On August 12, 2021 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that the company has executed an exclusive license agreement with Mayo Clinic for a novel technology that may be able to transform the administration of chimeric antigen receptor engineered T cell ("CAR T") therapies and potentially be used as an off-the-shelf therapy.

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The technology, developed by Larry R. Pease, Ph.D., principal investigator and former director of the Center for Immunology and Immune Therapies at Mayo Clinic, is a new platform to administer CAR T therapy using a two-step approach. First, a peptide is administered to the patient to drive the proliferation of the patient’s resident T cells. This is followed by the administration of a viral CAR construct directly into the lymph nodes of the patient. In turn, the viral construct infects the activated T cells and effectively forms CAR T cells in vivo in the patient. Successful implementation may lead to an off-the-shelf product with no need to isolate and expand patient T cells ex vivo.

"We are excited by the possibilities that this novel technology has to offer given our ongoing development of CAR T cell therapies in hematologic and solid tumor cancers," said Manuel Litchman, M.D., President and Chief Executive Officer of Mustang. "The potential use of this technology to facilitate how these treatments are delivered to patients can lead to earlier treatment post diagnosis, and using an off-the-shelf therapy may reduce the cost of care, all of which would help bring more innovative treatments to a broader base of patients in need."

Preclinical proof-of-concept has been established and the ongoing development of this technology will take place at Mayo Clinic.

"The immune cells are activated in vivo using the natural methods employed by the body to deal with infection rather than the artificial activation used to manufacture traditional CAR T cells ex vivo," said Dr. Pease. "This could potentially reduce the substantial toxicities that are characteristic of traditional CAR T therapy."

Mustang plans to file an Investigational New Drug ("IND") application for a multicenter Phase 1 clinical trial once a lead construct has been identified.

Mayo Clinic and Dr. Pease have financial interest in the technology referenced in this announcement. Mayo Clinic will use any revenue it receives to support its not-for-profit mission in patient care, education and research.

On August 12, 2021 Anavex Life Sciences Corp. ("Anavex" or the "Company") (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, reported financial results for its fiscal quarter ended June 30, 2021 (Press release, Anavex Life Sciences, AUG 12, 2021, View Source [SID1234586378]).

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"We are pleased with the rigorous and efficient execution of the confirmatory late-stage Precision Medicine ANAVEX2-73 Phase 2b/3 randomized, double-blind, placebo-controlled clinical trial with a total of 509 patients with Alzheimer’s disease and are excited to be able to provide top-line data by 2H 2022. The steadfast clinical trial execution capabilities of the Anavex team also extend to the Precision Medicine ANAVEX2-73 Phase 2/3 AVATAR clinical trial in patients with Rett syndrome, which exceeded the enrollment target, and currently topline results from this study are expected by 2H 2021," said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. "We are excited about the potential of our SIGMAR1 platform, and we continue working to advance new programs to the clinic, all with the aim of meeting the unmet clinical needs of patients with both degenerative and developmental neurological disorders around the world."

Recent Business Highlights:

On May 25, 2021, Anavex announced the appointment of Ms. Jiong Ma, PhD to its Board of Directors. Ms. Ma is a Senior Board executive with over 25 years of experience in investing, building, and scaling of companies with a focus on innovative product launches in digital health, technology, and new energy transition.
On June 8, 2021, Anavex announced that it exceeded its enrollment target for the confirmatory late-stage ANAVEX2-73 Phase 2b/3 randomized, double-blind, placebo-controlled trial in Alzheimer’s disease. The Company expects to announce topline results from this study by 2H 2022.
On June 21, 2021, Anavex reported complete data from its ANAVEX2-73 U.S. Rett syndrome Phase 2 study. Predictive biomarker of response established with SIGMAR1 mRNA expression correlates significantly with responses in primary clinical efficacy endpoints from the U.S. Phase 2 randomized, double-blind, placebo-controlled trial of ANAVEX2-73 in patients with Rett syndrome.
On June 24, 2021, Anavex announced the closing of $50 million registered direct offering to Deep Track Capital, an investment firm focused exclusively on the life sciences industry.
On June 28, 2021, Anavex reported complete data from its ANAVEX2-73 Parkinson’s disease dementia (PDD) Phase 2 study. Predictive biomarker of response established with SIGMAR1 mRNA expression correlates significantly with responses in primary and secondary clinical efficacy endpoints from the proof-of-concept randomized, double-blind, placebo-controlled Phase 2 trial of 132 patients with Parkinson’s disease dementia.
Further clinical milestones are provided in Anavex Life Sciences’ latest corporate presentation, available on anavex.com.
Financial Highlights:

Cash and cash equivalents of $157.6 million on June 30, 2021, an increase of over $128 million compared to $29.2 million from September 30, 2020.
Net loss of $10.2 million, or $0.14 per share for the quarter, compared to net loss of $6.5 million, or $0.11 per share in comparative quarter of fiscal 2020.
Research and development expenses of $9.0 million for the quarter, compared to $6.7 million for comparable quarter of fiscal 2020.
General and administrative expenses of $2.4 million for the quarter, compared to $1.4 million for comparable quarter of fiscal 2020.
The financial information for the fiscal quarter ended June 30, 2021, should be read in conjunction with the Company’s interim condensed consolidated financial statements, which will appear on EDGAR, www.sec.gov and will be available on the Anavex website at www.anavex.com.

Webcast / Conference Call Information:

The live webcast of the conference call can be accessed online at View Source

To join the conference call, live via telephone, interested parties within the U.S. should dial, toll-free, 1 (866) 939-3921 and international callers should dial 1 (678) 302-3550. Please use confirmation number 50210582, followed by the pound sign (#).

A replay of the conference call will also be available on www.anavex.com.

On August 12, 2021 Calliditas Therapeutics AB (publ) ("Calliditas" or the "Company") (Nasdaq Stockholm – CALTX; Nasdaq – CALT), a biopharma company focused on identifying, developing and commercializing novel treatments in orphan indications, reported its intention to carry out a directed issue of common shares of approximately SEK 300 million to Swedish and international institutional investors and sector specialist investors (the "Issue") (Press release, Calliditas Therapeutics, AUG 12, 2021, View Source [SID1234586424]).

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The Issue in brief

The Issue is intended to be carried out with deviation from the shareholders’ preferential rights in accordance with the authorization granted by the Annual General Meeting on May 27, 2021. The subscription price and the total number of new shares in the Issue will be determined through an accelerated book building procedure, which will commence immediately following this press release. The completion, pricing and allocation of the new shares is expected to take place before the beginning of trading on Nasdaq Stockholm at 09.00 CEST on August 13, 2021. By establishing the subscription price in the Issue through an accelerated book building procedure, it is the assessment of the Board of Directors that the subscription price will accurately reflect current market conditions and demand. The closing, pricing and allocation in the book building procedure is at the discretion of the Company and may be cancelled at any time. The Company will announce the outcome of the Issue in a subsequent press release after the book building procedure has been completed.

The Company intends to use the net proceeds from the Issue for:

ongoing clinical development;
pre-commercial development in the United States;
commercial activities for Nefecon, if approved for marketing by the FDA later this year; and
general corporate purposes.
The Company believes that using the flexibility provided by a non-pre-emptive placing is the most appropriate and optimal transaction structure at this time in order to raise capital for the development of ongoing projects in a time and cost-effective manner. Moreover, the Company will further strengthen the shareholder base with Swedish and international institutional investors and sector specialist investors.

In connection with the Issue, the Company has agreed to a lock-up undertaking, with customary exceptions, on future share issuances for a period of 90 days. In addition, members of the Board of Directors and management of Calliditas, who owns shares or warrants, have, in connection with the Issue, agreed not to sell any shares in the Company during a lock-up period of 90 days subject to customary exceptions.

Advisers

In conjunction with the Issue, the Company has engaged Jefferies GmbH, Carnegie Investment Bank AB (publ) and Kempen & Co as Joint Global Coordinators and Joint Bookrunners. Vinge act as legal adviser to the Company and Baker McKenzie act as legal adviser to the Banks.

The information in the press release is inside information that Calliditas is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact persons above, on August 12, 2021 at 17:57 (CEST).

On August 12, 2021 Chinook Therapeutics, Inc. (Nasdaq: KDNY), a biopharmaceutical company focused on the discovery, development and commercialization of precision medicines for kidney diseases, reported financial results for the second quarter ended June 30, 2021 (Press release, Aduro Biotech, AUG 12, 2021, View Source [SID1234586440]).

