On March 15, 2021 Veracyte, Inc. (Nasdaq: VCYT) reported it has completed its acquisition of Decipher Biosciences, Inc. to further solidify its global leadership in genomic diagnostics, with an expanded reach into 7 of the 10 most common cancers in the United States, while accelerating revenue growth (Press release, Veracyte, MAR 15, 2021, View Source [SID1234576662]).

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"Bringing Decipher into the Veracyte family marks a significant advance in achieving our vision of improving outcomes for patients all over the world at every step of their journey"

"Bringing Decipher into the Veracyte family marks a significant advance in achieving our vision of improving outcomes for patients all over the world at every step of their journey," said Bonnie Anderson, Veracyte’s chairman and chief executive officer. "By combining Decipher’s leadership in urologic cancers with our broad cancer genomic testing menu and best-in-class nCounter diagnostics platform, this combination further positions us to address key unmet needs across the care continuum for patients everywhere. We are thrilled to welcome the Decipher team as we embark on an exciting journey together."

In connection with the closing of the acquisition, Dr. Tina Nova, previously president and chief executive officer at Decipher, has been appointed general manager, urologic cancers, at Veracyte. She and her team will continue to direct the company’s San Diego operations, which include a state-of-the-art CLIA-certified and CAP-accredited laboratory.

Transaction Details

Under the terms of the transaction, Veracyte acquired Decipher Biosciences for $600 million in cash paid to Decipher security holders and Decipher Biosciences has become a wholly-owned subsidiary of Veracyte.

On March 15, 2021 OSE Immunotherapeutics (FR0012127173) and the French cooperative group ARCAGY-GINECO reported that the French National Agency for Medicines and Health Products Safety (ANSM) and the French Central Ethic Committee (CPP) approved the initiation of a new Phase 2 clinical trial evaluating Tedopi in patients with recurrent ovarian cancer (the TEDOVA trial) (Press release, OSE Immunotherapeutics, MAR 15, 2021, View Source [SID1234646983]). Tedopi will be evaluated alone and in combination with Merck’s Keytruda (pembrolizumab), an immune checkpoint inhibitor, as maintenance treatment in ovarian cancer patients after chemotherapy.

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The three arm TEDOVA study will evaluate neo-epitope-based vaccine Tedopi as a maintenance treatment, alone or in combination with anti-PD-1 Keytruda, versus the best supportive care in platinum-sensitive recurrent ovarian cancer patients with controlled disease after platinum-based chemotherapy.

The clinical trial is sponsored by the "Association de Recherche sur les CAncers dont GYnécologiques (ARCAGY-GINECO)" on behalf of GINECO, lead group for the TEDOVA trial of the European Network for Gynaecological Trial Groups (ENGOT). It will be supported in part by a research grant from the Investigator-Initiated Studies Program of MSD (Merck Sharp & Dohme Corp), a subsidiary of Merck & Co., Inc.", which will provide Keytruda (pembrolizumab), and by OSE Immunotherapeutics which will provide Tedopi for the study as well as partial financial support.

Alexis Peyroles, Chief Executive Officer of OSE Immunotherapeutics, comments: "This new clinical development program for Tedopi in ovarian cancer demonstrates the interest in exploring the potential of a PD-1-targeted checkpoint inhibitor combination strategy for combating oncology indications with significant unmet medical needs. We are very pleased to collaborate with the oncology group ARCAGY – GINECO to advance a new therapeutic pathway for patients suffering from a particularly aggressive cancer."

Dr Alexandra Leary, Chief Investigator of TEDOVA study from Gustave Roussy cancer center, adds: "Our patients with ovarian cancer do not respond to checkpoint inhibitors (ICI) alone because these tumors are ‘immune cold’. The objective of TEDOVA is to turn ovarian cancer into an ‘immune hot’ tumor using a combination of tumor associated neo-epitopes that have been optimized to break immunological selftolerance. TEDOVA is the first trial evaluating such an innovative approach in ovarian cancer and has received enthusiastic support from the international gynecological oncology community."

