On November 3, 2021 Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases, reported that financial results for the third quarter ending September 30, 2021 and provided an update on clinical programs (Press release, NewLink Genetics, NOV 3, 2021, View Source [SID1234594228]).

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"During our third quarter we continued to advance our LUM-201 program for the treatment of pediatric growth hormone deficiency (PGHD), with the opening of a number of new OraGrowtH210 Trial sites," said Rick Hawkins, Chairman and CEO of Lumos Pharma. "The screening and enrollment for both our OraGrowtH210 and OraGrowtH212 Trials are progressing well. We continue to expect the primary outcome data readout for our OraGrowtH210 trial in the second half of 2023. Beyond PGHD, we believe LUM-201 represents a pipeline-in-a-product and are encouraged by discussions with KOLs regarding the potential to expand our LUM-201 platform into other indications currently treated by injectable growth hormone."

Clinical Update:

OraGrowtH210 Trial of LUM-201 in PGHD

Enrollment in the Phase 2 OraGrowtH210 Trial of LUM-201 in PGHD continues, with the majority of the approximately 50 planned sites activated and open for enrollment. OraGrowtH210 is a global clinical trial and is evaluating orally administered LUM-201 in approximately 80 patients diagnosed with PGHD. The objective of the trial is to identify the optimal dose of LUM-201 based on annualized height velocity to be used in a Phase 3 registration trial and to prospectively confirm the preliminary validation of our Predictive Enrichment Marker (PEM) strategy. The Company continues to anticipate six-month data read-out for the OraGrowtH210 Trial in the second half of 2023, with additional 12-month data to be collected.

OraGrowtH212 Trial of LUM-201 in PGHD Initiated Q2 2021

The OraGrowtH212 Trial was initiated in June and is also continuing to enroll patients. OraGrowtH212 is a single site, open-label trial evaluating the pharmacokinetic (PK) and pharmacodynamic (PD) effects of LUM-201 in up to 24 PGHD patients at two dose levels, 1.6 and 3.2 mg/kg/day. Given the open-label design of this trial, the Company has the ability to perform an interim analysis at its discretion. The objective of OraGrowtH212 is to confirm prior clinical data demonstrating the amplified pulsatile release of endogenous growth hormone unique to LUM-201 and its potential for this mechanism of action to contribute to efficacy in PGHD. The primary endpoint is six months of PK/PD and height velocity data, with additional 12-month data to be captured.

LUM-201 Life-Cycle Management

Injectable recombinant human growth hormone (rhGH) and derivative products are currently approved for multiple indications, including PGHD. LUM-201, through its unique mechanism of promoting increased secretion of endogenous GH, may have the potential to be efficacious in many of these indications. Lumos Pharma is in advanced discussions with key opinion leaders and our Clinical and Scientific Advisory Board to expand our LUM-201 pipeline. The Company is actively reviewing the pathway for LUM-201 in other indications including Turner Syndrome, Prader-Willi Syndrome, Idiopathic Short Stature (ISS), and Children Born Small for Gestational Age (SGA).

Financial Results for the Quarter Ended September 30, 2021

Cash Position – Lumos Pharma ended the third quarter on September 30, 2021, with cash and cash equivalents totaling $100.7 million compared to $98.7 million on December 31, 2020. Cash on hand as of the end of Q3 2021 is expected to support operations through the primary outcome data readout from OraGrowtH210 and OraGrowtH212 Trials.
R&D Expenses – Research and development expenses were $4.1 million, an increase of $2.0 million for the three months ended September 30, 2021 compared to the same period in 2020, primarily due to increases of $1.8 million in clinical trial and contract manufacturing expenses, $0.4 million in personnel-related expenses and $0.1 million in stock compensation expenses, offset by a decrease of $0.3 million in supplies and other expenses.
G&A Expenses – General and administrative expenses were $3.4 million, a decrease of $1.8 million for the three months ended September 30, 2021, as compared to the same period in 2020, primarily due to decreases of $1.3 million in personnel-related expenses, $0.3 million in legal, consulting and other expenses and $0.2 million in operating expenses for insurance, rent, supplies, and depreciation.
Net Loss – The net loss for the third quarter ended September 30, 2021 was $7.5 million compared to net income of $1.8 million for the same period in 2020.
Lumos Pharma ended Q3 2021 with 8,357,391 shares outstanding.
Conference Call and Webcast Details

The Company has scheduled a conference call and webcast for 4:30 p.m. ET today to discuss its financial results and to give an update on clinical programs. There will also be a question-and-answer session following management’s prepared remarks.

