On May 13, 2021 Kinnate Biopharma Inc. (Nasdaq: KNTE) ("Kinnate"), a biopharmaceutical company focused on the discovery and development of small molecule kinase inhibitors for difficult-to-treat, genomically defined cancers, announced the closing of a $35 million Series A financing for a joint venture in China (Press release, Kinnate Biopharma, MAY 13, 2021, View Source [SID1234580009]). Established with OrbiMed Asia Partners, OrbiMed Private Investments and Foresite Capital, the joint venture will be headquartered in Shanghai and enable the potential development and commercialization of certain Kinnate targeted oncology product candidates across Greater China (mainland China, Hong Kong, Taiwan, and Macau). Kinnate Biopharma will be the majority shareholder in the joint venture. The company has also announced that veteran biopharmaceutical industry executive Wenn Sun, Ph.D., has been appointed as the joint venture’s Executive Chair.

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"Establishing operations in China creates a tremendous opportunity for Kinnate to build its global footprint and further advance our mission of expanding access to innovative targeted therapies for people battling cancer," said Nima Farzan, President and CEO of Kinnate Biopharma Inc. "OrbiMed Private Investments and Foresite Capital have been important partners in the growth of Kinnate and we are pleased to now have the support of OrbiMed Asia Partners who led this financing and brings tremendous expertise and connections in China to this new joint venture. I look forward to working with this leading team of investors and Dr. Sun to make precision medicine a reality for more people in Greater China, which is one of the world’s largest healthcare markets."

The initial focus of the joint venture is on advancing the development of KIN-2787 for the Greater China market. KIN-2787 is a Rapidly Accelerated Fibrosarcoma (RAF) inhibitor candidate being developed for the treatment of patients with lung cancer, melanoma, and other solid tumors. The joint venture will also pursue development of KIN-3248 for the Chinese market. KIN-3248 is a Fibroblast Growth Factor Receptors (FGFR) inhibitor candidate for the treatment of patients with intrahepatic cholangiocarcinoma (ICC), a cancer of the bile ducts in the liver, and urothelial carcinoma (UC), a cancer of the bladder lining. The joint venture, which will be subsequently named, has an exclusive license to one other Kinnate program and may also obtain rights to develop certain other new product candidates in Greater China from the Kinnate pipeline, as well as other third-party product candidates in China and other geographies.

"Since its founding, Kinnate has demonstrated impressive scientific discipline and exceptional execution in building a robust pipeline of targeted therapies for hard-to-treat cancers," said Steven D. Wang, Ph.D., CFA, Partner and Senior Managing Director, OrbiMed Asia Partners. "We are pleased to join them in establishing this joint venture in China and look forward to working closely with Dr. Sun and our other regional colleagues in bringing these potentially life-saving therapies to more patients."

Dr. Sun is the Founder and President of Precision Medicine Asia (PREMIA), an oncology-focused clinical genomic data company she founded in 2018. Previously, she was the Founder and Managing Partner for OxOnc Development, a venture company that, along with Pfizer Oncology, co-developed XALKORI in patients with ROS1 genetic alterations in Asia, including China. Dr. Sun also served as Head of Strategic Alliances for GSK Oncology, and helped build its alliances with various clinical research networks around the world. In 2003, in collaboration with the National Comprehensive Cancer Network (NCCN), she helped introduce the NCCN Guidelines to China. Dr. Sun was appointed Chief Business Development Officer at the Lurie Cancer Center of Northwestern University after her post-doctoral fellowship at University of Wisconsin-Madison.

"I am honored to join the Kinnate team and lead the joint venture’s efforts to help the patients in China for whom no genomically-targeted therapies exist or for which a resistance to targeted treatments has evolved," said Dr. Sun. "KIN-2787 has already demonstrated very promising results in pre-clinical studies and presents a significant opportunity to help address the tremendous demand for more effective cancer therapies across Greater China."

On May 13, 2021 Savara Inc. (Nasdaq: SVRA), a clinical stage biopharmaceutical company focused on rare respiratory diseases, reported financial results for the first quarter ending March 31, 2021 and provided a business update (Press release, Savara, MAY 13, 2021, View Source [SID1234580041]).

