On April 21, 2022 Race Oncology reported The March 2022 quarter (Q3 FY 2022) was highlighted by positive preclinical findings that Zantrene (bisantrene dihydrochloride) was found to kill kidney cancer cells both on its own and synergistically in combination with known anti-cancer drugs (ASX announcement: 10 March 2022) (Press release, Race Oncology, APR 21, 2022, View Source [SID1234613998]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A second highlight was the results of preclinical work in extramedullary AML, where Zantrene in combination with decitabine was shown to be highly effective in killing a diverse range of AML cells as well as in a mouse model of extramedullary AML (ASX announcement: 17 March 2022).

These results support the planned AML Phase 1 / 2 clinical trial (RAC-006) in extramedullary AML. Our planned extramedullary AML clinical trial was further enhanced with news that Astex Pharmaceuticals has partnered via a supply agreement, under which its oral decitabine and cedazuridine formulation ASTX727 will be provided free of charge to Race (ASX announcement: 30 March 2022). In sum, while the quarter saw some minor delays to planned programs, considerable progress has been made including post quarter Human Ethics approval and governance submission for the extramedullary AML clinical trial (ASX announcement: 6 April 2022)Key events of the quarter

 On 18 January 2022, Race announced that it had received a $708,000 R&D tax refund for the financial year ended 30 June 2021. This reflects investment in Australian based R&D projects and encourages us to utilise Australian based resources, where possible.

 On 23 February 2022, Race announced that MD Anderson Cancer collaborators had published an AML Preclinical study on Zantrene in the Journal Leukemia & Lymphoma. The study confirmed that Zantrene, when used in combination with the AML drugs venetoclax, panobinostat, decitabine and olaparib showed synergies in killing AML cells. This work further supports our extramedullary AML clinical trial plans where Zantrene will be used in combination with decitabine and cytarabine, with the objective of treating extramedullary AML more effectively.

 On 10 March 2022, Race announced results of a preclinical study that confirmed compelling kidney cancer results for Zantrene, both on its own and in combination with known cancer agents. Greater cell killing synergies were observed when Zantrene was combined with lenvatinib, cabozantinib and pazopanib. These results support advancing Zantrene into human kidney cancer trials.

 On 17 March 2022, Race announced AML mouse model results, that showed excellent effectiveness for Zantrene when used in combination with Decitabine, to target extramedullary tumours as well as in the bone marrow and spleen. The results showed that low dose Zantrene used in combination with decitabine killed AML tumours and this supports the planned extramedullary AML trial and possibilities for improved treatment for extramedullary AML patients.

Other news from the quarter  Race expanded the preclinical team through the employment of Emily Ryan as a Research Assistant. Emily is based at the University of Wollongong and is developing new formulations of Zantrene.  Race signed a new supply contract signed with Laurus Laboratories (India) for the large-scale production of Zantrene over the next 2 years.  Dr Daniel Tillett, Race CSO, visited the University of Wollongong (UOW) to formally launch the research collaboration between Race and UOW. This visit was covered by WIN Television News.  Race signed an additional preclinical breast cancer research program with Nikki Verrills of the University of Newcastle exploring novel combinations of Zantrene and breast cancer drugs. The results of this program is expected in Q3 CY 2022. Race initiated of a number of preclinical animal studies exploring the use of Zantrene in AML, multiple myeloma, kidney cancer and breast cancer models with a range of international and Australian contract research organisations. The results of these studies are expected to be reported over the following two quarters. Summary of cash flow and quarterly activity As of 31 March 2022, Race held cash and equivalents of $35.68 million, compared with $37.10 million on 31 December 2021.

The change in cash reserves reflects planned higher research expenditure, offset by an R&D grant of $708k (net change of $1.43m vs $1.79m in the prior quarter). There was a reduction in this quarter’s administrative expense driven by timing differences. Listing rule 4.7C.3 Payments during the quarter to Related Parties amounted to $153k, comprising payments of salaries and superannuation to executive directors of $110k and board fees to non-executive directors of $43k. Shareholders by holding range Race is pleased to report that shareholders totalled 9,423 as of 31 March 2022, showing continued shareholder interest in Race’s progress.

Post quarter news
 On 6 April 2022, Race announced receiving Human Ethics approval and submitting its governance application for its extramedullary AML & Myelodysplastic syndromes (MDS) trial. governance approval is the final step required before initiating the clinical trial and treating the first patient. Approval is expected Q2 CT 2022.
 On 12 April 2022, Race executives Mr Phillip Lynch (CEO & MD) and Dr Daniel Tillett (CSO & ED) agreed to increase their formal time commitment to 75% reflecting an increased in Race related workload over the last 12 months.Expected news In the current quarter, shareholders can expect updates on the following activities:  Pre-clinical in vitro – cell-based programs in breast cancer, multiple myeloma, melanoma, and kidney cancer, as well as in cardioprotection are underway and will report over the next two quarters.

