On April 20, 2023 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that it will host a conference call at 1:30 p.m. PDT/4:30 p.m. EDT on Thursday, May 4, 2023, following the release of its first quarter 2023 financial results (Press release, Puma Biotechnology, APR 20, 2023, View Source [SID1234630372]).

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The call may be accessed by dialing 1-877-709-8150 (domestic) or 1-201-689-8354 (international). Please dial in at least 10 minutes in advance and inform the operator that you would like to join the "Puma Biotechnology Conference Call." A live webcast of the conference call and presentation slides may be accessed on the Investors section of the Puma Biotechnology website at View Source A replay of the call will be available approximately one hour after completion of the call and will be archived on Puma’s website for 90 days.

On April 20, 2023 Daiichi Sankyo (TSE: 4568) reported that the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) of quizartinib in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, and as continuation monotherapy following consolidation, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3-ITD positive (Press release, Daiichi Sankyo, APR 20, 2023, View Source [SID1234630371]).

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The FDA has extended the Prescription Drug User Fee Act (PDUFA) action date by three months to July 24, 2023 to allow additional time to review requested updates to the proposed Risk Evaluation and Mitigation Strategies (REMS) included in this application. No additional efficacy or safety data has been requested.

"We are continuing to work with the FDA to facilitate completion of their review of the quizartinib new drug application in order to bring this important medicine to patients as soon as possible," said Mark Rutstein, MD, Global Head, Oncology Clinical Development, Daiichi Sankyo. "Quizartinib was shown to improve overall survival when added to standard chemotherapy and continued as monotherapy and has potential to change the standard of care for patients with newly diagnosed FLT3-ITD positive AML."

The NDA is based on results from the QuANTUM-First trial, which demonstrated that quizartinib combined with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, and continued as monotherapy following consolidation, resulted in a statistically significant and clinically meaningful improvement in overall survival in adult patients with newly diagnosed FLT3-ITD positive AML compared to chemotherapy alone. The results of QuANTUM-First were presented at the 2022 European Hematology Association (EHA) (Free EHA Whitepaper) Congress.1

The safety of quizartinib combined with intensive chemotherapy and as continuation monotherapy in QuANTUM-First was generally manageable with no new safety signals observed. The incidence of grade ≥3 QT prolongation events was low, with uncommon ventricular arrythmia events. Overall, the risk of QT prolongation was manageable with ECG monitoring, quizartinib dose modification and correction/elimination of additional risk factors.

About QuANTUM-First
QuANTUM-First is a randomized, double-blind, placebo-controlled global phase 3 study evaluating quizartinib in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, and as continuation monotherapy following consolidation, in adult patients aged 18-75 with newly diagnosed FLT3-ITD positive AML. Patients were randomized 1:1 into two treatment groups to receive quizartinib or placebo combined with anthracycline- and cytarabine-based regimens. Eligible patients, including those who underwent hematopoietic stem cell transplant (HSCT), continued with quizartinib or placebo for up to 36 cycles.

The primary study endpoint was overall survival. Secondary endpoints include event-free survival, post-induction rates of complete remission (CR) and composite complete remission (CRc), and the percentage of patients who achieve CR or CRc with FLT3-ITD minimal residual disease negativity. Safety and pharmacokinetics, along with exploratory efficacy and biomarker endpoints, also were evaluated. QuANTUM-First enrolled 539 patients at 193 study sites across Asia, Europe, North America, Oceania and South America. For more information, visit ClinicalTrials.gov.

About FLT3-ITD Positive Acute Myeloid Leukemia
More than 474,500 new cases of leukemia were reported globally in 2020 with more than 311,500 deaths.2 AML accounts for 23.1% of total leukemia cases worldwide and is most common in adults.3,4 In the U.S., an estimated 20,380 new cases of AML will be diagnosed in 2023 with the five-year survival rate reported at 30.5%.4,5

A number of gene mutations have been identified in AML, and FLT3 (FMS-like tyrosine kinase 3) mutations are the most common, observed in up to 37% of all newly diagnosed patients.6,7 Approximately 80% of FLT3 mutations in AML are FLT3-ITD (internal tandem duplications), an oncogenic driver mutation that presents with a high leukemic burden.7,6 Patients with FLT3-ITD positive AML tend to have a particularly unfavorable prognosis including increased risk of relapse and shorter overall survival.6 The five-year survival rate for patients with FLT3-ITD positive AML has been reported at approximately 20%.8

The conventional treatment for fit patients with newly diagnosed AML is intensive induction and consolidation chemotherapy, with or without targeted therapy, and HSCT for eligible patients.9

