On August 31, 2021 CorMedix Inc. (Nasdaq: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of infectious and inflammatory disease, reported financial results for the second quarter and six months ended June 30, 2021 and provided an update on recent developments (Press release, CorMedix, AUG 31, 2021, View Source [SID1234587038]).

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Recent Business Highlights:

CorMedix remains focused in its efforts to resolve the deficiencies sent to the third-party manufacturer in the Post-Application Action Letter and remains on schedule to re-submit the DefenCath New Drug Application in the fourth quarter of 2021.
CorMedix received approximately $1.3 million in net proceeds upon the closing of the sale of tax benefits pursuant to the New Jersey Technology Business Tax Certificate Transfer (NOL) program.
CorMedix has approximately $78.3 million in cash on hand and short-term investments as of June 30; we believe this is sufficient to fund operations at least through 2022.
Khoso Baluch, CorMedix CEO commented, "We are pleased with the progress we are making as we address the deficiencies identified by FDA at the third-party manufacturing facility. We remain confident in our efforts to bring DefenCath to hemodialysis patients as an important novel antimicrobial catheter lock solution to reduce catheter related blood stream infections in patients receiving hemodialysis via central venous catheters."

Second Quarter and Six Month 2021 Financial Highlights

For the second quarter 2021, CorMedix recorded a net loss of $4.6 million, or $0.12 per share, compared with a net loss of $3.8 million, or $0.14 per share, in the second quarter of 2020. The higher net loss recognized in 2021 compared with 2020 was due to increased personnel expenses and a lower tax benefit from the NJ NOL program, partially offset by raw materials purchased during 2Q of 2020. We recorded an increase in SG&A and a decrease in R&D expenses. We recognized a tax benefit of $1.3 million in 2021 from the sale of our NJ Net Operating Losses compared with a $5.2 million benefit recorded in 2020.

Operating expenses in the second quarter of 2021 decreased approximately 34% to $5.9 million, compared with $8.9 million in the second quarter of 2020. R&D expense decreased approximately 56% to $2.5 million, mainly due to a $3.4 million purchase of raw material during the second quarter of 2020 for the manufacturing of DefenCath prior to its potential marketing approval. SG&A expense increased approximately 4% to $3.4 million compared with $3.2 million in the second quarter of 2020. This increase was driven primarily by an increase in non-cash charges for stock-based compensation, and an increase in personnel expenses as a result of additional hires, partially offset by a decrease in consulting fees.

For the six months ended June 30, 2021, CorMedix recorded a net loss of $11.8 million, or $0.32 per share, compared with a net loss of $9.3 million, or $0.36 per share, in the first half of 2020. Operating expenses in the first half of 2021 were $13.1 million, compared to $14.6 million in the first half of 2020, a decrease of approximately 10%. This decrease was primarily due to the raw material purchased in 2nd quarter of 2020 partially offset by higher SG&A expenses throughout the organization.

Total cash on hand and short-term investments as of June 30, 2021 was $78.3 million, excluding restricted cash of $0.2 million. The Company believes that, based on the Company’s cash resources at June 30, 2021, it has sufficient resources to fund operations at least through 2022, after taking into consideration the costs for re-submission of the NDA and initial preparations for the commercial launch for DefenCath.

Conference Call Information

The management team of CorMedix will host a conference call and webcast today, August 12, 2021, at 4:30 PM Eastern Time, to discuss recent corporate developments and financial results. Call details and dial-in information is as follows:

On August 31, 2021 Eli Lilly and Company (NYSE: LLY) reported that it will participate in Citi’s 16th Annual BioPharma Virtual Conference on September 8 and 9, 2021 (Press release, Eli Lilly, AUG 31, 2021, View Source [SID1234587037]). Anat Ashkenazi, senior vice president and chief financial officer, will participate in a virtual fireside chat on Thursday, September 9 at 9:45 a.m., Eastern Time.

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A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s Investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.

