On March 9, 2021 Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage biopharmaceutical company focused on therapeutics for rare diseases,reported financial results for the year ended December 31, 2020 and provided an update on clinical activities and financial guidance for 2021 (Press release, NewLink Genetics, MAR 9, 2021, View Source [SID1234576364]).

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"The fourth quarter and full year 2020 were marked by significant achievements for Lumos Pharma," commented Rick Hawkins, Chairman, CEO and President of Lumos Pharma. "From the completion of our merger last spring, the subsequent sale of our PRV providing significant non-dilutive funds, to the initiation of our Phase 2b OraGrowtH210 Trial evaluating our orally administered therapeutic for PGHD, Lumos Pharma has built a solid foundation to advance our clinical and corporate strategy targeting PGHD, diseases of growth hormone deficiency, and other rare diseases in the coming year."

Clinical Updates

LUM-201 Data Published in Journal of Endocrine Society (JES) – The manuscript, "Development of a Predictive Enrichment Marker for Oral GH Secretagogue LUM-201 in Children with Growth Hormone Deficiency," by Bright, G., MD, et al, was published in the Journal of Endocrine Society late February. This peer-reviewed analysis of data from prior studies of LUM-201 in pediatric growth hormone deficiency (PGHD) supports the utility of specific predictive enrichment markers (PEMs) in identifying PGHD patients likely to respond to LUM-201. The two PEMs identified after a single dose of LUM-201, baseline IGF-1 cut-off level > 30 ng/ml and peak GH level ≥ 5 ng/mL, are being used to qualify PGHD patients for enrollment in our OraGrowtH210 Trial.
Predictive Enrichment Markers Define Subset of Moderate Growth Hormone Deficiency – The manuscript, "Corroboration Between Prediction Enrichment Markers for Height Velocity to rhGH and an Oral GH Secretagogue Treatment in Children with Moderate GHD," by Blum, W., PhD, et al, was published in the Journal of Endocrine Society late February. This peer-reviewed analysis of data on children with growth hormone deficiency (GHD) in a large legacy database (GeNeSIS data) corroborates that approximately 60% of the total pediatric GHD patient population meet the definition of moderately GHD deficient (PEM-positive) and are likely to respond to a growth hormone secretagogue.
Poster to be Presented at ENDO 2021 – The poster entitled, "LUM-201 Elicits Greater GH Response than Standard GH Secretagogues in Pediatric Growth Hormone Deficiency," (abstract 7102) will be presented at the Endocrine Society 2021 Annual Meeting, March 20th-23rd.
Phase 2b OraGrowtH210 Trial Advances – The Phase 2b OraGrowtH210 Trial initiated in Q4 2020 continues to advance. This global Phase 2b trial will evaluate orally administered LUM-201 in approximately 80 patients diagnosed with PGHD. The purpose of this trial will be to prospectively confirm our Predictive Enrichment Marker (PEM) strategy and to identify the optimal dose of LUM-201 to be used in a Phase 3 registration trial. The Company continues to anticipate data read-out for the OraGrowtH210 Trial mid-year 2022.
OraGrowtH212 Trial (PK/PD study) of LUM-201 in PGHD Initiation Anticipated Q2 2021 – This study will evaluate the pharmacokinetic and pharmacodynamic (PK/PD) effects of two dose levels of LUM-201 (1.6 and 3.2 mg/kg/day) in approximately 24 PGHD patients at a single specialized clinical site. The purpose of this study will be to confirm prior preclinical and clinical data supporting the increased pulsatile release of endogenous growth hormone peaks that characterizes the unique mechanism of action of LUM-201. Recently we were informed of a fire at the San Borja Arriaran Hospital in Santiago, Chile, which is the location of the OraGrowtH212 trial. While we had originally expected to initiate this trial in Q1 2021, we now anticipate the initiation of the OraGrowtH212 Trial to occur in Q2 2021 due to potential delays as the hospital addresses this incident. Our investigator’s clinic was not directly involved in the fire, and we continue to work with our local contacts to advance the trial. As we have previously stated, this trial is not on the critical path for regulatory approval of LUM-201, and we do not anticipate the fire will cause any delays to our previously stated regulatory approval timeline. We are exploring alternate sites to conduct the trial in the event that the original site is unable to proceed in a timely manner.
OraGrowtH211 Trial, a Long-Term Extension Study, is Announced – Lumos Pharma announced the OraGrowtH211 Trial, an extension study to determine the long-term safety, PK/PD markers and growth outcomes attributable to LUM-201 administered to children with growth hormone deficiency. The OraGrowtH211 Trial will be open to all eligible PGHD patients who have completed OraGrowtH210, OraGrowtH212 or other subsequent LUM-201 trials.
Business Development – The Company continues to pursue opportunities to expand our rare disease pipeline through the in-licensure or acquisition of another novel therapeutic candidate for those suffering from rare diseases.
Corporate Updates

