On December 20, 2021 EMD Serono, the Healthcare business sector of Merck KGaA, Darmstadt, Germany, in the U.S. and Canada, reported a strategically focused expansion of its neurology pipeline with the acquisition of the rights to develop cladribine for the treatment of generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD) (Press release, EMD Serono, DEC 20, 2021, View Source [SID1234597486]). The company entered into an agreement to secure the global rights by acquiring Chord Therapeutics, a Swiss-based biotech company focused on rare neuroinflammatory diseases.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Cladribine has a unique mechanism of action which is relevant in antibody-mediated disease such as NMOSD and gMG. In exploratory studies, cladribine demonstrated promising results in these diseases. These data have prompted us to initiate further development with cladribine in gMG and NMOSD to potentially bring a new therapeutic option to patients and expand our portfolio in this area," said Danny Bar-Zohar, MD, Head of Global Development for the biopharma business of Merck KGaA, Darmstadt, Germany.

Cladribine is a well characterized molecule that has been extensively studied across MS and oncologic disorders and has shown promising results in exploratory studies in gMG and NMOSD. EMD Serono plans to leverage its existing capabilities to further develop an oral cladribine product tailored specifically for these indications. The transaction is expected to be closed in early 2022 after satisfactory completion of customary closing conditions.

Generalized myasthenia gravis is a disease in which patients experience weakness in ocular, limb, and respiratory muscles and that has a significant impact on Quality of Life. It is estimated that 700,000 people around the world are living with gMG. NMOSD, also known as Devic´s disease, is an autoimmune disease, often misdiagnosed as MS due to similar symptoms. NMOSD is estimated to impact 200,000 people around the world.

On December 20, 2021 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company utilizing proprietary genetic engineering platform technologies to create cell and gene therapeutics with the capacity to cure, reported that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for P-MUC1C-ALLO1, the Company’s allogeneic CAR-T product candidate targeting multiple solid tumor indications (Press release, Poseida Therapeutics, DEC 20, 2021, View Source [SID1234597484]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Poseida announces FDA clearance of IND for P-MUC1C-ALLO1, a fully #allogeneic CAR-T targeting multiple solid tumors.
"We are excited to begin the P-MUC1C-ALLO1 trial, an evaluation of a fully allogeneic CAR-T product candidate with the potential to treat a wide range of solid tumors, including breast, ovarian and other cancers," said Eric Ostertag, M.D., Ph.D., Chief Executive Officer of Poseida Therapeutics. "The genetic edits in P-MUC1C-ALLO1 have been shown to reduce or fully eliminate alloreactivity, and our proprietary manufacturing process, which includes our booster molecule, has the potential to treat many patients from a single manufacturing run. We look forward to beginning this trial and to presenting initial clinical data in 2022."

P-MUC1C-ALLO1 is the Company’s second fully allogeneic CAR-T product candidate to receive IND clearance in 2021 following P-BCMA-ALLO1 for the treatment of relapsed/refractory multiple myeloma. P-MUC1C-ALLO1 is the first product candidate to be produced out of the Company’s new GMP facility, an internal pilot manufacturing plant located at its San Diego headquarters.

P-MUC1C-ALLO1 will be evaluated in a Phase 1 multi-center, open-label, dose escalation study in adults with locally advanced or metastatic epithelial-derived solid tumors refractory to standard of care therapy, or those deemed ineligible or refused another existing treatment option. The study will evaluate the safety, tolerability, and preliminary efficacy of P-MUC1C-ALLO1 and will follow a 3+3 design of dose-escalating cohorts. After a subject enrolls, P-MUC1C-ALLO1 allogeneic CAR-T cells will be administered, following a standard chemotherapy-based conditioning regimen. The study protocol allows for exploration of additional dosing regimens, including re-dosing once initial safety has been established.

About P-MUC1C-ALLO1
P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate in preclinical development for multiple solid tumor indications. P-MUC1C-ALLO1 has the potential to treat a wide range of solid tumors derived from epithelial cells, such as breast, colorectal, lung, ovarian, pancreatic and renal cancers, as well as other cancers expressing a cancer-specific form of the Mucin 1 protein, or MUC1C. P-MUC1C-ALLO1 is designed to be fully allogeneic, with genetic edits to eliminate or reduce both host-vs-graft and graft-vs-host alloreactivity. Poseida has demonstrated the elimination of tumor cells to undetectable levels in preclinical models of both triple-negative breast and ovarian cancer.

On December 20, 2021 Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing next-generation therapies that target genetic drivers of cancer, reported regulatory updates for its lead drug candidate, repotrectinib, a ROS1/TRK Inhibitor, and for elzovantinib (TPX-0022), its drug candidate targeting MET, CSF1R and SRC (Press release, Turning Point Therapeutics, DEC 20, 2021, View Source [SID1234597483]). These updates follow FDA meetings held earlier in December .

