Ligand Completes Acquisition of Icagen Core Assets, Partnered Programs and Ion Channel Technologies

On April 2, 2020 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported the completion of its acquisition of the core assets of Icagen’s North Carolina operations (Press release, Ligand, APR 2, 2020, View Source [SID1234556112]). As previously announced, the purchase price was $15 million in cash, and Icagen is entitled to receive additional cash payments based on certain revenue achievements.

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"We are very pleased to add the Icagen technologies, partnerships and this profitable business to Ligand," said John Higgins, Chief Executive Officer of Ligand. "We all know how extremely challenging the current business environment is, and I want to commend the teams on both sides who worked together to get this deal done. It’s a fantastic acquisition that will enable Ligand to expand its services to current partners and drive new collaborations. We expect the Icagen ion channel capabilities will have particular synergy with our Vernalis and OmniAb antibody discovery platforms. We welcome our new Icagen colleagues to Ligand."

Higgins continued, "While we adjust to conducting business during the pandemic, Ligand’s balance sheet remains very strong, and we continue to generate positive cash flow. We have a successful track record of acquiring innovative companies, and subsequently growing revenue, increasing our partner portfolio, and leveraging technology and customer synergies. Given our significant financial resources and talented operating team, we are well positioned to pursue acquisitions and new opportunities to expand our business."

The acquisition of Icagen brings Ligand the following:

Technology Platform. Icagen’s extensive biological capability focused on ion channels, transporters and x-ray fluorescence, along with a strong track record in novel drug discovery from screening to lead optimization. Ion channels are key components in a wide variety of biological processes that involve rapid changes in cells and have broad therapeutic applicability including cancer, metabolic disease, pain, neurological diseases, infectious diseases and others.
Roche Collaboration to develop and commercialize therapies for neurological diseases. The collaboration provides research funding, potential milestone payments of up to $274 million and tiered royalty payments should a drug be commercialized.
Cystic Fibrosis Foundation (CFF) Collaboration to discover therapeutics to treat patients with cystic fibrosis caused by specific genetic mutations. The CFF collaboration allows for up to $11 million in research funding, milestone payments of up to $59 million and tiered royalties on sales, should a product be commercialized.
Proprietary Service Unit to Drive New Collaborations and Revenue, including a 32-person R&D team based in Raleigh, N.C., now known as Icagen, a Ligand Company, focused on drug discovery of ion channels and transporters. Icagen provides Ligand with an East Coast operation to efficiently serve partners and brings a portfolio of current or recent/active collaboration agreements with over 30 biopharma companies plus an ongoing business development pipeline.
Novel, Unpartnered Programs that include six preclinical-stage assets applicable to a range of therapy areas including diabetes, Parkinson’s disease, pain and other disorders.

Cancer Vaccine Start Up, Calviri, Closes Successful Seed Funding Round

On April 2, 2020 Calviri, Inc., a biotech startup spun out of Arizona State University Biodesign Institute, focused on ending deaths from cancer, reported the closing of $2.25 million in seed round funding (Press release, Calviri, APR 2, 2020, View Source [SID1234556111]). The funding is from five private investors including Dr. Jacque Sokolov, a Calviri Board member, and Dr. Mitzi Krockover.

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Calviri was founded based on the discovery that tumors make frequent and recurrent errors in RNA transcription and processing that create highly immunogenic frameshift peptides (FSPs). Calviri has developed a manufacturing process that displays >400,000 FSPs on microchips; this enables patients’ blood to be simply screened for anti-FSP antibodies. This process is the basis for the design of off-the-shelf vaccines for any cancer, as well as diagnostics for the early detection of any cancer. Calviri holds exclusive rights, licensed from Skysong, to the diagnostic technology developed by the Biodesign Center at ASU.

Kathryn Sykes, VP of Research and Product Development, stated, "We will use the funding to demonstrate the strengths of this unique technology. We anticipate demonstrating an accurate predictive diagnostic for response to checkpoint inhibitors and a high sensitivity test for the early detection of breast and colon cancers."

Stephen Albert Johnston, CEO and founder, announced, "This funding is a vote of confidence for our ambitious goals. It comes at a time that will allow Calviri to meet some important valuation inflection milestones for both the diagnostics and vaccines."

Merrimack Divests Early Stage Asset for $2.25 Million; Provides Strategy Update

On April 2, 2020 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK) [("Merrimack" or the "Company")] reported receipt of $2.25 million in connection with the closing of a transaction in which the Company sold certain assets related to its preclinical nanoliposome programs to Celator Pharmaceuticals, Inc. Merrimack will not receive any further contingent consideration or royalties as a result of this transaction (Press release, Merrimack, APR 2, 2020, View Source [SID1234556110]).

