On December 16, 2020 Amphivena Therapeutics, a clinical-stage oncology company focused on developing a platform of bivalent T-cell engagers that restore anti-cancer immunity in patients, reported the first patient has been dosed in the Phase 1 dose expansion study evaluating AMV564, an investigational, first-in-class agent that depletes myeloid derived suppressor cells (MDSC) and activates T cells, in adult patients with selected solid tumor indications (Press release, Amphivena Therapeutics, DEC 16, 2020, View Source [SID1234572955]).

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The dose escalation phase of the first-in-human, multicenter, open-label study of AMV564 in solid tumors was initiated in October 2019 and established the safety, PK profile and clinical activity of AMV564 including a confirmed RECIST complete response.

The dose expansion study will explore AMV564 as a monotherapy in tumor-specific cohorts selected to enrich for patients with actionable antigens. The primary objectives of the study are to further characterize the safety and tolerability and to evaluate preliminary efficacy of AMV564 monotherapy administered subcutaneously.

"AMV564 has demonstrated an excellent safety profile and encouraging preliminary activity in heavily pretreated patients, and we look forward to seeing how this novel mechanism translates to clinical benefit in selected cancer patient populations including patients with inadequate benefit from checkpoint inhibitors, representing an area of high unmet need," said Curtis Ruegg, Ph.D., President and Chief Executive Officer of Amphivena Therapeutics.

The trial is being conducted at a planned 15 sites in the United States. Additional information on this trial, including comprehensive inclusion and exclusion criteria, can be accessed at www.ClinicalTrials.gov (NCT04128423).

About AMV564

AMV564 relieves immune suppression via targeted depletion of immunosuppressive MDSC and drives T cell activation and polarization to restore anti-cancer immunity. To date, over 95 patients have received AMV564 across three Phase 1 clinical trials for patients with solid tumors, acute myeloid leukemia (AML), and myelodysplastic syndromes (MDS).

On December 16, 2020 Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), reported that it has entered a collaboration enabling the use by AC Bioscience LTD of preclinical and clinical data for LX2931 without granting any right or license under any of Lexicon’s patent rights for the compound (Press release, Lexicon Pharmaceuticals, DEC 16, 2020, View Source [SID1234572939]). LX2931 is a small molecule sphingosine-1-phosphate (S1P) lyase inhibitor that is currently not in active development at Lexicon.

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"In line with our realignment around the rapid development of the LX9211 program, we continue to evaluate our pipeline and determine the most effective way to advance our broad library of compounds and targets," said Praveen Tyle, Ph.D., executive vice president of research and development. "We are pleased to collaborate with AC Bioscience to accelerate their efforts around S1P and look forward to learning additional information about this potentially interesting pathway."

Under the terms of the agreement, Lexicon will receive an upfront payment and is eligible to receive milestone payments totaling up to $5.3 million in the aggregate.

On December 16, 2020 Qualigen Therapeutics, Inc. (NASDAQ: QLGN) ("Qualigen" or the "Company"), reported it has entered into a definitive agreement with a single institutional investor for the purchase and sale for $12,000,000 of (i) 2,370,786 Common Shares, (ii) 1,000,000 Pre-Funded Warrants (iii) 1,348,314 Common Warrants exercisable immediately and (iv) 842,696 Common Warrants exercisable six (6) months after issuance at a combined purchase price of $3.56 in a registered direct offering (Press release, Qualigen, DEC 16, 2020, View Source [SID1234572956]). The Common Warrants will have an exercise price of $4.07 per share and have a term of two years. The closing of the offering is expected to occur on or about December 18, 2020, subject to the satisfaction of customary closing conditions.

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A.G.P./Alliance Global Partners is acting as sole placement agent for the offering.

