On January 10, 2022 Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, reported that Michael Gilman, Ph.D., Chief Executive Officer, will present a corporate overview at the 40th Annual J.P. Morgan Healthcare Conference on Thursday, January 13, 2022 at 8:30 a.m. ET (Press release, Arrakis Therapeutics, JAN 10, 2022, View Source [SID1234598527]).

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On January 10, 2022 Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) ("Takeda") reported the exercise of its option to acquire Adaptate Biotherapeutics ("Adaptate"), a UK company focused on developing antibody-based therapeutics for the modulation of variable delta 1 (Vδ1) gamma delta (γδ) T cells (Press release, Takeda, JAN 10, 2022, View Source [SID1234598526]). Through the acquisition, Takeda will obtain Adaptate’s antibody-based γδ T cell engager platform, including pre-clinical candidate and discovery pipeline programs. Adaptate’s γδ T cell engagers are designed to specifically modulate γδ T cell-mediated immune responses at tumor sites while sparing damage to healthy cells.

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The planned acquisition of Adaptate follows Takeda’s recently exercised option to acquire GammaDelta Therapeutics ("GammaDelta") and is intended to further accelerate the development of innovative γδ T cell-based therapies. Combining GammaDelta’s cell therapy-based platform and Adaptate’s antibody-based γδ T cell engager platform with Takeda’s strong research and development organization positions Takeda to be at the leading edge in deploying the full potential of γδ T cells in the fight against cancer. The planned acquisition complements Takeda’s ongoing efforts to research and develop cell engagers for solid tumor applications, bolstered by the novel T cell engager COBRA platform, which was acquired from Maverick Therapeutics in another successful build-to-buy collaboration.

"Partnering with early-stage innovators to access cutting-edge platforms in the fight against cancer is at the center of our R&D strategy," said Christopher Arendt, Ph.D., Head of Oncology Cell Therapy and Therapeutic Area Unit of Takeda. "Adaptate’s γδ T cell engager platform and the team’s deep understanding of γδ T cell biology gives us an opportunity to develop a new class of therapeutics that tap into powerful innate immune mechanisms. The planned acquisition will strengthen our immuno-oncology R&D efforts as part of our ongoing pursuit of life-transforming medicines for patients with cancer."

Adaptate was formed in 2019 as a spin-out company from GammaDelta with investment from Abingworth LLP and Takeda, in which Takeda received an exclusive right to purchase Adaptate for a pre-negotiated upfront payment. The acquisitions of Adaptate and GammaDelta are expected to be finalized in Q1 of Takeda’s fiscal year 2022, pending completion of review under applicable antitrust laws, including the Hart-Scott-Rodino (HSR) Antitrust Improvements Act of 1976 in the U.S.

"Our acquisition by Takeda recognizes the tremendous work put in over the last two years by Adaptate’s incredibly talented team," said Dr. Natalie Mount, CEO of Adaptate. "We have rapidly demonstrated, in preclinical models, the therapeutic potential of our novel Vδ1-targeting antibodies, and this move brings us an exciting step closer to realizing the full potential for Vδ1 T cell targeted therapies to improve treatment outcomes for cancer patients."

In addition, Tim Haines, Chair & Managing Partner at Abingworth noted, "Having played an instrumental role in creating Adaptate, we are delighted to see the impressive developments of its γδ T cell therapeutic antibody portfolio to date, under the leadership of Natalie Mount. We look forward to seeing Takeda progress Adaptate’s very promising therapeutic antibodies into the clinic."

Takeda’s oncology pipeline focuses on novel strategies that leverage the power of the immune system, with a focus on innate immunity. Innate immune responses serve as the body’s first defense mechanism against disease and involve the orchestration of a broad arsenal of mechanisms and cell types, including γδ T cells and natural killer (NK) cells, that may help to overcome cancer’s ability to evade immune recognition. Adaptate has discovered a unique set of antibodies that selectively modulate γδ T cell activity in the tumor microenvironment. The antibodies provide a precisely targeted signal to the immune system, thereby offering the opportunity for superior efficacy and safety compared to conventional immuno-oncology approaches in solid tumors.

