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Junshi Biosciences and Coherus Announce U.S. FDA Breakthrough Therapy Designation Granted for Toripalimab for 1st Line Treatment of Nasopharyngeal Carcinoma

On August 12, 2021 Shanghai Junshi Biosciences Co., Ltd ("Junshi Biosciences", HKEX: 1877; SSE: 688180) and Coherus BioSciences, Inc. ("Coherus", Nasdaq: CHRS) reported that the United States Food and Drug Administration ("FDA") has recently granted Breakthrough Therapy Designation ("BTD") for toripalimab in combination with chemotherapy (gemcitabine and cisplatin) for the 1st line treatment of recurrent or metastatic nasopharyngeal carcinoma ("NPC") (Press release, Coherus Biosciences, AUG 12, 2021, View Source [SID1234586462]). The FDA had earlier granted BTD for toripalimab monotherapy for patients with recurrent or metastatic NPC with disease progression on or after platinum-containing chemotherapy.

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BTD is intended to expedite the development and regulatory review of drugs where preliminary clinical evidence demonstrates substantial improvement over existing therapies for a severe or life-threating disease. Drugs with BTD will be granted closer FDA guidance – including that from senior FDA officials – and various forms of support to avail patients with new therapy as soon as possible.

Junshi Biosciences expects to complete the biologics license application ("BLA") submission for toripalimab plus chemotherapy for 1st line NPC and for toripalimab monotherapy for 2nd or 3rd line NPC later this quarter.

"We are pleased to have received Breakthrough Therapy designation for our novel PD-1 blocking antibody, toripalimab, for nasopharyngeal carcinoma, which is an aggressive cancer with no immuno-oncology treatment options approved in the United States," said Dr. Patricia Keegan, Chief Medical Officer of Junshi Biosciences. "We look forward to working closely with the FDA during the BLA review process and with our partner, Coherus, to bring toripalimab to NPC patients in the U.S., if approved."

The Breakthrough Therapy designation is supported by data from the Phase 3 clinical trial "JUPITER-02" evaluating toripalimab in combination with chemotherapy for the first-line treatment of NPC. In this study, toripalimab in combination with chemotherapy demonstrated a statistically significant and clinically meaningful improvement in progression free survival ("PFS") compared to chemotherapy alone (assessed by a blinded independent review committee ("BIRC") per RECIST v1.1). JUPITER-02 also met secondary endpoints of PFS assessed by the investigator and objective response rate assessed by BIRC. There was also a longer duration of response, a higher disease control rate, and higher one- and two-year survival rates for the toripalimab arm. The safety profile of toripalimab is consistent with that observed in previously reported toripalimab clinical trials and the safety profile of this class of drugs. The result of JUPITER-02 was recently presented at the ASCO (Free ASCO Whitepaper) plenary session (#LBA2) and full results can be found in the August 2021 on-line edition of Nature Medicine.

About toripalimab
Toripalimab is an anti-PD-1 monoclonal antibody developed for its ability to block PD-1 interactions with its ligands, PD-L1 and PD-L2, and for enhanced receptor internalization (endocytosis function). Blocking PD-1 interactions with PD-L1 and PD-L2 is thought to recharge the immune system’s ability to attack and kill tumor cells.

More than thirty company-sponsored toripalimab clinical studies covering more than fifteen indications have been conducted globally, including in China and the United States. Pivotal clinical trials are ongoing or completed evaluating the safety and efficacy of toripalimab for a broad range of tumor types including cancers of the lung, nasopharynx, esophagus, stomach, bladder, breast, liver, kidney and skin.

In China, toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing (approved in China as TUOYI). On December 17, 2018, toripalimab was granted a conditional approval from the National Medical Products Administration (NMPA) for the second-line treatment of unresectable or metastatic melanoma. In December 2020, toripalimab was successfully included in the updated National Reimbursement Drug List. In February 2021, the supplemental NDA for toripalimab in combination with chemotherapy for the first-line treatment of patients with advanced, recurrent or metastatic nasopharyngeal carcinoma was accepted by the NMPA. In the same month, the NMPA granted a conditional approval to toripalimab for the treatment of patients with recurrent or metastatic nasopharyngeal carcinoma (NPC) after failure of at least two lines of prior systemic therapy. In April, NMPA granted a conditional approval to toripalimab for the treatment of patients with locally advanced or metastatic urothelial carcinoma who failed platinum-containing chemotherapy or progressed within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.