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

REPOTRECTINIB, ROS1/TRK INHIBITOR

Based on the breakthrough therapy designation granted by the FDA earlier this quarter, the company participated in a Type B meeting with the FDA to discuss potential next steps for repotrectinib in NTRK-positive TKI-pretreated advanced solid tumor patients treated within expansion cohort 6 (EXP-6) of the registrational TRIDENT-1 study.

The FDA guided that a pre-NDA meeting should be requested to discuss the topline blinded independent central review (BICR) results from the Phase 2 TKI-pretreated EXP-6 and TKI-naïve EXP-5 patients, when responders have been followed for at least six months past onset of response. The FDA also acknowledged the company’s plan to submit to the pre-NDA meeting, BICR data from 40 patients from EXP-6 and BICR data from patients enrolled in EXP-5 cohort at that time (estimated at approximately 40 patients of the targeted 55 patients). The FDA noted that data from EXP-5 may be used to support the efficacy data for EXP-6, or potentially could be pooled with data from EXP-6 to support a broader indication. The FDA stated that whether the data from EXP-6 and EXP-5 are deemed potentially adequate to either support a broader indication or an indication in patients who have received a prior TKI across a wide range of tumor types will be determined at the time of the submission of the pre-NDA meeting package, and ultimately during the review of the NDA submission.

The company plans to provide guidance on the timing of the pre-NDA meeting for repotrectinib in patients with NTRK-positive advanced solid tumors after completion of enrollment of the targeted 40 EXP-6 patients is achieved.

ELZOVANTINIB (TPX-0022), MET/CSF1R/SRC INHIBITOR

The company participated in a Type B meeting with the FDA Division of Oncology 3 (DO3, the division responsible for oversight of gastric cancer therapeutic area). The purpose of the meeting was to discuss the proposed approach to identifying the recommended phase 2 dose (RP2D) and the planned Phase 2 portion of the SHIELD-1 study focused on the potential next steps for elzovantinib in patients with MET amplified gastric/GEJ cancer.

The FDA DO3 agreed with the approach for RP2D identification previously discussed with FDA Division of Oncology 2 (DO2, the division responsible for oversight of lung cancer therapeutic area).

The FDA also agreed with the company’s plan to proceed to the potentially registrational Phase 2 MET amplified gastric/GEJ cancer expansion cohorts of SHIELD-1 after RP2D determination. Based on guidance from the FDA, the company plans to submit data to the FDA from the Phase 2 to discuss the potential registrational aspect of the study. Currently, the company continues to enroll patients within Phase 1 dose expansion using 40 mg QD to 40 mg BID dosing and in Phase 1 dose escalation at the last anticipated dose level of 60 mg QD to 60 mg BID.

The company anticipates initiating the planned Phase 2 portion of SHIELD-1 in 2022.

"We are pleased with the feedback received from the FDA at our recent Type B meetings for repotrectinib and elzovantinib, and look forward to further advancing these important programs," said Mohammad Hirmand, M.D., Chief Medical Officer.

On December 20, 2021 KemPharm, Inc. (NasdaqGS: KMPH), a specialty pharmaceutical company focused on the discovery and development of proprietary prodrugs, reported its Board of Directors has authorized a program to repurchase up to $50 million of the Company’s outstanding stock (Press release, KemPharm, DEC 20, 2021, View Source [SID1234597481]). The share repurchase authorization is effective immediately and valid through December 31, 2023. This program is equivalent to approximately 18 percent of KemPharm’s current market capitalization.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The Board’s decision to establish this share repurchase program reflects the positive momentum underway across all elements of our business, including our outlook for the ongoing commercialization of AZSTARYS, our strong balance sheet, and the expectations we have for 2022 and beyond, coupled with the strong belief that our shares are currently undervalued," said Travis Mickle, Ph.D., President and Chief Executive Officer of KemPharm. "We will continue our efforts to create value for our shareholders by continuing to prioritize capital allocation initiatives that support our growth strategies, including the advancement of our serdexmethylphenidate (SDX) pipeline and the exploration of other opportunities to expand our product pipeline."

The shares may be repurchased from time to time in open market transactions, through privately negotiated transactions or by other means in accordance with federal securities laws. The Company intends to fund repurchases from available working capital and cash provided by operating activities. The timing, as well as the number and value of shares repurchased under the program, will be determined by the Company at its discretion and will depend on a variety of factors, including the market price of the Company’s common stock, general market and economic conditions and applicable legal requirements. The exact number of shares to be repurchased by the Company is not guaranteed and the program may be suspended, modified, or discontinued at any time without prior notice. Any repurchases will be made in compliance with the SEC’s Rule 10b-18.

About AZSTARYS:

AZSTARYS is an FDA-approved, once-daily product for the treatment of attention deficit hyperactivity disorder (ADHD) in patients age six years or older. AZSTARYS consists of SDX, KemPharm’s prodrug of d-methylphenidate (d-MPH), co-formulated with immediate release d-MPH. Corium, Inc., a portfolio company of Gurnet Point Capital, is leading all commercialization efforts for AZSTARYS in the U.S.

