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InDex Pharmaceuticals Holding AB (publ) interim report January – September 2022

On November 23, 2022 InDex Pharmaceuticals reported its interim report for January-September 2022(Press release, InDex Pharmaceuticals, NOV 23, 2022, View Source [SID1234624381]).

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Period January – September 2022
Net sales amounted to SEK 0.0 (0.0) million
Operating loss amounted to SEK –45.0 (–80.2) million
Result after tax amounted to SEK –44.0 (–80.2) million, corresponding to SEK –0.08 per share (–0.16) before and after dilution
Cash flow from operating activities amounted to SEK –95.2 (–80.1) million
Cash and cash equivalents at the end of the period amounted to SEK 398.3 (463.1) million
Number of employees at the end of the period was 7 (8)
Number of shares at the end of the period was 532,687,650
All comparative amounts in brackets refer to the outcome during the corresponding period 2021.

Significant events during the quarter

InDex got a new patent for cobitolimod granted in Europe
Significant events after the reporting period

InDex’s Board of Directors named Jenny Sundqvist as new CEO
Other events

InDex received positive feedback from the Japanese regulatory authority, the PMDA, regarding the clinical development plan for cobitolimod
CEO statement
It has been an eventful quarter, the Board has appointed a new CEO and InDex has received great interest in cobitolimod at Europe’s largest scientific meeting for gastroenterologists, UEGW. In addition, EMA’s safety committee has recently recommended new measures to limit the use of JAK inhibitors, which again highlights the need for new drugs for ulcerative colitis without severe side effects.

Patient recruitment for the phase III study CONCLUDE with cobitolimod is underway and a number of clinics have been very active in enrolling patients. However, the study has had a slower start-up than expected with several underlying reasons. Many clinics are still handling the effects of the covid pandemic, which has and continues to result in longer administrative processes. Russia’s invasion of Ukraine has had a clear impact, as the planned clinics in Russia have had to be replaced with clinics in other countries. The work continues at full speed to activate the remaining clinics in, among other places, Ukraine and not least to ensure that the initiated clinics include patients. We continue to take measures that we believe will have positive effect on the recruitment rate going forward. In our dialogue with the clinics, it is clear that there is a strong interest in participating in the CONCLUDE study with its new mechanism of action and a great medical need for new treatment options for patients with moderate to severe ulcerative colitis.

Based on the previously communicated successful interactions with the Japanese regulatory authority, the Pharmaceuticals and Medical Devices Agency (PMDA), we continue to plan to include Japanese patients in our second induction study in the phase III program for cobitolimod and are simultaneously evaluating the possibility of entering strategic collaborations in Japan. We have received encouraging feedback on the unique and positive decision from PMDA in our contacts with potential partners, and the decision also indicates great potential for cobitolimod.

In the beginning of October, InDex participated as an exhibitor at the United European Gastroenterology Week (UEGW) in Vienna, the largest scientific meeting for gastroenterologists in Europe. Informing healthcare professionals and other stakeholders about cobitolimod and the phase III study CONCLUDE is part of our planned activities. There was a great interest in cobitolimod and CONCLUDE, with many visitors to our booth, both from clinics already participating in our study and from new clinics expressing interest to join.

Our ongoing clinical pharmacokinetic study (PK study) with cobitolimod is progressing according to plan and the results are expected to be presented during the first quarter of 2023. The study will include at least 6 patients with moderate to severe ulcerative colitis treated with doses of 500 mg cobitolimod administered rectally. The aim of the study is to confirm that the systemic uptake of cobitolimod is limited, which has been shown in previous preclinical and clinical studies. A limited systemic uptake is a significant advantage compared to competing drugs for ulcerative colitis that act on the whole body and can cause severe side effects outside the inflamed colon.

On October 28, EMA’s safety committee recommended new measures for the use of JAK inhibitors due to their risk of severe side effects. The new measures imply that several patient groups should only use JAK inhibitors if there are no other treatment options available. The recommendations are another reminder that the safety profile of a drug is very important, which is good news for cobitolimod which has so far shown an excellent safety profile. FDA updated its safety warnings and restricted the use of JAK inhibitors already back in September 2021.

On October 10, InDex announced that the Board has appointed Jenny Sundqvist as the new CEO, effective from January 1, 2023. Jenny brings a broad experience from pharmaceutical development and business management, and we all look forward to working together with Jenny.

During the quarter, we have continued with our appreciated investor presentations. For those of you who did not have the opportunity to watch the presentations live, the recordings are available on our website. We will present InDex tomorrow on November 24, at Redeye Life Science Day and I hope to see you then!

Johan Giléus, acting CEO

For more information:
Johan Giléus, acting CEO
Phone: +46 8 122 038 50
E-mail: [email protected]

The full report is attached as a PDF and is available on the company’s website View Source

Publication
This information is information that InDex Pharmaceuticals Holding AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation (MAR). The information was submitted for publication through the agency of the contact person set out above at 8:00 CET on November 23, 2022.

