On August 10, 2021 Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), reported financial results for the second quarter ended June 30, 2021 and provided a corporate update (Press release, Regulus, AUG 10, 2021, View Source [SID1234586261]).

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"The team at Regulus accomplished a lot over the last several months, including the achievement of several major milestones in our ADPKD program, the completion of meeting preparations with FDA and extending our cash runway into Q4 of next year," commented Jay Hagan, CEO of Regulus. "In addition, we are pleased to have completed enrollment of the second cohort of our Phase 1b trial of RGLS4326 for ADPKD, for which we expect to report topline data in the next several months. Additionally, we look forward to obtaining FDA feedback on our approach for addressing the remaining partial clinical hold requirements through our meeting request."

Program Updates

RGLS4326 for ADPKD: In June 2021, the Company presented additional data from the first cohort of patients in its Phase 1b clinical trial of RGLS4326 for the treatment of ADPKD, as well as new preclinical data from relevant animal models of the disease, at PKD Connect 2021. The additional data presented at PKD Connect demonstrated clinical proof of mechanism by showing statistically significant increases in polycystin levels in ADPKD patients by targeting miR-17 in the kidneys. Levels of PC1 and PC2 have previously been shown to be inversely correlated with disease severity. RGLS4326 was well-tolerated with no serious adverse events.

The Phase 1b study is an adaptive design, open-label, multiple dose study in up to three cohorts of patients with ADPKD evaluating the safety, pharmacokinetics, and effects on pharmacodynamic biomarkers of multiple doses of RGLS4326. In the first cohort of the study, nine patients were enrolled and received four doses of 1mg/kg of RGLS4326 administered every other week.

The Company also presented new data from relevant preclinical models showing treatment with RGLS4326 resulted in increased gene and polycystin levels in vitro. Improvements in key disease markers including serum creatinine and BUN were demonstrated in the Pkd1(F/RC) mouse model that harbors a mutation in the Pkd1 gene equivalent to human ADPKD. Mutation to the Pkd1 gene is reported for 85% of diagnosed patients with ADPKD.

In late July 2021, the Company completed enrollment of the second cohort of patients in its Phase 1b clinical study of RGLS4326. The patients in the second cohort are being administered 0.3mg/kg of RGLS4326 every other week for four doses. The dose of RGLS4326 for the third and final cohort will be chosen based on the results of this second cohort and enrollment is expected to commence thereafter. The Company expects to report topline data from the second cohort in the fourth quarter.

The Company requested a Type A meeting with FDA to discuss the data supporting its approach to addressing the remaining partial clinical hold requirements. FDA typically responds to a sponsor’s request with the meeting being scheduled within 30 days from the receipt of the meeting request.

Corporate Highlights

Raised $15.4 Million in Gross Proceeds Through its ATM Facility: A total of 12,007,546 shares were sold and settled for gross proceeds of $15.4 million at a weighted average price per share of $1.28 under the ATM facility during the three months ended June 30, 2021. The Company expects its existing cash will provide cash resources to fund planned activities into Q4 2022.

Financial Results

Cash Position: As of June 30, 2021, Regulus had $41.4 million in cash and cash equivalents.

Research and Development (R&D) Expenses: Research and development expenses were $4.2 million and $7.5 million for the three and six months ended June 30, 2021, respectively, compared to $4.2 million and $7.4 million for the same periods in 2020, respectively. These amounts reflect internal and external costs associated with advancing our clinical and preclinical pipeline.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.5 million and $5.0 million for the three and six months ended June 30, 2021, respectively, compared to $2.3 million and $4.7 million for the same periods in 2020, respectively. These amounts reflect personnel-related and ongoing general business operating costs.

Net Loss: Net loss was $6.0 million, or $0.08 per share (basic and diluted), and $12.0 million, or $0.16 per share (basic and diluted), for the three and six months ended June 30, 2021, compared to $6.9 million, or $0.23 per share (basic and diluted), and $12.9 million, or $0.48 per share (basic and diluted), for the same periods in 2020.

