On August 4, 2021 EQRx, a company committed to developing and delivering important new medicines at radically lower prices, and AbCellera (Nasdaq: ABCL), a technology company with a centralized operating system for next-generation antibody discovery, reported a collaboration agreement for the discovery and development of therapeutic antibodies (Press release, EQRx, AUG 4, 2021, View Source [SID1234585740]). The collaboration will leverage AbCellera’s AI-powered antibody discovery technology to search and analyze natural immune responses to identify novel antibody drug candidates for multiple targets. The partnership, which spans several therapeutic areas, will help to rapidly expand EQRx’s early-stage pipeline of novel medicines. The deal also includes the option for additional investment from AbCellera at progressive stages of preclinical development, clinical development, and commercialization in exchange for an increased share of product sales. The initial programs will focus on targets in oncology and immunology.

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"This collaboration with AbCellera represents another important step to continue to expand our portfolio as we seek to lead the way in the future of affordable, accessible and high-impact medicines," said Carlos Garcia-Echeverria, Ph.D., chief of Rx creation at EQRx. "AbCellera’s operating system for antibody discovery addresses each step in the process to dramatically improve the cycle time, cost, and probability of success, and we look forward to working together to advance unique clinical candidates against key disease targets."

"AbCellera exists to create value by connecting technology with innovation of all kinds – innovation in biology, in new drug modalities, and in new commercial models. We’re proud to partner with EQRx on their bold mission to reimagine drug development and bring medicines to patients faster and at lower cost," said Carl Hansen, Ph.D., CEO of AbCellera. "This partnership is yet another example of how we can apply our business model, creating greater value and alignment through deals that include the option to invest in programs."

On August 4, 2021 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, reported that Emil D. Kakkis, M.D., Ph.D., the company’s Chief Executive Officer and President will participate in a panel titled Miss Con-GENE-iality – Updates in Gene Tx on Wednesday, August 11, 2021 at the Wedbush PacGrow Healthcare Conference at 12:00 PM ET (Press release, Ultragenyx Pharmaceutical, AUG 4, 2021, View Source [SID1234585739]).

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The live and archived webcast of the presentation will be accessible from the company’s website at View Source The replay of the webcast will be available for 90 days.

On August 4, 2021 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition") reported it will host a conference call on Thursday, August 12, at 8:00 a.m. Eastern time to discuss its financial and operating results for the second quarter of 2021, in addition to providing a business update (Press release, VolitionRX, AUG 4, 2021, View Source [SID1234585738]).

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Cameron Reynolds, President and Chief Executive Officer of Volition, will host the call along with Terig Hughes, Chief Financial Officer, Dr. Tom Butera, Chief Executive Officer of Volition Veterinary, and Scott Powell, Executive Vice President, Investor Relations. The call will provide an update on recent developments and Volition’s activities, including details of new and ongoing clinical trials, important events which have taken place in the second quarter of 2021, and milestones for 2021 and beyond.

A live audio webcast of the conference call will also be available on the investor relations page of Volition’s corporate website at View Source In addition, a telephone replay of the call will be available until August 26, 2021. The replay dial-in numbers are 1-844-512-2921 (toll-free) in the U.S. and Canada and 1-412-317-6671 (toll) internationally. Please use replay pin number 13722250.

On August 4, 2021 Vericel Corporation (NASDAQ:VCEL), a leader in advanced therapies for the sports medicine and severe burn care markets, reported financial results and business highlights for the second quarter ended June 30, 2021 (Press release, Vericel, AUG 4, 2021, View Source [SID1234585737]).