 Pre-clinical in vivo – the melanoma animal study will report during this quarter, with results to be shared as soon as the relevant IP protection process is in place. Animal work assessing cardioprotection and how Zantrene may offset anthracycline and carfilzomib induced heart damage are underway with results to be reported in Q2/3 CY 2022.  Clinical – an update on the relapsed / refractory AML trial in Israel which is in the dose escalation phase (6-12 patients) can be expected this quarter. Governance approval and first patient enrolment expected in Q2 CY 2022 for the extramedullary AML trial. Management commentary Race CEO Phillip Lynch said: "We are moving through CY 2022 with a comprehensive program of activities that will increasingly move into the clinic with expected AML results from Israel and commencement of the RAC-006 trial in Australia.

Our preclinical work is advancing to animal models, and we can expect this to support and guide clinical decisions. Importantly we remain well-resourced financially and in human capability to support our plans." Race CSO Daniel Tillett said: "It has been another busy quarter for Race, building on the new Three Pillar strategy. Zantrene continues to surprise us with positive results and I am looking forward to seeing its potential as we move to treating additional patients in the clinic." Race Chairman John Cullity said: "The strategy for Zantrene continues to form as the drug talks to us through our preclinical and clinical programs. We are coming up to an exciting time, reporting the first glimpses of data from the AML trial in Israel. My thanks goes to our clinical collaborators who continue to strongly support our efforts to bring Zantrene back to market, and to the Race team who are working overtime to realise the drug’s potential."

On April 21, 2022 Helix BioPharma Corp. (TSX: "HBP") ("Helix" or the "Company"), a clinical-stage biopharmaceutical company developing unique therapies in the field of immuno-oncology, based on its proprietary technological platform DOS47, reported that it has closed a private placement financing for net proceeds of $2,002,000 from the issuance of 7,700,000 common shares at a price of $0.26 per common share (Press release, Helix BioPharma, APR 21, 2022, View Source [SID1234613227]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Mr. Jerzy Wilczewski ("Mr. Wilczewski"), an insider of the Company, subscribed for 7,700,000 common shares issued under the private placement. As a result of the closing of the private placement, Mr. Wilczewski owns or exercises controlordirectionover31,167,153 commonshares, representingapproximately19.85% oftheissued and outstanding common shares of the Company on a non-diluted basis, or approximately 27.15% on a partially diluted basis, assuming the full exercise of the 15,739,500 common share purchase warrants that Mr. Wilczewski owns or exercises control or direction over.

The purchase of common shares by Mr. Wilczewski is considered a "related party transaction" within the meaning of Multilateral Instrument 61-101 – Protection of Minority Security Holders in Special Transactions ("MI 61-101"). The Company relied on exemptions from the formal valuation and minority approval requirements in sections 5.5(a) and 5.7(1)(a) of MI 61-101 in respect of Mr. Wilczewski ‘s purchase of common shares. The Company did not file a material change report in respect of the related party transaction less than 21 days prior to the closing of the private placement, which the Company deems reasonable in the circumstances so as to be able to avail itself of the proceeds of the private placement in an expeditious manner.

"In the last few months, I have increased my investment by another 3M as I have great confidence and trust in the new team and their strategy" said Mr. Wilczewski.

"We would like to thank Mr. Wilczewski for his strong support and confidence in Helix’s revised strategy, execution plans and team. We look forward to continued efforts toward this exciting program" said Mr. Gabor, CEO of Helix.

The Company intends to use the net proceeds of the private placement for working capital and advancing the Company’s L-DOS47 drug development program.