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"During the second quarter of 2021, we made strong progress across our pipeline of programs for kidney diseases, including driving enrollment in the phase 3 ALIGN and phase 2 AFFINITY trials of atrasentan, presenting interim clinical data for BION-1301 demonstrating initial proof-of-concept in patients with IgA nephropathy and advancing key IND-enabling studies for CHK-336," said Eric Dobmeier, president and chief executive officer of Chinook Therapeutics. "Our solid cash position and resourcing enable us to continue executing on key priorities to advance our pipeline and build Chinook into a leading kidney disease company."

Recent Highlights

Enrolled the first patient in the phase 2 AFFINITY basket trial of atrasentan, a highly potent and selective endothelin A receptor (ETA) antagonist (see www.clinicaltrials.gov, identifier NCT04573920).

Presented interim data from the first several patients with IgA nephropathy (IgAN) enrolled in Cohort 1 of Part 3 of the ongoing phase 1/2 study of BION-1301 at the 58th ERA-EDTA conference, demonstrating durable reductions in Gd-IgA1, IgA, IgM, and to a lesser extent, IgG levels, as well as clinically meaningful reductions in 24-hour proteinuria (UPCR), providing initial proof-of-concept for BION-1301 in IgAN.

Presented Gd-IgA1 biomarker data in healthy volunteers from Part 1 (single ascending dose) and Part 2 (multiple ascending dose) of the ongoing phase 1/2 study of BION-1301 at the ISN World Congress of Nephrology 2021, showing dose-dependent and durable reductions in Gd-IgA1 levels following administration of BION-1301.

Presented data from the BION-1301 phase 1 intravenous to subcutaneous bioavailability study in healthy volunteers at the ISN World Congress of Nephrology 2021.

Entered into an agreement for Sairopa B.V., or Sairopa, to acquire certain of Chinook’s non-renal assets in exchange for a 44 percent preferred equity position in Sairopa. Any future proceeds resulting from this transaction will be shared equally between the CVR holders and Chinook until October 4, 2030, after which 100 percent of the value will accrue to Chinook.

Announced promotions of Tom Frohlich to Chief Operating Officer and Andrew King, D.V.M., Ph.D., to Chief Scientific Officer.
Anticipated Upcoming Catalysts

Chinook expects to complete enrollment in Cohort 1 of Part 3 of the ongoing phase 1/2 study of BION-1301 in the third quarter of 2021 and to present additional patient data from this cohort at the ASN Kidney Week 2021 in November.

Chinook expects to initiate enrollment in Cohort 2 of Part 3 of the ongoing phase 1/2 study of BION-1301 in the third quarter of 2021. Patients in Cohort 2 will receive a subcutaneous dose of 600 mg of BION-1301 every two weeks for up to 52 weeks. Chinook expects to present patient data from Cohort 2 and provide an update on planned later-stage clinical trials of BION-1301 in the first half of 2022.

Chinook expects to present interim data from one or more patient cohorts of the ongoing phase 2 AFFINITY basket trial of atrasentan in the first half of 2022.

CHK-336 is currently in IND-enabling studies and is advancing towards expected initiation of a phase 1 clinical study in healthy volunteers in the first quarter of 2022 for the treatment of primary hyperoxaluria.
Second Quarter 2021 Financial Results

Cash Position – Cash, cash equivalents and marketable securities totaled $229.8 million at June 30, 2021, compared to $250.4 million at December 31, 2020.

Revenue – Total revenue increased by less than $0.1 million for the second quarter of 2021 and increased by $0.4 million for the six months ended June 30, 2021, compared to the same periods in 2020 due to revenue recognized related to research and development services provided under the collaboration agreement with Lilly, which was acquired under the merger with Aduro.

Expenses –

Research and development expenses were $22.8 million for the second quarter of 2021 and $48.5 million for the six months ended June 30, 2021, compared to $3.9 million and $6.7 million, respectively, for the same periods in 2020. The increase was primarily due to external clinical and manufacturing expenses related to the atrasentan and BION-1301 clinical programs; higher employee-related costs, including salaries, benefits and stock-based compensation expense associated with hiring staff to build out Chinook’s clinical and development capabilities; increased spending for consulting and outside services; and an increase in facilities and other costs. The six months ended June 30, 2021 also includes an upfront fee of $3.3 million due to Evotech International GmbH under a research collaboration and license agreement entered into in February 2021.