Takeda’s Commitment to Oncology

Our core R&D mission is to deliver novel medicines to patients with cancer worldwide through our commitment to science, breakthrough innovation and passion for improving the lives of patients. Whether it’s with our hematology therapies, our robust pipeline, or solid tumor medicines, we aim to stay both innovative and competitive to bring patients the treatments they need. For more information, visit www.takedaoncology.com

On January 10, 2022 Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported that recent corporate updates and highlighted upcoming priorities for its pipeline programs in 2022 (Press release, Scholar Rock, JAN 10, 2022, View Source [SID1234598525]).

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"2021 was another transformative year for Scholar Rock, with positive data from both of our clinical programs, including from the TOPAZ Phase 2 trial for apitegromab, which is being developed for the improvement of motor function in patients with SMA; and Part A of the DRAGON Phase 1 proof-of-concept trial for SRK-181, being developed to overcome resistance to check point inhibitor therapy in cancer patients," said Nagesh Mahanthappa, Ph.D., Interim CEO of Scholar Rock. "In 2022, we are thrilled to be advancing a pivotal Phase 3 trial of apitegromab and advancing our SRK-181 program to test our hypothesis that this highly selective and potent molecule can overcome resistance to checkpoint inhibitors thereby increasing the number of patients who may benefit from cancer immunotherapy. In addition, the preclinical pipeline has received a major boost as we have regained rights to assets discovered and developed during our research partnership with Gilead that have novel pharmacological profiles relevant to TGFβ-mediated diseases."

2022 Priorities:

Apitegromab is a selective inhibitor of myostatin activation being developed as the potential first muscle-directed therapy for the treatment of spinal muscular atrophy (SMA).

Robust Enrollment of the Phase 3 SAPPHIRE Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 Patients. Scholar Rock has initiated the SAPPHIRE study. The study design plans for approximately 156 patients aged 2-12 years old with non-ambulatory Type 2/3 SMA to be enrolled in the main efficacy population. Patients will be randomized 1:1:1 to receive for 12 months either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks added on top of background SMN treatment.
Progress TOPAZ Long-Term Extension to Two Year Readout. As of January 6, 55 of 57 patients remain in the long-term extension trial of apitegromab in Type 2 and 3 SMA.
Advance Development Activities to Include Patients with Type 1 and Ambulatory SMA.
SRK-181 is a potent and highly selective inhibitor of latent TGFβ1 activation being developed with the aim of overcoming primary resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.

Advance Progress in Part B of DRAGON Phase 1 Proof-of-Concept Trial. Based on the safety and pharmacokinetic data from Part A of the DRAGON Phase 1 trial, Scholar Rock has initiated the Part B dose expansion portion of the trial, which is evaluating SRK-181 dosed 1500 mg every three weeks (Q3W) in patients receiving an approved anti-PD-(L)1 therapy dosed Q3W and 1000 mg every two weeks (Q2W) in patients receiving an approved anti-PD-(L)1 therapy dosed Q2W. Part B will enroll and dose patients in multiple proof of concept cohorts conducted in parallel, including;
Urothelial carcinoma (UC),
Cutaneous melanoma (MEL),
Non-small cell lung cancer (NSCLC),
Clear cell renal cell carcinoma (ccRCC),
Other solid tumors.
Each cohort is expected to enroll up to 40 patients with various locally advanced or metastatic solid tumors who have demonstrated primary resistance to anti-PD-(L)1 therapy. Early efficacy and safety data are anticipated in 2022.