Received Final Tranche of Funds from PRV Sale – In January 2021, Lumos received the second and final tranche of $26.0 million from the total $60.0 million due to the Company from the PRV sale. We anticipate these funds will serve as additional capital to support the expansion of the Company’s pipeline through its business development efforts.
Financial Guidance for 2021 – The Company anticipates average cash use of approximately $8.0 to $9.0 million per quarter through 2021.
Management Changes – As previously disclosed, Eugene Kennedy, MD, Chief Medical Officer (CMO), departed Lumos Pharma March 4, 2021 to join a privately held company focused on developing therapeutics for patients suffering from cancer. Lumos Pharma will conduct a search for his replacement. John McKew, PhD, COO and CSO together with George Bright, MD, VP, Clinical Development and a pediatric endocrinologist, will cover all CMO responsibilities in the interim.
Appointed New Board Member with Rare Disease Background – February 16, 2021, Lumos Pharma announced the appointment of new Board member, An van Es-Johansson, M.D., with a wealth of experience in rare diseases. Dr. van Es-Johansson recently served as the Chief Medical Officer and Head of Development for AlzeCure Pharma, a Swedish pharmaceutical company with a primary focus on Alzheimer’s disease, where she now serves as a senior advisor. Dr. van Es-Johansson’s early work in the life science industry focused on the clinical development of recombinant human growth hormone (rhGH) therapeutics for Turner Syndrome and other endocrine disorders at both Eli Lily and Pharmacia Upjohn. Since then, Dr. van Es-Johansson has held leadership roles at several large and small biopharmaceutical companies and currently serves on the Board of Directors at Medivir AB, Savara Pharmaceuticals, PLUS Therapeutics, and Agendia BV. Dr. van Es-Johansson received a M.D. from Erasmus University, Rotterdam, The Netherlands.
Financial Results for the Year Ended December 31, 2020

Cash Position – Lumos Pharma ended the year on December 31, 2020, with cash and cash equivalents totaling $98.7 million compared to $5.0 million on December 31, 2019 and pro forma December 31, 2019 cash of $95.5 million, inclusive of NewLink Genetics. The Company expects its cash on hand is sufficient to fund current operations through the read-out of our Phase 2b OraGrowtH210 Trial and completion of the OraGrowtH212 Trial.
R&D Expenses – Research and development expenses for the year ended December 31, 2020 were $9.2 million, an increase of $3.5 million from $5.7 million for the same period in 2019. The increase is primarily due to increases of $2.4 million in clinical trial expenses, $1.4 million in personnel-related and stock compensation expenses, $0.8 million in supplies and other expenses and $0.4 million in write-off of the acquired NewLink’s in-process research and development costs, offset by a decrease of $1.5 million in contract manufacturing expense.
G&A Expenses – General and administrative expenses for the year ended December 31, 2020 were $17.3 million, an increase of $13.1 million from $4.2 million for the same period in 2019. The increase was due primarily to increases of $7.0 million in personnel-related and stock compensation expenses, $4.2 million in operating expenses for insurance, rent, supplies, and depreciation expenses, $1.6 million due to the Merger related expenses and $0.6 million in legal and consulting expenses, offset by a decrease of $0.3 million in travel expenses.
Net Loss – The net loss for the year ended December 31, 2020 was $5.7 million compared to a net loss of $9.7 million for the same period in 2019.
Lumos Pharma ended Q4 2020 with 8,305,269 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 and business development activities. 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 3735869. The replay will be available for two weeks from the date of the call.

