On March 1, 2022 United Therapeutics Corporation (Nasdaq: UTHR) reported that Michael Benkowitz, President and Chief Operating Officer, will provide an overview and update on the company’s business during a fireside chat session at the Cowen 42nd Annual Health Care Conference (Press release, United Therapeutics, MAR 1, 2022, View Source [SID1234609283]).

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The session will take place virtually on Tuesday, March 8, 2022, from 11:10 a.m. to 11:40 a.m., Eastern Standard Time, and can be accessed via a live webcast on the United Therapeutics website at View Source An archived, recorded version of the session will be available approximately 24 hours after the session ends and can be accessed at the same location for 90 days.

On March 1, 2022 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a leading clinical-stage precision oncology company enabled by its proprietary synthetic lethality approach to the discovery and development of novel therapeutics, reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Repare Therapeutics, MAR 1, 2022, View Source [SID1234609282]).

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"We achieved several key milestones in 2021 to advance our innovative, synthetic lethality-based pipeline across multiple clinical programs," said Lloyd M. Segal, President and Chief Executive Officer of Repare. "We are particularly pleased with important progress in our RP-3500 program and to have initiated our second clinical program, RP-6306, a first-in-class oral PKMYT1 inhibitor, and look forward to starting IND-enabling studies of our Polθ inhibitor program in the first half of 2022 as part of our growing clinical pipeline."

Mr. Segal added: "2022 is expected to be another exciting year for Repare, beginning in the first quarter 2022 with the Phase 2 (Module 2) expansion of the RP-3500 TRESR trial and the initiation of the TRESR Phase 1 monotherapy pediatric module in the first quarter of 2022. We anticipate comprehensive monotherapy TRESR Module 1 clinical data in the second quarter of 2022, and also look forward to initial data from the Phase 1 RP-6306 monotherapy MYTHIC trial in late 2022."

2021 Highlights and 2022 Outlook:

Announced initial Phase 1 RP-3500 monotherapy data from Phase 1/2 TRESR trial at AACR (Free AACR Whitepaper)-NCI-EORTC
Initial Phase 1 results provided clinical proof of concept and validated Repare’s SNIPRx platform for molecular selection of tumors for therapy with RP-3500.
Preliminary data showed that monotherapy RP-3500 appears safe and well tolerated, with compelling early efficacy signals across multiple genotypes and tumor types in heavily pretreated patients.
Recommended Phase 2 dose and schedule for further monotherapy RP-3500 evaluation was determined to be 160mg, taken weekly for 3 days on and 4 days off.
Key TRESR Milestones in 2022:
Initiated a monotherapy Phase 2 TRESR trial of RP-3500 for the treatment of solid tumors with specific synthetic-lethal genomic alterations including those in the ATM gene (ataxia telangiectasia mutated kinase), in tumors with ATM loss of function and in tumors with other STEP2 genomic alteration in February 2022.
Initiated recruitment in a Phase 1 pediatric module of TRESR trial of RP-3500 monotherapy and enrollment of a first patient is expected in the first quarter of 2022.
Comprehensive monotherapy Phase 1 (Module 1) clinical data from 120 patients enrolled in the Phase 1/2 TRESR trial of RP-3500 is expected in the second quarter of 2022.
Determination of recommended Phase 2 dose of RP-3500 in combination with gemcitabine, a trial that began enrolling patients in December 2021, is expected in the second half of 2022.
Early clinical data readout for PARPi combination from Phase 1/2 TRESR trial and ATTACC trial of RP-3500 in combination with, collectively, three marketed PARP inhibitors is expected in the third quarter of 2022.
Advanced RP-6306, a first-in-class, oral PKMYT1 inhibitor both as monotherapy and in combination with gemcitabine and in combination with FOLFIRI
In December 2021, the Company began dosing in a Phase 1 clinical trial of RP-6306, a first-in-class small molecule candidate targeting PKMYT1, in combination with gemcitabine for the treatment of molecularly selected advanced solid tumors.
In February 2022, the Company initiated recruitment in the Phase 1 MINOTAUR clinical trial of RP-6306 in combination with FOLFIRI for the treatment of molecularly selected advanced solid tumors.
Early readout of monotherapy Phase 1 clinical data is expected in late 2022.
Advanced the development of earlier stage discovery programs
The Company is expected to initiate IND-enabling studies of its third synthetic lethal asset, the Polθ inhibitor program, in the first half of 2022.
Recent Corporate Updates:

Appointed Philip Herman to Executive Leadership Team as EVP Commercial & New Product Development
In January 2022, the Company appointed Philip Herman as EVP Commercial & New Product Development. Mr. Herman was most recently Chief Commercial Officer of Y-mAbs Therapeutics and led the successful launch of DANYELZA (naxitamab).
In November 2021, the Company announced the closing of an upsized follow-on public offering yielding aggregate gross proceeds of approximately $101.2 million, or net proceeds of approximately $94.3 million, after deducting underwriting commissions and offering expenses of $0.8 million.
Fourth Quarter and Full Year 2021 Financial Results:

Cash and cash equivalents and marketable securities: Cash and cash equivalents and marketable securities as of December 31, 2021 were $341.9 million, including the proceeds from the follow-on public offering in November 2021. Repare believes its current cash and cash equivalents and marketable securities will be sufficient to fund planned operations through 2023.
Research and development expenses, net of tax credits (Net R&D): Net R&D expenses were $28.0 million and $90.0 million for the three and twelve month periods ended December 31, 2021, respectively, as compared to $12.4 million and $40.1 million for the three and twelve month periods ended December 31, 2020. The increase in Net R&D expenses for the three and twelve month periods were primarily due to increases in development costs related to the Company’s RP-3500 and RP-6306 programs, as well as increases in personnel related expenses, including share-based compensation.
General and administrative (G&A) expenses: G&A expenses were $7.6 million and $26.2 million for the three and twelve month periods ended December 31, 2021, respectively, as compared to $4.8 million and $14.3 million for the three and twelve month periods ended December 31, 2020, respectively. The increase in G&A expenses for the three and twelve month periods were due to personnel related costs, including share-based compensation and, for the twelve month period, the increase was also due to D&O insurance which increased as a result of the Company’s IPO in June 2020.
Net loss: Net loss was $28.3 million, or $0.70 per share and $106.9 million, or $2.83 per share, in the three and twelve month periods ended December 31, 2021, respectively, and $15.3 million, or $0.41 per share and $53.4 million, or $2.66 per share in the three and twelve month periods ended December 31, 2020, respectively.
About Repare Therapeutics’ SNIPRx Platform

Repare’s SNIPRx platform is a genome-wide CRISPR-based screening approach that utilizes proprietary isogenic cell lines to identify novel and known synthetic lethal gene pairs and the corresponding patients who are most likely to benefit from the Company’s therapies based on the genetic profile of their tumors. Repare’s platform enables the development of precision therapeutics in patients whose tumors contain one or more genomic alterations identified by SNIPRx screening, in order to selectively target those patients most likely to achieve clinical benefit from resulting product candidates.

On March 1, 2022 Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs), reported financial results for the fourth quarter and year ended December 31, 2021 and highlighted recent corporate progress (Press release, Avidity Biosciences, MAR 1, 2022, View Source [SID1234609281]).

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"2021 was a pivotal year for Avidity, the field of RNA therapeutics and for the DM1 community as the first patient was dosed with an AOC as part of the AOC 1001 MARINA trial," said Sarah Boyce, president and chief executive officer. "This milestone transitioned us into a clinical-stage biopharmaceutical company and we continue to make great strides with our advancing and expanding pipeline and AOC platform technology. By the end of 2022, we plan to have three programs in three different rare diseases in the clinic with AOC 1001, AOC 1020 and AOC 1044."

"We are in a strong financial position with $406 million at year-end bolstered through approximately $175 million raised in 2021, inclusive of $155 million of net proceeds from our first follow on financing. We are well funded into 2024 which we expect will allow us to complete the MARINA trial, advance the AOC 1044 and AOC 1020 programs into clinical development and to continue to invest in expanding our pipeline and the AOC platform in muscle and beyond," said Mike MacLean, chief financial officer.

