The European Myeloma Network and Karyopharm Announce Dosing of First Patient in Collaborative EMN29/XPORT-MM-031 Study

On May 25, 2022 The European Myeloma Network (EMN), an international collaborative network of expertise centers for multiple myeloma in Europe and Australia, and Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, reported the dosing of the first patient in the collaborative EMN29/XPORT-MM-031 study, a randomized, global Phase 3 study evaluating an all-oral regimen of selinexor, Karyopharm’s first-in-class, oral exportin 1 (XPO1) inhibitor, in combination with Pomalyst (pomalidomide) and low-dose dexamethasone (SPd) versus Empliciti (elotuzumab), pomalidomide, and dexamethasone (EPd) in patients with relapsed or refractory multiple myeloma (NCT05028348//EMN29) (Press release, Karyopharm, MAY 25, 2022, View Source [SID1234615031]).

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The Phase 3, two-arm, randomized, active comparator-controlled, open-label, multicenter study will compare the efficacy, safety, and the impact on health-related quality of life of SPd versus EPd in pomalidomide-naïve patients with relapsed or refractory multiple myeloma. Patients will have received one to four prior lines of therapy, including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD) and an anti-CD38 monoclonal antibody (mAb), prior to being enrolled in the study. Final dosing for the selinexor arm at 40 mg or 60 mg will be determined following an interim analysis of data from the first 60 patients. The primary endpoint of the study is progression-free survival (PFS). The study is sponsored by the European Myeloma Network and is expected to recruit approximately 280 patients.

"Despite the progress made in treating multiple myeloma, new treatment options remain a critical need as the majority of patients will relapse and eventually stop responding to current therapies," said Reshma Rangwala, MD, PhD, Chief Medical Officer at Karyopharm Therapeutics. "We look forward to collaborating with the EMN on this important study. We have encouraging data in patients who have been treated with an anti-CD38 based regimen and we are eager to see the result in this patient population."

"We are extremely pleased to have this important study underway and look forward to further elucidating the potential of the SPd triplet regimen for Multiple Myeloma patients," added Professor Katja Weisel, Deputy Director of the University Cancer Center in Hamburg (UCCH) and principal investigator of the EMN29/XPORT-MM-031 study. "We look forward to the top-line results in 2024."

The initiation of this Phase 3 study follows encouraging data from an all-oral arm of the Phase 1b/2 STOMP study (NCT02343042) and the Phase 2 study XPORT-MM-028 (NCT04414475) in which selinexor was evaluated in combination with Pomalyst and low-dose dexamethasone in patients with relapsed or refractory multiple myeloma who received at least two prior lines of therapy, including a PI and an IMiD.

About Multiple Myeloma

According to Clarivate Analytics, approximately 47,000 patients are diagnosed with relapsed or refractory multiple myeloma in the U.S. each year.1 It is most frequently diagnosed in people aged 65-74 years old.2 Despite recent therapeutic advances, there is currently no cure and most patients’ disease will typically progress following treatment with currently available therapies. According to the American Cancer Society, an estimated 12,640 deaths due to multiple myeloma are expected to occur in the U.S. in 2022.3

About EMN

The European Myeloma Network (EMN) is an international collaborative network of expertise centers for multiple myeloma in Europe and Australia, working together with many national cooperative groups. EMN is chaired by Professor Pieter Sonneveld and Professor Mario Boccadoro. It has offices in Rotterdam, the Netherlands and in Torino, Italy. EMN has organized more than 30 international prospective trials in multiple myeloma and related diseases in collaboration with international pharmaceutical companies. EMN strongly supports translational research in its trials, focusing on modes of action of the drugs under investigation.

