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DiCE Molecules Announces $80 Million Series C Financing

On January 8, 2021 DiCE Molecules, a biopharmaceutical company leveraging its proprietary DNA-encoded library platform to discover and develop next-generation therapeutics in immunology, reported the completion of an $80 million Series C financing (Press release, DiCE Molecules, JAN 8, 2021, View Source [SID1234573713]). The financing was led by RA Capital Management with participation from new investors including Eventide Asset Management, New Leaf Venture Partners, Soleus Capital, Driehaus Capital Management, Osage University Partners and Asymmetry Capital Management. Existing investors Northpond Ventures, Sands Capital, Sanofi Ventures, Alexandria Venture Investments, Altitude Life Science Ventures and Agent Capital also participated.

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DiCE’s drug discovery platform leverages a proprietary DNA-encoded library (DEL) technology, combined with unique structural insights, to generate small molecule antagonists against a range of protein-protein-interface (PPI) targets. Proceeds from the financing will support the progression of the Company’s first-in-class, oral IL-17 antagonist into clinical trials, as well as enable the advancement and expansion of its preclinical portfolio of additional PPI antagonists. The IL-17 family of cytokines are strong inducers of inflammation and are implicated in a variety of autoimmune diseases including psoriasis, psoriatic arthritis and ankylosing spondylitis.

"We are thrilled to have the support of this stellar group of investors as we continue to advance important new medicines for patients suffering from debilitating autoimmune diseases," said Kevin Judice, Ph.D., Chief Executive Officer of DiCE. "This financing will enable us to accelerate our lead IL-17 program through important milestones while advancing our other assets, including a pair of integrin inhibitors, and also to expand our pipeline using the same combination of technology and structural insights. We believe the immunology space is underserved by current small molecule approaches and we are excited about the opportunity to advance next-generation therapeutics for this patient population."

In connection with the financing, Jake Simson, Ph.D., will join the DiCE Board of Directors. As a Partner at RA Capital Management, Dr. Simson works on both public and private investments and serves as a Board Director for Xenikos, B.V., Tyra Biosciences and AavantiBio. He holds a B.S. in materials science and engineering from MIT and a Ph.D. in biomedical engineering from Johns Hopkins University.

"We are looking forward to working with RA Capital Management and our additional new investors as we continue to build DiCE into a leading player in small molecule immunology," said Richard Scheller, Ph.D., Chairman of the Board of DiCE. "We are fortunate to have these investors join the DiCE team, and we welcome Jake Simson to our Board."

"We believe the DiCE team is poised to disrupt the multibillion dollar IL-17 class with a molecule that combines a best-in-class efficacy and convenience profile," said Jake Simson, Ph.D., Partner at RA Capital Management. "Beyond IL-17, we are excited to partner with the DiCE team and this high quality investor syndicate to continue to identify and develop best-in-class oral molecules to disrupt biologic drugs across immunology indications."

SVB Leerink acted as exclusive financial advisor for DiCE Molecules’ Series C financing.

MANA Therapeutics Launches with $35 Million Series A Financing

On January 8, 2021 MANA Therapeutics, a clinical-stage company creating nonengineered, off-the-shelf allogeneic cell therapies that target multiple cancer antigens, reported a $35 million Series A financing (Press release, MANA Therapeutics, JAN 8, 2021, View Source [SID1234573712]). The financing was led by Cobro Ventures and Lightchain Capital, with participation from LifeSci Venture Partners and other undisclosed investors. MANA is using its EDIFY platform to develop a pipeline of proprietary and partnered off-the-shelf cell therapies for cancer patients across a broad range of liquid and solid tumors, with an initial focus on relapsed acute myeloid leukemia (AML).

