On May 14, 2021 Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses, reported financial results for the first quarter ended March 31, 2021 and provided a business update (Press release, Selecta Biosciences, MAY 13, 2021, View Source [SID1234579954]).

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"We are very pleased about the continued progress across all aspects of the company," said Carsten Brunn, Ph.D., president and chief executive officer of Selecta. "We regained exclusive rights to our MMA program and now have two proprietary gene therapy programs to rapidly follow our ongoing empty capsid study. Additionally, we continue to progress our enzyme program, with an expected IND filing by the end of 2021 in IgA nephropathy, and topline data from the Phase 3 DISSOLVE program for SEL-212 anticipated in the second half of 2022."

Recent Highlights and Anticipated Upcoming Milestones:

Enzyme Therapies:

SEL-212 for chronic refractory gout: Enrollment for the Phase 3 DISSOLVE clinical program for SEL-212 for the treatment of chronic refractory gout, which was licensed to Sobi, is progressing as planned with topline data expected in the second half of 2022.
Leveraging the success of SEL-212, Selecta expects to file an Investigational New Drug, or IND, application by the end of 2021 for a novel therapeutic approach that combines ImmTOR with an enzyme, IgA1 protease for the treatment of IgA nephropathy.
Gene Therapies:

First-in-human trial of SEL-399: In collaboration with AskBio, Selecta initiated the first-in-human, dose-escalation trial of SEL-399, an adeno-associated viral serotype 8 (AAV8) empty vector capsid (EMC-101) containing no DNA combined with ImmTOR. The trial aims to determine the optimal dose of ImmTOR to mitigate the formation of antibodies to AAV8 capsids used in gene therapies. Selecta and AskBio expect to report topline data in the fourth quarter of 2021.
MMA-101 for methylmalonic acidemia (MMA): Selecta regained exclusive rights to its lead gene therapy program in MMA from AskBio and expects to file an IND in MMA-101, in combination with ImmTOR, by the end of 2021. The phase 1/2 MMA-101 program, which is expected to commence in 2022, will evaluate biomarkers of efficacy, neutralizing antibodies and safety and tolerability.
SEL-313 for ornithine transcarbamylase deficiency (OTC deficiency): Selecta’s proprietary gene therapy product candidate, SEL-313, is being developed to treat OTC deficiency, a rare genetic urea cycle disorder that causes ammonia to accumulate in the blood due to mutations in the OTC gene. SEL-313 is currently in preclinical development and a clinical trial application, or CTA and/or IND filing are expected in 2022. A Pediatric Investigation Plan (PIP) for SEL-313 was submitted to the European Medicines Agency (EMA) pediatric committee in February 2021.
Sarepta Therapeutics program in Duchenne Muscular Dystrophy (DMD) and certain Limb-Girdle Muscular Dystrophies (LGMD) subtypes: Selecta has achieved a $3 million milestone payment related to the completion of a preclinical study under the Research License and Option Agreement.
Restoring Self-Tolerance in Autoimmune Diseases:

Selecta continues IND-enabling work on an ImmTOR-based approach to treating primary biliary cholangitis (PBC), a chronic, progressive autoimmune liver disorder that leads to inflammation, damage and scarring of the small bile ducts. Selecta expects to file an IND in PBC in the second half of 2022.
Corporate Updates:

Kristen Baldwin was appointed Chief People Officer. She brings 20 years of Human Resources and Consulting experience to the company. Most recently Ms. Baldwin served in dual capacity as the Chief People Officer for the LIVEKINDLY Collective, a high growth plant-based foods company, and as a Senior Partner at CEO.works. Ms. Baldwin has also held senior HR roles at Bayer and Otsuka Pharmaceuticals.

Satish Tripathi, Ph.D., was appointed Vice President of Global Regulatory Affairs. Dr. Tripathi has over 25 years of combined R&D, business, and global regulatory strategy experience. Dr. Tripathi most recently served as VP of Global Regulatory Affairs for AveXis which became Novartis Gene Therapies, where he led the regulatory strategy and implementation for the gene therapy product AVXS-101 for Spinal Muscular Atrophy. AVXS-101 is recognized as only one of the 3 drugs in the world to receive Breakthrough (US FDA), PRIME (EMA) and Sakigake (MHLW/PMDA) designations. Dr. Tripathi led the simultaneous submission of AVXS-101 in 2018 for global registration, which has been approved as Zolgensma for SMA in US, Europe, Japan, Canada, and Brazil.

Brad Dahms will be stepping down as Chief Financial Officer effective May 21st, 2021 to pursue another opportunity. Mr. Dahms’ departure is not related to Selecta’s operations, financial reporting, or controls. A search is currently underway for a successor.

Ann Donohue will be promoted to Vice President Finance, effective immediately, after having served as Controller of Selecta since December 2017.
First Quarter 2021 Financial Results:

Cash Position: Selecta had $149.2 million in cash, cash equivalents, marketable securities, and restricted cash as of March 31, 2021, which compares to cash, cash equivalents, and restricted cash of $140.1 million as of December 31, 2020. Selecta believes its available cash, cash equivalents, marketable securities, and restricted cash will be sufficient to meet its operating requirements into the second quarter of 2023.

Net cash used in operating activities was $12.1 million for the first quarter of 2021, as compared to $11.7 million for the same period in 2020.
Revenue: Revenue recognition for the first quarter of 2021 was $11.1 million, compared to no revenue recognition for the same period in 2020. Revenue was recognized under the license agreement with Sobi which began in July 2020 resulting from the shipment of clinical supply and the reimbursement of costs incurred for the Phase 3 DISSOLVE clinical program.

Research and Development Expenses: Research and development expenses for the first quarter 2021 were $13.0 million, which compares with $14.7 million for the same period in 2020. During the quarter ended March 31, 2021, there was a reduction in expenses for the SEL-212 clinical program and for the AskBio Collaboration, offset by an increase of expense for discovery and preclinical programs.

General and Administrative Expenses: General and administrative expenses for the first quarter 2021 were $5.2 million, which compares with $4.1 million for the same period in 2020. The quarterly increase in expense was the result of expenses for consulting and professional fees and salaries offset by reduced travel expenses.

Net Loss: For the first quarter 2021, Selecta reported a net loss of $24.6 million, or $0.22 per share, compared to a net loss of $19.6 million, or $0.21 per share for the same period in 2020.

Conference Call and Webcast Reminder:
Selecta management will host a conference call at 8:30 AM ET today to provide a corporate update and review the company’s first quarter 2021 financial results. Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10147801. Investors and the public can access the live and archived webcast of this call and a copy of the presentation via the Investors & Media section of the company’s website, www.selectabio.com.

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.