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm’s Selective Inhibitor of Nuclear Export (SINE) compounds to be approved for the treatment of cancer. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in multiple oncology indications, including: (i) in combination with Velcade (bortezomib) and dexamethasone (XVd) in patients with multiple myeloma after at least one prior therapy; (ii) in combination with dexamethasone in patients with heavily pre-treated multiple myeloma; and (iii) in patients with diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy. XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in a growing number of ex-U.S. territories and countries, including Europe, the United Kingdom, China, South Korea, Singapore and Israel, and is marketed in those areas by Karyopharm’s global partners. Selinexor is also being investigated in several other mid- and late-stage clinical trials across multiple high unmet need cancer indications, including endometrial cancer and myelofibrosis. For more information about Karyopharm’s products or clinical trials, please contact the Medical Information department at:

Tel: +1 (888) 209-9326
Email: [email protected]

XPOVIO (selinexor) is a prescription medicine approved:

In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy (XVd).

In combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti–CD38 monoclonal antibody (Xd).

For the treatment of adult patients with relapsed or refractory diffuse large B–cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma, after at least 2 lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
SELECT IMPORTANT SAFETY INFORMATION

Warnings and Precautions

Thrombocytopenia: Monitor platelet counts throughout treatment. Manage with dose interruption and/or reduction and supportive care.

Neutropenia: Monitor neutrophil counts throughout treatment. Manage with dose interruption and/or reduction and granulocyte colony–stimulating factors.

Gastrointestinal Toxicity: Nausea, vomiting, diarrhea, anorexia, and weight loss may occur. Provide antiemetic prophylaxis. Manage with dose interruption and/or reduction, antiemetics, and supportive care.

Hyponatremia: Monitor serum sodium levels throughout treatment. Correct for concurrent hyperglycemia and high serum paraprotein levels. Manage with dose interruption, reduction, or discontinuation, and supportive care.

Serious Infection: Monitor for infection and treat promptly.

Neurological Toxicity: Advise patients to refrain from driving and engaging in hazardous occupations or activities until neurological toxicity resolves. Optimize hydration status and concomitant medications to avoid dizziness or mental status changes.

Embryo–Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential and males with a female partner of reproductive potential, of the potential risk to a fetus and use of effective contraception.

Cataract: Cataracts may develop or progress. Treatment of cataracts usually requires surgical removal of the cataract.
Adverse Reactions

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive XVd are fatigue, nausea, decreased appetite, diarrhea, peripheral neuropathy, upper respiratory tract infection, decreased weight, cataract and vomiting. Grade 3–4 laboratory abnormalities (≥10%) are thrombocytopenia, lymphopenia, hypophosphatemia, anemia, hyponatremia and neutropenia. In the BOSTON trial, fatal adverse reactions occurred in 6% of patients within 30 days of last treatment. Serious adverse reactions occurred in 52% of patients. Treatment discontinuation rate due to adverse reactions was 19%.

The most common adverse reactions (≥20%) in patients with multiple myeloma who receive Xd are thrombocytopenia, fatigue, nausea, anemia, decreased appetite, decreased weight, diarrhea, vomiting, hyponatremia, neutropenia, leukopenia, constipation, dyspnea and upper respiratory tract infection. In the STORM trial, fatal adverse reactions occurred in 9% of patients. Serious adverse reactions occurred in 58% of patients. Treatment discontinuation rate due to adverse reactions was 27%.

The most common adverse reactions (incidence ≥20%) in patients with DLBCL, excluding laboratory abnormalities, are fatigue, nausea, diarrhea, appetite decrease, weight decrease, constipation, vomiting, and pyrexia. Grade 3–4 laboratory abnormalities (≥15%) are thrombocytopenia, lymphopenia, neutropenia, anemia, and hyponatremia. In the SADAL trial, fatal adverse reactions occurred in 3.7% of patients within 30 days, and 5% of patients within 60 days of last treatment; the most frequent fatal adverse reactions were infection (4.5% of patients). Serious adverse reactions occurred in 46% of patients; the most frequent serious adverse reaction was infection (21% of patients). Discontinuation due to adverse reactions occurred in 17% of patients.

FINAL RESULTS ANNOUNCEMENT for the year ended 31 December 2021

On May 25, 2022 ImmuPharma PLC (LSE:IMM), ("ImmuPharma" or the "Company"), the specialist drug discovery and development company, reported its final results for the twelve months ended 31 December 2021 (the "Period") (Press release, ImmuPharma, MAY 25, 2022, View Source [SID1234615030]).

