On February 25, 2021 Celsion Corporation (NASDAQ: CLSN), a clinical-stage development company focused on DNA-based immunotherapy and next-generation vaccines, reported an update on its Phase I/II OVATION 2 Study with GEN-1 in patients with advanced ovarian cancer, including interim observations (Press release, Celsion, FEB 25, 2021, View Source [SID1234575744]). GEN-1 is Celsion’s DNA-mediated interleukin-12 (IL-12) immunotherapy designed using TheraPlas, its proprietary, synthetic, non-viral nanoparticle delivery system platform.

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The OVATION 2 Study combines GEN-1 with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three adjuvant cycles of chemotherapy and up to nine additional weekly GEN-1 treatments, the goal of which is to delay progression and improve overall survival. The OVATION 2 Study is an open-label, 1-to-1 randomized trial, 80% powered to show the equivalent of a 33% improvement in progression-free survival (PFS) (HR=0.75), the primary endpoint, when comparing the treatment arm (standard of care + GEN-1) with the control arm (standard of care alone).

To date, the Company has enrolled approximately one-third, or 34 patients, of the anticipated 110 patients to be enrolled into the OVATION 2 Study, of which 20 are in the treatment arm and 14 are in the control. Currently, 27 patients have had their interval debulking surgery with the following results:

12 of 15, or 80%, of patients treated with GEN-1 had a R0 resection, which indicates a microscopically margin-negative complete resection in which no gross or microscopic tumor remains in the tumor bed.
7 of 12 patients, or 58%, of patients in the control arm had an R0 resection.
This interim data represents a 38% improvement in R0 resection rates for GEN-1- patients compared with control arm patients and is consistent with the reported improvement in resection scores noted in the encouraging Phase I OVATION I Study, the manuscript of which has been submitted for peer review publication.
"As the goal for surgical debulking is to eliminate microscopic disease, more ovarian cancer patients require neoadjuvant chemotherapy. However, little progress has been made in adding additional efficacious immunotherapy agents to standard neoadjuvant chemotherapy," said Premal H. Thaker, M.D., MSc., Professor in Gynecologic Oncology and Director of Gynecologic Oncology Clinical Research at Washington University School of Medicine in St. Louis and lead Principal Investigator for the OVATION 2 Study. "The results seen to date in the OVATION 2 Study are exciting and impactful for ovarian cancer patients."

The Company further reports that 22 clinical sites in the U.S. and Canada have been initiated, with three more sites expected to be added by the end of the first quarter. Clinical investigators met in early February in a virtual meeting and expressed excitement about the potential for GEN-1 to treat advanced ovarian cancer and, despite the challenges and earlier delays posed by the COVID-19 pandemic, they remain committed to completing enrollment in the study during the second half of 2021.

Commenting on the interim patient reports, Dr. Nick Borys, chief medical officer of Celsion, said, "We are gratified that such a high proportion of GEN-1 patients had no residual disease at the time of their debulking surgery. These results are consistent with what we observed in our Phase I (OVATION I) study. This is great news for the patients and the surgeons in our study as a R0 resection suggests a good clinical outcome. We are following our patients carefully to see how well they do long term. The OVATION 2 investigators remain enthusiastic about the potential of GEN-1 to treat late-stage ovarian cancer, which currently has few treatment options."

Celsion announced earlier this week that GEN-1 had received Fast Track designation from the U.S. Food and Drug Administration (FDA). This designation is intended to facilitate the development and expedite the regulatory review of drugs to treat serious conditions and fill an unmet medical need. According to the FDA, a Fast Track Drug must show some advantage over available therapy, including:

Showing superior effectiveness, effect on serious outcomes or improved effect on serious outcomes
Avoiding serious side effects of an available therapy
Decreasing a clinically significant toxicity of an available therapy that is common and causes discontinuation of treatment
About GEN-1 Immunotherapy

GEN-1, designed using Celsion’s proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anti-cancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The Company has previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy or a combination therapy in patients with advanced peritoneally metastasized primary or recurrent ovarian cancer, and recently completed a Phase Ib dose-escalation trial (OVATION 1 Study) of GEN-1 in combination with carboplatin and paclitaxel in patients with newly diagnosed ovarian cancer.

On February 25, 2021 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics to treat chronic diseases and extend healthy lifespan, reported that its Chief Executive Officer, Steven Engle, will present a company overview at the H.C. Wainwright Global Life Sciences Conference, being held virtually March 9 – 10, 2021 (Press release, CohBar, FEB 25, 2021, View Source [SID1234575743]).