About Quizartinib
Quizartinib is an oral, highly potent type II FLT3 inhibitor that selectively targets FLT3-ITD mutations and has been specifically developed for patients with FLT3-ITD positive AML.6

Regulatory applications for quizartinib in newly diagnosed FLT3-ITD positive AML are currently under review in Europe, Japan and the U.S. based on the results of the QuANTUM-First trial. The FDA has granted Priority Review and Fast Track Designation to quizartinib for the treatment of adult patients with newly diagnosed AML that is FLT3-ITD positive in combination with standard cytarabine and anthracycline induction and cytarabine consolidation chemotherapy. Orphan Drug Designation has been granted to quizartinib for the treatment of AML in Europe, Japan and the U.S.

Quizartinib is approved for use in Japan for the treatment of adult patients with relapsed/refractory AML that is FLT3-ITD positive, as detected by an approved test. Quizartinib is an investigational medicine in all countries outside of Japan. The quizartinib clinical development program includes a phase 1/2 trial in pediatric and young adult patients with relapsed/refractory FLT3-ITD positive AML in Europe and North America and several phase 1/2 combination studies as part of a strategic collaboration with The University of Texas MD Anderson Cancer Center.

On April 20, 2023 Prelude Corporation (PreludeDx), a leader in molecular diagnostics and precision medicine for early-stage breast cancer, reported it will be presenting data using DCISionRT to examine the decision impact on physician recommendation for adjuvant radiation therapy by race (White vs. Black) in women with ductal carcinoma in situ (DCIS) enrolled in the PREDICT study (Press release, PreludeDx, APR 20, 2023, https://www.prnewswire.com/news-releases/preludedx-to-present-comparative-analysis-of-treatment-recommendations-of-black-and-white-dcis-breast-cancer-patients-using-dcisionrt-at-asbrs-2023-annual-meeting-301802609.html [SID1234630369]). This race/ethnicity data will be presented at the 24th Annual Meeting of the American Society of Breast Surgeons (ASBrS), being held on April 26 – 30, 2023 at the John B. Hynes Veterans Memorial Convention Center in Boston, Massachusetts.

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Poster Presentation
Title: A Comparative Analysis of Changes in Treatment Recommendations for Black and White Patients with Ductal Carcinoma in Situ Using a 7-gene Predictive Biosignature: Analysis of the PREDICT Study
Presenter: Pat Whitworth, MD, FACS, FSSO, Director of the Nashville Breast Center
Date: Friday, April 28, 6:15 PM – 7:30 PM

"While there is a significant body of evidence addressing race/ethnic treatment differences among women with invasive breast cancer, there is limited evidence for DCIS. We are excited to present this new and valuable data for DCIS patients," says Dan Forche, President and CEO of PreludeDx. "We are confident that the new DCISionRT data will help guide physicians and their patients to make more informed treatment decisions."

About DCISionRT for Breast DCIS

DCISionRT is the only risk assessment test for patients with ductal carcinoma in situ (DCIS) that predicts radiation therapy benefit. Patients with DCIS have cancerous cells lining the milk ducts of the breast, but they have not spread into surrounding breast tissue. In the US, over 60,000 women are newly diagnosed with DCIS each year. DCISionRT, developed by PreludeDx on technology licensed from the University of California San Francisco, and built on research that began with funding from the National Cancer Institute, enables physicians to better understand the biology of DCIS. DCISionRT combines the latest innovations in molecular biology with risk-based assessment scores to assess a woman’s individual tumor biology along with other pathologic risk factors and provide a personalized recurrence risk. The test provides a Decision Score that identifies a woman’s risk as low or elevated. Unlike other risk assessment tools, the DCISionRT test combines protein expression from seven biomarkers and four clinicopathologic factors, using a non-linear algorithm to account for multiple interactions between individual factors in order to better interpret complex biological information. DCISionRT’s intelligent reporting provides a woman’s recurrence risk after breast conserving surgery alone and with the addition of radiation therapy. In turn, this new information may help patients and their physicians to make more informed treatment decisions.

On April 20, 2023 Samyang Holdings and LG Chem reported their recent execution of strategic partnership agreement on Thursday, April 20th, for development of novel mRNA-based cancer therapeutics that utilizes Samyang’s proprietary drug delivery technology (Press release, Samyang Biopharmaceuticals, APR 20, 2023, View Source [SID1234630370]).

