On November 9, 2022 Lipocine Inc. (NASDAQ: LPCN), a biopharmaceutical company focused on treating Central Nervous System ("CNS") disorders by leveraging its proprietary platform to develop differentiated products, reported financial results for the third quarter and nine months ended September 30, 2022 and provided a corporate update (Press release, Lipocine, NOV 9, 2022, View Source [SID1234623532]).

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"During the third quarter we announced a new strategic direction for Lipocine, which involves applying our validated technology to develop differentiated treatments for CNS disorders. Our initial focus is on endogenous neuroactive steroids which have broad applicability in treating various CNS conditions," said Dr. Mahesh Patel, President and CEO of Lipocine Inc. "Our most advanced neuroactive steroid ("NAS") candidate is LPCN 1154, which we are developing for the treatment of postpartum depression. We recently initiated a pilot PK bridge study (a prelude to a pivotal study required for NDA filing) and look forward to reporting results of the pilot study in the first half of 2023."

Third Quarter Highlights

Strategic realignment and focus on CNS

●In September, Lipocine announced a strategic realignment and forward focus on treating CNS disorders. The Company’s initial focus is to advance its pipeline of endogenous neuroactive steroids ("NAS") which have potential to treat various neuropsychiatric conditions.
●Lipocine’s CNS development portfolio includes LPCN 1154, a fast-acting oral antidepressant for postpartum depression ("PPD") with potential for outpatient use; LPCN 2101, with a novel mechanism of action, for women with epilepsy; and additional undisclosed CNS focused candidates.
●The Company is exploring partnering its portfolio of non-core assets (LPCN 1144 for treatment of non-cirrhotic NASH, LPCN 1148 for decompensated liver cirrhosis, LPCN 1107 for prevention of pre-term birth, and LPCN 1111, a once-a-day therapy for TRT), consistent with our strategy designed to diversify risk and potentially create opportunities for non-dilutive financing.

Corporate

●Appointed Spyros Papapetropoulos, M.D. Ph.D., as Lead Director and Chairman of the Board. Dr. Papapetropoulos has served on Lipocine’s board since April 2022.
●Appointed George Nomikos, M.D., Ph.D., as Chief Medical Officer. Dr. Nomikos is an R&D scientist and psychiatry-trained clinician with extensive academic and industry experience in neuropsychiatric, neurometabolic, neurohormonal, neuromuscular and chronic pain therapeutics.

Third quarter Ended September 30, 2022 Financial Results

Lipocine reported a net loss of $2.4 million, or ($0.03) per diluted share for the quarter ended September 30, 2022, compared with a net loss of $3.1 million, or ($0.04) per diluted share, in the quarter ended September 30, 2021.

There were no revenues in the quarter ended September 30, 2022. There was $55,000 in license revenue recorded in the quarter ended September 30, 2021 related to payments received from Spriaso under a licensing agreement which did not recur in 2022.

Research and development expenses were $2.1 million for the quarter ended September 30, 2022, compared with $2.4 million for the quarter ended September 30, 2021. The decrease in research and development expenses was primarily due to a decrease in expenses related to the LPCN 1154 clinical studies, a decrease in expenses and consulting costs related to the completion of the LPCN 1144 LiFT Phase 2 clinical study in NASH subjects, and a decrease in costs associated with TLANDO. These decreases were offset by an increase in costs related to the Phase 2 POC study in male cirrhotic subjects with LPCN 1148, an increase in LPCN 1111 manufacturing scale up, an increase in LPCN1107 clinical studies, and an increase in other research and development costs and personnel expenses.

General and administrative expenses were $0.8 million for the quarter ended September 30, 2022, compared with $1.2 million for the quarter ended September 30, 2021. The decrease in general and administrative expenses was primarily due to decreases in legal expenses, personnel costs, corporate insurance expense and other G&A expenses. The decreases were offset by an increase in directors’ fees resulting from the addition of two new directors, increases in professional and consulting fees and increases in travel related expense.

As of September 30, 2022, the company had $34.3 million of unrestricted cash, cash equivalents and marketable investment securities, compared to $46.6 million at December 31, 2021.

Nine Months Ended September 30, 2022 Financial Results

Lipocine reported a net loss of $8.5 million, or ($0.10) per diluted share, for the nine months ended September 30, 2022, compared with a net loss of $13.3 million, or ($0.16) per diluted share, in the nine months ended September 30, 2021.