Access to the live conference call is available five minutes prior to the start of the call by dialing (855) 469-0612 (U.S.) or (484) 756-4268 (international). The conference call will be webcast live and a link to the webcast can be accessed through the Lumos Pharma website at View Source in the "Investors & Media" section under "Events and Presentations" or through this link: View Source To ensure a timely connection, it is recommended that users register at least 10 minutes prior to the scheduled webcast. A replay of the call will be available approximately two hours after the completion of the call and can be accessed by dialing (855) 859-2056 (U.S.) or (404) 537-3406 (international) and using the passcode 2891824. The replay will be available for two weeks from the date of the call.

On November 3, 2021 Alkermes plc (Nasdaq: ALKS) reported plans to present a poster related to nemvaleukin alfa (nemvaleukin), the company’s novel, investigational, engineered interleukin-2 (IL-2) variant immunotherapy, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 36th Annual Meeting, taking place Nov. 10-14, 2021 (Press release, Alkermes, NOV 3, 2021, View Source [SID1234594252]). The poster will include data from the ION-01 study, a phase 2 trial evaluating intravenous nemvaleukin in combination with pembrolizumab in patients with recurrent or metastatic head and neck squamous cell carcinoma that had previously progressed on an anti-PD-(L)1 therapy.

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Details of the presentation are as follows:

Abstract: 432
Title: Nemvaleukin alfa, a novel engineered IL-2 cytokine, in combination with the anti-PD-1 antibody pembrolizumab in patients with recurrent/metastatic head and neck squamous cell carcinoma (ION-01 study)
Presenter: Brian Gastman, M.D., Surgical Director, Melanoma & High-Risk Skin Cancer Program at the Cleveland Clinic, Cleveland, Ohio
Presentation Date: The poster and presentation will be available on the SITC (Free SITC Whitepaper) virtual meeting platform beginning Nov. 12, 2021. The poster is scheduled to be presented onsite at the meeting on Nov. 13, 2021.

About Nemvaleukin Alfa ("nemvaleukin")
Nemvaleukin is an investigational, novel, engineered fusion protein comprised of modified interleukin-2 (IL-2) and the high affinity IL-2 alpha receptor chain, designed to preferentially expand tumor-killing immune cells while avoiding the activation of immunosuppressive cells by selectively binding to the intermediate-affinity IL-2 receptor complex. The selectivity of nemvaleukin is designed to leverage the proven anti-tumor effects of existing IL-2 therapy while mitigating certain limitations.

About the Nemvaleukin Clinical Development Program
ARTISTRY is an Alkermes-sponsored clinical development program evaluating nemvaleukin as a potential immunotherapy for cancer. The ARTISTRY program is comprised of multiple clinical trials evaluating intravenous and subcutaneous dosing of nemvaleukin, both as a monotherapy and in combination with the anti-PD-1 therapy KEYTRUDA (pembrolizumab) in patients with advanced solid tumors. Ongoing trials in the ARTISTRY program include: ARTISTRY-1, ARTISTRY-2, ARTISTRY-3, ARTISTRY-6 and ARTISTRY-7.

ION-01 is a phase 2 trial being conducted in collaboration with Fred Hutchinson Cancer Research Center and is designed to estimate the response rate to intravenous nemvaleukin in combination with the anti-PD-1 therapy pembrolizumab in patients with advanced or recurrent head and neck squamous cell carcinoma who did not achieve complete remission with an anti-PD-(L)1 antibody treatment.