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Second Quarter 2021 Financial Highlights

Total net revenue of $39.5 million, an increase of 97% compared to the second quarter of 2020 and 51% compared to the second quarter of 2019
MACI net revenue of $26.5 million, Epicel net revenue of $12.2 million, and NexoBrid revenue of $0.8 million related to the U.S. Biomedical Advanced Research and Development Authority (BARDA) procurement for emergency response preparedness
Gross margin of 68%, compared to 57% in the second quarter of 2020
Net loss of $3.8 million, or $0.08 per share, compared to $8.3 million, or $0.18 per share, in the second quarter of 2020
Non-GAAP adjusted EBITDA of $7.8 million, or 20% of net revenue, compared to adjusted EBITDA loss of $3.5 million in the second quarter of 2020
Operating cash flow of $4.8 million
As of June 30, 2021, the Company had $116 million in cash and investments, compared to $100 million as of December 31, 2020, and no debt
Business Highlights and Updates

MACI net revenue growth of 76% compared to the second quarter of 2020 and 27% compared to the second quarter of 2019
MACI biopsy growth of more than 50% in the first half of 2021 compared to the same period in 2020, with a record quarterly high in the number of biopsies and the number of surgeons taking biopsies in the second quarter
Record quarterly Epicel revenue, with growth of 148% compared to the second quarter of 2020 and 128% compared to the second quarter of 2019
Record quarterly high in the number of Epicel biopsies and grafting burn centers
"The Company continued to execute extremely well in the second quarter as we delivered another quarter of strong financial and commercial results," said Nick Colangelo, President and CEO of Vericel. "Based on the strength of the underlying growth drivers for MACI and Epicel, we believe that the Company is well-positioned to continue driving sustainable penetration into the addressable markets for both products in the years ahead."

Full-Year 2021 Financial Guidance

Total net revenue now expected to be in the range of $168-$171 million, compared to previous guidance of approximately $165-$168 million
Adjusted EBITDA margin now expected to be in the range of 23% to 25%, compared to previous guidance of 21.5% to 23.5%
Maintaining gross margin guidance of 70% to 71% and estimated operating expenses of approximately $115 million
Second Quarter 2021 Results
Total net revenue for the quarter ended June 30, 2021 increased 97% to $39.5 million, compared to $20.0 million in the second quarter of 2020. Total net product revenue for the quarter included $26.5 million of MACI (autologous cultured chondrocytes on porcine collagen membrane) net revenue and $12.2 million of Epicel (cultured epidermal autografts) net revenue, compared to $15.1 million of MACI net revenue and $4.9 million of Epicel net revenue, respectively, in the second quarter of 2020. Total net revenue for the quarter also included $0.8 million of revenue related to the procurement of NexoBrid (concentrate of proteolytic enzymes enriched in bromelain) by BARDA for emergency response preparedness.

Gross profit for the quarter ended June 30, 2021 was $26.9 million, or 68% of net revenue, compared to $11.4 million, or 57% of net revenue, for the second quarter of 2020.

Total operating expenses for the quarter ended June 30, 2021 were $30.6 million, compared to $19.7 million for the same period in 2020. The increase in operating expenses was primarily due to an increase in stock-based compensation expense driven by share price appreciation over the past year and lower spend in the prior year due to COVID-19-related factors.

Net loss for the quarter ended June 30, 2021 was $3.8 million, or $0.08 per share, compared to $8.3 million, or $0.18 per share, for the second quarter of 2020.

Non-GAAP adjusted EBITDA for the quarter ended June 30, 2021 was $7.8 million, or 20% of net revenue, compared to an adjusted EBITDA loss of $3.5 million in the second quarter of 2020. A table reconciling non-GAAP measures is included in this press release for reference.

As of June 30, 2021, the Company had $116 million in cash and investments, compared to $100 million as of December 31, 2020, and no debt.

Conference Call Information
Today’s conference call will be available live at 8:30am Eastern Time and can be accessed through the Investor Relations section of the Vericel website at View Source." target="_blank" title="View Source." rel="nofollow">View Source A slide presentation with highlights from today’s conference call will be available on the webcast and in the Investor Relations section of the Vericel website. Please access the site at least 15 minutes prior to the scheduled start time in order to download the required audio software, if necessary. To participate in the live call by telephone, please call (877) 312-5881 and reference Vericel Corporation’s second quarter 2021 investor conference call. If calling from outside the U.S., please use the international phone number (253) 237-1173.