About ADPKD

ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs. Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60.

About RGLS4326

RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 and preferentially target the kidney. Preclinical studies with RGLS4326 have demonstrated direct regulation of Pkd1 and Pkd2, reduction of cyst growth in human in vitro ADPKD models, and attenuation of cyst proliferation and improvement of kidney function in mouse models of ADPKD. The RGLS4326 IND is currently on a partial clinical hold for treatment of extended duration by FDA until the second set of requirements outlined by the agency have been satisfactorily addressed. The Company will use information from the Phase 1 clinical studies, including the first cohort of the Phase 1b together with information from the recently completed additional nonclinical studies generated in 2020, in its plan to address the second set of requirements outlined in the Partial Clinical Hold letter to support studies of extended duration. Regulus plans to discuss its approach to addressing the remaining Partial Clinical Hold requirements with FDA in the third quarter 2021. RGLS4326 received orphan drug designation from FDA in July 2020.

On August 10, 2021 ImmVira reported that it has initiated its lead oncolytic virus therapy program MVR-T3011 IV (also known as T3011) (Press release, Immvira, AUG 10, 2021, View Source [SID1234586259]). The first patient has been dosed, receiving MVR-T3011 intravenous (IV) administration in the U.S. on August 10, 2021. The Phase I clinical trial is being conducted at multiple well-established clinical institutions, including Sarah Cannon Research Institute, Mary Crowley Cancer Research, and Prisma Health (ClinicalTrials.gov Identifier: NCT04780217).

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Dr. Joy Zhu, Chief Medical Officer of ImmVira, stated that "MVR-T3011 IV officially entering clinical study is a breakthrough in the administration of the oncolytic herpes simplex virus. We are hopeful that MVR-T3011 IV will demonstrate good safety profile and promising clinical results. Systemic delivery of MVR-T3011 IV provides an opportunity to treat many advanced cancers that cannot be reached via intratumoral injection."

Dr. Joy Zhu joined ImmVira with more than 25 years of drug development experience. She held leadership positions in several biotech companies, as well as several start-up companies, including legacy SUGEN, Amgen, and Halozyme Therapeutics. Dr. Zhu has broad and in-depth experience in pre-IND planning, IND filings, and NDA submissions with the U.S. FDA and many other regulatory agencies. She was responsible for the clinical development of two blockbuster drugs, SUTENT (sunitinib malate) and XGEVA (denosumab), which are widely recognized in the broad market.

"We are excited about the prospects of IV administration of MVR-T3011 IV. After MVR-T3011 IV’s NMPA clinical trial approval in China, the completion of first dosing in the U.S. marks another significant step for ImmVira’s pipeline development. Leveraging the OvPENS new drug R&D platform aimed at next-generation cancer therapy evolution, ImmVira is expected to reshape the future of oncolytic virotherapy and bring hope to cancer patients and their families," Dr. Grace Zhou, CEO of ImmVira, concluded.

On August 10, 2021 Biodesix, Inc. (Nasdaq: BDSX), a leading data-driven diagnostic solutions company with a focus in lung disease, reported its financial and operating results for the second quarter ended June 30, 2021 and provided a corporate update (Press release, Biodesix, AUG 10, 2021, View Source [SID1234586258]).

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"It was a productive quarter with 180% year over year growth in total revenue. Our first four blood-based tests which make up our core lung diagnostic testing, grew 109% year over year and 20% over the prior quarter," said Scott Hutton, CEO of Biodesix. "We expect growth in core lung diagnostic testing to continue through 2021 driven by the U.S.’s emergence from the pandemic, productivity from our growing salesforce, and our efforts to build on the body of evidence supporting the use of our tests. Beyond our currently marketed products, we also continue to develop our Diagnostic Cortex Artificial Intelligence (AI) platform and expand our pipeline of products capable of providing broad lung diagnostics to address patients’ needs over the course of their disease. The launch of our 72-hour liquid NGS test is now projected for the first quarter of 2022 and will supplement our 36-hour VeriStrat and GeneStrat tests. With this accelerated launch and multiple studies underway for our Risk of Recurrence (ROR) and Primary Immune Response (PIR) tests as well as our ALTITUDE, BEACON, INSIGHT, and ORACLE prospective studies actively enrolling or following patients, we look forward to a productive and exciting second half of the year."