Advancing assets gained from the Gilead collaboration. In December 2018, Gilead Sciences and Scholar Rock entered into a three-year collaboration to discover and develop therapeutics that target TGFβ-driven signaling, a central regulator of fibrosis. Under the collaboration, Gilead had exclusive options to license worldwide rights to antibodies from certain TGFβ programs being developed by Scholar Rock. Scholar Rock received $80.0 million in proceeds upon signing the agreement and an additional $25.0 million preclinical milestone was achieved in December 2019 for the successful demonstration of efficacy in preclinical in vivo proof-of-concept studies. As of December 19, 2021 the collaboration period has concluded and on January 6, 2022, Gilead agreed that its option exercise period for all programs has been terminated.

Scholar Rock regains rights to a suite of antibodies with novel pharmacological profiles that were discovered over the course of the collaboration.
Of particular note, Scholar Rock has discovered antibodies that selectively inhibit the activation of latent TGFβ1 in the context of fibrotic extracellular matrix and that avoid perturbing TGFβ1 presented by cells of immune system. Such antibodies demonstrated significant antifibrotic activity in a variety of preclinical rodent models and safety at all doses tested in a non-GLP mouse safety study that we intend to publish in 2022.
"The novel anti-fibrotic antibodies discovered during this collaboration demonstrate the unique capabilities of the discovery platform we have built at Scholar Rock," said Gregory Carven, CSO of Scholar Rock. "We are excited to continue the advancement of these assets as a part of the company’s growing preclinical pipeline."

"We made great progress across our portfolio in 2021 and we’re carrying that momentum into 2022," said Ted Myles, CFO and Head of Business Operations of Scholar Rock. "We recently strengthened our balance sheet through the use of our ATM and taking the $25 million second tranche of our debt facility with Silicon Valley Bank and Oxford Finance so that we have greater flexibility to continue to advance our clinical and pre-clinical programs. We have high conviction in our platform based on the exciting clinical data to date and we believe this puts us in a unique position as we advance our programs to serve patients’ needs."

On January 10, 2022 Humanigen, Inc. (Nasdaq:HGEN) ("Humanigen"), a clinical-stage biopharmaceutical company focused on preventing and treating an immune hyper-response called ‘cytokine storm’ reported the next steps for the development of its lead candidate, lenzilumab, in the prevention of CAR-T therapy related toxicities including ICANS and CRS in patients with relapsed or refractory Non-Hodgkin lymphoma (rrNHL) (Press release, Humanigen, JAN 10, 2022, View Source [SID1234598524]).

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"Following recent constructive interaction with FDA on the design of the Phase 3 study, known as SHIELD (Study on How to Improve Efficacy and toxicity with Lenzilumab in DLBCL and other NHL patients treated with CAR-T therapy), we believe that we have collaboratively aligned on a potential registration study, recognizing the clinical and economic benefits for patients and healthcare systems," stated Adrian Kilcoyne, chief medical officer of Humanigen. "The SHIELD trial will build on the positive results from the ZUMA-19 study. The primary endpoint of SHIELD will focus on demonstrating a significant improvement in neurotoxicity associated with both YESCARTA and TECARTUS. In agreement with FDA, we will also seek to demonstrate the beneficial impact that lenzilumab may have on healthcare resource utilization."

CAR-T therapies have resulted in significant advances for patients but the clinical benefit observed often comes at a cost. In up to one-third of patients, significant toxicities of ICANS and CRS occur1. Currently, the widespread adoption of CAR-T therapy is limited, in part, by the requirement for treatment in centers that are experienced in managing the common toxicities of ICANS and CRS and by the financial and health burden that this creates.

"CRS and ICANS remain huge challenges for physicians and patients treated with CAR-T therapy," said Dr. Saad Kendarian, Consultant Hematologist at Mayo Clinic and the primary investigator for the SHIELD study. "They result in additional morbidity for patients, as well as significantly increased costs for healthcare providers. Treatments that can prevent ICANS and CRS could address a critical unmet need."

The SHIELD study will begin recruitment in H1 2022, with preliminary data potentially being shared at the American Society of Hematology (ASH) (Free ASH Whitepaper) congress in December 2022.

