On August 4, 2021 Celyad Oncology SA (Euronext & Nasdaq: CYAD) (the "Company") (Brussels:CYAD) (Paris:CYAD) (NASDAQ:CYAD), a clinical-stage biotechnology company focused on the discovery and development of chimeric antigen receptor T cell (CAR T) therapies for cancer, reported an update on its financial results and recent business developments for the fiscal quarter ended June 30, 2021 (Press release, Celyad, AUG 4, 2021, View Source [SID1234585708]).

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"We continue to blaze a path forward by developing new technologies to advance allogeneic CAR T therapies, including our proprietary shRNA platform for allogeneic CAR T production and ‘armored’ CAR capabilities with co-expression of secreting cytokines, starting with IL-18. The innovations we are making through our clinical development pipeline and new technologies were the focus of our R&D day last month. This is an exciting time in our Company’s history as we plan for a steady stream of milestones in the second half of 2021," commented Filippo Petti, Chief Executive Officer of Celyad Oncology. "We plan on announcing multiple clinical updates in the next six months that are expected to help further the progress of our lead programs and proprietary shRNA platform for the development of next-generation allogeneic CAR Ts."

Second Quarter 2021 and Recent Business Highlights

Dr. Charles Morris was appointed as Chief Medical Officer in April 2021.
Preliminary data from the Phase 1 IMMUNICY-1 trial of CYAD-211 for the treatment of r/r MM were announced at the European Hematology Association (EHA) (Free EHA Whitepaper) 2021 Virtual Congress.
Research & Development Day held on July 20, 2021, during which the management team provided:
Updates on the allogeneic CAR T clinical candidates CYAD-211 and CYAD-101.
Highlights from the latest research from its proprietary shRNA platform, including the introduction of CYAD-203 – a novel allogeneic, IL-18-armored CAR T candidate for solid tumor now in IND-enabling studies.
Acquisition of an exclusive license from the Moffitt Cancer Center for an antibody directed to Tumor-associated glycoprotein (TAG-72), which will form the basis of a T cell engager to be used with our shRNA platform technology.
Pipeline Update

CYAD-101 – Allogeneic TIM-based NKG2D CAR T for mCRC

CYAD-101 is the Company’s first-in-class, allogeneic CAR T candidate engineered to co-express a chimeric antigen receptor (CAR) based on the NKG2D receptor and the novel inhibitory peptide TCR Inhibitory Molecule (TIM).

To the Company’s knowledge, CYAD-101 is the first investigational allogeneic CAR T candidate to generate evidence of clinical activity for the treatment of a solid tumor indication. This is based on data reported from the dose-escalation segment of the alloSHRINK Phase 1 trial evaluating CYAD-101 following FOLFOX (combination of 5-fluorouracil, leucovorin and oxaliplatin) preconditioning chemotherapy for the treatment of advanced metastatic colorectal cancer (mCRC).
CYAD-101 following FOLFOX preconditioning chemotherapy was observed to be well-tolerated with no evidence of Graft-versus-Host Disease (GvHD). In addition, two of 15 patients from the dose-escalation segment of the alloSHRINK trial achieved a confirmed partial response (PR). Median progression-free survival (mPFS) and median overall survival (mOS) from the dose-escalation segment was 3.9 months and 10.6 months, respectively. In addition, tumor burden decrease based on RECIST 1.1 criteria was observed in eight of 15 patients, including six of nine patients at the recommended dose of 1×109 CYAD-101 cells per infusion.
In September 2020, the Company entered a clinical trial collaboration with MSD, a tradename of Merck & Co., Inc., Kenilworth, NJ., USA, through a subsidiary. The Company will conduct the Phase 1b KEYNOTE-B79 clinical trial, which will evaluate CYAD-101 following FOLFOX preconditioning chemotherapy, with MSD’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) in refractory mCRC patients with microsatellite stable (MSS) / mismatch-repair proficient (pMMR) disease. Initiation of the KEYNOTE-B79 trial is expected in the fourth quarter of 2021.
CYAD-211 – Allogeneic shRNA-based, anti-BCMA CAR T for r/r MM

CYAD-211 is an investigational, shRNA-based allogeneic CAR T candidate for the treatment r/r MM. CYAD-211 is engineered to co-express a B cell maturation antigen (BCMA) targeting CAR and a single shRNA, which interferes with the expression of the CD3ζ component of the T-cell receptor (TCR) complex.