If you are unable to participate in the live call, the webcast will be available at View Source until August 4, 2022. A replay of the call will also be available until 11:30am (EDT) on August 11, 2021 by calling (855) 859-2056, or from outside the U.S. by calling (404) 537-3406. The conference ID is 2969646.

On August 4, 2021 eFFECTOR Therapeutics, Inc. (eFFECTOR), a leader in the development of selective translation regulation inhibitors (STRIs) for the treatment of cancer, reported the initiation of dosing in the Phase 2a expansion portion of an ongoing Phase 1/2 trial of zotatifin (eFT226) in solid tumors (Press release, eFFECTOR Therapeutics, AUG 4, 2021, View Source [SID1234585736]). This followed conclusion of the Phase 1 dose escalation portion of the trial and selection of a recommended Phase 2 dose (RP2D). eFFECTOR expects to initiate multiple indication-specific expansion cohorts in ER+ breast cancer and KRAS-mutant non-small cell lung cancer (NSCLC). Zotatifin will be evaluated both as a single agent and in combination with targeted therapies in each indication.

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"Following successful conclusion of dose escalation and selection of the RP2D, we are expanding our zotatifin program with the initiation of several Phase 2a expansion cohorts in cancers with substantial unmet need, including ER+ breast cancer and KRAS-mutant NSCLC," said Steve Worland, Ph.D., president and CEO of eFFECTOR. "Zotatifin has been generally well-tolerated in our clinical trials to date and showed very compelling preclinical activity, including in combination with palbociclib for breast cancer and in combination with sotorasib for KRAS-mutant NSCLC, two indications that we plan to evaluate in expansion cohorts."

The Phase 2a expansion cohorts will evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD) and antitumor activity of zotatifin in subjects with biomarker-positive solid tumor malignancies, including ER+ breast cancer and KRAS-mutant NSCLC. Each of the Phase 2a monotherapy and combination expansion cohorts will utilize a Simon’s Two Stage design, in which seven patients will be enrolled in the first stage of the trial and assessed for activity prior to advancing to the second stage of the trial. Zotatifin will be administered as a 1-hour intravenous (IV) infusion at the selected RP2D of 0.07 mg/kg on Day 1 and Day 8 of a 21-day cycle. eFFECTOR expects to present additional data from the Phase 1 dose escalation portion of the trial, as well as preliminary response data from Phase 2a expansion cohorts, at a medical conference in 2022.

The primary objective of this study is to assess the safety, tolerability and activity of zotatifin as a monotherapy treatment and in combination with targeted agents in biomarker-specific patient populations. If positive activity is observed in a Phase 2a expansion cohort, the company plans to evaluate zotatifin, potentially as a combination in a randomized trial against a relevant comparator control group, or potentially in a single-arm monotherapy trial following demonstration of an appropriate overall response rate (ORR) in the Phase 2a expansion cohort.

About Zotatifin (eFT226)

Zotatifin is a potent and sequence-selective inhibitor of eukaryotic translation initiation factor 4A (eIF4A) mediated translation. eIF4A is responsible for unwinding complex structures in the non-coding 5’ untranslated region of messenger RNA. Zotatifin is designed to block this process, thereby inhibiting the translation of mRNAs encoding several important oncogenes and survival factors, including receptor tyrosine kinases (RTKs), KRAS, Cyclin D, CDK4/6 and MYC. In vivo studies have shown potent tumor regression in multiple tumor models dependent on these factors, including non-small cell lung cancer (NSCLC) and breast cancer. Since zotatifin inhibits the translation of mRNA by acting in the non-coding region of mRNAs, it is not limited to specific KRAS activating mutation subtypes such as KRAS G12C or KRAS G12D. Zotatifin is currently being evaluated as an intravenous (IV) infusion in a Phase 2a clinical trial that will include patients with breast cancer and KRAS-mutant NSCLC and in a Phase 1b clinical trial in patients with mild to moderate COVID-19 infections pursuant to grant sponsorship by the Defense Advanced Research Projects Agency (DARPA).