ABOUT OVARIAN CANCER
Worldwide, ovarian cancer is the seventh most common cancer and the eighth leading cause of cancer death in women. The five-year survival rate for ovarian cancer worldwide is 30-40%. In 2018, there were nearly 300,000 new cases diagnosed. Once the first relapse has occurred, ovarian cancer is managed as a chronic disease, requiring iterative lines of platinum-based chemotherapy. After 6 cycles, chemotherapy is stopped and one of the major priorities is to extend "chemotherapy-free" intervals for the patients by proposing maintenance strategies with targeted therapies (PARP inhibitors or bevacizumab). By the time patients with ovarian cancer present with first or second relapse, they will have received BOTH a PARP inhibitor and bevacizumab, thus patients progressing post-PARP inhibitors and bevacizumab represent an area of unmet medical need, they are offered chemotherapy alone with no maintenance strategy. The TEDOVA trial focuses on these women.

On March 15, 2021 TILT Biotherapeutics, a clinical-stage biotechnology company developing cancer immunotherapeutics, reported that it has achieved the primary endpoint of safety in its phase I trial with the first dose of its dual cytokine armed oncolytic adenovirus, TILT-123, at Herlev hospital in Denmark (Press release, TILT Biotherapeutics, MAR 15, 2021, https://www.prnewswire.com/news-releases/tilt-biotherapeutics-advances-cancer-immunotherapy-clinical-trial-achieving-primary-end-point-in-the-first-cohort-301246532.html [SID1234576615]). No serious adverse events (SAEs) were observed in the trial for TILT-123 as a monotherapy or in combination with tumour infiltrating lymphocytes (TILs). The company has received regulatory approval to open a second clinical trial site at CHU Nantes, France, with 15 patients in total to be enrolled across the two sites. The clinical trial will now proceed to the second (of five) dose levels and expects interim data by the end of this year.

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The ‘TUNINTIL’ clinical trial(1) is a phase 1, open-label, dose-escalation study of the company’s oncolytic adenovirus coding for Tumor Necrosis Factor Alpha (TNF alpha) and Interleukin 2 (IL-2) known as TILT-123. Metastatic melanoma patients receive TILT-123 as an initial monotherapy over one month, followed by up to two administrations of tumor infiltrating lymphocytes (TILs) in the second month as well as ongoing consecutive doses of TILT-123.

TILT Biotherapeutics’ CEO, Akseli Hemminki, a biotech entrepreneur and cancer clinician who has personally treated almost 300 patients with ten different oncolytic viruses, said, "We are delighted our first-in-human phase 1 trial in metastatic melanoma has successfully met its primary clinical endpoint of safety at the initial dose and is now progressing steadily to the next dose level. I am grateful for the excellent work by the team at Denmark’s prestigious National Center for Cancer Immune Therapy and look forward to continuing this and also working with the team in Nantes. Overall, the trial has the potential to increase the efficacy of adoptive T-cell therapy, remove the need for pre- and post-conditioning regimens, and deliver the combined anti-tumor benefits of armed oncolytic viruses and T-cell therapy."

The company has received approval in Denmark to extend this trial for up to two years to demonstrate long term survival benefit. As part of this, the company will be analyzing secondary end points for TILT-123 including response rate, overall survival, progression free survival, and immune response against the tumor, its persistence and shedding.

The heart of TILT’s approach revolves around the use of armed oncolytic adenoviruses, using cytokines to boost the patient’s immune response to better enable it to find and destroy cancer cells. The TUNINTIL trial’s primary objective is to evaluate the safety of TILT-123 and is designed to also deliver insights about the behavior of TILT-123 in humans, such as systemic tumor transduction following intravenous delivery and virus replication in the tumor, as well as immunological responses.

On March 15, 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 fourth quarter and full year 2020 financial results (Press release, Beam Therapeutics, MAR 15, 2021, View Source [SID1234576643]).