The Company recently announced preliminary data from the dose-escalation Phase 1 IMMUNICY-1 trial, evaluating the tolerability and clinical activity of a single infusion of CYAD-211 following preconditioning with cyclophosphamide (300 mg/m²) and fludarabine (30 mg/m²) given for three consecutive days.
In June 2021, preliminary data from the Phase 1 IMMUNICY-1 trial was presented at the EHA (Free EHA Whitepaper) congress that demonstrated no dose limiting toxicity (DLT), Graft-versus-Host disease (GvHD) or CAR T-cell-related encephalopathy syndrome (CRES) were observed in the first two dose levels (30×106 and 100×106 cells per infusion) of the trial. Two of the five evaluable patients at the first two dose levels achieved a partial response. In addition, CYAD-211 cells were detected by PCR-based methods in all six patients with evidence of a dose dependent increase in cell engraftment.
In July 2021, the Company reported data from the first patient at dose level three (300×106 cells per infusion) which continues to show dose dependent engraftment of CYAD-211 with no GvHD reported to date.
Enrollment in the trial is ongoing with plans to explore higher doses of preconditioning regimens in future cohorts.
CYAD-203 – Allogeneic shRNA-based, IL-18-armored NKG2D CAR T for Solid Tumors

CYAD-203 is the Company’s first armored CAR T candidate engineered to co-express the cytokine interleukin-18 (IL-18) with the NKG2D CAR receptor. To the Company’s knowledge, this therapy is on track to be the first IL-18 secreting allogeneic CAR T candidate. IL-18 is a proinflammatory cytokine that directly potentiates the anti-cancer activity of CAR T cells while also altering the balance of pro- and anti-inflammatory cells within tumor tissue.

Investigational New Drug (IND)-enabling studies are currently in-progress for the program. Submission of the IND application for CYAD-203 for treatment of solid tumors is expected in mid-2022.
CYAD-02 – Autologous NKG2D CAR-T for r/r AML and MDS

CYAD-02, the Company’s autologous CAR T candidate with shRNA technology that targets the NKG2D ligands MICA and MICB, is currently being evaluated for the treatment of r/r acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) in the Phase 1 CYCLE-1 dose-escalation trial.

To date, eleven patients have received treatment with CYAD-02 in the CYCLE-1 trial, with an enrollment of five patients at dose level three (1×109 cells per infusion).
Preliminary data from the dose level three cohort demonstrated that CYAD-02 was generally well-tolerated. One dose-limiting toxicity was reported at dose level three (cytokine release syndrome, grade 4), leading to expansion of that cohort to six patients. In addition, initial clinical activity has been observed which appears greater than that previously reported from the first-generation autologous NKG2D candidate consistent with a positive contribution from the shRNA-mediated reduction in MICA/B production.
Dose level three cohort of the CYCLE-1 trial is ongoing. Additional safety and efficacy data from the trial are expected by year-end 2021.
Upcoming Anticipated Milestones

Initiation of Phase 1b KEYNOTE-B79 trial evaluating CYAD-101 with KEYTRUDA for advanced mCRC patients with MSS / pMMR disease in fourth quarter of 2021.
Report additional data for the Phase 1 IMMUNICY-1 trial of CYAD-211 for r/r MM by year-end 2021.
Submission of an IND application for CYAD-203 for solid tumors in mid-2022.
Report additional data from the dose-escalation Phase 1 CYCLE-1 trial evaluating CYAD-02 in r/r AML and MDS by year-end 2021.
First Half 2021 Financial Results

Key financial figures for the first half of 2021, compared with the first half of 2020 and full year 2020, are summarized below:

On August 4, 2021 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for cancer and population sequencing, reported financial results for the second quarter ended June 30, 2021 (Press release, Personalis, AUG 4, 2021, View Source [SID1234585707]).