MANA’s EDIFY platform leverages natural immune system pathways to educate T-cells to target multiple cell surface and intracellular tumor-associated antigens. The multi-antigen targeting is uniquely achieved without the need for viral or non-viral delivery systems or genetic modifications. MANA was founded based on the research and human proof-of-concept clinical trials conducted by Catherine Bollard, M.D., MBChB and her team at Children’s National Hospital (CNH) along with colleagues at Johns Hopkins Medical Center. The initial CNH and Hopkins-led clinical trials in solid and hematologic tumors supported a strong safety profile, showed immunological anti-tumor activity and validated MANA’s initial set of tumor antigens. The product candidates used in these trials formed the basis of the Phase 1 candidate, MANA-312, an allogeneic donor-derived cell therapy for the treatment of AML in the relapsed/refractory post-hematopoietic stem cell transplant (HSCT) setting, and MANA-412, a preclinical off-the-shelf allogeneic cell therapy being developed for the treatment of transplant-ineligible AML and solid tumors.

"I co-founded MANA with Dr. Bollard and her colleagues from Children’s National Hospital because we believe off-the-shelf, allogeneic approaches are the future of cell therapy that will enable more patients to benefit from this breakthrough in cancer treatment," said Marc Cohen, Co-founder of Cobro Ventures and Co-founder and Executive Chairman of MANA Therapeutics. "MANA is building upon the strong foundational science established at CNH with a unique approach that promises to produce off-the-shelf allogeneic therapies that do not compromise on safety or efficacy. I look forward to continuing to support the MANA team as they advance their internal pipeline for the treatment of AML and select solid tumors, and expand the potential of EDIFY through strategic partnerships focused on new target antigens and cancer types."

MANA’s goal is to develop an inventory of off-the-shelf allogeneic products that will be able to treat the majority of patients in targeted cancer indications based on a simplified HLA matching protocol. Through multiple antigen targeting, MANA’s product candidates are designed to prevent immune escape and could provide superior efficacy to single antigen and other cell therapy approaches. MANA uses a simplified scalable manufacturing process that does not require genetic modification and has a high product yield, with the potential to generate tens of billions of cells from a single partial HLA-matched donor. The high safety profile supported by academic clinical trials and the nonengineered approach may also permit for repeat dosing of patients.

"Over the past decade we have seen tremendous progress in cancer research and treatment and are beginning to unlock the potential of cell therapy for a variety of tumor types," said Dr. Bollard, who chairs MANA’s Scientific Advisory Board. "The human proof-of-concept trials conducted by my team and colleagues showed potential for a nonengineered approach to educating T-cells to attack multiple tumor antigens, which MANA is expanding even further through refinement of the manufacturing process for an allogeneic product and application to a broader set of antigens in a variety of clinical indications and settings."

"This Series A funding is enabling rapid progress with our programs," said Martin Silverstein, M.D., President and CEO of MANA Therapeutics. "We recently initiated our Phase 1 clinical trial for MANA-312 in relapsed/refractory AML with the goal of establishing single-agent activity and safety of higher and multiple doses along with the assessment of key efficacy biomarkers in the post-transplant setting. This important study with an allogeneic donor-derived cell therapy is designed to inform and accelerate trials for our off-the-shelf allogeneic candidate, MANA-412, for which we plan to file an IND in late-2021."

As part of the financing, Drew Dennison of Lightchain Capital has joined the MANA Board of Directors. Locust Walk served as exclusive transaction advisor to MANA.

About EDIFY

MANA Therapeutics’ EDIFY platform constitutes the next-generation cell therapy approach by leveraging natural immune system pathways to educate T-cells to target the unique sets of antigens expressed by tumors, without the need for genetic modification. The EDIFY platform uses dendritic cells as antigen presenting cells. The dendritic cells are loaded with ManaMix antigens and stimulate and expand T-cells to generate product candidates. Product candidates developed from the EDIFY platform are designed to increase efficacy through multiple antigen targeting and to support a strong safety profile by utilizing a nonengineered approach that could permit for repeat dosing of patients. The EDIFY platform uses a simplified, high-yield manufacturing process.

