On March 15, 2022 Allarity Therapeutics, Inc. ("Allarity" or the "Company"), a clinical-stage pharmaceutical company developing novel oncology therapeutics together with drug-specific DRP companion diagnostics for personalized cancer care, reported an update on its lead dovitinib program (Press release, Allarity Therapeutics, MAR 15, 2022, View Source [SID1234610125]).

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On February 18, 2022, the Company announced that the U.S. Food and Drug Administration ("FDA") had provided the Company with Refusal to File ("RTF") letters regarding the new drug application ("NDA") for dovitinib, and its accompanying pre-market approval ("PMA") application for the DRP-Dovitinib companion diagnostic, for the third-line treatment of metastatic renal cell carcinoma ("mRCC"). In its announcement, Allarity stated that it intends to seek guidance concerning information, data, and specific deliverables that the agency would require for a resubmitted NDA and PMA to be deemed complete. The Company also stated that it anticipates that a new prospective clinical trial will be required to overcome the FDA’s outstanding objections.

Following several weeks of analysis by Company leadership together with clinical and regulatory experts, Allarity has now filed a formal request with the FDA for a "Type C" meeting to further discuss potential clinical paths to support approval of dovitinib, together with its DRP-Dovitinib companion diagnostic, in view of the FDA’s recent RTFs. According to FDA guidelines, "A Type C meeting is any meeting other than a Type A or Type B meeting between CBER or CDER and a sponsor or applicant regarding the development and review of a product." The Type C meeting is typically scheduled within 75 days of FDA receipt of the written meeting request. The Company anticipates providing a further update on the outcome of its FDA meeting and the future of the dovitinib program before the end of the third quarter of this year.

"Welook forward to working closely with the FDA and we remain highly confident in the clinical profile of dovitinib, together with the DRP-Dovitinib companion diagnostic. We are determined to further advance this product candidate as a potential new treatment option for cancer patients," said Allarity’s CEO Steve Carchedi. "With clarification fromthe FDA following our requested Type C meeting, we hope to have a clinical path forward with the goal of refiling our NDA and PMA once additional clinical data are in hand ."

" I remain enthusiastic about dovitinib, together with its DRP – Dovitinib companion diagnostic, as a promising new treatment option for mRCC patients , " stated Professor Roberto Pili, M.D., Associate Dean for Cancer Research and Integrative Oncology at the University at Buffalo Jacobs School of Medicine and Biomedical Sciences. "These patients, and their treating oncologists, are greatly in need of new precision medicines, coupled with validated companion diagnostics, to help select and treat the most likely responders. Although the landscape of treatment options for later-stage mRCC is evolving to include combination therapies, I continue to see a potential place for dovitinib with its DRP companion diagnostic in the treatment of these patients."

On March 15, 2022 Altimmune, Inc. (Nasdaq: ALT), a clinical-stage biopharmaceutical company, reported financial results for the fourth quarter and full year ended December 31, 2021 and provided a corporate update (Press release, Altimmune, MAR 15, 2022, View Source [SID1234610124]).

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"We expect the next 12 months to be a period of intense execution with value-creating data readouts from multiple clinical trials. We are extremely pleased with the progress of our pemvidutide (ALT-801) program as we showed double-digit weight loss after 12 weeks of treatment, good tolerability without the use of dose titration, and pronounced decreases in serum lipids commonly associated with cardiovascular disease. We also observed a remarkable reduction in liver fat content to undetectable levels after only 6 weeks of pemvidutide treatment in subjects with hepatic steatosis, or fatty liver," said Vipin K. Garg, Ph.D., President and Chief Executive Officer at Altimmune. "In addition to our ongoing 12-week Phase 1b NAFLD trial, we expect to initiate a 48-week Phase 2 trial of pemvidutide in obesity, the MOMENTUM trial, in the next few weeks and look forward to sharing data from both of these trials later this year. In addition, we expect to read out our HepTcell trial in the first half of 2023."

Recent Highlights and Anticipated Milestones:

Pemvidutide1 (ALT-801)

Enrollment in Phase 1b nonalcoholic fatty liver disease (NAFLD) trial is over 90% complete, and data readout is expected in Q3 2022

The 12-week trial is being conducted at 15 sites in the U.S., with Dr. Stephen A. Harrison serving as Principal Investigator. The trial will be comprised of 72 non-diabetic and diabetic subjects across four treatment arms (pemvidutide 1.2, 1.8, 2.4 mg and placebo).
The primary efficacy readouts of this trial are liver fat reduction and weight loss.
A 52-week biopsy driven Phase 2 non-alcoholic steatohepatitis (NASH) trial is expected to follow the conclusion of the NAFLD trial.