On August 31, 2021 BeyondSpring (the "Company" or "BeyondSpring") (NASDAQ: BYSI), a global pharmaceutical company focused on the development of cancer therapeutics, reported a late-breaking oral presentation at the European Society for Medical Oncology 2021 Congress taking place virtually September 16-21, 2021 (Press release, BeyondSpring Pharmaceuticals, AUG 31, 2021, View Source;utm_medium=rss&utm_campaign=beyondspring-announces-late-breaking-oral-presentation-of-final-phase-3-dublin-3-data-with-the-plinabulin-docetaxel-combination-versus-docetaxel-alone-in-2nd-3rd-line-non-small-cell-lung-cancer-patien [SID1234587036]). This will include the final intention-to-treat (ITT) dataset from the Company’s DUBLIN-3 Phase 3 registrational trial of their first-in-class lead asset, plinabulin, in combination with docetaxel vs. docetaxel alone for the treatment of 2nd/3rd line non-small cell lung cancer (NSCLC) patients with EGFR wild type.

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Additional Details:

Title: A Global Phase (Ph) 3 Trial with the Plinabulin/Docetaxel (Plin/Doc) combination vs. Doc in 2nd/3rd Line NSCLC Patients (pts) with EGFR-wild type (wt) Progressing on a Prior Platinum-Based Regimen
Session: Proffered Paper session – NSCLC, metastatic 2
Date: September 20, 2021 from 2:10 – 2:20 p.m. CEST
Location: Channel 4
Presentation Number: LBA48
Speaker: Baohui Han, M.D., Ph.D, Professor in the Department of Respiratory Medicine, Shanghai Chest Hospital, China

"The ESMO (Free ESMO Whitepaper) Congress is the perfect venue to present high-impact clinical oncology findings such as data from our successful DUBLIN-3 study that demonstrates plinabulin’s ability to improve overall survival in 2nd/3rd line NSCLC patients," said Dr. Lan Huang, co-founder, chairwoman and chief executive officer of BeyondSpring. "Importantly, the direct durable anti-cancer benefit shown by plinabulin in this study is a gateway to developing plinabulin as part of immuno-oncology combos in multiple cancer indications. In the coming months, BeyondSpring plans to apply for an NDA in the U.S. and China for the use of plinabulin in these NSCLC patients. This would add to the ongoing U.S. and China regulatory review of plinabulin in combination with G-CSF for the prevention of chemotherapy-induced neutropenia, which has a PDUFA date of November 30, 2021. We’ve had a tremendous year of milestones at BeyondSpring, and we are grateful to all of our collaborators for their great efforts in helping to bring plinabulin to cancer patients globally."

About Plinabulin
Plinabulin, BeyondSpring’s lead asset, is a selective immunomodulating microtubule-binding agent (SIMBA), which is a potent antigen presenting cell (APC) inducer. It is a novel, intravenous infused, patent-protected, NDA stage asset for CIN prevention and a Phase 3 anti-cancer candidate for non-small cell lung cancer (NSCLC). Plinabulin triggers the release of the immune defense protein, GEF-H1, which leads to two distinct effects: first is a durable anticancer benefit due to the maturation of dendritic cells resulting in the activation of tumor antigen-specific T-cells to target cancer cells, and the second is early-onset of action in CIN prevention after chemotherapy by boosting the number of hematopoietic stem/progenitor cells (HSPCs). Plinabulin received Breakthrough Therapy designation from both U.S. and China FDA for the CIN prevention indication. As a "pipeline in a drug," plinabulin is being broadly studied in combination with various immuno-oncology agents that could boost the effects of the PD-1/PD-L1 antibodies and re-sensitize PD-1/PD-L1 antibody-resistant patients.