In the United States, a rolling submission of the first toripalimab Biologics License Application (BLA) is underway for the treatment of recurrent or metastatic nasopharyngeal carcinoma (NPC). The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for toripalimab in combination with chemotherapy for the 1st line treatment of recurrent or metastatic nasopharyngeal carcinoma ("NPC") and also for toripalimab monotherapy in second or third line treatment of recurrent or metastatic NPC. There are currently no PD-1 blocking antibodies indicated for use in NPC in the United States. Additionally, FDA has granted Fast Track status for the development of toripalimab for the treatment of mucosal melanoma and orphan drug designation for NPC, mucosal melanoma and soft tissue sarcoma. Earlier in 2021 Coherus in-licensed rights to develop and commercialize toripalimab in the United States and Canada. Coherus and Junshi Biosciences plan to file additional toripalimab BLAs with the FDA over the next three years for multiple rare cancers and highly prevalent cancers.

Nimbus Therapeutics and Schrödinger Extend Longstanding Collaboration to Discover Novel Therapeutics for Select Targets

On August 12, 2021 Nimbus Therapeutics, a biotechnology company designing breakthrough medicines through structure-based drug discovery and development, reported the extension of its collaboration with Schrödinger, Inc. to discover and design small molecule therapeutics for a number of high-value targets using cutting-edge structure-based drug design approaches (Press release, Nimbus Therapeutics, AUG 12, 2021, View Source [SID1234586461]).

The agreement extends the collaboration between the two companies, which began in 2009, to advance the usage of computational methods in drug discovery. Since then, the collaboration has generated numerous promising therapeutic candidates, including small molecule inhibitors of acetyl–CoA carboxylase (ACC) and tyrosine kinase 2 (TYK2), which are now in the clinic, and a hematopoietic protein kinase 1 (MAP4K1/HPK1) inhibitor, which Nimbus plans to advance to the clinic later this year.

"Nimbus’ collaboration with Schrödinger over the past 12 years has been groundbreaking, deploying computational horsepower at an unprecedented scale in the industry," said Jeb Keiper, M.S., MBA, Chief Executive Officer of Nimbus. "Schrödinger’s deep computational capabilities, paired with Nimbus’ demonstrated expertise in structural biology, small molecule drug discovery and clinical development, provides fertile ground for designing breakthrough medicines over the next decade."

"We are very pleased to embark on this new chapter of Schrödinger’s collaboration with Nimbus and to further expand the frontiers of combining physics-based drug discovery with machine learning," said Ramy Farid, Ph.D., Chief Executive Officer at Schrödinger. "Our collaborative track record underscores the potential of our combined multidisciplinary drug discovery team to design medicines that could provide meaningful benefits for patients."

Kintara Therapeutics to Present at the Investor Summit Virtual Conference on August 17, 2021

On August 12, 2021 Kintara Therapeutics, Inc. (Nasdaq: KTRA), a biopharmaceutical company developing novel cancer therapies for patients who are failing or are resistant to current treatment regimens, reported that its Chief Executive Officer, Saiid Zarrabian, will present at the Investor Summit Conference being held August 17 – 18, 2021 (Press release, Kintara Therapeutics, AUG 12, 2021, View Source [SID1234586459]).

Mr. Zarrabian will deliver his corporate presentation on August 17, 2021 at 12:30 p.m.ET.

Investors can also request a one-on-one meeting with Mr. Zarrabian to be arranged following the conclusion of the conference.

Investors can register here: Q3 Investor Summit Investor Registration

Ocuphire Announces Financial Results for the Second Quarter 2021 and Provides Corporate Update

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]).

"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.

ProMIS Neurosciences Announces Second Quarter 2021 Results

On August 12, 2021 ProMIS Neurosciences, Inc. (TSX: PMN) (OTCQB: ARFXF) ("ProMIS or the Company"), a biotechnology company focused on the discovery and development of antibody therapeutics targeting toxic oligomers implicated in the development of neurodegenerative diseases, reported its operational and financial results for the three and six months ended June 30, 2021 (Press release, ProMIS Neurosciences, AUG 12, 2021, View Source [SID1234586457]).

ProMIS Neurosciences is applying its patented technology platform to build a portfolio of antibody therapies, therapeutic vaccines, and diagnostics in neurodegenerative diseases, including Alzheimer’s disease (AD) and other dementias, Parkinson’s Disease (PD), and amyotrophic lateral sclerosis (ALS).

These diseases share a common biologic cause – misfolded versions of proteins, that otherwise perform a normal function, kill neurons and produce disease.

ProMIS’ technology platform is an example of the advances in drug discovery enabled by computational power, in silico discovery, and/or artificial intelligence. This platform provides an advantage in either selectively targeting the toxic misfolded proteins with therapeutics or detecting them with diagnostics. This capability has given ProMIS a growing portfolio of potential "best in class" monoclonal antibodies (or corresponding therapeutic vaccines), including our lead program PMN310, targeting toxic oligomers of amyloid in AD.