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Under the terms of the agreement for the transaction, Celator, in addition to paying the base purchase price of $2.25 million, reimbursed the Merrimack for certain specified expenses and assumed certain liabilities with respect to the acquired assets. Further, under the agreement, the Company assigned to Celator the previously disclosed intellectual property license agreement among the Company and Ipsen S.A pursuant to which Ipsen granted the Company licenses to certain patents, and Celator also granted the Company an exclusive license to certain specified know-how and patents related to specific nano-liposome projects which remain in the Merrimack portfolio.

Completion of this transaction is a continuation of Merrimack’s strategy of extending its cash runway into 2027 and preserving its ability to capture the potential $450 million of remaining ONIVYDE-related contingent milestone payments resulting from its 2017 asset sale to Ipsen as well as the potential $54.5 million of remaining contingent milestone payments resulting from its 2019 sale of certain programs to Elevation Oncology, Inc. (formerly known as 14ner Oncology, Inc.).

"This asset sale transaction reflects our ongoing strategic focus on both the monetization of our remaining preclinical assets and the further reduction of our operating expenses," said Gary Crocker, Chairman of Merrimack’s Board of Directors. "Ipsen recently publicly announced that ONIVYDE is in Phase 3 clinical trials in two additional indications which we believe could, if successful, support the attainment of certain milestones. The proceeds from the Celator transaction provide an additional operational buffer in the event of any potential unanticipated contingencies, and enhance our ability to focus on our top priority, which is to preserve our ability to collect milestones and to maximize returns to shareholders. As opportunities arise we will continue to look to distribute excess cash not essential to our minimal operations."

MATEON THERAPEUTICS TEAM PUBLISHES A NEW PEER-REVIEWED ONCOLOGY ARTICLE ON THE POSITIVE CLINICAL STUDY RESULTS FOR OT-101 AGAINST RECALCITRANT RESISTANT ANAPLASTIC ASTROCYTOMA – A RARE FORM OF MALIGNANT BRAIN TUMOR

On April 2, 2020 Mateon Therapeutics Inc. (OTCQB:MATN) reported the publication of a peer-reviewed research article co-authored by Fatih Uckun MD PhD, Sanjive Qazi PhD, and Vuong Trieu, PhD in the oncology journal Cancer Reports and Reviews (Press release, Mateon Therapeutics, APR 2, 2020, View Source [SID1234556109]).

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We previously reported the preliminary findings of a Phase II study (NCT00431561) confirmed its favorable safety profile and showed that OT101 can offer early disease control to R/R high-grade glioma patients at 6 months at a rate comparable to that achieved with the standard alkylating chemotherapy drug temozolomide. We are now reporting our analysis of the long-term follow-up data on the recalcitrant/resistant anaplastic astrocytoma (R/RR AA) patient subpopulation treated in the NCT00431561 study as proof of concept for the clinical utility of the Convection Enhanced Delivery of OT-101 platform (CEDOT). Notably, OT101 administered intratumorally via the CEDOT platform exhibited clinically meaningful single-agent activity and induced durable complete response (CR), partial response (PR) in more than half of the treated R/R AA patients. The median overall survival of patients receiving the CEDOT-delivered experimental therapy 1136 (95% CI:811 – 1743) days which was significantly better than the 590 (95% CI:287 – NA) days median OS (Log Rank χ2=6.5, P-value=0.011) of the TMZ-treated patient population.

"This work demonstrates superiority of OT-101 against an approved chemotherapeutic agent against AA and emphasizes our commitment to find effective new therapies for difficult-to-treat cancers," stated Dr. Vuong Trieu, Chairman and Chief Executive Officer of Mateon Therapeutics.

OT-101 has received orphan drug designation for glioblastoma, melanoma, and pancreatic cancer. Furthermore, FDA recently granted Rare Pediatric Designation for OT-101 against diffuse intrinsic pontine glioma (DIPG). OT-101 is also effective against coronavirus including COVID-19 and being deployed against the COVID-19 epidemic.

