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.

On April 20, 2023 Edison Oncology Holding Corp. ("Edison Oncology"), a privately held biopharmaceutical company, reported the presentation of two scientific posters at the annual meeting of the American Association of Cancer Research held in Orlando, Florida from April 14-19 (Press release, Edison Oncology, APR 20, 2023, View Source [SID1234630365]).

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In a presentation entitled "A Pilot Clinical Trial of VAL-413 (Orotecan, oral irinotecan HCl) in Patients with Recurrent Pediatric Solid Tumors," the company reported that, to date in its ongoing Phase 1-2a clinical trial, of Orotecan, a novel oral formulation of irinotecan, no dose limiting toxicity has been observed in patients receiving Orotecan, and preliminary analysis of pharmacokinetic data suggests that plasma levels of irinotecan and its active metabolite, SN-38, are similar to levels observed following treatment with unformulated commercially available i.v. irinotecan given orally in the same patient.

In a second presentation entitled "Investigating the Therapeutic Efficacy of EO3001 in Clear Cell Carcinoma of the Ovary", Edison Oncology reported that in CRISPR/Cas9 generated isogenic pairs of ovarian cancer cell lines, EO3001 selectively kills ARID1A mutant cancer cells at low nanomolar concentrations compared to "wildtype" ovarian cancer cells that do not harbor the mutation. A mechanism of action of EO3001 in ARID1A mutant ovarian clear cell carcinoma (OCCC) is also proposed. Since ARID1A mutations are known to inactivate the SWI/SNF complex and expose the DNA into an "open chromatin" structure, such cells have high DNA replication and enhanced metabolic rates. To meet this enhanced energy need such cells become dependent on oxidative phosphorylation (OXPHOS). The poster describes that by directly inhibiting the action of certain mitochondrial proteins required for the function of the OXPHOS pathway, EO3001 may result in energy deprivation and apoptosis the of metabolically overactive ARID1A mutant tumor cells.
About Orotecan (oral irinotecan HCl)

Orotecan is a novel oral liquid formulation of irinotecan hydrochloride designed to deliver the drug orally with improved tolerability. Irinotecan is an intravenous (i.v.) topoisomerase inhibitor approved by the FDA for the treatment of colorectal cancer and used ‘off label’ in the treatment of a several types of cancer including gastric cancers, neuroendocrine and adrenal tumors, pancreatic cancer, small cell lung cancer, cervical cancer, ovarian cancer, esophageal cancer, soft tissue sarcomas and bone cancer.

A standard regimen of i.v. irinotecan for the treatment of childhood tumors involves daily infusions for five consecutive days, every two to three weeks leading to significant patient inconvenience, diminished quality of life and high cost to the healthcare system. "Orotecan’s oral formulation has been developed to improve tolerability and patient compliance in the treatment of rare childhood tumors, with an opportunity to expand into major adult cancers," said Jeffrey Bacha, Edison Oncology’s chief executive officer.

"The data we have observed in our clinical trial thus far supports our confidence that Orotecan has the opportunity to address significant unmet needs in a number of tumors where i.v. irinotecan is currently utilized as a single agent, or in combination with oral anti-cancer treatments."

To date, more than 200 patients have been treated with unformulated commercially available i.v. irinotecan delivered orally with promising results in the treatment of Ewing sarcoma and other pediatric cancers; however, poor palatability of the drug has limited the widespread adoption of this approach in clinical practice.

Edison Oncology’s ongoing clinical trial (clinicaltrials.gov identifier: NCT04337177) is designed to examine the safety and tolerability of Orotecan and provide data assess the pharmacokinetics of Orotecan compared to current oral regimen employing unformulated i.v. irinotecan given orally.

About EO3001

EO3001 is a novel small-molecule drug candidate that has been studied in prior clinical trials involving more than 1000 patients. Recent research conducted by Edison Oncology and academic researchers has demonstrated EO3001 exhibits potent and selective activity against ARID1A deficient cancer cells. Mutations in ARID1A are associated with treatment resistance and poor outcomes in several cancers including gynecologic malignancies, breast cancer, gastric cancer, colorectal cancer and pancreatic cancer.

Ovarian clear cell carcinoma (OCCC) is an orphan cancer indication representing approximately 10% of ovarian cancers in North America with a higher frequency in patients of east Asian descent. OCCC is inherently resistant to standard chemotherapy and radiation treatment and currently represents the worst prognosis of any form of epithelial ovarian cancer.