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Second Quarter and Recent Highlights

Record quarterly revenue of $21.7 million in the second quarter of 2021 compared with $19.5 million in the second quarter of 2020, an 11% increase
Revenue of $8.2 million from biopharma and all other customers, excluding the U.S. Department of Veterans Affairs Million Veteran Program (VA MVP), in the second quarter of 2021 compared with $4.7 million in the second quarter of 2020, a 72% increase
Ended the second quarter with cash, cash equivalents, and short-term investments of $328.9 million as of June 30, 2021
Achieved milestone of delivering more than 125,000 whole human genome sequences to the VA MVP
"I’m proud to say that we were able to report record revenue once again this quarter and that we achieved our twentieth consecutive quarter of growth, as we converted an increasing number of orders into revenue. Revenue from biopharma and all other customers grew 72% over the same period of the prior year, and increased sequentially for the seventh consecutive quarter," said John West, Chief Executive Officer. "Recently, we achieved a significant milestone of sequencing our 125,000th whole human genome for the VA MVP, which highlights our expertise and ability to scale. In addition, our development efforts for NeXT Personal, our Minimal Residual Disease (MRD) offering that we expect to launch in 2021, remain on-track."

Second Quarter 2021 Financial Results

Revenue was $21.7 million in the three months ended June 30, 2021, up 11% from $19.5 million in the same period of the prior year.

Gross margin was 37.7% in the three months ended June 30, 2021, compared with 24.0% in the same period of the prior year.

Operating expenses were $23.1 million in the three months ended June 30, 2021, compared with $14.2 million in the same period of the prior year.

Net loss was $15.0 million in the three months ended June 30, 2021 and net loss per share was $0.34 based on a weighted-average basic and diluted share count of 44.0 million, compared with a net loss of $9.3 million and a net loss per share of $0.29 on a weighted-average basic and diluted share count of 31.7 million in the same period of the prior year.

Business Outlook

Personalis expects the following for the third quarter of 2021:

Total revenue to be approximately $22.2 million
Revenue from biopharma and all other customers, excluding VA MVP, to be in the range of $7.5 million to $8.5 million
Net Loss to be in the range of $17 million to $18 million; estimated outstanding shares of 44 million
Personalis expects the following for the full year of 2021:

Total revenue to be approximately $85 million
Revenue from biopharma and all other customers, excluding VA MVP, to be in the range of $33 million to $34 million
Net Loss to be in the range of $65 million to $70 million; estimated outstanding shares of 44 million
Webcast and Conference Call Information

Personalis will host a conference call to discuss the second quarter 2021 financial results after market close on Wednesday, August 4, 2021 at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time. The conference call can be accessed live over the phone by dialing (866) 220-8061 for U.S. callers or (470) 495-9168 for international callers, using the conference ID: 8564509. The live webinar can be accessed at View Source

On August 4, 2021 Certara, the global leader in biosimulation, reported it will host the inaugural New Horizons in Pediatric Drug Development Symposium taking place October 28-29, 2021 (Press release, Certara, AUG 4, 2021, View Source [SID1234585706]). The two-day virtual symposium is being held to bring together thought leaders and innovators in pediatric drug development to share new developments in the field and to collaborate on new ideas to advance pediatric drug development into a new era.

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"We are thrilled to have such an amazing group of speakers and renowned experts to support the advancement of drug development in pediatrics," said Patrick Smith, PharmD., Senior Vice President of Integrated Drug Development at Certara and chair of the organizing committee. "Pediatric drug development is rapidly evolving, with exciting new tools and emerging technologies, which address some of the inherent complexities associated with developing drugs for children. We look forward to the exciting conversations this event will bring forth."

The symposium is spearheaded by an organizing committee comprised of regulatory and drug development experts in the biopharmaceutical industry. Keynote speaker, Lynne Yao, M.D., Director at the FDA Division of Pediatric and Maternal Health, Center for Drug Evaluation and Research, will discuss challenges and opportunities to accelerate global pediatric drug development. Additionally, the agenda features speakers who will speak about the regulatory environment, clinical trial innovation, and the changing landscape with the recent passing of the US FDA’s Research to Accelerate Cures and Equity for Children Act to expand and encourage pediatric drug development, specifically in oncology.

"Being passionate about paediatric development, I am very proud and honoured to be part of this symposium. This is the opportunity to bring together distinguished experts to share their views and experience, which will certainly benefit all involved," said Solange Corriol-Rohou, M.D., Senior Global Regulatory Policy Director at AstraZeneca. "It is amazing to see how paediatric development has evolved and been optimised these last few years. Multi-stakeholder collaboration has increased with the uptake of new tools and methods, such as digital technologies, extrapolation or quantitative approaches, for the benefit of children in need."

On August 4, 2021 BerGenBio’s senior management team reported that it will take place Tuesday 17th August 2021 at 10:00 am CET (Press release, BerGenBio, AUG 4, 2021, View Source [SID1234585705]).