General and administrative expenses were $7.8 million for the second quarter of 2021 and $17.3 million for the six months ended June 30, 2021, compared to $3.9 million and $5.2 million, respectively, for the same periods in 2020. The increase was primarily due to higher employee-related costs, including salaries, benefits and stock-based compensation expense associated with the addition of administrative staff to buildout Chinook’s public-company infrastructure; higher legal, consulting and outside services costs; and an increase in facilities and other costs.

Expenses due to the change in fair value of contingent consideration and contingent value rights liabilities were $19.6 million for the second quarter of 2021 and $21.4 million for the six months ended June 30, 2021, compared to nil for the same periods in 2020. These non-cash expenses are due to the quarterly revaluation of assets and liabilities related to the Sairopa transaction and an updated valuation of Chinook’s CVR liability under the Merck collaboration, as a result of the merger with Aduro. In the second quarter of 2021, Merck informed Chinook that it intends to explore the potential benefit of the product candidate MK-5890, previously out-licensed to Merck by Aduro, in a phase 2 clinical study for a new indication. This may result in potential milestone and royalty payments for the benefit of CVR holders.

Other –

The sale of certain of Chinook’s non-renal assets to Sairopa in the second quarter of 2021 resulted in a $7.2 million gain.
Net Loss – Net loss for the second quarter of 2021 was $42.6 million or $0.97 per share and $79.8 million or $1.86 per share for the six months ended June 30, 2021, compared to net loss of $7.7 million or $1.87 per share and $12.9 million or $3.12 per share, respectively, for the same periods in 2020.
Cash Used in Operations – For the six months ended June 30, 2021, cash used in operations totaled $55.5 million.
(Press release, Aduro Biotech, AUG 12, 2021, View Source [SID1234586440])

On August 12, 2021 Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for the treatment of refractive and retinal eye disorders, reported financial results for the second quarter of 2021 and provided a corporate update (Press release, Ocuphire Pharma, AUG 12, 2021, View Source [SID1234586458]).

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"The second quarter of 2021 proved to be highly productive for Ocuphire, with continued success in clinical trials, key pivotal data presentations featured at major medical meetings across both Nyxol and APX3330, and additional IP protection granted for our lead drug candidate Nyxol," said Mina Sooch, MBA, President and CEO of Ocuphire Pharma.

"Following our first quarter announcement of positive MIRA-2 Phase 3 results of Nyxol for the treatment of reversal of mydriasis (RM), we also announced positive top-line results from our VEGA-1 Phase 2 study, which evaluated Nyxol in combination with low-dose pilocarpine (LDP) for the treatment of presbyopia, the gradual loss of your eyes’ ability to focus on nearby objects. In our view, RM and presbyopia both represent attractive and large US and global market opportunities, with each indication having a significant unmet medical need with very few if any pharmaceutical treatment options. Following the recent ASCRS meeting, it is also clear that global pharmaceutical and consumer healthcare companies are aggressively pursuing innovative new medicines, further validating the significant commercial potential for the large presbyopia market. Based upon the success of our VEGA-1 trial, we are highly confident that Nyxol in combination with LDP presents a potentially best-in-class therapeutic solution that can address the needs of the presbyopia patient population."

"With our balance sheet recently strengthened in the second quarter combined with our capital efficient operations, Ocuphire is well positioned to deliver additional late-stage clinical milestones in 2022 for Nyxol and APX3330 while continuing our global business development efforts."

Key Anticipated Future Milestones

Reversal of Mydriasis (RM): Initiate second Phase 3 (MIRA-3) registration trial and a small pediatric trial (MIRA-4) in the second half of 2021 investigating Nyxol with results expected in early 2022; Planning to file NDA submission with FDA for Nyxol in RM indication in late 2022
Presbyopia: Initiate Phase 3 program (VEGA-2/3) in first half of 2022 investigating Nyxol and LDP
Night Vision Disturbances: Completion of enrollment expected by year-end 2021 and top-line data expected in early 2022 from Phase 3 (LYNX-1) registration trial investigating Nyxol
Diabetic Retinopathy and Diabetic Macular Edema: Completion of enrollment in Phase 2 (ZETA-1) trial investigating APX3330 and top-line data expected in 2022

Second Quarter and Recent Business Highlights

Clinical Development

In June, the Company announced successful results from the VEGA-1 Phase 2 trial of Nyxol plus low-dose pilocarpine (LDP) for the treatment of presbyopia; the trial met its primary endpoint of 3 lines of near vision improvement and multiple key secondary endpoints such as fast onset of action and durability with statistical significance and a favorable safety profile (including no headaches)
In April, the Company initiated the ZETA-1 Phase 2 clinical trial to evaluate oral APX3330 in non-proliferative diabetic retinopathy (NPDR) and mild proliferative diabetic retinopathy (mild PDR)