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"We made considerable progress in the first quarter of the year," said Matt Pauls, Chair and Chief Executive Officer, Savara. "This includes the strategic decision to wind down two pipeline programs, enabling us to focus solely on advancing our lead asset, molgramostim in aPAP. Additionally, we started the onboarding process of a global, full-service contract research organization that is partnering with us to execute the IMPALA-2 trial. Lastly, we strengthened our cash position through a $130M equity raise. With a balance sheet of approximately $193M, we believe our runway extends through 2025 – well beyond the expected top line results of IMPALA-2 in 2Q 2024."

"We are pleased to announce that the IMPALA-2 site activation process has begun," said Badrul Chowdhury, Chief Medical Officer, Savara. "Sites will be activated on a rolling basis, with approximately 10-15 expected to be active by the end of June. As sites launch, patients are evaluated, and those who are eligible will begin a six-week screening period. This means patients could be randomized and dosed by the end of 2Q or early 3Q 2021. We also recently strengthened our management team with the hire of Dr. Dhaval Desai as Head of Clinical Development and Brian Maurer as Head of Clinical Operations. Both are seasoned professionals with deep expertise in running global clinical trials."

First Quarter Financial Results (Unaudited)

Savara’s net loss attributable to common stockholders for the three months ended March 31, 2021 was $10.2 million, or $(0.13) per share, compared with a net loss attributable to common stockholders of $15.4 million, or $(0.27) per share, for the three months ended March 31, 2020.

Research and development expenses decreased by $5.6 million, or 42.5%, to $7.6 million for the three months ended March 31, 2021 from $13.2 million for the three months ended March 31, 2020. The decrease is largely attributable to $5.4 million of costs for the acquisition of inhaled liposomal ciprofloxacin (Apulmiq) in March 2020. There were no research and development costs incurred related to Apulmiq during the three months ended March 31, 2021.

General and administrative expenses decreased by approximately $0.2 million, or 6.8%, to $2.8 million for the three months ended March 31, 2021 from $3.0 million for the three months ended March 31, 2020. This was primarily due to a decrease in noncash stock-based compensation and personnel costs for the three months ended March 31, 2021.

As of March 31, 2021, Savara had cash, cash equivalents, and short-term investments of approximately $192.7 million and debt of approximately $25 million.

Pulmatrix has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-K/A [Amend], Pulmatrix, 2021, MAY 14, 2021, View Source [SID1234580083]).

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On May 13, 2021 Certara, Inc. (Nasdaq: CERT), the global leader in biosimulation, reported the launch of new versions of its Immunogenicity (IG) and Immuno-oncology (IO) Quantitative Systems Pharmacology (QSP) Simulators to help address the challenges in the development of biologics and cancer Therapies (Press release, Certara, MAY 13, 2021, View Source [SID1234579906]). These new versions further expand applications of QSP and advance the field of using virtual patients to conduct computer-based trials throughout drug discovery and development.

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"We have seen an increasing interest in the potential of QSP to guide regulatory decisions and anticipate that this will only continue to grow, much like physiologically-based pharmacokinetic (PBPK) modeling, which is now a critical component in many regulatory submissions," said William F. Feehery, Ph.D., CEO of Certara.

QSP addresses the most complex challenges in drug development by combining computational modeling and experimental data to examine the relationships between a drug, biological system, and disease process. Immunogenicity is a key challenge for developing biologics, including novel modalities such as gene and cell therapies. Researchers use Certara’s IG Simulator to simulate immunogenicity in virtual patients. Version 4.0 of the IG Simulator now allows for the extrapolation of ex vivo patient assays in addition to in vitro assays to better inform the model and predict clinical outcomes. The IG Simulator has now been used in more than 20 case examples. Certara and its customers will be presenting several of these case examples at an upcoming FDA workshop in June.