Seattle Genetics Announces Potential Accelerated Approval Pathway in the U.S. for PADCEV™ (enfortumab vedotin-ejfv) in Combination with Immune Therapy Pembrolizumab as First-Line Treatment for Advanced Urothelial Cancer

On April 2, 2020 Seattle Genetics, Inc. (Nasdaq:SGEN) reported an update on the phase 1b/2 multicohort EV-103 trial (also known as KEYNOTE-869) of PADCEVTM (enfortumab vedotin-ejfv) in combination with anti-PD-1 therapy pembrolizumab for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy in the first-line setting (Press release, Seattle Genetics, APR 2, 2020, View Source [SID1234556108]). Based on recent discussions with the U.S. Food and Drug Administration (FDA), data from the randomized cohort K, along with other data from the EV-103 trial evaluating PADCEV combined with pembrolizumab as first-line therapy for cisplatin-ineligible patients, could potentially support registration under accelerated approval regulations in the United States. PADCEV is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.1

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"We are excited that EV-103 provides PADCEV with a potential pathway for U.S. accelerated approval in first-line metastatic urothelial cancer," said Roger Dansey, M.D., Chief Medical Officer at Seattle Genetics. "Our initial data on the combination of PADCEV and pembrolizumab in previously untreated patients who could not receive cisplatin are encouraging."

EV-103 is a multi-cohort, open-label, multicenter phase 1b/2 trial of PADCEV alone or in combination, evaluating safety, tolerability and efficacy in muscle invasive urothelial cancer, and in locally advanced or metastatic urothelial cancer in first- or second-line settings. Cohort K from EV-103 is intended to enroll 150 patients randomized 1:1 to PADCEV monotherapy or PADCEV in combination with pembrolizumab in locally advanced or metastatic urothelial cancer patients who are ineligible for cisplatin-based chemotherapy. The primary outcome measure is objective response rate (ORR) per blinded independent central review (BICR) using RECIST 1.1 and duration of response (DoR).

In addition to EV-103, the recently initiated EV-302 phase 3 randomized clinical trial is intended to support global registrations and potentially serve as a confirmatory trial if accelerated approval is granted based on EV-103. The EV-302 trial is evaluating the combination of PADCEV and pembrolizumab with or without chemotherapy versus chemotherapy alone in patients with previously untreated locally advanced or metastatic urothelial cancer. Importantly, EV-302 includes metastatic urothelial cancer patients that are either eligible or ineligible for cisplatin-based chemotherapy. The trial is expected to enroll 1,095 patients and has dual primary endpoints of progression-free survival and overall survival. Both the EV-103 and EV-302 trials are being conducted in collaboration with Astellas and Merck.

FDA recently granted Breakthrough Therapy designation for PADCEV in combination with pembrolizumab for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy in the first-line setting based on initial results from the EV-103 trial.

PADCEV (enfortumab vedotin-ejfv) was approved by the FDA in December 2019 and is indicated for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a platinum-containing chemotherapy before (neoadjuvant) or after (adjuvant) surgery or in a locally advanced or metastatic setting. PADCEV was approved under the FDA’s Accelerated Approval Program based on tumor response rate. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.2

About Bladder and Urothelial Cancer

It is estimated that approximately 81,000 people in the U.S. will be diagnosed with bladder cancer in 2020.3 Urothelial cancer accounts for 90 percent of all bladder cancers and can also be found in the renal pelvis, ureter and urethra.4 Globally, approximately 549,000 people were diagnosed with bladder cancer in 2018, and there were approximately 200,000 deaths worldwide.5

About PADCEV

PADCEV is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.6,7 Nonclinical data suggest the anticancer activity of PADCEV is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis).8 PADCEV is co-developed by Astellas and Seattle Genetics.