Received U.S. Food and Drug Administration (FDA) clearance of pemvidutide investigational new drug application (IND) for obesity – Initiation of the Phase 2 MOMENTUM trial of pemvidutide in obesity expected in the first quarter of 2022

The trial is expected to enroll approximately 320 non-diabetic subjects with either obesity or overweight with at least one obesity-related complication. Subjects will be randomized 1:1:1:1 to receive either 1.2 mg, 1.8 mg, 2.4 mg pemvidutide or placebo administered weekly for 48 weeks.
The primary endpoint of the MOMENTUM trial is the relative (percent) change in body weight at 48 weeks compared to baseline, with additional readouts including metabolic and lipid profiles, cardiovascular measures and glucose homeostasis.
An interim analysis is planned to assess changes in body weight after 24 weeks of treatment, with an expected readout in Q4 2022.

Initiated a double-blind, placebo-controlled 12-week extension to the ongoing NAFLD Phase 1b Trial

This extension will allow subjects to receive up to a total of 24 weeks of pemvidutide or placebo and provide a read out on weight loss at 24 weeks.
Topline data from the extension trial expected in Q4 2022.
Completed 6-month and 9-month toxicology studies of pemvidutide in rats and non-human primates

No significant findings, including no ALT or blood glucose elevations, were reported.
Toxicology results support 24-week NAFLD extension and 48-week obesity studies.
Initiated a 12-week Phase 1 trial to characterize effects of pemvidutide on glucose control in diabetic population
This represents a follow-on to the evaluation of a pre-diabetic population in our first-in-human trial in which reductions of insulin resistance and maintenance of glucose control were observed.
Results of a drug-drug interaction trial of pemvidutide expected in the first half of 2022
HepTcell

Enrollment ongoing for the Phase 2 clinical trial in chronic hepatitis B subjects, with study readout expected H1 2023
Readouts from this trial are expected to include virological markers of hepatitis B infection and functional cure.
Financial Results for the Three Months Ended December 31, 2021

Altimmune had cash, cash equivalents, short-term investments and restricted cash totaling $190.3 million at December 31, 2021.
Revenue was $3.3 million for the three months ended December 31, 2021 compared to $2.3 million in the same period in 2020. The increase in revenue quarter over quarter was primarily due to the receipt of prior period rate adjustments under the Company’s U.S. government contract for NasoShield, partially offset by the discontinuation of development work under prior programs.
Research and development expenses were $20.2 million for the three months ended December 31, 2021, compared to $9.0 million in the same period in 2020. The change was primarily the result of the increased costs related to the development of pemvidutide and an increase in the contingent liability for stock-based milestone payments associated with the acquisition of pemvidutide, partially offset by the discontinuation of development work for prior clinical programs.
General and administrative expenses were generally consistent period-over-period with $3.8 million recognized for the three months ended December 31, 2021 and $4.1 million in the same period in 2020.
Net loss for the three months ended December 31, 2021 was $23.9 million, or $0.57 net loss per share, compared to $10.6 million in the same period in 2020, or $0.29 net loss per share, due to the factors noted above.
Conference Call Information

Date: Tuesday, March 15
Time: 8:30 am Eastern Time
Domestic Dial-in: (844) 615-6509
International Dial-in: (918) 922-3148
Conference ID: 4557398
Webcast: View Source
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.

About Pemvidutide
Pemvidutide is a novel, investigational, peptide-based GLP-1/glucagon dual receptor agonist in development for the treatment of obesity and NASH. Activation of the GLP-1 and glucagon receptors is believed to mimic the complementary effects of diet and exercise on weight loss, with GLP-1 suppressing appetite and glucagon increasing energy expenditure. By combining GLP-1 and glucagon activity in a single peptide, pemvidutide has the potential to achieve weight loss comparable to bariatric surgery. Pemvidutide incorporates the EuPortTM domain, a proprietary technology that increases its serum half-life for weekly dosing while slowing the entry of pemvidutide into the bloodstream, which may improve its tolerability. In a 12-week Phase 1 clinical trial, pemvidutide-treated subjects demonstrated striking reductions in body weight, liver fat and serum lipids commonly associated with cardiovascular disease.

About HepTcell
HepTcell is a novel, investigational, immunotherapeutic comprised of nine synthetic peptides representing conserved hepatitis B (HBV) sequences formulated with IC31, a TLR9-based adjuvant from Valneva SE. The HBV-directed peptides are designed to drive T cell responses against all HBV genotypes towards a functional cure for chronic HBV in patients of diverse genetic backgrounds.