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H.C. Wainwright Global Life Sciences Conference
Webcast: The presentation may be accessed via webcast on demand using the following link: View Source

On February 25, 2021 Altimmune, Inc. (Nasdaq: ALT), a clinical-stage biopharmaceutical company, reported financial results for the year ended December 31, 2020 and provided a corporate update (Press release, Altimmune, FEB 25, 2021, View Source [SID1234575742]).

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"The past year has been a transformative time for our Company as we made substantial progress in each of our five portfolio programs," said Vipin K. Garg, Ph.D., President and Chief Executive Officer. "During the year we initiated multiple clinical trials for several of our product candidates (T-COVID, ALT-801 and HepTcell) and completed preparations to begin a Phase 1 clinical trial of AdCOVID, which has begun enrolling volunteers. These achievements have set the stage for a busy and exciting year ahead, as we anticipate multiple data readouts from these programs over the coming months. With two promising technology platforms and five novel product candidates now advancing in clinical development, we believe 2021 has the potential to be a momentous year for Altimmune."

Program Highlights

AdCOVID:

Commenced enrollment in AdCOVID Phase 1 clinical trial evaluating a novel, needle-free intranasal delivery approach for COVID-19 vaccination
Altimmune has commenced enrollment in its Phase 1 clinical trial of AdCOVID, which is designed to evaluate a needle-free intranasal delivery approach for vaccination against COVID-19. Altimmune believes AdCOVID has the potential to become a leading candidate for COVID-19 vaccination based on its ease of administration, and the potential for reduced disease transmission, and cold chain-free vaccine distribution, if the product is demonstrated to have extended stability at room temperature. As demonstrated in the NasoShield and NasoVax clinical trials, the Company believes the expected attributes of AdCOVID make it ideally suited for use in a pediatric setting as the intranasal administration and expected tolerability profile are well suited to meet the needs of children.

The Phase 1 clinical trial will evaluate the safety and immunogenicity of AdCOVID in up to 180 healthy adult volunteers between the ages of 18 and 55. Subjects will receive AdCOVID at one of three dose levels administered as a nasal spray. In addition to the primary study endpoint of safety and tolerability, the immunogenicity of AdCOVID will be evaluated by serum IgG binding and neutralizing antibody titers, mucosal IgA antibody from nasal samples, and T cell responses. Altimmune anticipates having a full data readout from this Phase 1 study in Q2 2021.

Initiated development of additional AdCOVID vectors targeting emerging SARS-CoV-2 variants
The emergence of SARS-CoV-2 variants is raising concerns about the effectiveness of currently authorized vaccines and prompting vaccine developers to engineer new vaccine candidates to combat these viral mutations. Altimmune has initiated the development of vaccine candidates against several variants as one is likely to become dominant in the population in the coming months. Altimmune plans to have these new vaccine candidates ready for use in upcoming later-stage clinical trials.

Established a consortium of manufacturing partners for potential commercial supply of AdCOVID
Altimmune has executed agreements with three commercial manufacturing partners with significant experience in adenoviral vector production. The Company has also established relationships with leading drug product fill/finish partners with sufficient capacity to meet potential commercial demand. Together, the Company believes that this network of strategic manufacturing partners will ensure Altimmune’s commercial readiness to supply vaccine, assuming the clinical data support this advancement.

Furthered AdCOVID preclinical studies in collaboration with the University of Alabama at Birmingham (UAB) and Saint Louis University
Based on the promising preclinical data for AdCOVID published on the BioRxiv server, Altimmune continues preclinical studies of AdCOVID in collaboration with UAB and Saint Louis University to evaluate AdCOVID in additional animal models and to further evaluate heterologous prime boost regimens of AdCOVID in support of future clinical development activities. Data from these ongoing preclinical studies are expected in Q1 and Q2 2021.

ALT-801:

Commenced dosing in a Phase 1 clinical trial of ALT-801, a novel GLP-1/glucagon dual-agonist being evaluated for the treatment of NASH
Altimmune commenced dosing in a Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) clinical trial of ALT-801, a GLP-1/glucagon dual-agonist being developed for the treatment of NASH. This trial is being conducted in Australia and is expected to enroll approximately 100 volunteers. The primary pharmacodynamic endpoints in the trial are weight loss and reduction in liver fat, outcomes that have been associated with NASH resolution and fibrosis improvement in advanced clinical studies of other NASH therapeutics. The Company has successfully completed the initial phases of the study and anticipates a data read-out from the 6-week MAD study in Q2 2021, followed by 12-week data in Q3 2021.