Second Quarter 2021 Financial Results

For the three-month period ended June 30, 2021, as compared to the same period of 2020 (where applicable):

Total revenue of $11.9 million, an increase of 180%;

Core lung diagnostic revenue of $4.8 million, an increase of 109%;
Continued sequential recovery from initial rapid uptake of COVID-19 immunizations.
Growth driven primarily by Nodify XL2 and Nodify CDT.
Nodify XL2 growth triggered the previously disclosed milestone resulting in a $37 million obligation payable by us over six quarterly installments of approximately $4.6 million beginning January 2022 and a final payment of approximately $9.3 million in July 2023.
COVID-19 testing revenue of $6.1 million, an increase of 345%;
Sequential quarter over quarter decline commensurate with our prior commentary as US immunizations accelerated in second quarter.
Services revenue of $1.0 million, an increase of 76%;
Clinical trial services showed improvement and we expect further recovery in clinical trial enrollments in second half of 2021.

Gross profit of $4.8 million, an increase of 103%, and gross margin percentage of 40% as compared to 56% representing a decline primarily attributable to the lower gross margin COVID-19 testing;

Operating expenses (excluding direct costs and expenses) of $15.4 million, which includes an investment in the planned expansion of our sales force, increased 93% over second quarter 2020;
On track to double size of lung focused direct and dedicated sales force in 2021.
Non-cash stock compensation expense of $0.5 million as compared to $0.1 million.
Non-cash expense for change in fair value of contingent consideration of $0.6 million as compared to a gain of $1.0 million.

Net loss of $11.4 million, an increase of 38% over second quarter of 2020; and

Maintained fiscal discipline and strong liquidity with cash and cash equivalents of $56.3 million as of June 30, 2021 and expect to qualify for PPP loan forgiveness of $3.1 million later in 2021.
For a full list of Biodesix’s press releases and webinars, please visit Biodesix.com.

Conference call and webcast information

Management will host an investor conference call and webcast today, August 10, 2021 at 4:30 p.m. Eastern Time.

On August 10, 2021 Indapta Therapeutics, Inc., a biotechnology company developing and commercializing an NK (natural killer) cell therapy platform for the treatment of blood cancers and solid tumors, reported the publication of new preclinical data in Blood Advances demonstrating Indapta’s proprietary allogeneic FcεRIγ-deficient G-NK cells in combination with an FDA-approved monoclonal antibody (mAb) resulted in greater than 99.9% tumor reduction in a preclinical cancer regression model of multiple myeloma compared to conventional NK cells (Press release, Indapta Therapeutics, AUG 10, 2021, View Source [SID1234586257]). G-NK cells are a naturally occurring subset of NK cells that when expanded and "turbocharged" using Indapta’s proprietary manufacturing process, boost the efficacy of mAbs and can overcome the cost, inconsistency, and possible toxicities associated with autologous T-cell therapies commonly used in the treatment of multiple myeloma.

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"These results suggest Indapta’s G-NK cells have the potential to be a potent off-the-shelf NK cell therapy that could enhance the efficacy of most mAbs," said Guy DiPierro, founder and chief executive officer of Indapta. "The promising results showed G-NK cells, compared to conventional NK cells, significantly enhanced anti-tumor cell activity of two FDA-approved mAbs for multiple myeloma and were able to maintain this augmented activity, expand in vivo, and have greater persistence even after cryopreservation. Together with a simplified and consistent, high-yield commercial-scale manufacturing platform at Lonza, we have the potential to produce an NK cell therapy with outstanding efficacy at a lower cost than other engineered cell therapies."