OncoSec Announces First Patient Dosed in Phase 2 Trial of TAVO™ Plus OPDIVO® as Neoadjuvant Therapy for Melanoma

On January 8, 2021 OncoSec Medical Incorporated (NASDAQ:ONCS) (the "Company" or "OncoSec") reported the first patient was dosed in OMS-104, an investigator-initiated Phase 2 trial evaluating TAVO (tavokinogene telseplasmid), the Company’s intratumoral DNA plasmid-based interleukin-12 (IL-12) therapy administered using its gene delivery platform (gene electrotransfer), in combination with the anti-PD-1 checkpoint inhibitor OPDIVO (nivolumab) as a neoadjuvant therapy prior to surgery in patients with operable, locally or regionally advanced melanoma (Press release, OncoSec Medical, JAN 8, 2021, View Source [SID1234573711]). The trial is designed to evaluate if the addition of TAVO can improve clinical outcomes already observed when using nivolumab alone as a neoadjuvant therapy.

Anti-PD1 checkpoint inhibitors, when administered as a neoadjuvant therapy, have shown encouraging clinical results, but rapid recurrence remains an issue for many patients. TAVO in combination with OPDIVO may drive deep anti-tumor immune responses and complete elimination of tumors prior to surgery, leading to improved long-term clinical outcomes for a significant proportion of treated patients. TAVO in combination with another anti-PD-1 checkpoint inhibitor, KEYTRUDA (pembrolizumab), has already been shown to enhance overall response rate and partial tumor responses in patients with anti-PD-1 checkpoint-refractory metastatic melanoma in OncoSec’s KEYNOTE-695 registration directed Phase 2 clinical trial.

"While studies have shown relapse and overall survival advantages when checkpoint inhibitors are given alone following surgery, there is a need to investigate novel immunotherapeutic agents such as TAVO that can be given preoperatively in order to further enhance the clinical efficacy of immunotherapy in patients with advanced melanoma," said Armad A. Tarhini, M.D., Ph.D., Leader of the OMS-104 trial and Senior Member and Professor at the H. Lee Moffitt Cancer Center and Research Institute and the University of South Florida Morsani College of Medicine. "The neoadjuvant approach utilizing TAVO in combination with checkpoint inhibitors as being tested in this study may improve operability, pathologic tumor response and long-term disease control, which is highly desirable for these patients, who continue to have a high risk of recurrence and progression despite the use of standard therapy after surgery."

OMS-104 (NCT04526730) is a Phase 2 open-label, single arm study investigating intratumoral TAVO delivered by gene electrotransfer, or short electric pulses, plus nivolumab as neoadjuvant therapy in patients with operable locally-regionally advanced melanoma. The trial aims to enroll 33 patients and consists of three phases:

1) Neoadjuvant phase, where TAVO will be administered intratumorally using gene electrotransfer in three cycles on days one and eight every four weeks and nivolumab will be administered after TAVO on day eight of each cycle via 30-minute intravenous (IV) infusion;

2) Surgical phase consisting of a definitive surgery that will be scheduled 2-4 weeks after the last dose of nivolumab following radiologic and clinical assessment; and

3) Adjuvant phase, where nivolumab monotherapy will begin 2-4 weeks after surgery and will be administered for up to nine four-week cycles.

The primary endpoint is pathological complete response, estimated based on the proportion of participants with no viable tumor on histologic assessment at definitive surgery after the 12-week neoadjuvant period.

Daniel J. O’Connor, President and Chief Executive Officer of OncoSec, added, "TAVO delivers DNA plasmid-based IL-12 directly into the tumor using gene electrotransfer, which demonstrably enhances the immunogenicity of the treated tumors to yield productive ‘in situ’ vaccines. This principle has yielded striking results in post-PD-1 patients and is likely relevant in this earlier clinical setting. We look forward to exploring the utility of TAVO as a potential neoadjuvant therapy in a variety of solid tumor settings for patients in need of more effective treatment options."