This offering is being made pursuant to an effective shelf registration statement on Form S-3 (File No. 333-232798) previously filed with the U.S. Securities and Exchange Commission (the "SEC"). A prospectus supplement describing the terms of the proposed offering will be filed with the SEC and will be available on the SEC’s website located at View Source Electronic copies of the prospectus supplement may be obtained, when available, from A.G.P./Alliance Global Partners, 590 Madison Avenue, 28th Floor, New York, NY 10022, or by telephone at (212) 624-2060, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On December 16, 2020, in connection with the consummation of the previously reported offering and sale by Lexicon Pharmaceuticals, Inc. (the "Company") of 20,312,500 shares of the Company’s common stock, par value $0.001 per share, in a registered direct offering to Artal International S.C.A. ("Artal"), an affiliate of Invus, L.P. ("Invus"), and certain other investors, the Company entered into a Supplement No. 3 (the "Supplement") with Invus and Invus C.V. supplementing the terms of the Company’s stockholders’ agreement ("Stockholders’ Agreement") and registration rights agreement ("Registration Rights Agreement") with Invus, each dated June 17, 2007 (as previously amended, supplemented or otherwise modified, the "Transaction Agreements") (Filing, 8-K, Lexicon Pharmaceuticals, DEC 16, 2020, View Source [SID1234573069]).

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The Supplement amends (i) the definition of "Investor" in the Stockholders’ Agreement to include Artal and its affiliates and (ii) the definition of "Holders" in the Registration Rights Agreement to include Artal and its affiliates. Artal is an affiliate of Invus, the Company’s largest stockholder.

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

On December 16, 2020 Novartis reported that the U.S. Food and Drug Administration (FDA) granted iptacopan (LNP023) Breakthrough Therapy Designation (BTD) in paroxysmal nocturnal hemoglobinuria (PNH) and Rare Pediatric Disease (RPD) Designation in C3 glomerulopathy (C3G) (Press release, Novartis, DEC 16, 2020, https://www.novartis.com/news/media-releases/novartis-investigational-oral-therapy-iptacopan-lnp023-receives-fda-breakthrough-therapy-designation-pnh-and-rare-pediatric-disease-designation-c3g [SID1234634302]).

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The breakthrough designation is intended to expedite the development and review of medicines for serious conditions to address unmet medical need, where early clinical evidence indicates a drug may demonstrate substantial improvement over available therapy on clinically significant endpoints. The FDA granted BTD to iptacopan for the treatment of PNH based on positive interim results from two ongoing Phase II studies, where iptacopan showed substantial benefits both in patients who remained anemic and dependent on transfusions despite standard of care anti-complement treatment8, as well as monotherapy in anti-C5 naïve PNH patients.

PNH is a rare and life threatening blood disorder characterized by complement-driven hemolysis, thrombosis and impaired bone marrow function9,10, resulting in anemia, fatigue and other debilitating symptoms that can impact patients’ quality of life1–3. Despite current standard of care – anti-C5 therapy eculizumab or ravulizumab – a large proportion of PNH patients remain anemic and dependent on transfusions1,2,9,11,12.

FDA grants the rare pediatric designation for serious or life-threatening diseases primarily affecting individuals aged 18 years or younger and impacting fewer than 200,000 people. C3G, an ultra-rare and severe form of primary glomerulonephritis4,13, is characterized by complement dysregulation. It has a poor prognosis; about 50% of patients progress to end-stage renal disease (ESRD) within 10 years, and 50–70% experience disease recurrence post kidney transplant14. It has a worldwide annual incidence of 1-2 per million15.

About iptacopan
Iptacopan (LNP023) is a first-in-class, orally administered, potent and highly selective factor B inhibitor of the alternative complement pathway6,7. It is currently in clinical development for PNH, as well as C3G and a number of other renal conditions with complement system involvement where significant unmet needs exist, including IgA nephropathy (IgAN), atypical hemolytic uremic syndrome (aHUS), and membranous nephropathy (MN).

Positive Phase II data from one study in PNH were presented at the European Society for Blood and Marrow Transplantation (EBMT) congress in August8, and Phase II interim analysis results in C3G were presented at the virtual 2020 Annual Meeting of the American Society of Nephrology (ASN) in October. Novartis is planning to initiate Phase III studies in several indications.

Iptacopan has the potential to become the first complement pathway inhibitor to slow disease progression in a number of complement-driven diseases. Based on disease prevalence and the positive interim data from Phase II studies, iptacopan has also received orphan drug designations from the FDA and EMA in C3G and PNH16, as well as EMA PRIME designation for C3G17, and EMA orphan drug designation in IgAN18.