Austin Bigley, Indapta’s vice president, research and development, said, "This data suggests our G-NK cell therapy, which has been optimized with a donor phenotyping and genotyping selection process and our propriety expansion method produces a highly functional, anti-tumor NK cell product when coupled with a therapeutic antibody."

This preclinical study examined the ability of G-NK cells to improve the efficacy of mAbs in multiple myeloma. Results from in vitro assays showed G-NK cells had six-fold higher anti-myeloma cell-killing activity compared with conventional NK cells when combined with the mAb daratumumab or elotuzumab (p<0.001). Separately, in a murine persistence study, cryopreserved G-NK cells showed more than 10 times greater persistence in peripheral blood (p<0.001) and spleen (p<0.001) compared with conventional NK cells at Day 31 post-infusion. Persistence in bone marrow was also higher (p<0.05). Additionally, in a mouse model of multiple myeloma that investigated tumor regression, the combination of G-NK cells and daratumumab led to a greater than 99.9% reduction in tumor burden compared with the combination of conventional NK cells and daratumumab (p<0.001). At Day 57 post-tumor inoculation, all seven mice treated with the G-NK cells in combination with daratumumab were alive, and the myeloma tumor burden was eliminated in five of seven mice. In contrast, no mice treated with conventional NK cells and daratumumab survived to Day 57. Further, Indapta allowed the cancer to grow for two weeks post-inoculation until therapy was started, resulting in a cancer regression model as opposed to a cancer suppression model, as is often seen in other pre-clinical NK cell models.

"We believe these findings underscore the promise of Indapta’s naturally occurring G-NK cell therapy to augment the effects of therapeutic antibodies more than conventional NK cells," said Nina Shah, M.D., study investigator and professor, Department of Medicine, University of California, San Francisco. "The data suggest that Indapta’s G-NK cell therapy have the potential to be a much needed, universal allogeneic cell source that could amplify the effect of therapeutic antibodies in patients, such as those with relapsing or refractory multiple myeloma."

Indapta plans to file an Investigational New Drug (IND) application for its G-NK cell therapy in combination with a mAb in multiple myeloma by the end of 2021 and expects to initiate clinical trials in early 2022.

About Indapta’s G-NK Cell Therapy

Indapta Therapeutics is developing a universal, allogeneic G-NK cell therapy designed to substantially improve the cytotoxicity of monoclonal antibody (mAb) therapy in multiple cancers. G-NK cells are a specific and potent subset of NK (natural killer) cells with specialized anti-tumor activity resulting from an epigenetic change rather than engineering. Indapta has further enhanced G-NK cells via specific G-NK cell subset selection and its proprietary manufacturing process, which, when combined, produce a G-NK cell therapy that demonstrates higher efficacy, persistence and enhanced cryopreservation.

When a mAb binds to the tumor target and Indapta’s G-NK cell therapy, it initiates the release of dramatically more cancer-killing compounds than conventional NK cells, allowing for increased efficacy and potentially less frequent dosing. Indapta’s off-the-shelf G-NK cell therapy is further differentiated from other NK cell therapies in that it is a cell banked product with low variability. In vivo studies have demonstrated the safety and efficacy of Indapta’s G-NK cell therapy.

On August 10, 2021 Blue Earth Diagnostics, a Bracco company and recognized leader in the development and commercialization of innovative PET radiopharmaceuticals, reported that presentations at the upcoming 2021 Society of Neuro-Oncology (SNO) Conference on Brain Metastases, to be held in a virtual format from August 19 to 20, 2021 (Press release, Blue Earth Diagnostics, AUG 10, 2021, View Source [SID1234586256]). The conference, the Third Annual Conference on Brain Metastases, is conducted by SNO and the American Association of Neurological Surgeons (AANS)/CNS Section on Tumors. Details of the presentations to be given by Blue Earth Diagnostics and its collaborators are listed below.

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Note: 18F-fluciclovine, under the tradename Axumin (fluciclovine F 18) injection, is an FDA-approved and commercially available molecular imaging radiopharmaceutical for use in PET imaging in men with suspected prostate cancer recurrence based on elevated blood prostate specific antigen (PSA) levels following prior treatment. The safety and efficacy of 18F-fluciclovine PET imaging for the detection of recurrent brain metastases has not been established.