Pacylex Granted FDA Fast Track Designation for PCLX-001 for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

On November 22, 2022 Pacylex, a clinical-stage N-myristoyltransferase (NMT) inhibitor company developing first-in-class therapies for leukemia, lymphoma, and solid tumor cancers, reported that the U.S. Food and Drug Administration (FDA) has granted PCLX-001 Fast Track Designation for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) (Press release, Pacylex Pharmaceuticals, NOV 22, 2022, View Source [SID1234645058]). Fast Track is a process to facilitate drug development and expedite the review of new drugs and vaccines intended to treat or prevent serious conditions and address unmet medical needs.

The FDA’s decision was informed by the results of Pacylex’s nonclinical studies and an ongoing Phase 1/2 clinical study of PCLX-001 in non-Hodgkin Lymphoma (NHL) and solid tumor patients, which showed that treatment with PCLX-001 had a favorable safety and tolerability profile. "Receiving Fast Track designation from the FDA reinforces Pacylex’s belief in PCLX-001 as a potential new treatment for AML," said Michael Weickert, PhD, CEO of Pacylex.

"Because Acute myeloid leukemia is a rapidly progressing, difficult-to-treat blood cancer with a poor prognosis, and Pacylex’s nonclinical data with PCLX-001 suggests that AML may be the cancer type most sensitive to NMT inhibitors, we have been eager to advance PCLX-001 into clinical studies in AML patients in the near future," said John Mackey, MD, CMO of Pacylex.

Earlier this month the FDA cleared the IND for PCLX-001 for Phase 1/2 clinical study to start in AML patients in the coming months. The FDA also recently granted PCLX-001 Orphan Drug Designation for AML. PCLX-001 is currently being studied in NHL and solid tumor cancer patients at 4 sites in Canada. Pacylex has the first and only NMT inhibitor in clinical studies.

In the ongoing clinical studies of PCLX-001, seventeen patients have been accrued through 5 dose levels of oral, once-per-day PCLX-001 with no dose limiting toxicities observed. The ongoing clinical PCLX-001 trial in NHL and solid tumor patients is registered at ClinicalTrials.gov Identifier: NCT04836195. Data on the scientific rationale for PCLX-001 in AML patients will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition from 10-13 December 2022.

About Acute Myeloid Leukemia
In 2020, more than 474,500 new cases of leukemia were reported globally with more than 311,500 deaths (Source: 1). In 2017, AML accounted for 23.1% of total leukemia cases worldwide, and it is one of the most common types of leukemia in adults (Source: 2,3). In the U.S., an estimated 20,050 new cases of AML will be diagnosed in 2022 with the five-year survival rate reported at 30.5% (Source: 2,4).
The conventional treatment for newly diagnosed AML is intensive induction and consolidation chemotherapy with HSCT for eligible patients (Source: 5). The introduction of new targeted therapies in recent years has added to the standard of care and improved outcomes for some patients with molecularly defined AML subtypes (Source: 6). However, there remains a need to improve survival for the majority of patients with AML (Source: 5).

PCLX-001
PCLX-001 (aka DDD86481) is a first-in-class, small molecule NMT inhibitor originally developed by the University of Dundee Drug Discovery Unit as part of a program to treat African sleeping sickness, funded by Welcome Trust. Pacylex is developing PCLX-001 in the form of a once-a-day pill initially to treat leukemia and lymphoma. PCLX-001 has also been shown to inhibit the growth of lung and breast cancer tumors in animal models. In leukemia, lymphoma and breast cancer patients, the levels of NMT2 are correlated with survival, suggesting an important biological role in these cancers.

Zhongsheng Shangjian "SG1408 for Injection" Phase I Clinical Trial Completed the First Subject’s First Administration

On November 22, Sumgen Biotech reported that the "Safety, Tolerability and Preliminary Effectiveness of SG1408 for Injection in Subjects with Advanced Malignant Solid Tumors" sponsored by Zhongsheng Shangjian Biomedicine (Hangzhou) Co., Ltd. (hereinafter referred to as Zhongsheng Shangjian) Phase I clinical study of "Phase I Clinical Research Center of Zhejiang Cancer Hospital completed the first administration of the first subject (Press release, Sumgen Biotech, NOV 22, 2022, View Source;a=nav&id=293 [SID1234625273]). This is another key milestone in the clinical research of SG1408 following the successful kick-off meeting held in Zhejiang Cancer Hospital on November 3.

Ms. Li Na, clinical director and vice president of Shangjian Biology and Zhongsheng Shangjian, said that SG1408 for injection is an innovative drug targeting PD-L1/VEGF independently developed by Zhongsheng Shangjian. The Drug Administration approved clinical trials in patients with advanced solid tumors. The project can overcome the impact of the new crown epidemic and start and advance quickly. I would like to thank Secretary Cheng Xiangdong, Director Song Zhengbo of Zhejiang Cancer Hospital, leaders at all levels of the hospital, and China Biotech for their strong support, and thank the project team for their joint efforts; look forward to everyone’s joint efforts Under the circumstances, the project can make further positive progress and benefit more cancer patients.

