Growth Disorders

HYOGO, Japan--(BUSINESS WIRE)-- JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced that it has achieved the first research milestone criterion in a collaboration with Alexion, AstraZeneca Rare Disease (“Alexion”) to develop an undisclosed initial therapeutic molecule that applies JCR’s proprietary J-Brain Cargo®, blood-brain barrier penetration technology, for the treatment of a neurodegenerative disease. (Related release is here). The achievement of a predefined research milestone triggers a milestone payment to JCR. JCR recently strengthened its collaboration with Alexion to address unmet needs by initiating a second research collaboration in December 2023 to apply the same technology to oligonucleotide therapeutics. (Related release is here). The milestone payment has a minor impact on JCR’s consolidated financial results for this fiscal year ending on March 31, 2024. About the J-Brain Cargo® Platform Technology J-Brain Cargo® is a proprietary blood-brain barrier (BBB)-penetrating technology to bring biotherapeutics into the central nervous system. The first drug developed based on this technology and approved in Japan for a lysosomal storage disorder (LSD) is IZGARGO® (INN: pabinafusp alfa) for the treatment of Mucopolysaccharidosis (MPS) type II (Hunter Syndrome). Based on the same platform technology, JR-141(INN: pabinafusp alfa), JR-171 for the treatment of MPS I (INN: lepunafusp alfa) and JR-441 for the treatment of MPS IIIA have advanced into global clinical trials. JCR intends to start clinical trials with four additional programs from its LSD pipeline by FY2028. About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is redefining expectations and expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 49-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, Fabry disease, acute graft-versus host disease, MPS II, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II (Hunter syndrome), MPS III A and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical progress at a global level. Our core values – reliability, confidence, and persistence – benefit all our stakeholders, including employees, partners, and patients. Together we soar. For more information, please visit Cautionary Statement Regarding Forward-Looking Statements This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as “believe,” “estimate,” “anticipate,” “intend,” “plan,” “will,” “would,” “target” and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors’ pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future.

Carcinoid syndrome occurs in around 20% of patients with neuroendocrine tumors, a rare, slow-growing cancer that usually originates in the digestive tract. As Crinetics prepares to make a tilt for FDA approval in a rare growth disorder later this year, a topline readout from a phase 2 study suggests the same drug also has the potential to treat symptoms associated with a neuroendocrine tumor. The phase 2 study enrolled 36 patients with carcinoid syndrome who initially received either 40 mg or 80 mg daily doses of the oral somatostatin receptor type 2 (SST2) agonist, paltusotine, for eight weeks. Thirty patients completed the randomized treatment phase, with one participant from the 40 mg cohort and five from the 80 mg group discontinuing treatment. The trial showed “rapid and sustained reductions in flushing episodes and bowel movement,” Crinetics said in a March 12 release. This included a 63% reduction from baseline in mean flushing frequency for the 24 patients with more than one bowel movement (BM) a day. For the 16 patients experiencing more than three BMs a day—considered the upper limit of normal—there was a 60% mean reduction in mean excess frequency, the company added. Across all the patients, there was a 61% reduction in flushing severity and a 64% reduction in BM urgency. Reductions in the frequency and severity of symptoms were observed from less than two weeks of the treatment beginning and were sustained through the eight-week regimen in both treatment naïve patients and those who had switched over from injectable somatostatin receptor ligand (SRL) therapy, which is the current mainstay treatment for carcinoid syndrome. No treatment-related severe or serious adverse events (AEs) were reported, with diarrhea, abdominal pain, nausea and headache being the most commonly reported AEs across both dose cohorts. Crinetics CEO Scott Struthers, Ph.D., said the latest results “confirm our decision to move expeditiously toward phase 3.” “These results highlight the potential of paltusotine to deliver significant benefits to patients living with the debilitating symptoms of carcinoid syndrome,” Struthers added. “We plan to engage with the FDA to align on a phase 3 study design and have begun preparations to enable the initiation of a phase 3 program by the end of the year.” Carcinoid syndrome occurs in around 20% of patients with neuroendocrine tumors, a rare, slow-growing cancer that usually originates in the digestive tract. If the cancer metastasizes to the liver it can cause carcinoid syndrome, which is typically associated with diarrhea and flushing. While injectable SRL therapies are currently used to treat the condition, these injections are “associated with considerable treatment burden and offer inadequate relief of carcinoid syndrome symptoms for many patients,” according to Crinetics. Paltusotine is also being developed for acromegaly, a rare hormone disorder that causes abnormal growth and swelling in adults. A phase 3 study suggested patients were able to switch over “seamlessly” to the new treatment from their usual treatment of injectable depot somatostatin drugs. The September 2023 data showed that insulin-like growth factor 1 (IGF-1) levels were maintained in 83% of patients taking the drug, compared to just 4% on placebo, which was the main goal of the study. Results from another phase 3 trial in acromegaly are expected by the end of the month, ahead of a planned approval submission to the FDA in the growth disorder indication in the second half of the year. To help bankroll these plans, Crinetics announced a private placement financing two weeks ago that brought in around $350 million. The biotech expects these funds to provide a cash runway through 2028.