Amended clinical trial protocol to extend MAD cohorts to incorporate 12-week Phase 1b study in Australia
Altimmune amended the clinical trial protocol for the ALT-801 Phase 1 clinical development program to incorporate its planned 12-week extension trial in patients with non-alcoholic fatty liver disease or NAFLD within the ongoing Phase 1 SAD/MAD trial in Australia. The Company believes that by incorporating the 12-week extension into this trial, it can avoid any potential impact of COVID-19 and maintain study timelines. Pending the results of this trial, Altimmune plans to transition rapidly to a 52-week, Phase 2, biopsy-trial based on NASH endpoints in early 2022. In parallel with these efforts, Altimmune continues to plan to file an Investigational New Drug (IND) application for ALT-801 in the United States in mid-2021.

Initiated chronic toxicology studies of ALT-801 to enable 52-week Phase 2 clinical study
Altimmune completed 6-week and 13-week GLP toxicology studies of ALT-801 with no significant toxicity or GI adverse events. The Company has initiated 6-month and 9-month GLP toxicology studies to support the planned 52-week biopsy-driven Phase 2 trial planned for early 2022.

T-COVID:

Completed Cohorts 1 and 2 in the Phase 1/2 trial of T-COVID in patients with early COVID-19
Altimmune, working with the Department of Defense, has completed the two safety cohorts in the EPIC (Efficacy and Safety of T-COVID in the Prevention of Clinical Worsening in COVID-19) study, a Phase 1/2 clinical trial of T-COVID, an investigational intranasally-administered therapeutic for the treatment of early COVID-19 infection. The trial is being overseen by an independent Data Safety Monitoring Committee, and no significant safety findings have been observed to date.

Cohort 3 is an efficacy and safety cohort that will include patients at higher-risk for severe COVID-19 infection, such as those 65 years or older, or those with one or more risk factors for severe COVID-19 complications. To ensure that a sufficient number of higher risk patients are enrolled, the study protocol was recently modified to require that a minimum number of patients meet one or more of these criteria in this final cohort. Additional enrichments of the study population are currently being evaluated to increase the event rates in the trial. While these modifications could extend the study timeline, the Company believes they could significantly enhance the probability of a meaningful trial outcome. Based on these changes, data from this trial is now expected in Q2 2021.

HepTcell:

Commenced dosing in a multinational Phase 2 clinical trial of HepTcell
In December, Altimmune began a multinational Phase 2 clinical trial of HepTcell, which is being conducted in the United States, Canada and Europe. The trial is a double-blind, randomized, placebo-controlled trial of 80 adult patients with HBeAg-negative inactive CHB and HBsAg ≤ 100 IU/mL.

HepTcell will be administered in 6 doses at 4-week intervals for 24 weeks, and patients will be followed for one year to evaluate safety and durability of response. The primary efficacy endpoint is virological response, defined as a 1-log reduction in HBsAg levels from baseline. Secondary efficacy endpoints include reactivation of anti-HBV T cell responses, HBsAg clearance, and other assessments of virologic response. Altimmune anticipates a data read-out from this trial in 1H 2022.

Financial Results for the Year Ended December 31, 2020

Altimmune had cash, cash equivalents and short-term investments of $216.0 million at December 31, 2020 compared to $37.3 million at December 31, 2019. The increase of $178.7 million is attributable to $213.5 million of net receipts during the year due primarily to its 2020 public offering, full utilization of the at-the-market offering program, and receipts from warrant exercises, offset by $34.4 million of cash used for operating activities.
Revenue was $8.2 million for the year ended December 31, 2020 compared to $5.8 million in the prior year period, an increase of $2.4 million. The change was primarily due to an increase in revenue under the Company’s U.S. government contracts due to timing of manufacturing and clinical trials for the NasoShield and T-COVID programs.
Research and development expenses were $49.8 million for the year ended December 31, 2020, compared to $17.8 million in the prior year period, representing an increase of $32.0 million. The increase was primarily attributable to increased costs related to development of AdCOVID, T-COVID and ALT-801 and an increase in the contingent liability for stock-based milestone payments associated with the acquisition of ALT-801.
General and administrative expenses were $13.2 million for the year ended December 31, 2020 compared to $8.5 million in the prior year period, an increase of $4.7 million. The increase is attributable to additional employee compensation as Altimmune’s workforce grew in 2020 along with an increase in professional costs.
Income tax benefit was $5.4 million for the year ended December 31, 2020, as compared to $59,000 for the same period in 2019. The increase is attributable to the Coronavirus Aid, Relief, and Economic Security Act (the "CARES Act") passed on March 27, 2020 which made temporary changes regarding the utilization and carry back of net operating losses.
Net loss attributed to common stockholders for the year ended December 31, 2020 was $49.0 million, or $1.91 net loss per share, compared to $21.0 million in the prior year, or $1.60 net loss per share. The difference in net loss is primarily attributable to higher research and development expenses and general and administrative expenses, offset by higher revenue and an increase in income tax benefit.
Conference Call Information