"We are very encouraged with the outcome of our meeting with FDA," said Dr. Cameron Durrant, Chairman and CEO of Humanigen. "Defining a potential registration pathway across multiple indications is an important next step in our development of lenzilumab. The PREACH-M study in chronic myelomonocytic leukemia has begun dosing patients. Both the SHIELD study in CAR-T and the RATinG study in acute graft vs. host disease are planned to begin enrolling in the first half of 2022. The NIH-sponsored ACTIV-5/BET-B study has reached target recruitment and there are additional COVID studies being initiated in Korea and Australia. Further strengthening the Humanigen pipeline is our Phase 1 program focused on ifabotuzumab in solid tumors."

Lenzilumab is an investigational product and is not authorized or approved in any country.

On January 10, 2022 Iksuda Therapeutics (Iksuda), the developer of a new generation of antibody drug conjugates (ADCs) with raised therapeutic index, reported further expansion of its differentiated ADC pipeline through a license and commercialisation agreement with LegoChem Biosciences (LCB), a Korean biopharmaceutical company focused on the development of next-generation novel therapeutics utilising its proprietary medicinal drug discovery technologies (Press release, Iksuda Therapeutics, JAN 10, 2022, View Source [SID1234598523]).

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The agreement provides Iksuda with exclusive world-wide rights (excluding Greater China and South Korea) to LCB’s Her2 ADC programme, LCB14. The agreement includes development, regulatory and commercial milestone payments to LCB contingent on successful achievement of certain milestones as well as royalties on commercial sales.

Iksuda anticipates that LCB14, now designated IKS014, will enter a phase 1 clinical trial programme in the US during Q3 2022.

LCB14 is under license from LCB to Fosun Pharma for Greater China where it is designated FS-1502. Fosun is currently conducting a phase 1 clinical trial in China in patients with breast cancer.

This agreement with LCB allows Iksuda to further expand its clinical development pipeline. The Company’s IKS03 programme, a best-in-class CD19-targeting ADC, is also expected to enter phase 1 clinical trials in H1 2022.

Iksuda is also rapidly expanding its pre-clinical pipeline. These assets centre on delivering ADCs with raised therapeutic index through both improved safety and efficacy, conferred by tumour activated, prodrug payloads in combination with stable conjugation technologies, including its proprietary novel PermaLink platform.

Iksuda announced the completion of its Series A financing round in June 2021, co-led by Celltrion Inc and Mirae Asset Capital, further recognising the Company’s expertise in ADCs and underpinning its commitment to targeting the treatment of cancers with currently limited treatment options.1

Dr Dave Simpson, Chief Executive Officer, Iksuda Therapeutics, said:
"This agreement further demonstrates Iksuda’s commitment to the development of differentiated ADCs that will potentially bring benefit to patients living with cancer. Importantly, it is also further recognition of the unmatched expertise of our team.

"We continue to focus on delivering our strategy of driving potentially valuable therapies through the clinic for cancer indications with high burden and for where there are limited treatment options. Our wholly-owned pipeline is supplemented with in-licensed assets where we been able leverage our expertise to recognise promising opportunities from others to partner and then develop further, utilising our armoury of [proprietary] payload and conjugation platforms where necessary."

Dr. Young-Lag Cho, Chief Development Officer and VP of LegoChem Biosciences, said:
"Iksuda is the best partner for the expedited development of HER2 ADC. Learning from Iksuda’s world class expertise and experience on ADC development by jointly conducting phase 1 trial in US will strengthen our internal development capabilities while accelerating advancement of our other ADC programs."

Yong-Zu Kim, CEO, LegoChem Biosciences, commented: "Recognising Iksuda’s depth of knowledge within the field of ADCs we are pleased to once again expand our relationship and our continued efforts to develop a broad pipeline of pre-clinical and clinical opportunities."

Press release: Iksuda Therapeutics closes $47 million financing round (07 June 2021)