In immuno-oncology, the sheer number of possible therapy combinations requires a quantitative framework to integrate the complex and dynamic factors that determine efficacy and historically have led to the selection of suboptimal combinations. Certara’s IO Simulator uses virtual patients to quickly test these different combinations to determine the optimal combination of therapies and dosing regimens. Version 3.0 of the IO Simulator vastly expands the number of targets and cell types including cytokines, immune cell types, and tumor neoantigens. It can also now test combinations of chemotherapy and radiotherapy. The IO Simulator has correctly predicted therapeutic outcomes of using drugs in various cancer types, including solid tumors and blood cancers.

"QSP is demonstrating its capacity to improve biopharmaceutical R&D, improving confidence in both the drug compound and target to increase the likelihood of success," said Piet van der Graaf, Ph.D., Senior Vice President, QSP at Certara. "The IG Simulator can use first-in-human data to design Phase II/III trials, predict impact of disease and co-medication, extrapolate to new populations, and predict if IG can be managed via dosing regimens. In tandem with the rise of IO therapeutics, we’ve expanded the number of drug targets and cell types in our IO Simulator enabling us to better predict optimal drug combinations and dosing regimens for more patient populations."

In recent years, the emergence of QSP has attracted increasing interest and is now becoming an essential part in model-informed drug discovery and development. The U.S. Food and Drug Administration (FDA) has seen an increase in evaluating QSP approaches in regulatory submissions.

On May 13, 2021 Humanigen, Inc. (Nasdaq: HGEN) ("Humanigen"), a clinical stage biopharmaceutical company focused on preventing and treating an immune hyper-response called ‘cytokine storm’ with its lead drug candidate, lenzilumab, reported financial results for the first quarter ending March 31, 2021 and provided a regulatory update on lenzilumab (Press release, Humanigen, MAY 13, 2021, View Source [SID1234579922]).

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"We are encouraged by the achievements Humanigen has made since the beginning of 2021 and by our progress on the emergency use authorization application," stated Cameron Durrant, MD, MBA, Chief Executive Officer, Humanigen. "We successfully completed our Phase 3 study of lenzilumab, referred to as LIVE-AIR, for the treatment of newly hospitalized and hypoxic COVID-19 patients. Trial results showed that patients who received lenzilumab and other treatments, including steroids and/or remdesivir, had a 54% greater relative likelihood of survival without the need for IMV compared with patients receiving placebo and other treatments. We believe this statistically significant result, along with data regarding additional endpoints and further analysis from the study, support the submission of applications for emergency use authorization to the U.S. Food and Drug Administration and conditional marketing authorization in the United Kingdom and the European Union. As is typical with COVID-19 study results, a pre-print of the LIVE-AIR study was published on-line. Positive results from the Phase 1b study of lenzilumab in combination with CAR-T gave further encouragement to our therapeutic approach to breaking the linkage between efficacy and toxicity in CAR-T, and we are designing a Phase 2 study of lenzilumab combined with all commercially available CD19 CAR-T therapies in diffuse large B-cell lymphoma patients."

Highlights from the First Quarter of 2021 Include:

Clinical – Lenzilumab in COVID-19

The Phase 3 results from the LIVE-AIR study were announced, demonstrating that lenzilumab improves survival without the need for mechanical ventilation in hospitalized, hypoxic patients with COVID-19.
Results from the LIVE-AIR Phase 3 study were published in MedRxiv, (View Source) showing additional analysis from the trial, including patients treated with remdesivir and/or steroids, and a second analysis which showed patients under 85 years of age with C-reactive protein ("CRP"), a widely-utilized inflammatory marker, less than 150 mg/L, derived the greatest benefit of treatment with lenzilumab.
With the report of positive top-line results from the LIVE-AIR study in March 2021, the company met the first of two specified milestones under the South Korea license agreement with KPM Tech Co., Ltd. and its affiliate, Telcon RF Pharmaceutical, Inc., and received $6.0 million (or $4.5 million net of withholding taxes and other fees and royalties) in the second quarter of 2021.
In preparation for potential launch under emergency use authorization ("EUA") and conditional marketing authorization ("CMA"), Humanigen entered into several supply agreements with contract manufacturing organizations ("CMOs") to supply bulk drug, fill/finish, and commercial packaging.
Clinical – CAR-T and Oncology