2021 Key Highlights

AOC 1001 Achievements

The Company initiated the Phase 1/2 MARINA trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1).
The FDA and the European Medicines Agency (EMA) granted AOC 1001 Orphan Designation and the FDA granted AOC 1001 Fast Track Designation.
The MARINA trial is on track for a preliminary assessment of safety, tolerability and key biomarkers in approximately half of the trial participants in Q4 2022.
Pipeline Advancements

AOC 1044 was named as the clinical development candidate for the Duchenne Muscular Dystrophy (DMD) program targeting Exon 44. AOC 1044 is in IND-enabling studies and is expected to enter the clinic by the end of 2022.
AOC 1020 was named as the clinical development candidate for the facioscapulohumeral muscular dystrophy (FSHD) program. AOC 1020 is in IND-enabling studies and is expected to enter the clinic by the end of 2022.
Avidity also entered a collaboration with the FSHD Clinical Trial Research Network (FSHD CTRN) to support a natural history study called the Motor Outcomes to Validate Evaluations Plus (MOVE+) Study to enhance the understanding of how to utilize whole-body MRI and other tools to identify specific biomarkers for FSHD that can potentially accelerate and support future clinical trial design.
Organizational Highlights

Avidity recently appointed Steve Hughes, M.D. as chief medical officer. Dr. Hughes brings over 20 years of experience in the biotechnology industry and has extensive experience in RNA-based treatments and rare diseases.
Fourth Quarter and Year-End 2021 Financial Results

Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled $405.5 million as of December 31, 2021, which reflects $174.7 million raised in 2021, inclusive of $155.1 million of net proceeds from our first follow on financing.

Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity’s partnership with Eli Lilly and Company and totaled $1.9 million for the fourth quarter of 2021 compared with $2.1 million for the fourth quarter of 2020, and $9.3 million for the full year 2021 compared with $6.8 million for the full year 2020.
Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were $33.0 million for the fourth quarter of 2021 compared with $13.6 million for the fourth quarter of 2020, and $101.2 million for the full year 2021 compared with $37.6 million for the full year 2020. The increases were primarily driven by the advancement of AOC 1001, AOC 1020 and AOC 1044, as well as costs related to the expansion of the company’s overall research capabilities.
General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs and patent filing and maintenance fees. These expenses were $7.4 million for the fourth quarter of 2021 compared with $4.8 million for the fourth quarter of 2020, and $26.2 million for the full year 2021 compared with $13.5 million for the full year 2020. The increases were primarily due to higher personnel costs. The full year increase was also due to higher professional fees and insurance costs.

On March 1, 2022 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a leader in the field of conditionally activated oncology therapeutics, reported fourth quarter and full year 2021 financial results, and provided a business update (Press release, CytomX Therapeutics, MAR 1, 2022, View Source [SID1234609280]).

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"2021 was a successful year of sustained execution and delivery on CytomX’s commitment to destroying cancer differently, highlighted by the significant progress advancing our ongoing Phase 2 studies of praluzatamab ravtansine and CX-2029. Patient enrollment for the Phase 2 study of praluzatamab ravtansine in breast cancer remains on track and an initial data readout is anticipated in the second half of 2022. This approaching milestone follows our recent announcement of preliminary results for CX-2029 in the treatment of squamous lung and head and neck cancers," said Sean McCarthy, D.Phil., chief executive officer and chairman of CytomX. "Our progress with CX-2029, announced in December, underscores our leadership as the first company to report results from a Phase 2 study for a conditionally activated biologic and we look forward to sharing additional data from our ambitious pipeline in the second half of 2022."

Dr. McCarthy added, "2021 was also marked by considerable progress in the earlier CytomX pipeline. CX-904, our conditionally activated T-cell-engaging bispecific antibody targeting EGFR and CD3, was advanced towards a first-in-human study and, as a result, CytomX now has six investigational products in clinical development across three therapeutic modalities. We also deepened our efforts in conditionally activated cytokines, a field in which we see broad and differentiated clinical opportunity. We have entered 2022 intensely focused on pipeline execution and plan to deliver important milestones this year."