On March 9, 2021 GeneCentric Therapeutics, a company making precision medicine more precise, reported the granting of Patent No. 10,934,595 by the United States Patent and Trademark Office (USPTO), protecting another one of its novel RNA-based lung cancer gene signatures (Press release, GeneCentric Therapeutics, MAR 9, 2021, View Source [SID1234576311]). This new patent provides further demonstration of the novel RNA-based technology achievements of the GeneCentric team and its growing pipeline of predictive response signatures developed using its Tumor and Immune Micro-Environment (T(I)ME) Explorer platform.

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The patent covers the use of reduced RNA gene sets to characterize tumors from patients diagnosed with non-squamous non-small cell lung cancer (NSCLC), which are foundational to the development of the company’s predictive response signatures and diagnostics for this important patient population. The use of RNA-based gene signatures allows for a deeper understanding of the T(I)ME than DNA mutations alone, often allowing for the identification of a broader patient population that may benefit from targeted therapy compared to use of traditional DNA mutation analysis.

"Those of us who study the genomics of lung cancer have known for more than a decade that there is a rich and reproducible story to be told though gene expression assays," said Neil Hayes, MD, GeneCentric co-founder, and Scientific Director, University of Tennessee West Institute for Cancer Research. "The signatures inherent to clinically relevant subgroups are opaque to the microscope but revealed through the genome. We are thrilled to open the door to applications applicable to prognosis and response for traditional cytotoxic chemotherapy, targeted therapies, and emerging immune-oncologic approaches."

GeneCentric continues to make significant progress with its growing pipeline of RNA-based predictive response signatures and molecular diagnostics, with the lung cancer program as its cornerstone. Further updates will be provided as the company presents additional data validating its novel RNA-based signatures and diagnostic tests across multiple tumor types at upcoming scientific meetings and in peer-reviewed publications.

"This is an important time for GeneCentric as we clinically validate and prepare to launch this diagnostic test," said Dr. Mike Milburn, President, and CEO of GeneCentric Therapeutics. "The granting of this latest lung cancer patent by the USPTO demonstrates the utility and uniqueness of our growing pipeline of RNA-based diagnostics,"

On March 9, 2021 TG Therapeutics, Inc. (NASDAQ: TGTX) reported the publication of results from the UNITY-NHL Phase 2b trial evaluating UKONIQ (umbralisib), the Company’s inhibitor of PI3k-delta and CK1-epsilon, in patients with relapsed or refractory indolent non-Hodgkin Lymphoma (NHL) in the Journal of Clinical Oncology (JCO) (Press release, TG Therapeutics, MAR 9, 2021, View Source [SID1234576327]).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer stated, "We are extremely pleased that the results of the UNITY-NHL trial which supported the recent approval of umbralisib, now called UKONIQ, in relapsed or refractory marginal zone and follicular lymphoma, have been published in the prestigious Journal of Clinical Oncology. The data published yesterday, and previously presented at the ASH (Free ASH Whitepaper) 2020 conference, highlight the utility of UKONIQ across these diseases. As the first and only inhibitor of both PI3K-delta and CK1-epsilon, which is now commercially available, we believe UKONIQ offers an important new treatment option for patients."

Pier Luigi Zinzani, MD, PhD, Professor, Institute of Hematology, "L. e A. Seràgnoli", University of Bologna, and Global Chair of the UNITY-NHL Phase 2b study stated, "The data published yesterday as well as the recent U.S. FDA approval of umbralisib in relapsed or refractory marginal zone lymphoma and follicular lymphoma, are encouraging for patients suffering from these diseases, especially given the lack of a standard of care in these settings. As we see from the UNITY-NHL publication, umbralisib offers meaningful clinical activity across both marginal zone and follicular lymphoma and a manageable safety profile with relatively low rates of immune mediated toxicities and discontinuations due to adverse events."

The manuscript includes data from 208 patients with relapsed or refractory iNHL, including 69 marginal zone lymphoma (MZL), 117 follicular lymphoma (FL), and 22 small lymphocytic lymphoma (SLL) patients who were unresponsive to prior treatments (≥1 MZL; ≥2 FL/SLL), including anti-CD20–based therapy. Patients were administered umbralisib 800 mg orally once-daily until disease progression, unacceptable toxicity, or study withdrawal. The primary end point was overall-response-rate (ORR) as assessed by an independent review committee (IRC) based on the Lugano classification.