"According to published studies up to 60% of OCCC patients harbor ARID1-A mutations, providing a readily available biomarker for patient selection in clinical trials," said Mr. Bacha. "We look forward to advancing EO3001 as a potential treatment for this important unmet medical need."

On April 20, 2023 Fusion Pharmaceuticals Inc. (Nasdaq: FUSN), a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines, reported that the Company will present in a company presentation at the Bloom Burton & Co. Healthcare Investor Conference on Tuesday, April 25, 2023 at 3:00 p.m. ET (Press release, Fusion Pharmaceuticals, APR 20, 2023, View Source [SID1234630364]). Presenting on behalf of Fusion will be Chief Executive Officer John Valliant, Ph.D.

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A live webcast of the event will be available on the "Events and Presentations" page in the "Investors & Media" section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 90 days following their respective presentation dates.

On April 20, 2023 Blueprint Medicines Corporation (NASDAQ: BPMC) reported that it will host a live conference call and webcast at 8:00 a.m. ET on Thursday, May 4, 2023 to report its first quarter financial results and provide a corporate update (Press release, Blueprint Medicines, APR 20, 2023, View Source [SID1234630363]).

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To access the live conference call, please dial 833-470-1428 (domestic) or 929-526-1599 (international), and refer to conference ID 668091. A webcast of the call will also be available under "Events and Presentations" in the Investors & Media section of the Blueprint Medicines website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the call.

On April 20, 2023 Rakovina Therapeutics Inc. (the "Company") (TSXV:RKV) reported the presentation of new data describing the progress of the Company’s kt-3000 drug development program at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in Orlando, Florida (Press release, Rakovina Therapeutics, APR 20, 2023, View Source [SID1234630362]).

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The Company’s data was presented in the DNA Damage Response section at the AACR (Free AACR Whitepaper) annual meeting in a poster entitled "A novel bi-functional agent targeting PARP and HDAC in Ewing sarcoma".

Ewing sarcoma is a highly aggressive bone and soft tissue tumor affecting mainly children and young adults, with a dismal 5-year survival rate of 15-30% for metastatic disease. Previous studies have demonstrated that Ewing sarcoma cells are sensitive to FDA-approved PARP inhibitors, but clinical trials have failed to produce a durable treatment response.

Rakovina Therapeutics’ kt-3000 series is a novel class of DNA-damage response inhibitors with dual PARP HDAC inhibitor functionality.

The combination of a PARP inhibitor with an HDAC inhibitor have shown potential synergy in laboratory studies. However, the clinical treatment of patients with the combination to date has been associated with significant side effects, limiting the adoption of this therapeutic strategy.

The kt-3000 series was designed based on the hypothesis that combining both HDAC and PARP inhibition into a single molecule would provide a more viable approach to providing clinical benefit to patients, while retaining efficacy and limiting side effects.

Data presented at the meeting demonstrate that Rakovina Therapeutics’ kt-3000 prototype lead candidate exhibits higher PARP-1 vs. PARP-2 selectivity compared to the FDA-approved PARP inhibitor, olaparib. Selectivity against PARP1 is believed to correlate with an improved safety profile vs. first-generation PARP inhibitors.

The data also demonstrate that the dual functional kt-3000 prototype lead candidate is more effective against Ewing sarcoma tumor cells than either a PARP inhibitor or HDAC inhibitor alone. This is achieved despite reduced potency at HDAC compared to the FDA-approved HDAC inhibitor, vorinostat.

The kt-3000 lead candidate effectively reduced lung metastases in mice inoculated with Ewing sarcoma tumor cells. The most common site where Ewing sarcoma metastasizes, or spreads, in patients is to their lungs, which is a leading cause of morbidity and mortality.

"The kt-3000 series compounds were designed with an aim of achieving synergistic PARP+HDAC activity against treatment of resistant tumors while improving safety and tolerability of treatment," said Prof. Mads Daugaard, Rakovina Therapeutics’ president and chief scientific officer.

"We believe that this profile offers potential as a new treatment for tumors traditionally resistant to therapy, particularly in the recurrent disease setting for Ewing sarcoma and major cancers such as breast and ovarian cancer that has become resistant to treatment with FDA-approved PARP inhibitors," he added.

Select kt-3000 lead candidates are being evaluated for pharmacokinetics and safety in vivo as part of the process to select a primary lead candidate for advancement to human clinical trials.