On April 21, 2022 Hansa Biopharma, the pioneer in immunomodulatory enzyme technology for rare IgG mediated diseases, reported its business update and interim report for January to March 2022 (Press release, Hansa Biopharma, APR 21, 2022, View Source;march-2022-301529853.html [SID1234612887]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Highlights for the first quarter 2022

Solid sales growth in the first quarter with SEK 24.2m in product sales; total revenue amounted to SEK 30.3m.
Commercial launch activities and market access efforts for Idefirix in Europe continued to progress as planned during Q1 2022 with market access secured in France through a reimbursed Early Access Program and in Germany through commercial access on negotiated terms. Additional market access procedures are ongoing in 11 countries, including Spain, Italy and the U.K.
Hansa and Medison Pharma announced that a marketing authorization in Israel for Idefirix has been granted for desensitization treatment of highly sensitized kidney transplant patients.
Key data from a Phase 2 program of imlifidase in anti-Glomerular Basement Membrane (anti-GBM) disease patients were published in the Journal of American Society of Nephrology (JASN). The publication recognizes the study’s significance in autoimmune diseases as it suggests that deactivation of autoantibodies could alter the course of an autoimmune disease.
In January, Hansa and AskBio entered into an agreement to evaluate the potential use of imlifidase as a pre-treatment prior to the administration of AskBio’s investigational gene therapy in Pompe disease in a preclinical and clinical feasibility program for patients with pre-existing neutralizing antibodies (NAbs). As part of the agreement, Hansa received a USD 5 million upfront payment, while AskBio has received an exclusive option to negotiate a full development and commercialization agreement.
The partnership with Sarepta investigating imlifidase in gene therapy and the preclinical collaboration with argenx exploring the potential for combination therapy with imlifidase moved forward according to plan.
Clinical pipeline update

U.S. ConfIdeS: 16 patients have been enrolled for randomization in our pivotal U.S. open-label, randomized, controlled trial "ConfIdeS" with the aim of completing enrollment by the end of this year, as previously guided.
AMR: In the Antibody Mediated Rejection (AMR) Phase 2 trial, 28 out of a target of 30 patients have been enrolled, and completion of enrollment is expected in the first half of 2022, as previously guided.
GBS: In the Guillain Barré Syndrome (GBS) Phase 2 trial, 16 patients out of a target of 30 patients have been enrolled. The COVID-19 pandemic has significantly impacted the enrollment rate in our GBS trial at the participating hospitals. To accelerate enrollment rate, we have implemented a number of initiatives to address the current situation and we expect these intiatives to support the completion of enrollment of GBS patients in H2 2022.
Events after the reporting period

Anti-GBM: On April 19, 2022, Hansa announced that the US FDA has accepted Hansa’sInvestigational New Drug (IND) application to proceed with a Phase 3 study of imlifidase in 50 patients across EU and the U.S. The first patient is expected to be enrolled in 2022, as previously guided.
Financial summary

SEKm, unless otherwise stated – unaudited

Søren Tulstrup, President and CEO of Hansa Biopharma, comments

"Hansa’s commercial launch activities and market access efforts for Idefirix in Europe continue to progress as planned. During the first quarter of 2022, we have seen additional key transplant centers becoming both clinically and commercially ready to use Idefirix and solid sales growth. Market access were secured in two of the five major European markets, namely in France on an early access basis and in Germany – two countries with more than 5,600 kidney transplants annually, of which approximately 75% are transplanted from a deceased donor.

We are very pleased to have reached these important agreements with both the German payer association, National Association of Statutory Health Insurance Funds (GKV-SV), and the early access granted by the French Haute Autorité de Santé (HAS). We expect to complete additional agreements in the course of the year as we have market access procedures ongoing in 11 countries, including Spain, Italy and the U.K. During 2021, market access was secured in Sweden and the Netherlands, as well as on an individual hospital basis in Finland and Greece.

Looking beyond our core markets, I am also pleased to see that our new collaboration with Medison Pharma is off to a good start with the recent marketing authorization obtained in Israel for Idefirix for the treatment of highly sensitized kidney transplant patients. Beyond Israel, our collaboration with Medison also covers Poland, Hungary, Croatia and Slovenia.

In the beginning of March, key data from the investigator-initiated open-label Phase 2 study of imlifidase in patients with anti-glomerular basement membrane (anti-GBM) disease were published in the leading nephrology publication Journal of the American Society of Nephrology (JASN). The publication recognizes the study’s significance in autoimmune diseases as it suggests that deactivation of autoantibodies could alter the course of an autoimmune disease, allowing restoration of kidney function. These results highlight the potential of imlifidase as we expand beyond kidney transplantation.

Speaking about anti-GBM, we are also pleased to share the positive news that the U.S. FDA recently accepted Hansa’s Investigational New Drug (IND) application to proceed with a pivotal Phase 3 study of imlifidase in approximately 50 patients across EU and the U.S. The first patient is expected to be enrolled later this year, as previously guided.