About TAVO
OncoSec’s gene delivery technology combines TAVO (tavokinogene telseplasmid), a DNA plasmid-based interleukin-12 (IL-12), with an intra-tumoral gene delivery platform (gene electrotransfer) to achieve endogenous IL-12 production in the tumor microenvironment that enables the immune system to target and attack tumors throughout the body. TAVO has demonstrated a local and systemic anti-tumor response in several clinical trials, including the pivotal Phase 2b trial KEYNOTE-695 for metastatic melanoma and the KEYNOTE-890 Phase 2 trial in triple negative breast cancer (TNBC). TAVO has received both Orphan Drug and Fast-Track Designation by the U.S. Food & Drug Administration for the treatment of metastatic melanoma.

KemPharm Announces Pricing of $50 Million Public Offering of Common Stock and Warrants and Uplisting to The Nasdaq Capital Market

On January 8, 2021 KemPharm, Inc. (NASDAQ: KMPH), a specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, reported the pricing of an underwritten public offering of 7,692,307 shares of its common stock (or pre-funded warrants to purchase common stock in lieu thereof) and accompanying warrants to purchase up to 7,692,307 shares of common stock (Press release, KemPharm, JAN 8, 2021, View Source [SID1234573710]). Each share of common stock (or pre-funded warrant in lieu thereof) is being sold together with one warrant to purchase one share of common stock at a combined public offering price of $6.50. The aggregate gross proceeds from the offering are expected to total $50.0 million, before deducting the underwriting discounts and commissions and estimated offering expenses payable by KemPharm and without giving effect to proceeds from any subsequent exercise of warrants.

KemPharm’s common stock will be listed on The Nasdaq Capital Market and will trade under the ticker symbol "KMPH" beginning today, January 8, 2021. The offering is expected to close on or about January 12, 2021, subject to customary closing conditions. In addition, KemPharm has granted to the underwriter a 45-day option to purchase up to 1,153,846 additional shares of its common stock and/or warrants to purchase up to 1,153,846 shares of its common stock, in any combination thereof, at the public offering price, less the underwriting discount.

Roth Capital Partners is acting as sole manager for the offering.

The securities described above are being sold by KemPharm pursuant to a registration statement filed by KemPharm with the Securities and Exchange Commission (the "SEC"), which was declared effective on January 7, 2021. The securities will be sold only by means of a prospectus, forming a part of the effective registration statement. Electronic copies of the accompanying prospectus may be obtained, when available, by contacting Roth Capital Partners, 888 San Clemente, Newport Beach, CA 92660, Attn: Prospectus Department, telephone: 800-678-9147, or email at [email protected], or by visiting the SEC’s website at View Source

argenx Announces 2021 Corporate Priorities and Highlights Recent Achievements Across Immunology Pipeline

On January 8, 2021 argenx (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases and cancers, reported its 2021 corporate priorities and highlighted recent achievements from its late-stage immunology pipeline driven by its FcRn antagonist, efgartigimod (Press release, argenx, JAN 8, 2021, View Source,of%20its%20clinical%2Dstage%20autoimmune [SID1234573709]). Additionally, the Company announced interim data from the Phase 2 CULMINATE trial of cusatuzumab in development with Cilag GmbH International, an affiliate of Janssen, and provided financial guidance for 2021.

argenx previously announced an exclusive license agreement with Zai Lab Limited ("Zai Lab") for the development and commercialization of efgartigimod in Greater China and the acceleration of efgartigimod development through Phase 2 proof-of-concept trials in new autoimmune indications. Zai Lab will also contribute Chinese patients to argenx’s global Phase 3 trials of efgartigimod. Under the terms of the agreement, argenx will receive $175 million in collaboration payments comprised of upfront Zai Lab equity, a guaranteed development cost-sharing payment, and a milestone payment upon U.S. efgartigimod approval. argenx will also be eligible for tiered royalties based on annual net sales of efgartigimod in Greater China.