On March 15, 2022 MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the JASDAQ Market of the Tokyo Stock Exchange (Code Number: 4875), reported that Geoffrey O’Brien, JD/MBA, Vice President and Executive Officer, will present a corporate overview at the 2022 Virtual Growth Conference, presented by Maxim Group LLC and hosted by M-Vest, on March 28 – 30, 2022 from 9:00 a.m. – 5:00 p.m. EDT (Press release, MediciNova, MAR 15, 2022, View Source [SID1234610123]).

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On March 15, 2022 Newron Pharmaceuticals S.p.A. ("Newron") (SIX: NWRN, XETRA: NP5), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system, reported its financial results and operational highlights for the year ended December 31, 2021, and provided an outlook for 2022 (Press release, Newron Pharmaceuticals SpA, MAR 15, 2022, View Source [SID1234610122]).

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Highlights 2021:

Evenamide (Schizophrenia)

-Two short-term explanatory studies of evenamide both met their primary objectives of demonstrating absence of arrhythmia risk (TQT-Study 010) in healthy volunteers, and the absence of EEG/neurological abnormalities (EEG Study 008) in patients with schizophrenia
– Newron initiated:
– an open-label study of evenamide as add-on to antipsychotics in patients with treatment resistant schizophrenia (TRS) and its extension (studies 014 and 015)
– study 008A, the first randomized, placebo-controlled, adequate, well-controlled, potentially pivotal study of evenamide in patients with chronic schizophrenia not responding adequately to second-generation antipsychotics (non-TRS)
– Newron continues to evaluate strategic commercial and development partnering options for evenamide
Xadago/safinamide (Parkinson’s disease)

– Newron signed a partnership agreement with Zambon to initiate a potentially pivotal study with safinamide in Parkinson’s disease patients with levodopa-induced dyskinesia (PD LID)
– Newron and its partners Zambon and Supernus continue to work to protect intellectual property rights associated with Xadago/safinamide in the US, responding to Paragraph IV Notice Letters regarding Abbreviated New Drug Applications submitted from generic pharmaceutical manufacturers
Corporate

– Newron received the fourth and fifth tranches of funding from the European Investment Bank (EIB), each totaling EUR 7.5 million; total financing from the EIB since 2019 is now EUR 40 million, and has thus been fully drawn
– Newron continues to explore a number of potential opportunities to expand its pipeline in central nervous system diseases
Stefan Weber, CEO of Newron, commented:

"We are pleased to share the progress made by Newron throughout 2021, as well as to provide an update on what we hope to achieve as we move forward into 2022. Most significantly, our team has succeeded in initiating study 008A with evenamide, launching our Phase II/III program and representing the first potentially pivotal study in patients with schizophrenia who are inadequate responders to antipsychotics. Our team continues to explore a number of strategic opportunities and potential commercial partnerships to expand our pipeline in central nervous system diseases, including opportunities to in-licence."

Evenamide

In 2021, Newron announced the results from two short-term explanatory studies of evenamide, study 010 and study 008, which both met their primary objective of safety. Study 010 was a short-term safety study of the effects of two doses of evenamide (30mg and 60mg) in 56 healthy volunteers, and study 008 was a four-week Phase II study in 138 outpatients with chronic schizophrenia currently being treated with a second-generation atypical antipsychotic. The results showed that evenamide is safe at all doses investigated (with no systematic pattern of adverse effects on the central nervous system), is devoid of any arrhythmic effect (a risk associated with antipsychotics) and can be safely taken with other antipsychotics. Following recent discussions with the U.S. Food and Drug Administration (FDA), Newron will address the remaining FDA issues once data from studies 014 and 008A studies are available.

After the encouraging results from study 008, Newron initiated study 008A, a four-week, randomized, double-blind placebo-controlled study to assess the efficacy, tolerability, and safety (including electroencephalogram effects) of the therapeutic BID dose of 30mg in patients with chronic schizophrenia currently being treated with a second-generation antipsychotic. This study represents the first part of Newron’s Phase II/III clinical trial program that targets patients with schizophrenia experiencing worsening of psychosis who are inadequately responding to therapeutic doses of second-generation antipsychotics (non TRS). Study 008A involves treatment centers across twelve countries in Europe, Asia and Latin America, and results are expected around the end of 2022. Subject to positive results, study 008A would be the first randomized, placebo-controlled, adequate, well-controlled, potentially pivotal study of evenamide in schizophrenia patients who are inadequate responders to atypical anti-psychotic treatment.