Revenues in the nine months ended September 30, 2022 were $0.5 million, compared with revenues of $55,000 in the comparable period in 2021. The increase in revenue was primarily related to a non-refundable cash fee received from Antares for consideration of a 90 day extension to exercise its option to license LPCN 1111.

Research and development expenses for the nine months ended September 30, 2022, were $6.9 million compared to $5.4 million for the comparable period in 2021. The increase in research and development expenses was due to an increase in expenses related to the Phase 2 POC study in male cirrhotic subjects with LPCN 1148, an increase in costs related to LPCN 1154 clinical studies, an increase in expenses related to LPCN 1111 scale up activities and a food effect study in LPCN 1107, an increase in personnel expense and other research and development costs. These increases were offset by a decrease in expenses related to the completion of the LPCN 1144 LiFT Phase 2 clinical study in NASH subjects, and a decrease in costs associated with TLANDO.

General and administrative expenses for the nine months ended September 30, 2022 were $3.2 million compared to $4.3 million for the comparable period in 2021. The decrease in general and administrative expenses was primarily due to a decrease in legal fees, a decrease in personnel costs and a decrease in other general and administrative expenses. These decreases were offset by an increase in professional fees related to the recruitment of additional directors to our Board and various other consulting services, an increase related to proxy solicitation and distribution services, an increase in corporate insurance expenses and travel related expenses, as well as an increase in royalty expense related to the net sales of TLANDO resulting from its commercial launch in June 2022.

On November 9, 2022 Merrimack Pharmaceuticals, Inc. (Nasdaq: MACK) [("Merrimack" or the "Company")] announced that Ipsen, SA ("Ipsen") reported its primary analysis of the results of its Phase III NAPOLI 3 trial of Onivyde (irinotecan liposome injection) plus 5 fluorouracil/leucovorin and oxaliplatin (NALIRIFOX regimen) as a treatment of first line metastatic pancreatic ductal adenocarcinoma (mPDAC) (Press release, Merrimack, NOV 9, 2022, View Source [SID1234623531]).

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The Ipsen press release indicates that the trial "met its primary endpoint demonstrating clinically meaningful and statistically significant improvement in overall survival compared to nab-paclitaxel plus gemcitabine in 770 previously untreated patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) and key secondary efficacy outcome of progression-free survival (PFS) also showed significant improvement over the comparator arm. The safety profile of Onivyde in the NAPOLI 3 trial was consistent with those observed in the previous phase I/II mPDAC study."

Ipsen also indicated in its update that it intends to file a supplemental New Drug Application with the U.S. Food and Drug Administration for Onivyde in combination with oxaliplatin plus 5- fluorouracil/leucovorin for the treatment of patients with previously untreated mPDAC following the Fast Track Designation granted in 2020.

"We are encouraged by these results which indicate progress toward a potential future milestone payment from Ipsen," said Gary Crocker, Chairman and CEO of Merrimack Pharmaceuticals. "We congratulate the Ipsen team and look forward to continuing to await further public announcements from Ipsen regarding progress with this important program."

On November 9, 2022 GlycoMimetics, Inc. (Nasdaq: GLYC), a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers and inflammatory diseases, reported its financial results and highlights for the third quarter ended September 30, 2022 (Press release, GlycoMimetics, NOV 9, 2022, View Source [SID1234623530]). Cash and cash equivalents as of September 30, 2022, were $51.6 million.

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"Blinded pooled survival data in our pivotal Phase 3 study show patients living longer than what would be expected based on publicly available historical data. We approached the FDA as we felt an ethical obligation to conduct an interim analysis and have the independent DMC assess whether the prolonged survival observed is linked to treatment with uproleselan," said Harout Semerjian, Chief Executive Officer. "We are pleased the FDA is aligned with our position. Whether the DMC recommends that we continue the study as originally planned or immediately unblind for full analysis, we remain confident and excited in the potential of uproleselan to improve outcomes in relapsed/refractory AML."

The statistical plan cleared with FDA is for the independent DMC to review efficacy and safety data at around 80% of planned survival events. The company has initiated the required regulatory and Institutional Review Board (IRB) approvals to amend the protocol to conduct the interim utility analysis and share the data with the DMC by end of Q1 2023. The DMC is then expected to meet to review the data and recommend whether the study should continue as planned or should be immediately unblinded for full analysis, which could occur if the efficacy data from treatment with uproleselan in combination with standard chemotherapy is observed to be compelling.

As part of its ongoing monitoring of blinded pooled survival data within the Phase 3 pivotal study, the company is now projecting the final survival events trigger to occur around year-end 2023. This change in timeline is the result of patients continuing to live longer than observed in historical benchmarks used to design the study and was the primary rationale for the company to approach the FDA regarding conduct of an interim utility analysis.