On November 3, 2021 Memgen, Inc. reported that the U.S. Food and Drug Administration (FDA) has accepted its investigational new drug (IND) application for MEM-288, the company’s wholly-owned cancer immunotherapy candidate for the treatment of multiple solid tumors (Press release, Memgen, NOV 3, 2021, View Source [SID1234594268]). Memgen intends to initiate screening of patients by year-end in a Phase 1 first-in-human study.

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"The FDA’s acceptance of our IND for MEM-288 is an important milestone in our pursuit of developing life-saving cancer immunotherapies in collaboration with leading cancer centers including the Moffitt Cancer Center and the Duke Cancer Institute," said Mark Cantwell, Ph.D., Chief Scientific Officer of Memgen. "We expect to initiate this first-in-human study in patients with non-small cell lung cancer, as well as those with other types of cancer, by year-end."

Memgen’s Phase 1 trial is an open-label, dose escalation study designed to evaluate the safety, tolerability, biologic activity and anti-tumor effects of MEM-288. In addition to non-small cell lung cancer (NSCLC), the trial may enroll patients with triple-negative breast cancer, pancreatic cancer, head and neck cancer, melanoma, cutaneous squamous-cell carcinoma, and Merkel cell carcinoma. Except for patients with pancreatic cancer, these are patients whose disease has progressed following treatment with anti-PD-1/PD-L1 therapy, and who have tumor lesions accessible for injection.

Duke Cancer Institute and the H. Lee Moffitt Cancer will be the initial clinical trial sites. Memgen expects patient recruitment to begin by December 31, 2021. Additional information about the study, including patient inclusion and exclusion criteria, can be found at ClinicalTrials.gov under study identifier NCT05076760.

The study will evaluate the oncolytic effect of MEM-288 as well as the activation of the patients’ immune system including T-cells. While the focus of the initial Phase 1 study will be patients with NSCLC, MEM-288 has shown robust anti-tumor effects in 13 different types of cancer. Once safety and tolerability have been evaluated in this trial, Memgen plans to expand clinical development of MEM-288 across multiple cancers and in combination with an immune checkpoint inhibitor.

About MEM-288
MEM-288 is an oncolytic adenovirus encoding transgenes for human interferon beta (IFNß) and the company’s proprietary recombinant chimeric CD40 ligand. MEM-288, armed with Memgen’s proprietary CD40 ligand, leverages a validated target that powerfully activates the patient’s immune system. The company’s proprietary CD40 ligand has been evaluated in earlier clinical studies and demonstrated significant immune activation without the toxicity that had previously prevented development of safe CD40-based therapeutics. Because the CD40 system works as a master "on switch" for the immune system upstream from the immune checkpoint inhibitors, it holds promise in those patients whose disease has progressed despite treatment with immune checkpoint inhibitors.

On November 3, 2021 Insmed Incorporated Inc. (Nasdaq: INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, reported that management will participate in the following virtual investor conferences (Press release, Insmed, NOV 3, 2021, View Source [SID1234594285]):

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The Credit Suisse 30th Annual Virtual Healthcare Conference on November 9, 2021 at 10:30 a.m. ET in a fireside chat
The Stifel 2021 Virtual Healthcare Conference on November 17, 2021 at 8:40 a.m. ET in a fireside chat
The Evercore ISI 4th Annual HealthCONx Virtual Conference on November 30, 2021 at 8:00 a.m. ET in a fireside chat
Each fireside chat will be webcast live and can be accessed by visiting the investor relations section of the Company’s website at www.insmed.com. Each webcast will be archived for a period of 30 days following the conclusion of each live event.