In the second indication of its Phase II/III development plan for evenamide, treatment-resistant schizophrenia (TRS), Newron has initiated pilot study 014. This is a six-week, open-label, randomized, rater-blinded, multi-centre study with sites in Italy, India, Sri Lanka and Malaysia. The study was designed to evaluate the safety, tolerability, and preliminary efficacy of fixed doses of evenamide of 7.5 mg BID, 15 mg BID and 30 mg BID as add-on treatment in patients with moderate to severe TRS. Currently, 110 of the intended 150 patients have been enrolled to study 014. Completers are eligible to continue treatment with the randomized dose in an extension study (015) for up to 46 weeks. Newron intends to announce first results from study 014 in Q2 2022.

The pilot study in patients suffering from TRS would be followed by the second potentially pivotal study with evenamide in patients with treatment resistant schizophrenia on a second-generation antipsychotic. Importantly, if approved, evenamide would be the first add-on therapy for schizophrenia. Its glutamatergic inhibition mechanism of action represents an innovative alternative to common dopaminergic or serotonergic drugs, potentially offering a new therapeutic option for patients who are not or inadequately responding to existing second-generation antipsychotics.

Xadago/safinamide

As Newron looks to further develop its marketed product, Xadago/safinamide, in 2021 the company announced it had signed a partnership agreement with Zambon to begin a potentially pivotal study in patients with Parkinson’s disease and levodopa-induced dyskinesia (PD LID). Under this partnership agreement, Newron will sponsor the study and be responsible for its development and execution, and lead on all regulatory interactions. Newron and Zambon will share the costs of the study evenly. The double-blind, placebo-controlled study is intended to be performed in the US, Europe and Asia/Australia, with the aim of a label extension for safinamide in key markets.

In May 2021, Newron announced that it had received several Paragraph IV Notice Letters regarding the submission by generic manufacturers of an Abbreviated New Drug Application to the FDA, seeking approval to engage in the commercial manufacture, use or sale of safinamide mesylate drug product in the US before expiration of certain US patents. Newron and its partners Zambon and Supernus have responded by filing an infringement suit against the generic manufacturers to secure a 30-month stay of the ANDAs approval, and thus to protect its intellectual property rights relating to Xadago/safinamide tablets. Xadago (safinamide) tablets are currently protected by three patents listed in the FDA’s Approved Drugs Product List (Orange Book) that expire no earlier than 2027.

Financial Key takeaways 2021:

– In 2021, Newron reported a net loss of EUR 14.9 million, compared to EUR 21.0 million in 2020
– Cash used in operating activities has decreased to EUR 11.5 million from EUR 15.6 million in 2020
– Xadago revenues from Zambon increased from EUR 5.3 million in 2020 to EUR 5.8 million in the reporting period.
– Newron’s R&D expenses have fallen to EUR 10.7 million from EUR 14.9 million in 2020
– G&A expenses reached EUR 7.4 million in 2021 versus EUR 8.1 million in 2020
– Cash and Other current financial assets as at December 31, 2021 were at EUR 34.6 million, compared to EUR 31.3 million at the beginning of the year

Newron’s Annual Report 2021 is available for download on the Company’s website: www.newron.com/investors/reports-and-presentation/year/2021

Outlook 2022:

"We look forward to completing study 008A evaluating the efficacy of evenamide in patients with schizophrenia, with results expected towards the end of 2022. We look forward to results from our open-label study of evenamide as add-on to antipsychotics in patients with treatment resistant schizophrenia, and plan to follow up on this study by investigating evenamide in a Phase III study as a new therapeutic option for patients who are considered to have treatment-resistant schizophrenia. In 2022, we will also continue to progress towards initiating the label-extension study for safinamide in patients with Parkinson’s disease and levodopa-induced dyskinesia with our partner Zambon. Newron’s total available cash resources will fund the Company’s planned development programs and operations into 2024," outlined Stefan Weber, CEO of Newron.

2022 Shareholders’ Meeting Agenda:

Newron’s Board of Directors has approved the below agenda for the April 5, 2022, Shareholders’ meeting, which will take place at the Company’s registered office (Via Antonio Meucci 3) in Bresso (Mi), Italy, starting at 10 am CET. The formal invitation to shareholders will be issued and disclosed in the statutory papers on or around March 15.