About SG1408 Phase I Clinical Trial

PD-L1 and VEGF are important targets for tumor therapy. The SG1408 double antibody can specifically bind PD-L1 and VEGF at the same time. On the one hand, by binding to PD-L1 on the surface of tumor cells, it can specifically block the PD-1/PD-L1 immunosuppressive signaling pathway and activate the body’s immune response; On the one hand, by combining with VEGF molecules, it effectively blocks the interaction of VEGF/VEGFR, inhibits the formation of tumor angiogenesis, and inhibits the proliferation and migration of tumor cells; the two mechanisms can exert a synergistic anti-tumor effect. Preclinical studies have shown that SG1408 has a clear anti-tumor effect, can exert a synergistic effect of "1+1>2", and has good safety. At present, there are no dual-antibody drugs targeting both PD-1/PD-L1 and VEGF approved for marketing at home and abroad. SG1408, as the first double antibody project developed by Zhongsheng Shangjian, is a manifestation of the further improvement of the company’s antibody research and development system.

SG1408 phase I clinical trial population is patients with relapsed or refractory advanced malignant solid tumors, focusing on chest tumors (lung cancer, thymus cancer), gynecological tumors (cervical cancer, endometrial cancer), digestive tract tumors (liver cancer, esophageal cancer) , colorectal cancer), etc., the project has been launched in the phase I clinical center of Zhejiang Cancer Hospital, and many centers have recently started, welcome to consult for details.

Race Receives Positive Guidance on Zantrene via Pre-IND Meeting with US FDA

On November 23, 2022 – Race Oncology Limited ("Race") is pleased to report that it has received confidential and constructive guidance by way of a pre-Investigational New Drug (pre-IND) meeting with the US Food and Drug Administration (FDA) for Zantrene (bisantrene dihydrochloride). Race submitted a pre-IND meeting package to the FDA in September 2022, which included a summary of the preclinical and clinical data for Zantrene, an overview of the proposed clinical development plan, and specific questions on the requirements to open an Investigational New Drug (IND) application (Press release, Race Oncology, NOV 22, 2022, View Source [SID1234625139]).

Opening an IND is a requirement before undertaking clinical trials in the United States and is a key step in the process of obtaining a New Drug Application (NDA) and marketing approval for a new drug. In written correspondence, the FDA provided insightful and helpful feedback on the proposed clinical development plan for Zantrene and acknowledged that Acute Myeloid Leukemia (AML) continues to have significant unmet clinical needs which require new treatment solutions.

Importantly, the FDA agreed that Race had adequately addressed outstanding clinical hold issues from an earlier IND application submitted in 2019 (ASX announcement: 25 April 2019). Under current US regulations, if the FDA does not place a clinical hold within 30 days of an IND submission the trial can proceed. The FDA also confirmed that the FDA505(b)(2) application pathway is a possible regulatory pathway for Zantrene, whereby some of the data from studies completed on Zantrene in the public domain can contribute to the full regulatory package required for a New Drug Application (NDA).

The FDA provided guidance on the data requirements for opening an IND for Zantrene, including helpful counsel related to the AML patient populations that should be studied.

While Race has no current plans to undertake any clinical trials in the US, receiving timely guidance from the FDA provides valuable commercial optionality as Race’s clinical activities and partnership discussions continue to advance. Race Oncology Ltd ABN 61 149 318 749 Registered Office: L36, 1 Macquarie Place, Sydney NSW 2000 www.raceoncology.com

Race Oncology Chief Scientific Officer, Dr Daniel Tillett said: "The clear guidance the FDA has provided Race on the regulatory requirements needed to undertake future clinical trials of Zantrene in the US is highly encouraging. While we are not planning on running clinical trials in the US in the short to medium term, having the option to run US trials, when appropriate, is extremely valuable.

Race Oncology Chief Executive Officer, Phil Lynch said: "As Race becomes increasingly clinical in its operations, FDA guidance is invaluable to ensuring our readiness, and to having the necessary optionality, when we seek to pursue our USA based clinical plans."

Pacylex Granted FDA Fast Track Designation for PCLX-001 for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia

On November 22, 2022 Pacylex, a clinical-stage N-myristoyltransferase (NMT) inhibitor company developing first-in-class therapies for leukemia, lymphoma, and solid tumor cancers, reported that the U.S. Food and Drug Administration (FDA) has granted PCLX-001 Fast Track Designation for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) (Press release, Greenfire, NOV 22, 2022, View Source [SID1234624533]). Fast Track is a process to facilitate drug development and expedite the review of new drugs and vaccines intended to treat or prevent serious conditions and address unmet medical needs.

The conventional treatment for newly diagnosed AML is intensive induction and consolidation chemotherapy with HSCT for eligible patients (Source: 5). The introduction of new targeted therapies in recent years has added to the standard of care and improved outcomes for some patients with molecularly defined AML subtypes (Source: 6). However, there remains a need to improve survival for the majority of patients with AML (Source: 5).