"We are excited to enter a new chapter for argenx as we look toward commercialization and achieving our mission of reaching patients with debilitating rare diseases. We’ve submitted a BLA to the FDA for efgartigimod in gMG and expect to have global efgartigimod trials ongoing this year in six indications and two formulations. We hope to continue to demonstrate the broad opportunity of our FcRn antagonist within autoimmune diseases in 2021 and beyond," said Tim Van Hauwermeiren, Chief Executive Officer of argenx. "In parallel, establishing global commercial infrastructure within the U.S. and Japan continues to be a top priority. Now through our collaboration with Zai Lab in China and with the appointment of a general manager in Europe, we’ve solidified and accelerated our capabilities to bring efgartigimod and our future immunology candidates to patients worldwide."

2021 Corporate Priorities and Recent Progress

The Company will continue its transition to a fully integrated immunology company by executing on three corporate priorities in 2021, including: preparation for the potential FDA approval and U.S. commercial launch of efgartigimod for the treatment of patients with gMG; the progression of its clinical-stage autoimmune pipeline; and the continued growth of its broad and differentiated pipeline through its Immunology Innovation Program.

Preparation for potential FDA approval and global commercial launch of efgartigimod for the treatment of patients with gMG

Submitted BLA to FDA for efgartigimod for treatment of gMG and continued preparations for other global regulatory submissions

On track to submit application to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) in first half of 2021
On track to submit to European Medicines Agency (EMA) in second half of 2021
Submission in China expected to occur shortly following potential approval in U.S.
Initiated bridging study for SC efgartigimod in gMG based on association between total IgG reduction and clinical benefit, and feedback from the FDA. The study is a registrational, non-inferiority trial comparing the pharmacodynamic effect of 1000mg SC efgartigimod with 10mg/kg IV efgartigimod and is expected to enroll approximately 50 patients.
Commercial preparation activities are underway and on track for potential 2021 launch, including continued engagement with key stakeholders, commercial inventory build, milestone-based hiring of field force around potential BLA acceptance and FDA approval, and development of a patient services program.
Appointed Anant Murthy, Ph.D., as General Manager of argenx Europe. In this role, Dr. Murthy will establish the commercial infrastructure for a European launch and lead market development activities in advance of a potential European Medicines Agency (EMA) approval of efgartigimod. Dr. Murthy brings ~20 years of international experience to argenx, most recently as Head of Market Access for EMEA and Canada and the General Manager of multiple European countries for Alnylam Pharmaceuticals.
Progress clinical-stage autoimmune pipeline, including seven expected global trials of efgartigimod and Phase 1 trial of first-in-class C2 antibody ARGX-117

ADVANCE (IV) and ADVANCE SC trials ongoing evaluating IV and SC efgartigimod in patients with primary immune thrombocytopenia (ITP); global program expected to support registration of both formulations
ADDRESS registrational trial ongoing evaluating SC efgartigimod in patients with pemphigus vulgaris (PV) and pemphigus foliaceous (PF)
ADHERE trial ongoing evaluating SC efgartigimod in chronic inflammatory demyelinating polyneuropathy (CIDP);

Completed enrollment of first 30 patients
Decision whether to expand enrollment up to 130-140 patients expected in first quarter of 2021
Clinical trials in fifth and sixth indications of efgartigimod to begin enrollment in 2021
Ongoing Phase 1 healthy volunteer trial of IV and SC ARGX-117, a first-in-class C2 antibody, to evaluate safety and tolerability and establish dosing regimen

Data expected in mid-2021, after which argenx plans to launch Phase 2 proof-of-concept trials in severe autoimmune diseases, including multifocal motor neuropathy (MMN)
Continued investment in broad and differentiated pipeline through Immunology Innovation Program

Preclinical work ongoing in early-stage pipeline, including continued progress on ARGX-118 and ARGX-119, and the optimization of ARGX-120
Commitment to expand pipeline at cadence of one new candidate per year from Immunology Innovation Program

Interim Data from Cusatuzumab Phase 2 CULMINATE Trial

Development of cusatuzumab in acute myeloid leukemia (AML) remains ongoing as part of a global collaboration and license agreement with Cilag GmbH International, an affiliate of Janssen.