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"Beam has made significant progress in the last 12 months," said John Evans, chief executive officer of Beam. "Since our IPO in February 2020, we named three development candidates from our portfolio; initiated IND-enabling studies for BEAM-101; presented data highlighting our broad set of gene editing technologies; expanded access to novel delivery modalities through the acquisition of Guide Therapeutics; secured and initiated work on our build-to-suit manufacturing facility; enhanced our leadership team with the addition of Amy Simon as chief medical officer; expanded our board with the appointment of Kate Walsh; and further strengthened our capital position and operating runway. In the remainder of 2021, we plan to submit our first Investigational New Drug application for BEAM-101, initiate IND-enabling studies for BEAM-102 and BEAM-201, and nominate our first development candidate from our liver portfolio, positioning Beam to become a clinical stage company with an expansive pipeline moving quickly behind our lead assets. We are well positioned today to advance our platform and pipeline, and remain focused on achieving our vision of providing life-long cures for patients suffering from serious diseases."

Recent Business Highlights

Acquisition of Guide Therapeutics (GuideTx) Expands Novel Base Editing Capabilities: In February 2021, Beam acquired GuideTx, a developer of nonviral drug delivery vehicles for genetic medicines, further expanding the potential reach of Beam’s genetic medicines into target tissues beyond the liver. GuideTx’s technology enables the screening of hundreds of nanoparticles in a single experiment, potentially generating in vivo drug delivery data at significantly greater rates compared to traditional screens. Beam believes that the proprietary screening method will enable identification of lipid nanoparticles with novel biodistribution and high selectivity for target cells. Using this platform and a proprietary library of cationic lipids, GuideTx has identified a broad array of novel lipid nanoparticle formulations.
Balance Sheet Enhanced with $260 Million Private Placement: In January 2021, Beam sold 2,795,700 shares of its common stock to certain institutional investors in a private placement. Aggregate gross proceeds from the offering were approximately $260 million, before deducting fees to the placement agents and other offering expenses payable by the Company.
Leadership Team Strengthened by the Addition of Amy Simon, M.D., as Chief Medical Officer: In March 2021, Beam appointed Amy Simon, M.D., as chief medical officer. Dr. Simon joins Beam from Alnylam Pharmaceuticals, where she spent over a decade in various roles with increasing responsibility for the clinical development of numerous RNAi-based medicines, most recently serving as vice president, clinical development and the lead clinician developing GIVLAARI (givosiran) for acute hepatic porphyria, which was approved by FDA in 2019. Dr. Simon holds a B.A. in history and science from Harvard University and received her M.D. from Tufts University School of Medicine.
Leading Healthcare Executive, Kate Walsh, Appointed to Board of Directors: In January 2021, Kate Walsh, president and chief executive officer of the Boston Medical Center (BMC) health system, joined Beam’s board of directors. BMC is a private, not-for-profit, academic medical center with a community-based focus and is the primary teaching affiliate of Boston University School of Medicine.
Base Editing Progress

Updated Data from Novel Sickle Cell Disease Approaches Presented at 62nd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH 2020): In December 2020, Beam presented updated data from the company’s complementary base editing approaches to treat hemoglobinopathies, BEAM-101 and BEAM-102, during poster sessions at ASH (Free ASH Whitepaper) 2020. The BEAM-101 data presented demonstrated precise base editing with no off-target editing observed, supporting the company’s planned IND submission in the second half of 2021. Additionally, in vivo data from the company’s Makassar base editing program demonstrated long-term engraftment and editing retention at 16 weeks, with BEAM-102 achieving greater than 90% bi- and mono-allelic Makassar editing in SCD CD34+ HSPCs in vitro.