On August 31, 2021 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported preliminary results from its Phase 1 clinical trial of P-PSMA-101, the Company’s solid tumor autologous CAR-T product candidate to treat patients with metastatic castrate-resistant prostate cancer (mCRPC) (Press release, Poseida Therapeutics, AUG 31, 2021, View Source [SID1234587035]). These data will be presented at the 6th Annual CAR-TCR Summit virtual meeting at 10:00am ET today in a presentation entitled, "P-PSMA-101 is a High-Tscm Autologous CAR-T Targeting PSMA Producing Exceptionally Deep and Durable Responses in Castration-Resistant Metastatic Prostate Cancer."

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"We are excited about the preliminary data from our Phase 1 trial of P-PSMA-101, which provides further evidence of the effectiveness of our CAR-T platform for solid tumor cancers," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer of Poseida, who will present at the CAR-TCR Summit. "To date, other CAR-T therapeutics have not had much success outside of hematologic malignancies. The deep and durable responses in our trial demonstrate that CAR-T products have the potential to work well against solid tumors, even at low doses, when using the appropriate technology platform."

Efficacy:

As of the cutoff date, the study had enrolled a total of nine patients with mCRPC: five patients at Dose A who each received a single treatment of 0.25X10E6 cells/kg (an average of about 20M cells), and four patients at Dose B, who each received a single treatment of 0.75X10E6 cells/kg (an average of about 60M cells). All patients received a lymphodepletion regimen consisting of 30 mg/m2 fludarabine + 300 mg/m2 cyclophosphamide. Patients were heavily pre-treated, having received an average of six prior lines of therapy with a median time since diagnosis of 6.4 years.

Key findings included:

Five patients dosed showed measurable declines in PSA levels

Three patients treated showed a greater than 50% decline in PSA levels and had concordant improvements in PSMA-PET imaging

One patient demonstrated evidence of complete tumor elimination and remains in a durable response of greater than five months at the time of this presentation

"This innovative Poseida PSMA-directed CAR T cell platform has demonstrated a robust anti-tumor response in patients with metastatic castration resistant prostate cancer," commented Susan F. Slovin, M.D., Ph.D., Associate Vice Chair of Academic Administration at Memorial Sloan Kettering Cancer Center and investigator on the trial. "This is the first time that I have seen such impressive responses with an immunotherapy product. The responses of my patients in the trial are far beyond my expectations."

Safety and Tolerability:

P-PSMA-101 demonstrated a favorable safety and tolerability profile. After a previously reported case of Macrophage Activation Syndrome (MAS) exacerbated by patient non-compliance, only three cases of possible Cytokine Release Syndrome (CRS) were observed, which were all low grade (1/2) and were managed well with early treatment. No cases of neurotoxicity (CRES/ICANS) were observed as of the cutoff date.

The Phase 1 trial is an open label, multi-center, 3+3 dose-escalating study designed to assess the safety of P-PSMA-101 in up to 40 adult subjects with mCRPC. The primary objectives of this study are to determine the safety, efficacy, and maximum tolerated dose of P-PSMA-101. Additional information about the study is available at www.clinicaltrials.gov using identifier: NCT04249947.

"We believe the key to success in solid tumors is a product with a high percentage of desirable stem cell memory T cells (Tscm)," said Matthew Spear, M.D., Chief Medical Officer of Poseida. "In this study, we have demonstrated that a high-percentage Tscm CAR-T product can home to the bone marrow and, in at least one case, completely eliminate tumor. This bone marrow homing property may be particularly important for bone avid diseases such as prostate adenocarcinoma. Importantly, the favorable tolerability associated with our Tscm CAR-T products has carried over to prostate cancer where we have so far seen manageable cytokine release syndrome and no neurotoxicity."

Company-Hosted Conference Call and Webcast Information

Poseida’s management team will host a conference call and webcast today, August 31, 2021 at 11:00am ET. The dial-in conference call numbers for domestic and international callers are (866) 939-3921 and (678) 302-3550, respectively. The conference ID number for the call is 50220147. Participants may access the live webcast and the accompanying presentation materials on Poseida’s website at www.poseida.com in the Investors section under Events and Presentations. An archived replay of the webcast will be available for 30 days following the event.