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The live webcast link will be available at www.bergenbio.com in the Investors/Financial Reports section. A recording will be available shortly after the webcast has finished.

The second quarter report and presentation will be available on the Company’s website in the Investors/Financial Reports section from 7:00 am CET the same day.

On August 4, 2021 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported its financial results for the second quarter of 2021 and recent business achievements (Press release, Ionis Pharmaceuticals, AUG 4, 2021, View Source [SID1234585703]).

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"Since our last quarterly update, we continued to execute on our strategic objectives to prepare for multiple Ionis commercial launches, expand our drug delivery capabilities and advance new products towards the market. Biogen completed dosing in the tofersen Phase 3 VALOR study and began offering tofersen to SOD1-ALS patients on an individual compassionate use basis. We achieved full enrollment in the eplontersen Phase 3 NEURO-TTRansform study and 50 percent enrollment in the pelacarsen Phase 3 Lp(a) HORIZON study. Additionally, we licensed Bicycle Therapeutics’ technology to expand the capabilities of our LICA technology," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "Looking ahead, we expect data from multiple pipeline programs, including additional data supporting the potential for our IONIS-PKK-LRx program to change the standard of care for patients with hereditary angioedema. And by this fall, we expect data from the Phase 3 VALOR study of tofersen in patients with SOD1-ALS. If results from the VALOR study are positive, we expect tofersen to be our next commercial medicine. These key recent achievements and upcoming catalysts keep us on track for a regular cadence of Phase 3 data and new drug applications, leading to 12 or more products on the market in 2026."

Second Quarter 2021 and Recent Summary Financial Results

Second quarter results reflect focus on Ionis’ strategic objectives
$126 million in total revenues
$154 million of operating expenses on a non-GAAP basis(1) and $199 million on a GAAP basis
Net loss of $36 million on a non-GAAP basis(1) and $81 million on a GAAP basis
Well capitalized with cash and investments of $2.1 billion at the end of the second quarter
"In addition to advancing our pipeline and expanding our drug discovery capabilities, we have taken multiple steps to streamline our operations in support of our wholly owned medicines. We have completed the integration of Akcea, entered distribution arrangements with Sobi and restructured our commercial operations. These steps enabled us to unlock significant resources that we are redirecting towards our highest priority programs," said Elizabeth L. Hougen, chief financial officer of Ionis. "We remain on track to achieve our 2021 revenue guidance of more than $600 million. We continue to expect increased R&D revenue in the second half of this year. Already in the third quarter, we earned $25 million from Novartis for the pelacarsen enrollment milestone. We are revising our 2021 operating expense and net loss guidance because of our license of Bicycle’s technology. Importantly, we remain well-capitalized with the resources we need to achieve our strategic objectives."

Revised 2021 Financial Guidance

All non-GAAP amounts referred to in this press release exclude non-cash compensation expense related to equity awards and expenses related to the Akcea acquisition and restructured commercial operations and the related tax effects. Please refer to the section below titled "Financial Impacts of Akcea Acquisition and Restructured Commercial Operations" for a summary of the costs specific to these transactions. Additionally, please refer to the detailed reconciliation of non-GAAP and GAAP measures, which is provided later in this release.

Second Quarter 2021 Marketed Products Highlights

SPINRAZA: The global market leader for the treatment of spinal muscular atrophy (SMA) patients of all ages
$500 million in worldwide sales in the second quarter
More than 11,000 patients worldwide on therapy at the end of the second quarter across commercial, expanded access and clinical trial settings
New data presented at CureSMA reinforce the potential for higher-dose SPINRAZA to improve SMA patient outcomes and further support SPINRAZA’s potential long-term benefit for SMA patients of all ages
TEGSEDI and WAYLIVRA: important medicines approved for the treatment of patients with severe rare diseases
Successfully completed the transition of North American TEGSEDI operations to Swedish Orphan Biovitrum AB (Sobi)
Second Quarter 2021 and Recent Events