In the U.S., our pivotal ConfIdeS trial in kidney transplantation is progressing with 16 out of a target of 64 patients enrolled for randomization. The ConfIdeS study is evaluating imlifidase as a potential desensitization therapy to enable kidney transplants in highly sensitized patients waiting for a deceased donor kidney through the U.S. kidney allocation system. We have now initiated enrollment at nine sites and expect participation by up to 15 leading transplantation centers across the U.S., with the aim of completing enrollment by the end of this year.

Turning to our ongoing Phase 2 programs for GBS and AMR, we have enrolled 28 out of a target of 30 patients in the AMR study, while 16 out of a target of 30 patients have been enrolled in the GBS study.

With respect to our GBS program, we have seen how the impact of the COVID-19 pandemic and the emergence of the new variants have negatively affected the enrollment rate across a number of trial centers. To mitigate this situation we have recently implemented several significant initiatives to increase the enrollment rate and we expect these initiatives will support the completion of enrollment of GBS patients in the second half of 2022.

Last, we were pleased to announce at the beginning of January that Hansa and AskBio, a subsidiary of Bayer AG, have entered into a collaboration to evaluate imlifidase in a preclinical and clinical feasibility program as pre-treatment ahead of gene therapy in Pompe disease in patients with pre-existing neutralizing antibodies (NAbs). We see significant potential for our antibody-cleaving enzyme technology to help overcome this barrier in gene therapy as NAbs against adeno-associated virus remain a major challenge.

We have commenced another exciting year with several important milestones to be achieved across our platform and franchises, and I look forward to making further progress in the remainder of the year towards the vision that we are pursuing with single-minded focus: A world where patients with rare immunologic diseases can lead long and healthy lives."

Upcoming milestones and news flow

H1 2022 AMR Phase 2 study: Complete enrollment

2022 NiceR: Completion of GLP tox studies

2022 Anti-GBM: Initiation of phase 3 study

H2 2022 GBS Phase 2 study: Complete enrollment

H2 2022 Kidney transplantation US: Complete enrollment

H2 2022 AMR Phase 2 study: First data read out

H1 2023 GBS Phase 2 study: First data read out

2023 Long-term follow-up data 5-years out in kidney transplantation

H2 2023 Kidney transplantation US: complete 12 months follow-up

H1 2024 Kidney transplantation US: BLA submission

Conference call details

Hansa Biopharma will host a telephone conference today Thursday April 21 14:00 CET / 8:00am EST.

The presentation will be held in English and be hosted by Hansa Biopharma’s CEO, Søren Tulstrup, and CFO, Donato Spota. Slides used in the presentation will be live on the company website during the call under "Events & Presentation" and will also be made available online after the call.

On April 21, 2022 Servier, a leader in oncology committed to bringing the promise of tomorrow to the patients we serve, reported the publication of results from the Phase 3 trial of TIBSOVO (ivosidenib tablets) in the New England Journal of Medicine (NEJM) (Press release, Servier, APR 21, 2022, View Source [SID1234612886]). The AGILE trial is a global Phase 3 double blinded placebo-controlled study of TIBSOVO in combination with the chemotherapy azacitidine in adults with previously untreated IDH1-mutated acute myeloid leukemia (AML) compared to azacitidine in combination with placebo. The study met the primary and all key secondary endpoints including overall survival. Servier is actively working with the FDA and health authorities across the globe to potentially bring this new indication to market.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Patients with IDH1-mutated AML have a poor prognosis and have few, if any, treatment options, especially for newly diagnosed patients who are not eligible for intensive chemotherapy," said Susan Pandya, M.D., Vice President Clinical Development and Head of Cancer Metabolism Global Development Oncology & Immuno-Oncology, Servier Pharmaceuticals. "The publication of the compelling Phase 3 AGILE study data in NEJM, reinforces the clinical importance of these results and supports Servier’s ongoing pursuit to serve patients with IDH1-mutated malignancies."

AML is a cancer of the blood and bone marrow marked by rapid disease progression and is the most common acute leukemia affecting adults with approximately 20,000 new cases estimated in the U.S. each year.1,2 The majority of patients with AML eventually relapse. Relapsed or refractory AML has a poor prognosis.3 The five-year survival rate is approximately 29.5%.1 IDH mutations are present in about 6 to 10 percent of AML cases.4

"We have significantly strengthened our position in oncology following the successful acquisition of the Agios Pharmaceuticals’ oncology business in 2021," said Claude Bertrand, Executive Vice President, Research and Development, Servier. "Servier has placed oncology among its priorities and allocates more than 50% of its R&D budget to fighting cancer. This strategy initiated by the Group is now bringing results with new treatments and future indications for patients with hard-to-treat cancers."