The Phase 2 CULMINATE trial (NCT04023526) is evaluating cusatuzumab in combination with azacitidine in newly-diagnosed, elderly AML patients who are ineligible for intensive chemotherapy. A total of 103 patients were randomized to receive either 10mg/kg (n=51) or 20mg/kg (n=52) cusatuzumab plus azacitidine as part of a dose identification. The 20mg/kg dose has been selected for ongoing and future trials.

A pre-planned interim analysis was conducted of the 52 patients (46.2% adverse ELN risk classification) receiving 20mg/kg cusatuzumab plus azacitidine treatment (intent-to-treat population (ITT)). The results from the ITT analysis showed a complete remission (CR) rate of 27% (14/52) and composite complete remission (CRc), including CRs with incomplete hematologic recovery, rate of 40% (21/52). The 30-day mortality rate of the ITT population was 9.6% (5/52). In a cohort where patients received at least two treatment cycles (20mg/kg cusatuzumab plus azacitidine), 42% (14/33) achieved CR and 64% (21/33) achieved CRc.

Cusatuzumab was observed to be well-tolerated and the safety profile was consistent with prior studies. Final results from the CULMINATE trial will be presented in a peer-reviewed forum.

The decision to initiate additional studies in the development of cusatuzumab, under the collaboration, will be determined following review of data from the ongoing Phase 1b ELEVATE trial (NCT04150887), which is evaluating cusatuzumab in combination with venetoclax and azacitidine in newly-diagnosed, elderly patients with AML who are ineligible for intensive chemotherapy.

Financial Guidance

As of December 31, 2020, argenx had approximately $2.0 billion in cash, cash equivalents and current financial assets. This preliminary cash balance does not include expenses or proceeds from recently announced business development transactions, including the purchase of a priority review voucher from Bayer HealthCare Pharmaceuticals, Inc. and the exclusive license agreement with Zai Lab for efgartigimod in Greater China.

Based on current plans to fund anticipated operating expenses and capital expenditures, argenx expects its cash burn to increase significantly in 2021, approximately doubling compared to 2020. The increased spend will support the Company’s transition to an integrated immunology company in 2021, including the build-out of global commercial infrastructure and drug product inventory ahead of the expected launch of efgartigimod in gMG in the U.S, the advancement of its clinical-stage pipeline, including seven expected global trials of efgartigimod, and the continued investment in its Immunology Innovation Program.

J.P. Morgan Healthcare Conference Presentation and Webcast

argenx CEO Tim Van Hauwermeiren, will present these updates at the virtual 39th Annual J.P. Morgan Healthcare Conference on Monday, January 11, 2020 at 8:20 a.m. ET, followed by a question and answer session.

The live webcast of the presentation and question and answer session that follows may be accessed on the homepage of the argenx website at www.argenx.com. A replay of the webcast will be available for 90 days on the argenx website.

About Efgartigimod

Efgartigimod is an investigational antibody fragment designed to reduce disease-causing immunoglobulin G (IgG) antibodies and block the IgG recycling process. Efgartigimod binds to the neonatal Fc receptor (FcRn), which is widely expressed throughout the body and plays a central role in rescuing IgG antibodies from degradation. Blocking FcRn reduces IgG antibody levels representing a logical potential therapeutic approach for several autoimmune diseases known to be driven by disease-causing IgG antibodies, including: myasthenia gravis (MG), a chronic disease that causes muscle weakness; pemphigus vulgaris (PV), a chronic disease characterized by severe blistering of the skin; immune thrombocytopenia (ITP), a chronic bruising and bleeding disease; and chronic inflammatory demyelinating polyneuropathy (CIDP), a neurological disease leading to impaired motor function.