Phase 3 Pipeline: Six Phase 3 studies on track for a regular cadence of data readouts beginning this year
Completed dosing in the Phase 3 VALOR study of tofersen in patients with SOD1-ALS, with data expected by this fall
Opened individual compassionate use access for SOD1-ALS patients with the most rapidly progressive disease
Achieved full enrollment in the Phase 3 NEURO-TTRansform study of eplontersen in patients with TTR polyneuropathy, with data expected by mid-2022
Achieved 50 percent enrollment in the Phase 3 Lp(a) HORIZON study of pelacarsen for patients at risk for Lp(a)-driven cardiovascular disease, resulting in a $25 million payment from Novartis
Advanced ION363 into a Phase 3 study in patients with FUS-ALS

Mid-stage Pipeline: multiple medicines with potential to change the standard of care for patients with severe diseases
Continued to advance the Phase 2b RE-THINc ESRD study of IONIS-FXI-LRx, with data expected in the first half of 2022
Reported data from the Phase 1/2 study of IONIS-MAPTRx in patients with Alzheimer’s disease, demonstrating durable, time and dose-dependent reductions in CSF tau protein; IONIS-MAPTRx was generally well tolerated
Advanced the ongoing Phase 2 study of ION541 in patients with ALS regardless of family history, resulting in a $10 million payment from Biogen
Advanced ION224 into a Phase 2b study in patients with non-alcoholic steatohepatitis (NASH)
Advanced ION373 into the Phase 2 portion of a pivotal study in patients with Alexander disease
Strategic and Business Events
Entered a license agreement with Bicycle Therapeutics for exclusive rights to Bicycle’s peptide technology to expand the capabilities of Ionis’ LICA technology
Announced changes to the Ionis board of directors
Joseph Loscalzo, M.D., Ph.D., appointed as chairman and Allene M. Diaz as a member of the board
Joseph Wender appointed as lead independent director
Ionis founder and executive chairman, Stanley T. Crooke M.D., Ph.D. and Breaux B. Castleman retired from the board
Upcoming 2021 Pipeline Catalysts(2)

Second Quarter 2021 Financial Results

Revenue

Ionis’ revenue was comprised of the following (amounts in millions):

In the second quarter of 2021, the Company successfully completed the transition of its TEGSEDI operations in North America to Sobi. As a result, the Company’s commercial revenue from product sales shifted to distribution fees based on net sales generated by Sobi.

The Company’s R&D revenue decreased in the second quarter of 2021 compared to the same period last year primarily because the Company earned more milestone payments in the second quarter of 2020 than the same period this year. The Company expects its R&D revenue to increase in the second half of 2021 compared to the first half as its partnered programs advance. Already in the third quarter of 2021, the Company earned a $25 million milestone payment from Novartis when Novartis achieved 50 percent enrollment in the Phase 3 Lp(a) HORIZON study of pelacarsen.

Financial Impacts of Akcea Acquisition and Restructured Commercial Operations

In the second quarter of 2021, the Company incurred $15 million of costs in conjunction with the Akcea acquisition and restructuring of the Company’s commercial operations. The Company excluded these costs from its non-GAAP amounts for the period. Refer to the detailed reconciliation of non-GAAP and GAAP measures that is provided later in this release.

Operating Expenses

Ionis’ operating expenses for the second quarter of 2021 increased slightly compared to the same period last year driven by an increase in R&D expenses, partially offset by a decrease in SG&A expenses. Ionis’ increased R&D expenses were primarily driven by the Company’s investments in advancing its late-stage wholly owned pipeline. Ionis’ decreased SG&A expenses were primarily from operating efficiencies achieved from integrating Akcea and restructuring the Company’s commercial operations.

Net Loss Attributable to Ionis Common Stockholders

Ionis’ net loss attributable to Ionis’ common stockholders for the second quarter of 2021 increased compared to the same period in the prior year for the reasons discussed above. Additionally, the Company recognized an $8.6 million non-cash loss from the early retirement of a significant portion of its 1 percent senior convertible notes.

Balance Sheet

Ionis ended June 2021 with cash, cash equivalents and short-term investments of $2.1 billion, compared to $1.9 billion at December 31, 2020. In April 2021, Ionis issued $632.5 million of 0 percent senior convertible notes due in April 2026 and repurchased $247.9 million of its 1 percent senior convertible notes. The Company’s remaining $62 million of 1 percent senior convertible notes mature in November 2021.

The Company revised its 2020 amounts to reflect the simplified convertible instruments guidance the Company adopted retrospectively on January 1, 2021.

Webcast

Today, at 11:30 a.m. Eastern Time, Ionis will conduct a live webcast to discuss this earnings release and related activities. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address.