Presentations and Publications

In July, the Company announced publication in the Journal of Cellular Signaling featuring Ocuphire’s novel oral Ref-1 inhibitor APX3330 in Phase 2 trial for the treatment of retinal disease which highlighted the favorable safety profile of APX3330 and its unique anti-angiogenic and anti-inflammatory mechanism of action properties relevant to a broad range of retinal diseases
In July at the 2021 American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting, Dr. Jay S. Pepose, Medical Advisor and Board Director, presented two papers featuring the positive results Phase 2 Presbyopia (VEGA-1) and Phase 3 Reversal of Mydriasis (MIRA-2)
In July, Mina Sooch, CEO, participated in the presbyopia drug therapy panel at the Eyecelerator held on July 22nd and in the Eye on Innovation panel at the Virtual Salon Series held on July 28th
In late May, Ocuphire hosted a Key Opinion Leader Event on Nyxol as a potential new treatment option for reversing pharmacologically induced mydriasis, highlighting recent positive Phase 3 results from the MIRA-2 Phase 3 registrational study
In May at the 2021 Association for Research in Vision and Ophthalmology (ARVO) Virtual Annual Meeting, Ocuphire presented data for APX3330 on pre-clinical ocular data and predictive human retina and plasma model data

Corporate

Announced closing of a $15 million registered direct offering and just over $4 million from ATM, extending cash runway into late 2022
Appointed Jay S. Pepose, M.D., Ph.D. to the Company’s Board of Directors
Received two new U.S. patent grants covering Nyxol including Nyxol plus LDP claims for the treatment of presbyopia through 2039
Entered into a license agreement granting Processa Pharmaceuticals (Nasdaq: PCSA) an exclusive license to develop, manufacture and commercialize globally RX-3117 (Rexahn legacy oncology intellectual property), excluding China, Hong Kong, Macau, Republic of Singapore and Taiwan (already licensed to BioSense Global LLC ("BioSense"))
Ocuphire (Nasdaq: OCUP) added to the Russell Microcap Index

Second Quarter and Year to Date 2021 Financial Highlights

As of June 30, 2021, the Company had cash and cash equivalents of approximately $24.2 million. Net cash used in operating activities for the three and six months ended June 30, 2021, was $4.3 million and $10.1 million, respectively.

Collaborations revenue was $0.1 million for the three and six months ended June 30, 2021. Revenue during the periods was derived from the license agreement with BioSense related to certain technology transfers. There was no collaborations revenue recognized during the comparable prior year periods.

General and administrative expenses for the three and six months ended June 30, 2021, were $3.4 million and $5.1 million, respectively, compared to $0.6 million and $0.9 million, respectively, for the three and six months ended June 30, 2020. The increases from the comparable periods in 2020 were attributable to increased costs primarily in administrative employee headcount, stock-based compensation, insurance, legal and settlement costs, and costs associated with operating as a public company subsequent to the reverse merger.

Research and development expenses for the three and six months ended June 30, 2021, were $3.8 million and $7.3 million, respectively, compared to $0.7 million and $0.9 million, respectively, for the three and six months ended June 30, 2020. The increases from the comparable periods in 2020 were primarily attributable to four new clinical trials and manufacturing activities for Nyxol and APX3330 as well as regulatory, preclinical, and other development activities.

There were no acquired in-process research and development expenses in the current six-month period. In the prior year in connection with the sublicense agreement with Apexian for the continued research, development and potential commercialization of APX3330, the Company recorded acquired in-process research and development expenses of $2.1 million during the six-month period ended June 30, 2020.

The total loss from operations for the three and six months ended June 30, 2021 was $7.1 million and $12.3 million, respectively, compared to $1.3 million and $4.0 million for the three and six months ended June 30, 2020, respectively. This included non-cash stock-based compensation expense of $0.5 million and $1.0 million during the three and six months ended June 30, 2021, respectively, and $0.3 million and $0.4 million during the three and six months ended June 30, 2020, respectively.

For further details on Ocuphire’s financial results, including results for the three and six month periods ended June 30, 2021 refer to the Company’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, to be filed with the Securities and Exchange Commission.