On November 3, 2021 Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to non-opioid pain management and regenerative health solutions, reported financial results for the third quarter of 2021 (Press release, Pacira Pharmaceuticals, NOV 3, 2021, View Source;991.htm [SID1234594354]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Third Quarter 2021 Financial Highlights

Total revenues of $127.7 million
GAAP net income of $17.7 million, or $0.40 per share (basic) and $0.39 per share (diluted)
Adjusted EBITDA (non-GAAP) of $48.1 million
"Throughout the third quarter we made significant progress advancing our corporate growth strategy. We delivered solid topline growth and EXPAREL continues to significantly outpace the recovery of the elective surgery market, which is facing ongoing pandemic-related challenges. This underscores the growing adoption of EXPAREL in outpatient settings, non-elective procedures and beyond. October saw a positive shift in market dynamics, and we expect the fourth quarter to return to more robust year-over-year topline growth," said David Stack, chairman and chief executive officer of Pacira. "Looking ahead, we are on track to achieve our 5-year objective for top-line annual growth in at least the high teens with operating margins greater than 50 percent. Our proposed acquisition of Flexion Therapeutics is expected to further diversify and grow our topline while providing meaningful synergies that deliver substantial accretion to our cash flows and earnings beginning in 2022. Importantly, this proposed acquisition strengthens our leadership position in opioid-sparing pain management."

The company provides weekly EXPAREL utilization and elective surgery data within its investor presentation, which is accessible at investor.pacira.com.

Third Quarter 2021 Financial Results

Total revenues were $127.7 million in the third quarter of 2021, versus the $117.5 million reported for the third quarter of 2020.
EXPAREL net product sales were $121.9 million in the third quarter of 2021, versus the $113.7 million reported for the third quarter of 2020. The number of EXPAREL selling days were 64 in the third quarter of 2021, versus 65 in the third quarter of 2020.
Third quarter 2021 iovera° net product sales were $4.2 million, versus the $2.7 million reported for the third quarter of 2020.
Sales of bupivacaine liposome injectable suspension to a third-party licensee for use in veterinary practice were $0.7 million in the third quarter of 2021, versus the $0.4 million reported for the third quarter of 2020.
Third quarter 2021 royalty and collaborative licensing and milestone revenues were $0.9 million, versus the $0.6 million reported for the third quarter of 2020.
Total operating expenses were $96.3 million in the third quarter of 2021, versus the $99.9 million reported for the third quarter of 2020.
Research and development (R&D) expenses were $11.6 million in the third quarter of 2021, compared to $14.7 million in the third quarter of 2020. R&D expenses included $4.7 million and $5.6 million of product development and manufacturing capacity expansion costs in the third quarters of 2021 and 2020, respectively.
Selling, general and administrative (SG&A) expenses were $48.8 million in the third quarter of 2021, compared to $52.6 million in the third quarter of 2020.
GAAP net income was $17.7 million, or $0.40 per share (basic) and $0.39 per share (diluted), in the third quarter of 2021, compared to GAAP net income of $130.1 million, or $3.03 per share (basic) and $2.94 per share (diluted), in the third quarter of 2020. Included in GAAP net income in the third quarter of 2020 was a $124.6 million income tax benefit related to the release of a valuation allowance on deferred tax assets.
Non-GAAP net income was $32.5 million, or $0.73 per share (basic) and $0.72 per share (diluted), in the third quarter of 2021, compared to non-GAAP net income of $29.9 million, or $0.70 per share (basic) and $0.68 per share (diluted), in the third quarter of 2020.
Adjusted EBITDA (non-GAAP) was $48.1 million in the third quarter of 2021, compared to $34.2 million in the third quarter of 2020.
Pacira ended the third quarter of 2021 with cash, cash equivalents and short-term available-for-sale investments ("cash") of $693.9 million. Cash provided by operations was $60.3 million in the third quarter of 2021, compared to $39.8 million in the third quarter of 2020.
Pacira had 44.5 million basic and 45.5 million diluted weighted average shares of common stock outstanding in the third quarter of 2021.
See "Non-GAAP Financial Information" below.

Recent Business Highlights

Acquisition of Flexion Therapeutics further expanding leadership position in non-opioid pain management. On October 11, 2021, Pacira and Flexion Therapeutics, Inc. announced a definitive agreement for Pacira to acquire Flexion for $8.50 per share in cash, plus one non-tradeable contingent value right (CVR) worth up to $8.00 per share in cash. The CVR is payable (subject to certain terms and conditions) in the event certain sales and/or regulatory milestones are achieved.