The data from the global Phase 3 AGILE study show that TIBSOVO is the first IDH1 mutation specific targeted therapy to demonstrate improved event-free survival (EFS) and overall survival (OS) in combination with azacitidine compared to azacitidine plus placebo. Treatment with TIBSOVO in combination with azacitidine demonstrated a statistically significant improvement in EFS (hazard ratio [HR] = 0.33, 95% confidence interval [CI] 0.16, 0.69, 1-sided P = 0.0011).5,6 The combination of TIBSOVO with azacitidine showed a statistically significant improvement in OS (HR = 0.44 [95% CI 0.27, 0.73; 1-sided P = 0.0005), with a median OS of 24.0 months vs. 7.9 months in the placebo + azacitidine arm.

In addition, the complete remission (CR) rate was 47.2% (n = 34/72) for TIBSOVO in combination with azacitidine vs. 14.9% (n = 11/74) for placebo plus azacitidine (P < 0.0001). CR + complete remission with partial hematologic recovery rate (CR + CRh rate) was 52.8% (n = 38/72) for TIBSOVO in combination with azacitidine vs. 17.6% (n = 13/74) for placebo plus azacitidine (P < 0.0001).The objective response rate (ORR) was 62.5% (n = 45/72) for TIBSOVO in combination with azacitidine vs. 18.9% (n = 14/74) for placebo plus azacitidine (P < 0.0001).

TIBSOVO[*] is currently approved in the U.S. as monotherapy for the treatment of adults with IDH1-mutant relapsed or refractory AML, and for adults with newly diagnosed IDH1-mutant AML who are ≥75 years old or who have comorbidities that preclude the use of intensive induction chemotherapy. Recently, TIBSOVO was approved as a first and only targeted therapy for patients with previously treated IDH1-mutated cholangiocarcinoma.

About the NCT03173248 AGILE Phase 3 AML Trial
The AGILE trial is a global, Phase 3, multicenter, double-blind, randomized, placebo-controlled clinical trial designed to evaluate the efficacy and safety of TIBSOVO in combination with azacitidine compared with placebo in combination with azacitidine, in adults with previously untreated IDH1-mutated acute myeloid leukemia (AML) who are not candidates for intensive chemotherapy (≥75 years old or who have comorbidities that preclude the use of intensive induction chemotherapy). The study’s primary endpoint is EFS, defined as the time from randomization until treatment failure, relapse from remission, or death from any cause, whichever occurs first. Treatment failure is defined as failure to achieve complete remission (CR) by Week 24.

Key secondary endpoints included CR rate, defined as the proportion of participants who achieve a CR; overall survival (OS), defined as the time from date of randomization to the date of death due to any cause; CR and complete remission with partial hematologic recovery (CRh) rate, defined as the proportion of participants who achieve a CR or CRh; and objective response rate (ORR), defined as the rate of CR, CR with incomplete hematologic recovery (CRi) (including CR with incomplete platelet recovery [CRp]), partial remission (PR), and morphologic leukemia-free state (MLFS).

About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) a cancer of blood and bone marrow characterized by rapid disease progression, is the most common acute leukemia affecting adults, with approximately 20,000 new cases in the U.S., and 43,000 cases in Europe each year.1,2,7 AML incidence significantly increases with age, and the median age of diagnosis is 68.1 The vast majority of patients do not respond to chemotherapy and progress to relapsed/refractory AML.3 The five-year survival rate is approximately 29.5%.1 For 6 to 10 percent of AML patients, the mutated IDH1 enzyme blocks normal blood stem cell differentiation, contributing to the genesis of acute leukemia.4

On April 21, 2022 Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, reported that it will release its first quarter 2022 financial results on Thursday, May 5, 2022 (Press release, Insmed, APR 21, 2022, View Source [SID1234612885]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Insmed management will host a conference call for investors beginning at 8:30 a.m. ET on Thursday, May 5, 2022 to discuss the financial results and provide a business update.

Shareholders and other interested parties may participate in the conference call by dialing (844) 200-6205 (U.S. toll free), (646) 904-5544 (U.S. local), or +1-929-526-1599 (international) and referencing access code 388457. The call will also be webcast live on the company’s website at www.insmed.com.

A replay of the conference call will be accessible approximately 1 hour after its completion through June 4, 2022, by dialing (866) 813-9403 (U.S. toll free), (929) 458-6194 (U.S. local), or +44-204-525-0658 (international) and referencing access code 252664. A webcast of the call will also be archived for 90 days under the Investor Relations section of the company’s website at www.insmed.com.