oral solid small-to-mid-size batch needs. This capacity is particularly important for customers with R&D pipelines featuring accelerated, orphan, and rare disease programs for oncology and other important therapeutic areas," said Dr. Aris Gennadios, Group President of Catalent’s Pharma & Consumer Health segment.

"Over the past several years, Metrics has undergone a period of transformational change to expand its footprint and service offering, becoming a global end-to-end novel oral solid CDMO. Catalent, a global leader in advanced drug development and manufacturing, is well-positioned to continue to invest in and accelerate the growth of Metrics and we believe this transaction will be extremely positive for our Greenville team and customers," said Scott Richards, Chief Executive Officer of Mayne Pharma.

The new facility will seamlessly integrate into Catalent’s industry-leading oral development and manufacturing network, which includes flagship sites for large-scale and controlled release oral solids manufacturing in Winchester, Kentucky; softgel development and manufacturing in St. Petersburg, Florida; and additional facilities with bioavailability enhancement technologies and complex oral solids manufacturing platforms.

The acquisition is expected to close before the end of this calendar year, subject to customary closing conditions, and the entire team of over 400 employees will join Catalent. Mayne Pharma and Catalent have also agreed on the terms of a long-term supply agreement whereby the Greenville facility will continue to manufacture multiple Mayne Pharma products. Catalent will pay the purchase price for this all-cash acquisition using a combination of cash on hand, existing credit facilities, and, depending on market conditions, potentially new debt financing. The closing of the acquisition is not contingent on any financing activity.

Heron Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission .

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On August 9, 2022 Blue Water Vaccines Inc. ("BWV" or "Blue Water Vaccines" or "the Company"), a biopharmaceutical company developing transformational vaccines to address significant global health challenges, reported that it has entered into definitive agreements with several healthcare-focused institutional investors for the purchase of 3,683,280 shares of common stock (or common stock equivalents in lieu thereof) in a private placement priced at-the-market under Nasdaq rules (Press release, Onconetix, AUG 9, 2022, View Source [SID1234641112]). The Company will also issue to the investors unregistered preferred investment options (the "investment options") to purchase up to an aggregate of 4,972,428 shares of common stock. The purchase price for one share of common stock (or common stock equivalent) and one investment option to purchase one share of common stock is $2.715. The investment options will have an exercise price of $2.546 per share, will be exercisable immediately upon issuance, and will have a term equal to five years following the issuance date.

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H.C. Wainwright & Co. is acting as the exclusive placement agent for the offering.

The gross proceeds from the private placement are expected to be approximately $10 million, before deducting placement agent fees and other offering expenses. The Company intends to use the net proceeds from the private placement for the research and development of its pipeline as well as for working capital and other general corporate purposes. The Company’s current cash position including the expected gross proceeds from this private placement is approximately $30.3 million. The private placement is expected to close on or about August 11, 2022, subject to the satisfaction of customary closing conditions.

The offer and sale of the foregoing securities are being made in a transaction not involving a public offering and the securities have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or applicable state securities laws. Accordingly, the securities may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws. Pursuant to a registration rights agreement with investors, BWV has agreed to file a resale registration statement covering the securities described above.

In addition, the investors in the private placement agreed to cancel preferred investment options to purchase up to an aggregate of 1,180,812 shares of the Company’s common stock issued in April 2022.

This press release does not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.