Following the conclusion of the call, the webcast will be available for replay on the Investor Relations page of the Company’s website at www.altimmune.com. The company has used, and intends to continue to use, the IR portion of its website as a means of disclosing material non-public information and for complying with disclosure obligations under Regulation FD.

On February 25, 2021 LIDDS reported that Interim report January – December 2020 (Press release, Lidds, FEB 25, 2021, View Source [SID1234575741])

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JANUARY – DECEMBER 2020

Net sales amounted to MSEK 0.3 (0.0)
Operating expenses amounted to MSEK -33.0 (-31.4)
Profit/loss before and after tax amounted to MSEK -32.3 (-31.4)
Earnings per share amounted to SEK -1.20 (-1.34)
Cash flow from operating activities amounted to MSEK -27.4 (-31.2)
Equity amounted to MSEK 42.8 (15.5) and the debt/equity ratio was 79% (72%)
OCTOBER – DECEMBER 2020

Net sales amounted to MSEK 0.3 (0.0)
Operating expenses amounted to MSEK -12.9 (-8.5)
Profit/loss before and after tax amounted to MSEK -12.5 (-8.5)
Earnings per share amounted to SEK -0.42 (-0.36)
Cash flow from operating activities amounted to MSEK -10.1 (-9.8)
Equity amounted to MSEK 42.8 (15.5) and the debt/equity ratio was 79% (72%)
SIGNIFICANT EVENTS DURING THE FORTH QUARTER 2020

Intratumoral injection of NZ-TLR9, NanoZolid formulation of a Toll-Like Receptor 9 (TLR9), results in strong antitumoral efficacy combined with prominent antitumoral immune responses in mouse tumor models. NZ-TLR9 forms an intratumoral depot which releases the TLR9 agonist for least 6 weeks and thus minimizes the need for repeated injections.
The warrants programme was finalized in October 2020 and 0.8 percent of the warrants issued was exercised.
A newly revised guideline from the Chinese National Medical Products Administration (NMPA) has extended the requirements to a full registration dossier for the conditional market approval (CMA). LIDDS licensee Jiangxi Puheng Pharma is therefore aiming to submit the application for CMA for the prostate cancer drug candidate Liproca Depot in China during Q1, 2021.
A review article in OncoTargets and Therapy journal defines LIDDS as a key player in TLR9 agonist research field and being the only provider having a sustained release product in development.
LIDDS announced that the company will apply for a relisting of its shares from Nasdaq First North to Nasdaq Stockholm Main Market in 2021.
Nina Herne will assume the role of CEO for LIDDS as from April 19, 2021. Monica Wallter will continue in LIDDS as Senior Adviser.
SIGNIFICANT EVENTS FOLLOWING THE END OF THE PERIOD

The Phase I study where NanoZolid is combined with docetaxel continues with dose escalation. New patients are in recruitment to receive injection in solid tumors with new dose of docetaxel.
European Urology Focus has accepted a scientific article describing LIDDS Phase IIb study results with Liproca Depot.
LIDDS has filed a patent application for local intracranial treatment of brain tumors. The NanoZolid technology enables controlled and sustained release of oncology drugs. Preclinical study has showed that NanoZolid injected as a depot in brain is tolerable and safe.
LIDDS has signed a manufacturing agreement for NZ-TLR9 with Pharmidea in Latvia.

The interim report is available on the company’s website, go to View Source

On February 25, 2021 argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancer, reported that it will host a conference call and audio webcast on March 4, 2021 at 2:30 p.m. CET (8:30 a.m. ET) to discuss its full year 2020 financial results and provide a fourth quarter business update (Press release, argenx, FEB 25, 2021, View Source [SID1234575740]).

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A webcast of the live call may be accessed on the Investors section of the argenx website at argenx.com/investors. A replay of the webcast will be available on the argenx website for one year following the call.