Key highlights from this manuscript include:

The ORR was 47.1% across all relapsed or refractory iNHL patients treated (n=208)
At a median follow-up of 27.8 months patients with relapsed or refractory MZL demonstrated:
• 49.3% ORR with 16% Complete response (CR) rate (IRC assessed)
• Median duration of response (DOR) was not reached (95% CI, 10.3 – not estimable) and
• Median Progression Free Survival (PFS) was not reached (95% CI, 12.1 – not estimable)
At a median follow-up of 27.5 months patients with relapsed or refractory FL demonstrated:
• 45.3% ORR with 5.1% achieving a CR (IRC assessed)
• Median DOR of 11.1 months (95% CI, 8.3–15.6)
• Median PFS was 10.6 months
Grade ≥3 treatment emergent adverse events (TEAEs) reported in ≥10% of patients included: neutropenia (11.5%) and diarrhea (10.1%). Increased ALT/AST (grade ≥3) occurred in 6.7%/7.2% of patients.
Other AEs of special interest included pneumonitis (1.4%; grade >3 1.0%) and non-infectious colitis (1.9%; grade >3 0.5%).
A total of 31 patients (14.9%) discontinued due to a treatment-related adverse event.
These data are described further in the manuscript entitled, "Umbralisib, a Dual PI3Kδ/CK1ε Inhibitor in Patients with Relapsed/Refractory Indolent Lymphoma," which was published online yesterday in the Journal of Clinical Oncology. The online version of the article can be accessed at View Source

On March 9, 2021 AngioDynamics, Inc. (NASDAQ: ANGO), a leading provider of innovative, minimally invasive medical devices for vascular access, peripheral vascular disease, and oncology, reported that it will report financial results for the third quarter of fiscal year 2021 before the market open on Tuesday, March 30, 2021 (Press release, AngioDynamics, MAR 9, 2021, View Source [SID1234576345]). The Company’s management will host a conference call at 8:00 a.m. ET the same day to discuss the results.

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To participate in the conference call, dial 1-877-407-0784 (domestic) or +1-201-689-8560 (international) and refer to the passcode 13717367.

This conference call will also be webcast and can be accessed from the "Investors" section of the AngioDynamics website at www.angiodynamics.com. The webcast replay of the call will be available at the same site approximately one hour after the end of the call.

A recording of the call will also be available from 11:00 a.m. ET on Tuesday, March 30, 2021, until 11:59 p.m. ET on Tuesday, April 6, 2021. To hear this recording, dial 1-844-512-2921 (domestic) or +1-412-317-6671 (international) and enter the passcode 13717367.

On March 9, 2021 Pacira BioSciences, Inc. (Nasdaq: PCRX), the industry leader in its commitment to non-opioid pain management and regenerative health solutions, reported preliminary unaudited net product sales of EXPAREL (bupivacaine liposome injectable suspension) and iovera° of $35.6 million and $0.7 million, respectively, for the month of February 2021 (Press release, Pacira Pharmaceuticals, MAR 9, 2021, View Source [SID1234576293]). EXPAREL average daily sales were 103 percent of the prior year for February 2021.

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"We are off to a terrific start for the year, with February EXPAREL sales surpassing a very strong comparative month in 2020 amid recent severe weather-related shutdowns in key markets and ongoing COVID-19 related challenges. EXPAREL growth continues to significantly outpace the recovery in the elective surgery market as utilization increases across a wide range of anesthesia-driven regional blocks and enhanced recovery after surgery protocols shift more complex, painful elective surgeries to the outpatient setting. We also continue to see a strong uptake in regional blocks for non-elective procedures given the market’s critical need for opioid-sparing approaches. Looking ahead, Pacira remains well-positioned to deliver robust top- and bottom-line growth as we further entrench EXPAREL as the leader in non-opioid postsurgical pain management and advance iovera° in key orthopedic settings," said Dave Stack, chairman and chief executive officer of Pacira BioSciences.

The company’s 2021 product sales continue to be negatively impacted by the COVID-19 pandemic, which mandated 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. In order to provide greater transparency, the company will continue to report monthly intra-quarter unaudited net product sales until it has gained enough visibility around the impacts of COVID-19. The company is also providing weekly EXPAREL utilization and elective surgery data within its investor presentation, which is accessible at investor.pacira.com. The financial information included in this press release is preliminary, unaudited and subject to adjustment. It does not present all information necessary for an understanding of the company’s financial results for the first quarter or full year 2021.