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Under the terms of the agreement, Samyang Holdings grants non-exclusive rights to "NanoReady" technology and is responsible for supplying its key components to LG Chem, and LG Chem will be implementing "NanoReady" technology to develop innovative mRNA-based cancer therapeutics with maximal therapeutic effects. In return, LG Chem is required to pay upfront and a series of milestone payments upon completion of each developmental objective as in the terms agreed by both parties.

mRNA harnesses genetic information that directs the synthesis of specific proteins. Protective drug delivery systems that safely carry the mRNA to the target cell and mediate effective protein expression are crucial in developing mRNA therapeutics.

"NanoReady" is one of Samyang Holdings’ proprietary drug delivery technologies, characterized by its high adaptability to different mRNA molecules. Provided as a pre-made drug delivery formulation, it is expected to significantly reduce development time and time-to-patient, making an impact in the application of personalized therapeutics by streamlining the rigorous mixing process when being compounded with mRNA therapeutic molecules developed by LG Chem.

"LG Chem has exceptional R&D expertise in innovative drug development and a number of promising new drug pipelines," said Young-Joon Lee., CEO, Samyang Holdings Biopharm Division, "Taking this recent deal as a driving force, we will continue to strengthen our partnership with LG Chem. By leveraging the capabilities of the two companies, we hope to accelerate the development of innovative cancer therapy to significantly improve the lives of suffering cancer patients."

Ji-Woong Son, head of LG Chem’s Life Sciences company said, "We hope to maximize success through active collaboration between the two companies, with the aim to provide innovative mRNA-based cancer therapeutics to patients worldwide."

On April 20, 2023 Hansa Biopharma, the pioneer in immunomodulatory enzyme technology for rare IgG mediated diseases, reported its business update and interim report for January to March 2023 (Press release, Hansa Biopharma, APR 20, 2023, View Source [SID1234630366]).

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Highlights for the first quarter of 2023

Total Q1 revenue of SEK 24.2m including SEK 14.3m in product sales and SEK 9.9m in revenue recognition mainly under the agreement with Sarepta.
Received positive reimbursement decision in Spain, completing market access in the five largest European markets. Market access has now been secured in 12 European countries. Market Access procedures are ongoing in additional eight countries including Portugal, Belgium, and Switzerland.
Expanded commercialization partnership with Medison Pharma for Idefirix for kidney transplantation to cover the Baltics.
Announced completion of enrollment in phase 2 study of imlifidase in Guillain-Barré Syndrome (GBS).
Initiated clinical trial of HNSA-5487 with dosing in the first healthy volunteers.
Appointed Matthew Shaulis as Chief Commercial Officer and U.S. President of Hansa Biopharma.
Clinical pipeline update

U.S. ConfIdeS: As of April 20, 2023, 62, out of a target of 64 patients, have been enrolled in our pivotal U.S. open label, randomized controlled trial of imlifidase in kidney transplantation. Hansa continues to see strong interest among clinics and will continue enrollment to accelerate randomization. We expect to add further centers up to a total of 20.
On March 31, 2023, Hansa announced completion of enrollment in the phase 2 study of imlifidase in GBS. The first high level data read-out is expected in the second half of 2023.
Anti-GBM: Hansa’s pivotal phase 3 study in anti-GBM disease has been initiated with the first sites being activated at end of 2022 and several more sites to be activated before summer 2023. The study will target 50 patients with anti-GBM disease across the U.S., U.K. and EU, as previously communicated.
HNSA-5487, the second-generation lead molecule, is progressing. A new clinical phase 1 trial has started with dosing of the first healthy volunteers.
Financial summary

SEKm, unless otherwise stated – unaudited

Q1 2023

Q1 2022

12M 2022

Revenue

24.2

30.3

154.5

SG&A expenses

(103.3)

(80.4)

(337.9)

R&D expenses

(92.8)

(70.9)

(346.2)

Loss from operation

(182.3)

(135.0)

(588.6)

Loss for the period

(205.4)

(138.4)

(611.1)

Net cash used in operations

(207.0)

(130.5)

(502.7)

Cash and short-term investments

1,286.8

753.7

1,496.2

Shareholders’ equity

414.7

636.0

602.9

EPS before and after dilution (SEK)

(3.92)

(3.11)

(13.60)

Number of outstanding shares

52,443,962

44,473,452

52,443,962

Weighted avg. number of shares before and after dilution

52,443,962

44,473,452

44,923,998

Number of employees at the end of the period

159

141

150

Søren Tulstrup, President and CEO of Hansa Biopharma, comments

"Hansa’s commercial efforts for Idefirix in Europe continue to progress as planned. During the first quarter of 2023, we were pleased to announce a positive reimbursement decision in Spain, where more than 3,000 kidney transplantations are performed annually with approximately 90 percent of transplanted organs coming from deceased donors and where one in five on the kidney waitlist are classified as highly sensitized.