The company’s cash runway now extends to year-end 2023 as a result of the decrease in expenses from the transition of the Phase 3 relapsed/refractory AML clinical trial to follow-up, the completion of key uproleselan commercial manufacturing activities, and the realization of savings from a headcount reduction earlier this year. The company’s allocation of its capital resources will continue to prioritize the advancement of the uproleselan development program, including key regulatory and pre-commercial activities.

The pivotal Phase 3 trial evaluating uproleselan in addition to a standard chemotherapy regimen in patients with relapsed/refractory AML completed enrollment in November of 2021. A total of 388 patients across 70 sites in nine countries were randomized in the clinical trial, which has a primary endpoint of overall survival.

Operational Highlights

● Following alignment with the FDA on conducting an interim analysis, GlycoMimetics initiated the required regulatory and IRB approvals to update the protocol of the company’s pivotal Phase 3 study of uproleselan in relapsed/refractory AML. These efforts are ongoing and are expected to be completed in Q1 2023.
● Initial clinical data from two investigator-sponsored trials studying the use of uproleselan in combination with other treatments in patients with different forms of AML have been accepted for poster presentation at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH Press Release). These studies examine the safety and potential efficacy of uproleselan to benefit patients across the AML spectrum, including older, unfit treatment naïve patients and those with treated secondary AML.
● The National Cancer Institute (NCI) continues to prepare for its planned interim analysis of event-free survival of the 267 patients in its Phase 2/3 clinical trial evaluating uproleselan in newly diagnosed older adults with AML who are fit for chemotherapy. When available, the company intends to share the outcome of the NCI’s interim analysis of the Phase 2 data and whether the trial will reopen for Phase 3 recruitment. The NCI will determine the timing and medical meeting to present the full results of the Phase 2 study.

Third Quarter 2022 Financial Results:

Cash position: As of September 30, 2022, GlycoMimetics had cash and cash equivalents of $51.6 million as compared to $90.3 million as of December 31, 2021.

Revenue: There was no revenue recognized during the three months ended September 30, 2022, compared to $87,000 of revenue recognized during the three months ended September 30, 2021.

R&D Expenses: Research and development expenses decreased to $4.9 million for the quarter ended September 30, 2022, as compared to $13.3 million for the same period in 2021. The significant decrease in expenses was primarily due to lower clinical trial and development costs related to the pivotal Phase 3 clinical trial of uproleselan in individuals with relapsed/refractory AML as patient enrollment ended in November 2021.

G&A Expenses: General and administrative expenses decreased to $3.8 million for the quarter ended September 30, 2022, as compared to $4.1 million for the third quarter of 2021 primarily due to lower professional fees.

Shares Outstanding: Shares of common stock outstanding as of September 30, 2022, were 52,423,944.

The company will host a conference call and webcast today at 8:30 a.m. ET. To access the call by phone, please go to this registration link and you will be provided with dial in details. Participants are encouraged to connect 15 minutes in advance of the scheduled start time.

A live webcast of the call will be available on the "Investors" tab on the GlycoMimetics website. A webcast replay will be available for 30 days following the call.

About Uproleselan

Discovered and developed by GlycoMimetics, uproleselan is an investigational first-in-class, E-selectin antagonist. Uproleselan (yoo’ pro le’se lan), currently in a comprehensive Phase 3 development program in acute myeloid leukemia (AML), has received Breakthrough Therapy designation from the U.S. FDA and from the Chinese National Medical Products Administration for the treatment of adult AML patients with relapsed or refractory disease. Uproleselan is designed to block E-selectin binding and stimulation of myeloid cells. E-selectin is expressed on the surface of blood vessels, and its binding to myeloid cells confers a pro-survival effect via NF-kB signaling. Uproleselan is designed to provide a novel approach to disrupting established mechanisms of leukemic cell resistance.

On November 9, 2022 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 third quarter ended September 30, 2022 (Press release, Vericel, NOV 9, 2022, View Source [SID1234623529]).