On December 20, 2021 Cytokinetics, Incorporated (Nasdaq: CYTK) and Ji Xing Pharmaceuticals Limited (JI XING), a biopharmaceutical company based in Shanghai and backed by RTW Investments, LP (RTW), reported an expansion of their collaboration by entering into an exclusive license and collaboration agreement to develop and commercialize omecamtiv mecarbil for the proposed treatment of heart failure with reduced ejection fraction (HFrEF) in Greater China (Press release, Cytokinetics, DEC 20, 2021, View Source [SID1234597480]). In addition to the license and collaboration agreement with JI XING, Cytokinetics has also entered into Common Stock Purchase Agreements that provide for the sale and issuance to entities affiliated with RTW of 511,182 of shares of Cytokinetics common stock at a price per share of $39.125.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Pursuant to these transactions, Cytokinetics will receive committed capital of $70 million, comprised of $50 million from JI XING of upfront and near-term payments under the collaboration agreement and $20 million from RTW as proceeds for the sale of common stock. In addition, Cytokinetics will be eligible to receive up to $330 million from JI XING in additional milestone payments plus tiered royalties on the net sales of omecamtiv mecarbil in Greater China, subject to certain reductions.

"We are pleased to expand our current relationship with JI XING to now include omecamtiv mecarbil, and believe that their expertise in drug development and commercialization in China can help bring omecamtiv mecarbil to substantially more patients in an important cardiovascular market," said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. "JI XING shares our strategic vision of building a business franchise by leveraging investments in science, people and infrastructure as can benefit the potential commercialization of both omecamtiv mecarbil as well as aficamten which was the subject of our initial collaboration. Together, we believe more patients in China may benefit from both drug candidates."

"We have been impressed by our productive partnership to date focused on aficamten and are pleased to expand our collaboration to now include omecamtiv mecarbil," said Joseph Romanelli, CEO of JI XING. "Despite the advancement of heart failure treatments in China, patients continue to need novel therapies to reduce the risk of disease. With the addition of omecamtiv mecarbil to our growing cardiovascular pipeline, we have the opportunity to potentially bring a much needed, novel heart failure medicine to patients in Greater China."

"The expansion of this collaboration further brings together the expertise from both companies to support the global commercialization of omecamtiv mecarbil and represents a tremendous opportunity to create a larger footprint to impact more people with heart failure," said Roderick Wong, M.D., Chairman of JI XING and Managing Partner, RTW Investments, LP. "The two companies have demonstrated how well they work together, and we are pleased to lend further support in the interests of better cardiovascular health."

Conference Call and Webcast Information

Members of Cytokinetics’ senior management team will host a conference call and webcast today, December 20, at 8:30 AM Eastern Time. The webcast can be accessed through the Investors & Media section of the Cytokinetics website at www.cytokinetics.com. The live audio of the conference call can also be accessed by telephone by dialing either (866) 999-CYTK (2985) (United States and Canada) or (706) 679-3078 (international) and typing in the passcode 8997061.

An archived replay of the webcast will be available via Cytokinetics’ website until January 3, 2022. The replay will also be available via telephone by dialing (855) 859-2056 (United States and Canada) or (404) 537-3406 (international) and typing in the passcode 8997061 from December 20, 2021 at 11:30 AM Eastern Time until January 3, 2022.

About Omecamtiv Mecarbil

Omecamtiv mecarbil is an investigational, selective, small molecule cardiac myosin activator, the first of a novel class of myotropes1 designed to directly target the contractile mechanisms of the heart, binding to and recruiting more cardiac myosin heads to interact with actin during systole. Omecamtiv mecarbil is designed to increase the number of active actin-myosin cross bridges during each cardiac cycle and consequently augment the impaired contractility that is associated with heart failure with reduced ejection fraction (HFrEF). Preclinical research has shown that omecamtiv mecarbil increases cardiac contractility without increasing intracellular myocyte calcium concentrations or myocardial oxygen consumption.2-4

The development program for omecamtiv mecarbil is assessing its potential for the treatment of HFrEF and includes GALACTIC-HF and METEORIC-HF, a Phase 3 clinical trial designed to evaluate the effect of treatment with omecamtiv mecarbil compared to placebo on exercise capacity.

About Heart Failure

Heart failure is a grievous condition that affects more than 64 million people worldwide5 (and well over 10 million in China6) about half of whom have reduced left ventricular function.7,8 It is the leading cause of hospitalization and readmission in people age 65 and older.9, 10 Despite broad use of standard treatments and advances in care, the prognosis for patients with heart failure is poor.11 An estimated one in five people over the age of 40 are at risk of developing heart failure, and approximately 50 percent of people diagnosed with heart failure die within five years of initial hospitalization.12,13 More than 2 million people in the U.S. are estimated to have an ejection fraction <30%, indicating they may have severe heart failure.14