Fourth Quarter Business Highlights and Recent Developments

Praluzatamab ravtansine – Praluzatamab ravtansine is a CD166-directed conditionally activated antibody-drug conjugate (ADC) wholly-owned by CytomX. Patient enrollment continued in the three-arm Phase 2 study of praluzatamab ravtansine in breast cancer. Arms A and B are evaluating praluzatamab ravtansine, as monotherapy, in patients with hormone receptor-positive, human epidermal growth factor receptor 2 (HER2)-non-amplified breast cancer and triple-negative breast cancer (TNBC), respectively. Arm C is examining the combination of praluzatamab ravtansine and pacmilimab, CytomX’s proprietary conditionally activated anti-PD-L1 therapeutic candidate, in patients with TNBC.
CX-2029 – CX-2029 is a CD71-directed conditionally activated ADC being co-developed by CytomX and AbbVie. Preliminary data from the Phase 2 expansion study of CX-2029 were announced in December 2021, showing an objective response rate of 18.8 percent and disease control rate of 87.5 percent in 16 efficacy-evaluable patients with sqNSCLC. The safety profile in the expansion phase was consistent with previous Phase 1 observations, with no new safety signals identified and anemia was the most common Grade 3 or higher treatment-related adverse event.
CX-904 – CX-904 is a conditionally activated T-cell-engaging bispecific antibody (TCB) targeting the epidermal growth factor receptor (EGFR) on tumor cells and the CD3 receptor on T cells, and is being developed in collaboration with Amgen. In January 2022, the investigational new drug application for CX-904 was allowed to proceed by the U.S. Food and Drug Administration and CytomX is in the process of initiating a first-in-human Phase 1 study in patients with advanced solid tumors.
Ipilimumab Probody Program – BMS-986249 and BMS-986288 are Probody versions of the anti-CTLA4 antibody, ipilimumab and non-fucosylated ipilimumab, respectively. CytomX’s partner, Bristol Myers Squibb, continued to study BMS-986249 in a randomized Phase 2 study in combination with nivolumab, the anti-PD-1 antibody, versus ipilimumab plus nivolumab in patients with previously-untreated advanced melanoma. This novel combination is also being evaluated in advanced hepatocellular carcinoma, castration-resistant prostate cancer, and TNBC. Bristol Myers Squibb also continued to evaluate BMS-986288, as monotherapy and in combination with nivolumab, in a Phase 1 study in advanced solid tumors.
Preclinical Programs – CytomX continued to work on broadening the potential application of its versatile and tunable Probody platform to other product candidates, including potential first-in-class ADCs directed toward the epithelial cell adhesion molecule (EpCAM/Trop-1) and a conditionally activated cytokine, interferon alpha-2b.
Publication – CytomX continued to publish key results supporting its platform and pipeline, taking the total preclinical and clinical manuscripts published since 2021 to seven. First-in-human data in patients with advanced solid tumors was published in February 2022 in the peer-reviewed journal Clinical Cancer Research, validating CD166, for the first time, as a potential ADC target View Source
Priorities for 2022

Initiate a Phase 1 study of CX-904 in advanced solid tumors in the first half of 2022
Continue patient enrollment in the expansion phase of the Phase 2 study of CX-2029 and provide additional data updates in the second half of 2022
Report initial data from Arms A and B in the Phase 2 study of praluzatamab ravtansine in patients with breast cancer in the second half of 2022
Fourth Quarter and Full Year 2021 Financial Results
Cash, cash equivalents and investments totaled $305.2 million as of December 31, 2021, compared to $316.1 million as of December 31, 2020.

Total revenues were $19.7 million and $69.6 million for the three and twelve months ended December 31, 2021, respectively, compared to $16.4 million and $100.4 million for the corresponding periods in 2020. The increase in total revenues during the three months ended December 31, 2021 was largely related to the CD71 collaboration with AbbVie. The decrease in total revenues for the twelve months ended December 31, 2021 was mainly due to the $40.0 million milestone payment earned in the first quarter of 2020 under the CD71 Co-Development and Licensing Agreement with AbbVie.