Additional CAR-TCR Summit Highlights

Presentation: "Developing CAR-T Cells for Multiple Myeloma: From Autologous to Allogeneic"

Session Date/Time: Wednesday, September 1, 2021, 4:00pm ET

Presenter: Matthew Spear, M.D., CMO, Poseida Therapeutics

This presentation will outline Phase 1 and 2 development of the Company’s lead autologous P-BCMA-101 CAR-T therapy and insights that were used to develop a fully allogeneic version, P-BCMA-ALLO1 that is expected to enter the clinic soon. The presentation will be part of the afternoon session on the Clinical Management Track.

Presentation: "Advancing Nonviral Manufacturing for Multi-Product Allogeneic T-Cell Therapies"

Session Date/Time: Wednesday, September 1, 2021, 4:30pm ET

Presenter: Devon Shedlock, Ph.D., SVP Research & Development, Poseida Therapeutics

This presentation will discuss how Poseida’s piggyBac DNA Delivery System, Cas-CLOVER Site-specific Gene Editing System and Booster Molecule are used to manufacture multi-product, fully allogeneic T-cell therapies. The Company will also discuss how efficient multiplexed Cas-CLOVER gene editing exhibits low to no off-target editing or translocations as determined by next-generation sequencing, and how the Company’s Booster Molecule helps to protect against the "allo tax," maintaining a favorable high-stem cell memory T cell (Tscm) product and enabling up to hundreds of doses in a single manufacturing run. This presentation will be part of the afternoon session on the Manufacturing Track.

Presentation: "Developing ‘Off-the-Shelf’ CAR-T Cells for Bone Marrow Transplant Conditioning"

Session Date/Time: Thursday, September 2, 2021, 9:00am ET

Presenter: Nina Timberlake, Ph.D., Associate Director, Research (Gene Therapy), Poseida Therapeutics

This presentation will discuss leveraging the piggyBac DNA Delivery System and Cas-CLOVER Site-specific Gene Editing System to generate off-the-shelf fully allogeneic CAR-T cells to specifically target hematopoietic cells in the bone marrow. This potential therapeutic could be used as a non-myeloablative conditioning regimen for hematopoietic stem cell transplant or as a therapeutic for the treatment of acute myeloid leukemia (AML). The presentation will occur as part of the conference’s Focus Day, "CAR-TCR Beyond Oncology: Fundamental Biology & Mechanisms of Action Beyond Oncology."

The full presentations at the CAR-TCR Summit will be made available on Poseida’s website at their respective session times.

On August 31, 2021 Eagle Pharmaceuticals, Inc. (Nasdaq: EGRX) ("Eagle" or the "Company") reported that the U.S. Patent and Trademark Office has granted the Company U.S. Patent No. 11,103,483, entitled "Formulations of Bendamustine (Press release, Eagle Pharmaceuticals, AUG 31, 2021, View Source [SID1234587030])." Eagle is submitting the ‘483 patent for listing in the U.S. Food and Drug Administration’s Orange Book for both BENDEKA and BELRAPZO.

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Eagle had asserted several Orange Book-listed patents against Slayback Pharma LLC, Apotex Inc. et al, Mylan Laboratories Limited, and Fresenius Kabi USA, LLC, related to their respective abbreviated new drug applications referencing BENDEKA. On July 6, 2020, the District Court for the District of Delaware had held these asserted patents both valid and infringed. Apotex, Mylan, and Fresenius appealed this ruling. Previous to the appellate hearing, Eagle settled the Fresenius litigation. On August 13, 2021, the United States Court of Appeals for the Federal Circuit affirmed that the asserted patents were both valid and infringed. Both the asserted patents and the ‘483 patent expire in 2031.

"We are pleased that this appellate decision, as well as the newly issued patent, continue to strengthen Eagle’s intellectual property rights for both BENDEKA and BELRAPZO," stated Scott Tarriff, Chief Executive Officer of Eagle Pharmaceuticals.