Important Safety Information

Warnings and Precautions

Hyperglycemia occurred in patients treated with PADCEV, including death and diabetic ketoacidosis (DKA), in those with and without pre-existing diabetes mellitus. The incidence of Grade 3-4 hyperglycemia increased consistently in patients with higher body mass index and in patients with higher baseline A1C. In one clinical trial, 8% of patients developed Grade 3-4 hyperglycemia. Patients with baseline hemoglobin A1C ≥8% were excluded. Closely monitor blood glucose levels in patients with, or at risk for, diabetes mellitus or hyperglycemia. If blood glucose is elevated (>250 mg/dL), withhold PADCEV.
Peripheral neuropathy (PN),predominantly sensory, occurred in 49% of the 310 patients treated with PADCEV in clinical trials; 2% experienced Grade 3 reactions. In one clinical trial, peripheral neuropathy occurred in patients treated with PADCEV with or without preexisting peripheral neuropathy. The median time to onset of Grade ≥2 was 3.8 months (range: 0.6 to 9.2). Neuropathy led to treatment discontinuation in 6% of patients. At the time of their last evaluation, 19% had complete resolution, and 26% had partial improvement. Monitor patients for symptoms of new or worsening peripheral neuropathy and consider dose interruption or dose reduction of PADCEV when peripheral neuropathy occurs. Permanently discontinue PADCEV in patients that develop Grade ≥3 peripheral neuropathy.
Ocular disorders occurred in 46% of the 310 patients treated with PADCEV. The majority of these events involved the cornea and included keratitis, blurred vision, limbal stem cell deficiency and other events associated with dry eyes. Dry eye symptoms occurred in 36% of patients, and blurred vision occurred in 14% of patients, during treatment with PADCEV. The median time to onset to symptomatic ocular disorder was 1.9 months (range: 0.3 to 6.2). Monitor patients for ocular disorders. Consider artificial tears for prophylaxis of dry eyes and ophthalmologic evaluation if ocular symptoms occur or do not resolve. Consider treatment with ophthalmic topical steroids, if indicated after an ophthalmic exam. Consider dose interruption or dose reduction of PADCEV for symptomatic ocular disorders.
Skin reactions occurred in 54% of the 310 patients treated with PADCEV in clinical trials. Twenty-six percent (26%) of patients had maculopapular rash and 30% had pruritus. Grade 3-4 skin reactions occurred in 10% of patients and included symmetrical drug-related intertriginous and flexural exanthema (SDRIFE), bullous dermatitis, exfoliative dermatitis, and palmar-plantar erythrodysesthesia. In one clinical trial, the median time to onset of severe skin reactions was 0.8 months (range: 0.2 to 5.3). Of the patients who experienced rash, 65% had complete resolution and 22% had partial improvement. Monitor patients for skin reactions. Consider appropriate treatment, such as topical corticosteroids and antihistamines for skin reactions, as clinically indicated. For severe (Grade 3) skin reactions, withhold PADCEV until improvement or resolution and administer appropriate medical treatment. Permanently discontinue PADCEV in patients that develop Grade 4 or recurrent Grade 3 skin reactions.
Infusion site extravasation Skin and soft tissue reactions secondary to extravasation have been observed after administration of PADCEV. Of the 310 patients, 1.3% of patients experienced skin and soft tissue reactions. Reactions may be delayed. Erythema, swelling, increased temperature, and pain worsened until 2-7 days after extravasation and resolved within 1-4 weeks of peak. One percent (1%) of patients developed extravasation reactions with secondary cellulitis, bullae, or exfoliation. Ensure adequate venous access prior to starting PADCEV and monitor for possible extravasation during administration. If extravasation occurs, stop the infusion and monitor for adverse reactions.

Embryo-fetal toxicity PADCEV can cause fetal harm when administered to a pregnant woman. Advise patients of the potential risk to the fetus. Advise female patients of reproductive potential to use effective contraception during PADCEV treatment and for 2 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with PADCEV and for 4 months after the last dose.
Adverse Reactions

Serious adverse reactions occurred in 46% of patients treated with PADCEV. The most common serious adverse reactions (≥3%) were urinary tract infection (6%), cellulitis (5%), febrile neutropenia (4%), diarrhea (4%), sepsis (3%), acute kidney injury (3%), dyspnea (3%), and rash (3%). Fatal adverse reactions occurred in 3.2% of patients, including acute respiratory failure, aspiration pneumonia, cardiac disorder, and sepsis (each 0.8%).

Adverse reactions leading to discontinuation occurred in 16% of patients; the most common adverse reaction leading to discontinuation was peripheral neuropathy (6%). Adverse reactions leading to dose interruption occurred in 64% of patients; the most common adverse reactions leading to dose interruption were peripheral neuropathy (18%), rash (9%) and fatigue (6%). Adverse reactions leading to dose reduction occurred in 34% of patients; the most common adverse reactions leading to dose reduction were peripheral neuropathy (12%), rash (6%) and fatigue (4%).

The most common adverse reactions (≥20%) were fatigue (56%), peripheral neuropathy (56%), decreased appetite (52%), rash (52%), alopecia (50%), nausea (45%), dysgeusia (42%), diarrhea (42%), dry eye (40%), pruritus (26%) and dry skin (26%). The most common Grade ≥3 adverse reactions (≥5%) were rash (13%), diarrhea (6%) and fatigue (6%).

Lab Abnormalities

In one clinical trial, Grade 3-4 laboratory abnormalities reported in ≥5% were: lymphocytes decreased, hemoglobin decreased, phosphate decreased, lipase increased, sodium decreased, glucose increased, urate increased, neutrophils decreased.

Drug Interactions

Effects of other drugs on PADCEV: Concomitant use with a strong CYP3A4 inhibitor may increase free MMAE exposure, which may increase the incidence or severity of PADCEV toxicities. Closely monitor patients for signs of toxicity when PADCEV is given concomitantly with strong CYP3A4 inhibitors.
Specific Populations

Lactation: Advise lactating women not to breastfeed during treatment with PADCEV and for at least 3 weeks after the last dose.
Hepatic impairment: Avoid the use of PADCEV in patients with moderate or severe hepatic impairment.