Research and development expenses increased by $14.6 million and $1.3 million during the three and twelve months ended December 31, 2021, respectively, to $36.6 million and $114.2 million, compared to $22.0 million and $112.9 million for the corresponding periods in 2020. The increases were primarily driven by personnel expenses, clinical trial activities, and consulting and contract services to support our pre-clinical and clinical portfolio, which, for the twelve months ended December 31, 2021, was mostly offset by a decrease in licensing expenses.

General and administrative expenses increased by $0.3 million and $3.1 million during the three and twelve months ended December 31, 2021, respectively, to $9.5 million and $39.2 million, compared to $9.1 million and $36.0 million for the corresponding periods in 2020. The increase was mainly attributable to increase in personnel related and recruiting expenses.

Conference Call & Webcast Information
CytomX management will host a conference call and a simultaneous webcast today at 5:00 p.m. ET (2:00 p.m. PT) to discuss the financial results and provide a business update. To join the conference call, please dial (877) 809-6037 (domestic) or (615) 247-0221 (international) and reference the conference ID 8454049. A live webcast of the call can be accessed on the Events and Presentations page of CytomX’s website at View Source An archived replay of the webcast will be available on the Company’s website until March 8, 2022.

On March 1, 2022 Allakos Inc. (the "Company") (Nasdaq: ALLK), a biotechnology company developing lirentelimab (AK002) and AK006 for the treatment of allergic and inflammatory diseases, reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, Allakos, MAR 1, 2022, View Source [SID1234609279]).

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Recent Events

Initiated a Phase 2 randomized, double-blind, placebo-controlled study of subcutaneous lirentelimab in patients with moderate-to-severe atopic dermatitis in the fourth quarter of 2021.

Reported topline data from ENIGMA 2, a Phase 3 randomized, double-blind, placebo-controlled study of lirentelimab in patients with eosinophilic gastritis/eosinophilic duodenitis ("EG"/"EoD") in the fourth quarter of 2021.

Reported topline data from KRYPTOS, a Phase 2/3 randomized, double-blind, placebo-controlled study of lirentelimab in patients with eosinophilic esophagitis ("EoE") in the fourth quarter of 2021.
Upcoming Milestones

Hold an End-of-Phase 2 meeting with the FDA during second quarter of 2022 to discuss the Phase 2/3 KRYPTOS data and the development path with subcutaneous lirentelimab in patients with EoE.

Report topline data from the Phase 3 study of lirentelimab in patients with EoD (EoDyssey) in the third quarter of 2022.

Initiate a Phase 2b randomized, double-blind, placebo-controlled study of subcutaneous lirentelimab in patients with chronic spontaneous urticaria in the middle of 2022.

Complete IND-Enabling studies of AK006 during 2022 and initiate the first-in-human study in the first half of 2023.
Fourth Quarter and Full Year 2021 Financial Results

Research and development expenses were $72.9 million in the fourth quarter of 2021 as compared to $28.5 million in the same period in 2020, an increase of $44.4 million. Research and development expenses were $196.3 million for the full year 2021 as compared to $105.5 million in the same period in 2020, an increase of $90.8 million.

General and administrative expenses were $23.2 million in the fourth quarter of 2021 as compared to $15.8 million in the same period in 2020, an increase of $7.4 million. General and administrative expenses were $75.1 million for the full year 2021 as compared to $51.5 million in the same period in 2020, an increase of $23.6 million.

Allakos reported a net loss of $94.4 million in the fourth quarter of 2021 as compared to $44.3 million in the same period in 2020, an increase of $50.1 million. Net loss per basic and diluted share was $1.73 for the fourth quarter of 2021 compared to $0.86 in the same period in 2020. Net loss was $269.9 million for the full year 2021 as compared to $153.5 million in the same period in 2020, an increase of $116.4 million. Net

loss per basic and diluted share was $5.01 for the full year 2021 compared to $3.10 in the same period in 2020.

Allakos ended the fourth quarter of 2021 with $424.2 million in cash, cash equivalents and marketable securities.