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Key Highlights (including post Period review)

Loss for the Period of £8.2m (£6.9m at 31 December 2020)
Research and development expenses of £3.7m (31 December 2020: £2.4m)
Administrative expenses of £1.0m (31 December 2020: £1.8m)
Exceptional items of £1.4m (31 December 2020: £Nil), representing corporate reorganisation costs
Expected cost savings after corporate reorganisation (commencing from 2022) of approximately £1.1m per annum in committed overheads cost (excluding R&D project cost), a decrease of around 50%, including reduction of costs relating to Board and connected parties of £0.5m per annum
Cash balance at 31 December 2021 of £1.6m (31 December 2020: £5.9m)
Successful subscription and placing, raising in total £3.55m (gross) – December 2021
Lanstead derivative financial asset of £0.9m (31 December 2020: £1.2m)
Incanthera financial asset of £1.2m (£1.8m at 31 December 2020) and warrants financial asset of £0.2m (£0.6m at 31 December 2020)
Convertible loan notes of £Nil (£0.6m at 31 December 2020). Convertible loan notes repaid, totalling £0.8m (with accrued interest)
‘Autoimmunity’: Lupuzor ("P140")

P140 Pharmokinetic ("PK") study successfully completed with key endpoints met. Subcutaneous injection of P140 in 200 mcg and 800 mcg doses showed a clear time and dose-related PK profile, detectable in the blood of human volunteers and applicable for all potential clinical dosing regiments of P140
P140 was safe and well tolerated across all doses and in all subjects
Discussions continue with potential partners for Lupuzor (P140) outside of US in key territories
P140 for CIDP which is in active preparation for a phase 2/3 clinical study has now been initiated and specialist CRO appointed. Commercial partnering discussions ongoing
‘Anti-Infection’

BioAMB – further pre-clinical studies expected in second half of 2022. Commercial partnering discussions ongoing
BioCin – further pre-clinical studies expected in second half of 2022
Commenting on the statement and outlook Tim McCarthy, CEO, said: "2021 brought significant changes in the leadership of ImmuPharma. We have created positive and constructive developments within the business, with a focus on delivery of pipeline progression, meeting key future milestones and having a much more commercially driven corporate strategy.

"With now a fully reviewed and assessed R&D development pipeline, we remain focused on bringing our two late-stage clinical assets, Lupuzor and P140 for CIDP closer to the market. Specifically, on Lupuzor, our partner Avion, is committed to moving this program into Phase 3 as soon as possible, following final discussions with the FDA and based on the positive readout of the recent PK study. We are also focused on ensuring earlier stage assets, specifically within anti-infectives, progress, with a key strategy on securing partnering opportunities over the medium term.

We were delighted to secure the successful fundraising in late 2021, as it demonstrated that our corporate repositioning efforts, since the Board changes, were recognised by our existing shareholders and partner, Avion (Alora Pharmaceuticals).

"In closing, we look forward to sharing value enhancing newsflow over the next period, including progress within Lupuzor and our P140 platform. We would also like to thank our shareholders for their continuing support, particularly through the significant changes made over the last year, as well as our staff, corporate and scientific advisers and our partners including, CNRS and Avion."

DTx Europe

On May 25, 2022 EVERSANA reported to be attending this year’s DTx Europe Summit taking place 28-29 June 2022 in London, UK and online (Press release, EVERSANA, MAY 25, 2022, View Source [SID1234615029]). Schedule a meeting today to connect with digital therapeutic innovators,

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Join EVERSANA and other leaders in the industry, at the upcoming DTx Europe conference as we advance the conversation on:

Commercializing digital therapeutics by navigating the local regulatory pathways to approval
Advancing the adoption of companion apps
Securing broad payer coverage and reimbursement for digital therapies
Be sure to check out the following panel to hear from Martin Culjat, Senior VP, Regulatory Innovation & Digital Medicine, EVERSANA and other panelists discuss the need for innovation in policy of clinical trials.