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Third Quarter 2022 Financial Highlights
•Total net revenue of $38.6 million
•MACI net revenue of $31.0 million, Epicel net revenue of $7.3 million, and NexoBrid revenue of $0.2 million
•Gross margin of 65%
•Net loss of $6.6 million, or $0.14 per share
•Non-GAAP adjusted EBITDA of $3.3 million
•Operating cash flow of $4.1 million
•As of September 30, 2022, approximately $133 million in cash and investments, and no debt

Business Highlights and Updates
•Third-quarter MACI revenue growth of 30% compared to the prior year and 8% sequential growth versus the prior quarter, representing the highest quarterly revenue outside of the seasonally-high fourth quarter since the launch of MACI
•Ninth straight quarter of positive adjusted EBITDA and operating cash flow
•FDA’s review of NexoBrid BLA is progressing, with inspections of manufacturing facilities in Taiwan and Israel underway
•Type C meeting with the FDA scheduled for December to discuss MACI arthroscopic delivery program

"The Company executed well in the third quarter, delivering strong MACI revenue growth, record third-quarter total revenue, and continued quarterly profitability and positive operating
1
Exhibit 99.1
cash flow," said Nick Colangelo, President and CEO of Vericel. "We also continued to advance important pipeline programs with planned meetings with the FDA for both the MACI arthroscopic delivery and ankle development programs, as well as continued pre-launch activities for the potential launch of NexoBrid, which we believe positions the Company for continued strong growth in the years ahead."

Full-Year 2022 Financial Guidance Update
•Total net revenue expected to be in the range of $164 to $166 million
•MACI revenue expected to be approximately $130 to $132 million
•Total burn care revenue, including Epicel and NexoBrid, expected to be approximately $34 million
•Gross margin expected to be in the mid-60% range
•Adjusted EBITDA margin in the mid-teens % range

Third Quarter 2022 Results
Total net revenue for the quarter ended September 30, 2022 increased 12% to $38.6 million, compared to $34.5 million in the third quarter of 2021. Total net product revenue for the quarter included $31.0 million of MACI (autologous cultured chondrocytes on porcine collagen membrane) net revenue and $7.3 million of Epicel (cultured epidermal autografts) net revenue, compared to $23.9 million of MACI net revenue and $9.8 million of Epicel net revenue, respectively, in the third quarter of 2021. Total net revenue for the quarter also included $0.2 million of revenue related to the procurement of NexoBrid (concentrate of proteolytic enzymes enriched in bromelain) by the U.S. Biomedical Advanced Research and Development Authority (BARDA) for emergency response preparedness, compared to $0.8 million in the third quarter of 2021.

Gross profit for the quarter ended September 30, 2022 was $25.2 million, or 65% of net revenue, compared to $22.1 million, or 64% of net revenue, for the third quarter of 2021.

Total operating expenses for the quarter ended September 30, 2022 were $32.0 million, compared to $27.1 million for the same period in 2021. The increase in operating expenses was primarily due to an increase in employee expenses, continued investment in commercialization initiatives and additional stock-based compensation expense.

Net loss for the quarter ended September 30, 2022 was $6.6 million, or $0.14 per share, compared to $4.9 million, or $0.11 per share, for the third quarter of 2021.

Non-GAAP adjusted EBITDA for the quarter ended September 30, 2022 was $3.3 million, or 9% of net revenue, compared to $4.3 million, or 12% of net revenue, for the third quarter of 2021. A table reconciling non-GAAP measures is included in this press release for reference.
As of September 30, 2022, the Company had approximately $133 million in cash and investments, compared to approximately $129 million as of December 31, 2021, and no debt.

2
Exhibit 99.1
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 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 by telephone, please register here to receive dial-in details and your personal passcode. A replay of the webcast will be available on the Vericel website until November 9, 2023.

On November 9, 2022 RAPT Therapeutics, Inc. (Nasdaq: RAPT), a clinical-stage, immunology-based biopharmaceutical company focused on discovering, developing and commercializing oral small molecule therapies for patients with significant unmet needs in inflammatory diseases and oncology, reported that members of the RAPT management team will participate in the following investor conferences in November (Press release, RAPT Therapeutics, NOV 9, 2022, https://investors.rapt.com/news-releases/news-release-details/rapt-therapeutics-participate-several-upcoming-investor-0 [SID1234623528]).

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Guggenheim 4th Annual Immunology and Neurology Day – Tuesday, November 15, 2022, at 2:10 p.m. ET in New York, NY
Piper Sandler 33rd Annual Healthcare Conference – Tuesday, November 29, 2022, at 8:00 a.m. ET in New York, NY
Evercore ISI 5th Annual Virtual HealthCONx Conference – Wednesday, November 30, 2022, at 3:55 p.m. ET
To access the live webcasts or subsequent archived recordings of the company presentations, please visit the RAPT Therapeutics website at https://investors.rapt.com/events-and-presentations.