Corporate Highlights

On May 21, 2021, we re-initiated the path to Investigational New Drug application for PMN310 in AD with the start of producer cell line development. This key first step in the manufacturing of antibody therapeutics is being carried out by Selexis, SA, using Selexis’ proprietary SUREtechnology Platform.
On June 3, 2021, the Company announced that it had filed a preliminary Prospectus with the securities regulators in each of the provinces and territories of Canada, except Quebec. The Prospectus will allow the Company to make offerings of common shares, warrants, units, debt securities, subscription receipts, convertible securities or any combination thereof for up to an aggregate total of US$50 million during the 25-month period that the Prospectus is effective.
On July 8, 2021, the Company announced that it had filed, and obtained a receipt for the Prospectus with the securities regulators in each of the provinces and territories of Canada, except Quebec.
On July 2, 2021, the Company announced the voting results of the Company’s annual meeting of shareholders held on June 30, 2021, in Vancouver, British Columbia, Canada. All resolutions described in the Management Proxy Circular and placed before the meeting were approved by the shareholders.
People

On May 12, 2021, Dr. Rudolph Tanzi, Ph.D., was appointed as the Chair of the Company’s Scientific Advisory Board. Dr. Tanzi is the Joseph P. and Rose F. Kennedy Professor of Neurology at Harvard University and Vice-Chair of Neurology, Director of the Genetics and Aging Research Unit, and Co-Director of the Henry and Allison McCance Center for Brain Health at Massachusetts General Hospital.
On May 13, 2021, we appointed Neil K. Warma, to the Company’s Board of Directors. Neil Warma has been a healthcare entrepreneur for over 25 years having managed and advised numerous biotechnology and pharmaceutical companies.
On May 25, 2021, the Company appointed Owen Dempsey to lead the commercialization program for its COVID-19 serology assay.
On May 27, 2021, Dr. David Wishart, Distinguished University Professor in the Departments of Biological Sciences and Computing Science at the University of Alberta, was appointed as Chief Physics Officer at ProMIS.
Financial Results

Results of Operations – Three months ended June 30, 2021 and 2020

Net Loss for the three months ended June 30, 2021 were $297,346 compared to $1,650,218 for the three months ended June 30, 2020. Included in the net loss amount for the three months ended June 30, 2021, was non-cash expenses/(income) of ($1,049,745) representing the change in the fair value of the embedded derivative of ($1,245,388), share-based compensation of $180,392, amortization of property and equipment and an intangible asset of $12,252 compared to $76,310 for the three months ended June 30, 2020, consisting of share-based compensation of $74,642 and amortization of an intangible asset of $1,668.

Operating loss for the three months ended June 30, 2021 was $1,378,603, as compared to $1,650,218 in the three months ended June 30, 2020. The decrease in the operating loss for the three months ended June 30, 2021, reflects decreased contracted salaries and associated costs of $350,622 due to reduction in compensation to management and attrition of contract staff, decreased investor relations of $203,971 due to scale down of investor relations activities and consultants and foreign exchange gains of $233,874 due to the foreign exchange on US denominated assets and liabilities offset by increased costs associated with external contract research organizations for internal programs of $172,518 as the company restarts the internal programs, share-based compensation of $105,749 due to the grant of share options, increased patent expense of $40,162 due to increased maintenance fees, increased legal expense of $45,752, increased consulting expense of $137,522, increase in amortization of property and equipment and intangible asset of $13,584 and decreased revenue of $1,565.

Research and development expenses for the three months ended June 30, 2021 were $1,065,197, as compared to $898,887 in the three months ended June 30, 2020. The increase in research and development expense for the three months ended June 30, 2021, compared to the same period ended June 30, 2020, is primarily attributed to increased costs associated with external contract research organizations for internal programs of $172,518 as the company restarts the internal programs, increased share-based compensation of $28,579 due to the grant of share options, increased patent expense of $40,162 due to increased maintenance fees ,increased outside consultants of $137,706 and increase in amortization of property and equipment and intangible asset of $13,584 offset by decreased contracted research salaries and associated costs of $226,238 due to reduction in compensation to management and attrition of contract staff.