Exploring a Need for Change of Clinical Trials in Digital Medicine
Tuesday, 28 June – 2:20pm BST

The current framework for clinical trials in digital health falls under an umbrella of one size fits all. There is an unwritten dichotomy between regularly updated software based digital health products and how regulatory frameworks are designed. This panel of experts will dive deep into discussing the need for innovation in policy of clinical trials.

What are the key challenges facing the European market in clinical trials today?
How can we utilise decentralised clinical trials to bring value to the DTx industry?
How do decentralized clinical trials compare to traditional studies?
Where can changes be made within the clinic to develop more efficient trials for DTx?
What changes must be made in regulation to accommodate for new digital medicine?
Companies are trying to innovate software fast in DTx but regulatory issues are getting in the way, how can we combat this?
How much of an algorithm can change for its regulatory classification to change?

Elevation Oncology Announces FDA Fast Track Designation Granted to Seribantumab for the Tumor-Agnostic Treatment of Solid Tumors Harboring NRG1 Gene Fusions

On May 25, 2022 Elevation Oncology, Inc. (Nasdaq: ELEV), a clinical stage biopharmaceutical company focused on the development of precision medicines for patients with genomically defined cancers, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to seribantumab for the tumor-agnostic treatment of advanced solid tumors that harbor NRG1 gene fusions (Press release, Elevation Oncology, MAY 25, 2022, View Source;utm_medium=rss&utm_campaign=elevation-oncology-announces-fda-fast-track-designation-granted-to-seribantumab-for-the-tumor-agnostic-treatment-of-solid-tumors-harboring-nrg1-gene-fusions [SID1234615028]). Seribantumab is currently being evaluated in the ongoing Phase 2 CRESTONE study, for which initial data will be presented in an oral presentation at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2022 Annual Meeting on Tuesday, June 7, 2022.

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"There are currently no approved therapies that specifically target NRG1 fusions, and therefore, receipt of Fast Track designation in a tumor-agnostic setting is a significant step in addressing this unmet need," said Shawn M. Leland, PharmD, RPh, Founder and Chief Executive Officer of Elevation Oncology. "NRG1 fusions are a type of genomic alteration that causes unregulated cell growth and proliferation in a variety of solid tumors, and we look forward to working closely with the FDA as we continue exploring the potential of seribantumab to improve outcomes for patients whose tumor harbors this unique oncogenic driver."

Fast Track is an FDA process designed to facilitate the development and expedite the review of potential therapies that seek to treat serious conditions and fill an unmet medical need. A drug candidate that receives Fast Track designation is afforded greater access to the FDA for the purpose of expediting the drug’s development, review and potential approval. Additionally, the designation allows for eligibility for Accelerated Approval and Priority Review, if relevant criteria are met, as well as a Rolling Review, which means a drug company can submit completed sections of its New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be submitted for review.

About Seribantumab and NRG1 Gene Fusions

Seribantumab is a fully human IgG2 monoclonal antibody that binds to human epidermal growth factor receptor 3 (HER3). HER3 is traditionally activated through binding of its primary ligand, neuregulin-1 (NRG1). The NRG1 gene fusion is a rare genomic alteration that combines NRG1 with another partner protein to create chimeric NRG1 "fusion proteins". The NRG1 fusion protein is often also able to activate the HER3 pathway, leading to unregulated cell growth and proliferation. Importantly, NRG1 gene fusions are predominantly mutually exclusive with other known genomic driver mutations and are considered a unique oncogenic driver event associated with tumor cell survival.

NRG1 fusions have been identified in a variety of solid tumors, including lung, pancreatic, gallbladder, breast, ovarian, colorectal, neuroendocrine, cholangiocarcinomas, and sarcomas. In preclinical experiments, seribantumab prevented the activation of HER3 signaling in cells that harbor an NRG1 gene fusion and destabilized the entire ERBB family signaling pathway including the activation of HER2, EGFR, and HER4. In addition to extensive nonclinical characterization and testing, seribantumab has been administered to over 800 patients across twelve Phase 1 and 2 studies, both as a monotherapy and in combination with various anti-cancer therapies. Seribantumab was granted Fast Track designation from the FDA for the tumor-agnostic treatment of patients whose solid tumors harbor NRG1 fusions and is currently being evaluated in the Phase 2 CRESTONE study for patients with solid tumors of any origin that have an NRG1 fusion.