With Spain secured, Idefirix now has market access in the five largest markets in Europe, representing approximately 15,000 kidney transplants per year. This is great news for the thousands of people who are in urgent need of more personalized and innovative desensitization options like Idefirix which can enable incompatible kidney transplantation.

Our goal in kidney transplantation is to change the approach to desensitization and organ allocation by integrating Idefirix into clinical practice as a new standard-of-care (SOC) for highly sensitized patients. With this novel therapy, we are changing the transplantation ecosystem and advancing a treatment regime from one that has been solely focused on compatibility, to one that is more patient-centric – accommodating transplants for incompatible patients, who previously had no other choice than to wait and hope.

On the development side, we continue to drive progress across our pipeline. At the end of March, we announced the completion of enrollment in our phase 2 study of imlifidase in Guillain-Barré Syndrome (GBS). GBS is an acute autoimmune attack on the peripheral nervous system, which affects approximately one to two patients per 100,000 people, annually. The first high-level data read-out is expected in the second half of 2023, while the outcome of the comparative efficacy analysis to an externally matched cohort from the International GBS Outcome Study (IGOS) database is expected to be shared in 2024.

Patient enrollment in the U.S. continues to progress in our pivotal ConfIdeS trial in kidney transplantation. As of April 20, 2023, a total of 62 out of a targeted 64 patients were enrolled. Hansa continues to see strong interest among clinics and will continue enrollment to accelerate randomization and add additional centers up to a total of 20. This will help build valuable, real-world clinical experience in desensitization of highly sensitized patients among key transplantation centers and specialists in preparation for a planned launch in the market.

Lastly, I am pleased to announce we have dosed the first healthy volunteers with HNSA-5487, our lead molecule from our second-generation IgG antibody cleaving enzyme program. Moving HNSA-5487 into the clinic is a major accomplishment of our R&D team and an important milestone for the Company. HNSA-5487 represents an opportunity to substantially expand the potential indications in rare immunologic diseases that can be targeted, including indications where patients may benefit from more than one dose of an IgG-modulating enzyme.

On the organizational side, we are very excited to welcome Matthew Shaulis as the new Chief Commercial Officer and U.S. President. Matthew joins Hansa from Pfizer, where he held several senior executive roles including President, Inflammation and Immunology for the International Developed Markets; President, North America Oncology; and, most recently, Senior Vice President responsible for the company’s global commercial and medical go-to-market model transformation. With over 20 years of international experience in the pharmaceutical industry, Matthew will further strengthen our commercial and in-market leadership team and create a U.S.-focused organization that will help deliver our goal of bringing imlifidase to patients and clinicians in the U.S.

We continue to make solid strides in delivering on our mission of developing innovative, life-saving and life-altering immunomodulating therapies by leveraging our unique IgG-cleaving enzyme technology platform for people with rare diseases who have limited to no treatment options available."

Upcoming milestones and news flow

H1 2023 Anti-GBM Phase 3: First patient enrolled

H1 2023 U.S. Kidney transplantation (ConfIdeS): Complete enrollment

2023 Sarepta DMD pre-treatment: Commence clinical study

H2 2023 Long-term follow-up study in kidney transplantation: 5-year data readout

H2 2023 AMR Phase 2: Full data readout

H2 2023 GBS Phase 2: First data readout

H2 2023 U.S. Kidney transplantation (ConfIdeS): Complete randomization

2024 GBS Phase 2: Outcome of the comparative efficacy analysis to IGOS data

2024 U.S. Kidney transplantation (ConfIdeS): BLA submission

Updated financial calendar 2023

April 20, 2023 Interim Report for January-March 2023

June 14, 2023 2023 Annual General Meeting

July 20, 2023 Half-year Report for January-June 2023

October 18, 2023 Interim Report for January-September 2023

Conference call details

Hansa Biopharma will host a telephone conference today Thursday April 20, 2023, 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.

To participate in the telephone conference, please use the dial-in details provided below:

Sweden: +46 10 884 80 16

United Kingdom: +44 20 3936 2999

United States: +1 646 664 1960

Participant access code: 574936

The webcast will be available on View Source

The interim report and latest investor presentation can be downloaded from our web:

Interim report January to March 2023 View Source

Investor road show presentation Q1, 2023 View Source

This is information that Hansa Biopharma AB is obliged to make public pursuant to the Securities Markets Act.