Highlighted 18F-Fluciclovine Scientific Presentations

All 2021 SNO Conference on Brain Metastases presentations are available beginning 11:00 a.m. ET, Thursday, August 19, 2021.

Title:

Trial in Progress: A Prospective, Multicenter Phase 2b Study to Establish Image Interpretation Criteria for 18F-Fluciclovine PET in Detecting Recurrent Brain Metastases after Radiation Therapy (PURSUE)
Presenter: Rupesh Kotecha, MD, Baptist Health South Florida, Miami, FL, USA
Abstract:

TRLS-01

Title:

Trial in Progress: A Multicenter Phase 3 Study to Establish the Diagnostic Performance of 18F-Fluciclovine PET in Detecting Recurrent Brain Metastases after Radiation Therapy (REVELATE)
Presenter: Alain Chaglassian, MD, Blue Earth Diagnostics Inc., Burlington, MA
Abstract:

TRLS-02
Blue Earth Diagnostics invites participants at the SNO Conference on Brain Metastases 2021 to attend the presentations above. For more information about the meeting, please see the online program here.

Blue Earth Diagnostics has two clinical studies underway to investigate the use of 18F-fluciclovine PET in the detection of recurrent brain metastases. The Phase 2 PURSUE trial is designed to establish image interpretation criteria for 18F-fluciclovine PET in detecting recurrent brain metastases. REVELATE is a Phase 3 study designed to evaluate its diagnostic performance in the detection of recurrent brain metastases in patients previously treated with radiation therapy. Further information about these trials, including a current list of clinical trial sites, can be found on www.clinicaltrials.gov (PURSUE, NCT04410367; REVELATE, NCT04410133).

About 18F-Fluciclovine PET

18F-Fluciclovine PET is a novel diagnostic imaging radiopharmaceutical for PET imaging to visualize the increased amino transport that occurs in malignant tumors. It consists of a synthetic amino acid that is preferentially taken up by cancer cells compared with surrounding normal tissues and is labeled with the radioisotope 18F for PET imaging. 18F-Fluciclovine is under investigation by Blue Earth Diagnostics for potential use for the detection of recurrent brain metastases in adult patients who have previously undergone radiation therapy. 18F-Fluciclovine was invented at Emory University, in Atlanta, Ga., with much of the fundamental clinical development carried out by physicians at Emory University’s Department of Radiology and Imaging Sciences. Blue Earth Diagnostics licensed 18F-fluciclovine from GE Healthcare and is investigating the molecule for other potential cancer indications, including in neuro-oncology.

U.S. Indication and Important Safety Information About Axumin

INDICATION

Axumin (fluciclovine F 18) injection is indicated for positron emission tomography (PET) imaging in men with suspected prostate cancer recurrence based on elevated blood prostate specific antigen (PSA) levels following prior treatment.

IMPORTANT SAFETY INFORMATION

Image interpretation errors can occur with Axumin PET imaging. A negative image does not rule out recurrent prostate cancer and a positive image does not confirm its presence. The performance of Axumin seems to be affected by PSA levels. Axumin uptake may occur with other cancers and benign prostatic hypertrophy in primary prostate cancer. Clinical correlation, which may include histopathological evaluation, is recommended.
Hypersensitivity reactions, including anaphylaxis, may occur in patients who receive Axumin. Emergency resuscitation equipment and personnel should be immediately available.
Axumin use contributes to a patient’s overall long-term cumulative radiation exposure, which is associated with an increased risk of cancer. Safe handling practices should be used to minimize radiation exposure to the patient and health care providers.
Adverse reactions were reported in ≤ 1% of subjects during clinical studies with Axumin. The most common adverse reactions were injection site pain, injection site erythema and dysgeusia.
To report suspected adverse reactions to Axumin, call 1-855-AXUMIN1 (1-855-298-6461) or contact FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.