First Data Highlighting Base Editing Program for Glycogen Storage Disease Type Ia Presented at American Association for the Study of Liver Diseases’ (AASLD) The Liver Meeting Digital Experience: In November 2020, Beam presented the first data highlighting its novel base-editing strategy for correcting disease-causing mutations underlying Glycogen Storage Disease Type Ia (GSDIa) during a poster session at AASLD, demonstrating in vivo correction of the R83C and Q347X mutations by ABEs in the livers of two strains of transgenic mice, each carrying one of the two G6PC mutations. Next-generation sequencing data from whole liver extracts revealed significant correction for both R83C and Q347X, with nearly 40% and approximately 70% A-to-G conversion efficiency, respectively, of each mutation back to the normal gene sequence. These significant levels of mutation correction greatly surpassed those expected to restore glucose homeostasis, and functional studies are ongoing to correlate pathophysiology to extent of mutation correction by base-editing. Further, these levels of in vivo correction for GSDIa by base-editing were achieved without detectable creation of double-stranded breaks. In total, these data support base-editing technology as a promising approach for precise correction of causative mutations in GSDIa.
Fourth Quarter and Full Year 2020 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $299.7 million as of December 31, 2020, compared to $91.8 million as of December 31, 2019. This cash balance does not include the proceeds from the January 2021 private placement.
Research & Development (R&D) Expenses: R&D expenses were $32.5 million for the fourth quarter of 2020 and $103.2 million for the full year ended December 31, 2020, compared to $20.2 million for the fourth quarter of 2019 and $54.6 million for the full year ended December 31, 2019.
General & Administrative (G&A) Expenses: G&A expenses were $8.4 million for the fourth quarter of 2020 and $29.6 million for the full year ended December 31, 2020, compared to $6.2 million for the fourth quarter of 2019 and $20.6 million for the full year ended December 31, 2019.
Net Loss: Net loss attributable to common stockholders was $95.5 million, or $1.69 per share, for the fourth quarter of 2020 and $195.9 million, or $4.19 per share, for the full year ended December 31, 2020, compared to $31.1 million, or $4.35 per share, for the fourth quarter of 2019 and $91.0 million, or $14.05 per share, for the full year ended December 31, 2019.

On March 15, 2021 Savara Inc. (Nasdaq: SVRA), an orphan lung disease company, reported the closing of its previously announced underwritten public offering of 57,479,978 shares of its common stock, including 11,694,150 shares sold pursuant to the exercise in full by the underwriters of their option to purchase additional shares, at a price to the public of $1.45 per share (Press release, Savara, MAR 15, 2021, View Source [SID1234576663]). In addition, in lieu of shares of common stock, Savara sold to certain investors pre-funded warrants to purchase an aggregate of 32,175,172 shares of common stock at a purchase price of $1.449 per warrant, which represents the per share public offering price for the common stock less the $0.001 per share exercise price for each pre-funded warrant. As a result of the underwriters’ full option exercise, the aggregate gross proceeds of the offering to Savara, before deducting underwriting discounts and commissions and other offering expenses, were approximately $130.0 million.

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Savara intends to use the net proceeds from this offering for working capital to support operations, including but not limited to clinical development, manufacturing, regulatory, and commercial activities related to its molgramostim nebulizer solution (molgramostim, formerly referred to as Molgradex) in autoimmune pulmonary alveolar proteinosis (aPAP) program and the IMPALA 2 trial, and general and administrative expenses.

Jefferies LLC and Piper Sandler acted as joint book-running managers and representatives of the underwriters for the offering. Oppenheimer & Co. Inc. acted as the lead manager and H.C. Wainwright & Co., LLC acted as co-manager for the offering.

The securities described above were issued and sold pursuant to Savara’s existing shelf registration statement (File No. 333-225994) filed with the Securities and Exchange Commission (SEC) on June 29, 2018 and declared effective on July 13, 2018. The final prospectus supplement describing the terms of the offering was filed with the SEC on March 12, 2021. Copies of the final prospectus supplement and accompanying prospectus relating to the offering may be obtained by contacting Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, 2nd Floor, New York, NY 10022, or by telephone at (877) 547-6340, or by email at [email protected] or Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, or by telephone at (800) 747-3924, or by email at [email protected]. An electronic copy of the final prospectus supplement and accompanying prospectus relating to the offering will be available on the SEC website at www.sec.gov.

This press release does not constitute an offer to sell or the solicitation of offers to buy any securities of Savara, and shall not constitute an offer, solicitation, or sale of any security in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.