About the Phase 2 CRESTONE Study

CRESTONE (Clinical Study of Response to Seribantumab in Tumors with Neuregulin-1 (NRG1) Fusions; NCT04383210) is a Phase 2 tumor-agnostic study evaluating seribantumab in patients with solid tumors that harbor an NRG1 fusion and have progressed after at least one prior line of standard therapy. The primary objective of the study is to describe the anti-tumor activity and safety of seribantumab as a monotherapy specifically in patients whose solid tumor is uniquely driven by an NRG1 gene fusion. CRESTONE offers a clinical trial opportunity for patients with advanced solid tumors who have not responded or are no longer responding to treatment. Patients are encouraged to talk to their doctor about genomic testing of their tumor. CRESTONE is open and enrolling today in the United States, Australia, and Canada. For more information visit www.NRG1fusion.com.

Entry into a Material Definitive Agreement

On May 25, 2022, Celsion Corporation ("Celsion") reported that entered into an At the Market Offering Agreement (the "Agreement") with H.C. Wainwright & Co., LLC, as sales agent ("Wainwright"), pursuant to which Celsion may offer and sell, from time to time, through Wainwright shares of common stock, par value $0.01 per share, of Celsion having an aggregate offering price of up to $ 7,500,000 (the "Shares") (Filing, 8-K, Celsion, MAY 25, 2022, View Source [SID1234615027]). Celsion intends to use the net proceeds from the offering, if any, for general corporate purposes, including research and development activities, capital expenditures and working capital. Pending the application of the net proceeds, Celsion intends to invest the net proceeds in short-term, investment grade, interest-bearing securities.

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Celsion is not obligated to sell any Shares under the Agreement. Subject to the terms and conditions of the Agreement, Wainwright will use commercially reasonable efforts, consistent with its normal trading and sales practices and applicable state and federal law, rules and regulations and the rules of The Nasdaq Capital Market, to sell Shares from time to time based upon Celsion’s instructions, including any price, time or size limits or other customary parameters or conditions Celsion may impose.

Under the Agreement, Wainwright may sell Shares by any method deemed to be an "at the market offering" as defined in Rule 415 promulgated under the Securities Act of 1933, as amended.

The Agreement may be terminated by Wainwright or Celsion at any time upon notice to the other party, or by Wainwright at any time in certain circumstances, including the occurrence of a material adverse change in Celsion. Unless earlier terminated, the Agreement will automatically terminate upon the issuance and sale of all of the Shares subject to the Agreement through Wainwright on the terms and subject to the conditions set forth in the Agreement.

Celsion will pay Wainwright a commission of 3.0% of the aggregate gross proceeds from each sale of Shares and has agreed to provide Wainwright with customary indemnification and contribution rights. Celsion has also agreed to reimburse Wainwright for legal fees and disbursements, not to exceed $35,000 in the aggregate, in connection with entering into the Agreement.

The foregoing summary of the Agreement does not purport to be complete and is qualified in its entirety by reference to the full text of the Agreement, a copy of which is attached as Exhibit 10.1 hereto and incorporated herein by reference.

The purchasers in and placement agent for the Company’s January 2022 preferred stock offerings provided consents under their respective purchase and placement agent agreements for the At the Market Offering program.

The Shares will be issued pursuant to Celsion’s previously filed and effective Registration Statement on Form S-3 (File No. 333-254515), the base prospectus dated March 30, 2021, filed as part of such Registration Statement, and the prospectus supplement dated May 25, 2022, filed by Celsion with the Securities and Exchange Commission. This Current Report on Form 8-K shall not constitute an offer to sell or the solicitation of an offer to buy Shares, nor shall there be any sale of the Shares in any state in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state.