New EXPAREL Patents. In October 2021, Pacira received two Notices of Allowance from the United States Patent and Trademark Office. One patent claims chemical composition of EXPAREL and the other claims a novel manufacturing process. After issuance, Pacira will submit the composition patent for listing in the FDA Approved Drug Products with Therapeutic Equivalence Evaluations (the Orange Book). The patents will have an expiration date of January 22, 2041.

Commercial production underway with enhanced EXPAREL manufacturing process at Swindon facility. In September 2021, Pacira announced that it had successfully completed process validation and EXPAREL commercial production is underway at its custom 200-liter manufacturing suite in Swindon, England.

Publication of Phase 3 study of EXPAREL infiltration in pediatric patients undergoing spinal or cardiac surgeries. In September 2021, Pacira announced that full results of its Phase 3 PLAY study of EXPAREL administered via infiltration in pediatric patients undergoing spinal or cardiac surgeries have been published in Journal of Clinical Anesthesia. The study, which was designed to establish the safety and pharmacokinetics (PK) of EXPAREL in a pediatric population, found the PK profile was comparable across age groups and generally consistent with the profile in adult patients.
Financial Guidance

The company’s net product sales were negatively impacted by the COVID-19 pandemic in 2020 due to the significant postponement or suspension in the scheduling of elective surgical procedures resulting from public health guidance and government directives. Elective surgery restrictions began to lift on a state-by-state basis in April 2020, allowing EXPAREL sales to return to year-over-year growth in June 2020. However, while many restrictions have since eased and COVID-19 vaccines become more widely available and administered to the general public, it is still unclear how long it will take the elective surgery market to normalize, or if restrictions on elective procedures will recur due to COVID-19 variant strains or otherwise. In order to provide greater transparency, the company is reporting monthly intra-quarter unaudited net product sales until it has gained enough visibility around the impacts of COVID-19. The company reports the number of selling days for EXPAREL to normalize for differences in reporting period growth rates. The company also provides weekly EXPAREL utilization and elective surgery data within its investor presentation, which is accessible at investor.pacira.com.

Today’s Conference Call and Webcast Reminder

The Pacira management team will host a conference call to discuss the company’s financial results and recent developments today, Wednesday, November 3, 2021, at 8:30 a.m. ET. To participate in the conference call, dial 1-877-845-0779 and provide the passcode 5562638. International callers may dial 1-720-545-0035 and use the same passcode. In addition, a live audio of the conference call will be available as a webcast. Interested parties can access the event through the "Events" page on the Pacira website at investor.pacira.com.

For those unable to participate in the live call, a replay will be available at 1-855-859-2056 (domestic) or 1-404-537-3406 (international) using the passcode 5562638. The replay of the call will be available for one week from the date of the live call. The webcast will be available on the Pacira website for approximately two weeks following the call.

Non-GAAP Financial Information

This press release contains financial measures that do not comply with U.S. generally accepted accounting principles (GAAP), such as non-GAAP net income, non-GAAP net income per common share, non-GAAP cost of goods sold, non-GAAP research and development (R&D) expense, non-GAAP selling, general and administrative (SG&A) expense and adjusted EBITDA, because these non-GAAP financial measures exclude the impact of items that management believes affect comparability or underlying business trends.

These measures supplement the company’s financial results prepared in accordance with GAAP. Pacira management uses these measures to better analyze its financial results, estimate its future cost of goods sold, R&D expense and SG&A expense outlook for 2021 and to help make managerial decisions. In management’s opinion, these non-GAAP measures are useful to investors and other users of our financial statements by providing greater transparency into the operating performance of Pacira and its future outlook. Such measures should not be deemed to be an alternative to GAAP requirements or a measure of liquidity for Pacira. Non-GAAP measures are also unlikely to be comparable with non-GAAP disclosures released by other companies. See the tables below for a reconciliation of GAAP to non-GAAP measures.