The full invitation and supporting material will be made available on the Company’s website (www.newron.com/investors/shareholders-meeting) on the same date. The agenda is as follows:

1. Approval of the balance sheet as at 31 December 2021. Connected and consequent resolutions
2. Redefinition of the number of the Board members; connected and consequent resolutions
3. Appointment of the statutory auditors for the three fiscal-year time 2022-2024 and, therefore, until the approval of the balance sheet as at 31 December, 2024, and determination of their fees. Connected and consequent resolutions
4. Appointment of the auditing company; connected and consequent resolutions
Dial-in details to the media/analyst/investor conference on March 15, 2022, 3 pm CET

The Newron management team will present the 2021 full-year results and provide an update and guidance for 2022. The conference call can be accessed via the following dial-in numbers:

The slide deck is available at www.newron.com/investors/reports-and-presentation/year/2021

Upcoming events

– AGM 2022: April 5, 2022
– Half-year report 2022: September 15, 2022

On March 15, 2022 EXUMA Biotech, Corp., a clinical-stage biotechnology company discovering and developing cell and gene therapies for liquid and solid tumors, announced that preclinical data from its novel, subcutaneous CAR-TaNK therapy for hematological tumors, and in vivo engineered CAR gene therapy program (GCAR), will be presented in two posters at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, taking place April 8-13, in New Orleans, LA (Press release, EXUMA Biotechnology, MAR 15, 2022, View Source [SID1234610120]).

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EXUMA is currently conducting IND-enabling studies of the first products on its CAR-TaNK cell therapy platform, targeting CD19 and CD22. CAR-TaNKs are potent cancer killers with T and NK features, which preclinically have been observed to reproducibly and robustly expand in vivo in lymphoreplete animal models following the generation of synthetic lymph nodes post subcutaneous injection. Based on the absence of a lymphodepleting preparatory regimen, and favorable preclinical CAR-TaNK blood cytokine profile, EXUMA plans to pursue development of its CD19 and CD22 cell therapies for use in the community oncology setting.

Additionally, EXUMA presented robust B cell aplasia results obtained by directly injecting a CD3-targeting lentiviral vector (GCAR) into two different lymphoreplete humanized animal models. Further pre-clinical studies are ongoing to characterize the pharmacokinetic and pharmacodynamic effects of EXUMA’s GCAR gene therapy.

"We are excited to share data for the first time from our in vivo GCAR platform, as we continue our work to bring this program into the clinic. We will also be presenting important biological and mechanistic findings from our subcutaneous rPOC CAR-TaNK program, which is currently undergoing IND enabling studies. CAR-TaNK therapy holds great promise as a cancer treatment modality. EXUMA’s goal is to make engineered cell and gene therapies more scalable, affordable, and accessible to patients across the globe," said Sid Kerkar, M.D., VP of R&D.

Abstract Title ("rPOC"): "Generation of tertiary lymphoid structures and CD3 + CD8+ CD56+ NKG2D+ CAR-TaNK cells following subcutaneous injection of CD3- directed lentiviral-loaded PBMCs"
Session: Adoptive Cell Therapy 1
Abstract Number: 5673
Presentation Date & Time: Sunday, April 10th, 1:30 pm-5:00 pm
Presenter: Dr. Sid P. Kerkar, M.D.
Location: Poster Section 36

Abstract Title ("GCAR") : "In vivo delivery of a novel CD3-targeted lentiviral vector generates CD19 CAR-T cells in two different humanized mouse models and results in complete B cell depletion"
Session: Gene and Vector-Based Therapy
Abstract Number: 6345
Presentation Date & Time: Tuesday, April 12th, 1:30 pm-5:00 pm
Presenter: Frederic Vigant, Ph.D.
Location: Poster Section 24

About rPOC SC CAR-TaNK platform

EXUMA’s next-generation "rPOC" platform is being developed for subcutaneous administration without the need for lymphodepleting chemotherapy or long-term immunosuppression. The unique CAR-TaNK cell type producing less CRS-associated cytokines, combined with the lack of lymphodepleting chemotherapy, may result in fewer side effects and hospitalizations thereby potentially allowing for administration in a community oncology setting.

About GCAR in vivo gene therapy platform

"GCAR" is a novel in vivo CAR gene therapy platform that holds the potential to directly deliver CAR constructs specifically into CD3+ cells, while simultaneously providing an activation signal to enable efficient gene integration in vivo without the need to modify cells ex vivo and without the need for lymphodepleting chemotherapy. This platform has the potential to address many of the challenges of existing cell therapy including manufacturing cost and complexity, time, scalability, and safety.