General and administrative expenses for the three months ended June 30, 2021 were $313,406, as compared to $752,896 in the three months ended June 30, 2020. The decrease for the three months ended June 30, 2021, compared to the same period in 2020, is primarily attributable to a reduction in contracted corporate salaries and associated costs of $124,383 due to reduction in compensation to management and attrition of contracted staff, decreased investor relations of $203,971 due to scale down of investor relations activities and consultants and foreign exchange gains of $233,874 due to the foreign exchange on US denominated assets and liabilities offset by share-based compensation of $77,171 due to the grant of share options, and increased legal expense of $45,752.

Results of Operations – Six months ended June 30, 2021 and 2020

Net loss for the six months ended June 30, 2021 were $7,896,763 compared to $3,412,137 for the six months ended June 30, 2020. Included in the net loss amount for the six months ended June 30, 2021, was non-cash expense $5,909,542, representing the change in the fair value of the embedded derivative of $5,766,915, share-based compensation of $112,123, amortization of property and equipment and an intangible asset of $30,504 for the six months ended June 30, 2021, compared to $290,048 for the six months ended June 30, 2020, consisting of share-based compensation of $286,712 and amortization of an intangible asset of $3,336.

Operating loss for the six months ended June 30, 2021 was $1,960,934, as compared to $3,412,137 in the six months ended June 30, 2020. The decrease in the operating loss for the six months ended June 30, 2021, reflects decreased contract salaries and associated costs of $924,565 due to reduction in compensation to management and attrition of contracted staff, decreased investor relations of $380,959 due to scale down of investor relation activities and consultants, decreased share-based compensation of $174,589 due to forfeiture of unvested/vested share options due to termination of consulting arrangement and foreign exchange gains of $293,012 due to the foreign exchange on US denominated offset by increased costs associated with external contract research organizations for internal programs of $88,673 as the company restarts the internal programs, increased patent expense of $3,617 due to increased maintenance fees, increased legal expense of $99,572, increased consulting expense of $101,315, increase in amortization of property and equipment and intangible asset of $27,168 and decreased revenue of $1,578.

Research and development expenses for the six months ended June 30, 2021 were $1,259,120, as compared to $1,872,473 in the six months ended June 30, 2020. The decrease in research and development expense for the six months ended June 30, 2021, compared to the same period ended June 30, 2020, reflects the conservation of cash resources and decreased contract salaries and associated costs of $673,785 due to reduction in compensation to management and attrition of contracted staff and decreased share-based compensation of $145,459 due to forfeiture of unvested/vested share options due to termination of consulting arrangement offset by increased costs associated with external contract research organizations for internal programs of $88,673 as the company restarts the internal programs, increased patent expense of $3,617 due to increased maintenance fees, increased consulting expense of $86,435 and increase in amortization of property and equipment and intangible asset of $27,168.

General and administrative expenses for the six months ended June 30, 2021 were $701,814, as compared to $1,541,242 in the six months ended June 30, 2020. The decrease for the six months ended June 30, 2021, compared to the same period in 2020, is primarily attributable to a reduction in contract salaries and associated costs of $250,780 due to reduction in compensation to management and attrition of contracted staff, decreased investor relations of $380,959 due to scale down of investor relation activities and consultants, decreased share-based compensation of $29,129 due to forfeiture of unvested/vested share options due to termination of consulting arrangement and foreign exchange gains of $293,012 due to the foreign exchange on US-denominated offset by the increased legal expense of $99,572 and increased consulting expense of $14,880.

Outlook

Going forward ProMIS will focus on accelerating or re-initiating programs in our core business area, best-in-class therapeutics for neurodegenerative diseases. In addition, we will continue to expand the application of our unique discovery platform, with which we can "rationally design" antibodies or vaccines to be selective for only mis-folded, pathogenic proteins involved in disease.

In Alzheimer’s we will restart IND enabling work for PMN310, our antibody highly selective for toxic oligomers of amyloid. That selectivity may prove to give PMN310 significant competitive advantages in safety and efficacy over products from Biogen, Lilly, and Eisai that appear to provide benefit slowing the progression of Alzheimer’s disease. In addition, starting with the same proprietary technology that creates selective antibodies ("passive" immunotherapy), we are moving forward our program to create therapeutic vaccines ("active" immunotherapy) targeting toxic oligomers of amyloid. Therapeutic vaccines may be a preferred therapy for Alzheimer’s prevention; the ultimate goal in Alzheimer’s treatment is to detect disease in the ~20 year window before symptoms arise and treat to prevent symptoms of cognitive decline.

In ALS we will advance our program targeting toxic TDP-43 with further in vitro and in vivo validation, and we will build on the significant scientific advances we have made targeting RACK1 (Receptor for A Activated C Kinase 1). We will also further advance our alpha-synuclein program with further in vivo and in vitro validation, targeting diseases like Parkinson’s disease and Multiple System Atrophy.