The positive data from the Phase 1 study of ifabotuzumab in glioblastoma multiforme was presented at the AACR (Free AACR Whitepaper) Annual Meeting 2021.
The CAR-T Phase 1b study results in diffuse large B-cell lymphoma("DLBCL") with lenzilumab were announced, showing 100% Objective Response Rate ("ORR") and no severe cytokine release syndrome or severe neurotoxicity at the recommended dose.
With the positive Phase 1b results, the company terminated its clinical collaboration agreement with Kite, a Gilead Company, and announced plans to initiate a Phase 2 study with all commercially available CD19 CAR-T therapies for DLBCL patients.
Corporate

Dr. Adrian Kilcoyne was appointed to the newly created role of Chief Medical Officer.
Entered into a loan facility with Hercules Capital which will provide the company up to $80 million of secured debt financing.
The company launched a public offering of common stock which closed after quarter-end, resulting in net proceeds to Humanigen of $94.1 million.
Two patents were issued for the use of lenzilumab, expanding the company’s anti-GM-CSF patent portfolio.
Lenzilumab Regulatory Update

The company recently held a meeting with FDA to discuss the filing of an EUA for lenzilumab for hospitalized, hypoxic COVID-19 patients, where topline data from the LIVE-AIR study were reviewed, along with the timeline for submission of additional clinical and manufacturing data for lenzilumab. The company plans to submit an EUA application at the end of May 2021. The company has also been in discussion with the Medicines and Healthcare Products Regulatory Agency ("MHRA") for the use of lenzilumab in COVID-19 patients in the United Kingdom and plans to initiate a rolling CMA submission before the end of the second quarter of 2021. The company also plans to submit for CMA to the European Medicines Agency ("EMA") for the use of lenzilumab in the European Union. Further, the company is reviewing the possibility of similar submissions for approval or compassionate use in other territories or countries worldwide.

The company intends to submit a Biologics License Application ("BLA") to FDA in 2022, for the use of lenzilumab in hospitalized, hypoxic COVID-19 patients. Since BLAs typically require more than one study, the company is currently evaluating the extent to which ACTIV-5/BET-B may serve as a basis for a BLA-confirmatory study for lenzilumab.

First Quarter Ended March 31, 2021 Financial Results

Net loss for the three months ended March 31, 2021 was $65.6 million or $1.25 per share as compared to $2.5 million or $0.11 per share for the three months ended March 31, 2020. The increase in net loss for the first quarter 2021 as compared to the first quarter 2020 was largely due to an increase in total expenses, mainly Research and Development expense ("R&D") of $59.2 million from $0.7 million for the three months ended March 31, 2020 to $59.9 million for the three months ended March 31, 2021. The increase in R&D is primarily due to an increase of $51.4 million of expense in lenzilumab manufacturing costs and $7.5 million for clinical trial expenses related to the LIVE-AIR study, both of which began after the first quarter of 2020. The costs incurred for the production of lenzilumab will continue to be included in R&D until lenzilumab is authorized or approved for commercial use, at which point the amounts expended for production will be classified as inventory.

Cash and Cash Equivalents

Net cash used in operating activities, net of balance sheet changes, was $35.8 million for the three months ended March 31, 2021. During the three months ended March 31, 2021, the company raised net proceeds of $36.1 million from the sale of shares of common stock under its At-the-Market offering program. The company drew the first tranche of $25.0 million under its credit facility with Hercules Capital, providing net proceeds of $24.4 million. As of March 31, 2021, the company had cash and cash equivalents of $92.9 million. The company also completed a public offering in the second quarter of 2021 with net proceeds of $94.1 million. The proforma balance of cash and cash equivalents at March 31, 2021 with the proceeds from the public offering is $187.0 million. The company expects to continue to use its funds on development and manufacturing of lenzilumab in anticipation of its potential commercialization under EUA or other conditional marketing authorizations. In the second quarter of 2021 the company anticipates the amount of spending on lenzilumab production will be at least the same level as the first quarter of 2021. If an EUA or CMA for lenzilumab is not received by mid-2021, the company will seek to decrease or eliminate spending on the production of lenzilumab for commercial use.