End of Phase 2 Meeting to Occur in Q2 2024, Initiation of LUM-201 Phase 3 Trial Expected in Q4 2024 Previously Announced Topline Data from Phase 2 OraGrowtH210 and OraGrowtH212 Trials of LUM-201 in Moderate PGHD Met All Primary and Secondary Endpoints Pisit “Duke” Pitukcheewanont, MD, Appointed Chief Medical Officer Conference Call Today at 4:30 PM EST AUSTIN, Texas, March 07, 2024 (GLOBE NEWSWIRE) -- Lumos Pharma, Inc. (NASDAQ:LUMO), a late-stage biopharmaceutical company advancing an oral therapeutic candidate for moderate Pediatric Growth Hormone Deficiency (PGHD), today announced financial results for the year ended December 31, 2023 and provided an update on clinical and regulatory activity. “The past year was a highly successful one for Lumos, culminating in the announcement of topline results from our Phase 2 OraGrowtH210 and OraGrowtH212 trials, which met all of their primary endpoints and provided substantial support for the advancement of LUM-201 toward a registrational Phase 3 trial in moderate PGHD,” said Rick Hawkins, Chairman and CEO of Lumos Pharma. “We expect to present full twelve-month and longer-term data in a subset of patients from these trials at a medical meeting in the second quarter of this year. Next steps for this program are also on track, with our End-of-Phase 2 Meeting with the FDA scheduled for next quarter, and preparation for our Phase 3 pivotal trial of LUM-201 well underway. We expect to be in position to initiate this registrational trial in the fourth quarter, pending positive feedback from the FDA. As evidenced by the Key Opinion Leader event we held last December, there is broad support among the pediatric endocrinology community for our novel approach to treating growth hormone disorders, and we remain confident in this asset’s potential to become the first oral therapeutic for the treatment of PGHD.” Upcoming Milestones Full 12-month data from OraGrowtH210 Trial to be presented in Q2 2024 at major medical meeting End of Phase 2 Meeting to occur in Q2 2024 Company expects to initiate Phase 3 pivotal trial of LUM-201 in Q4 2024 Recent Highlights Topline Data from Phase 2 OraGrowtH210 and OraGrowtH212 Trials of LUM-201 in PGHD Met All Primary and Secondary Endpoints OraGrowtH210 results showed LUM-201 dose of 1.6 mg/kg achieved annualized height velocities (AHV) of 8.2 cm/yr at 6 months and 8.0 cm/yr at 12 months, similar to growth rates for moderate PGHD population Delta at 6 and 12-month AHV between optimal LUM-201 dose of 1.6 mg/kg and rhGH comparator arm was within the non-inferiority margin (< 2 cm/yr) suggested by FDA for recent approvals Initial 24-month LUM-201 data from combined OraGrowtH210 and OraGrowtH212 Trials demonstrated a sustained AHV effect from Year 1 to Year 2 OraGrowtH212 demonstrated that, with only 20% of the GH concentration of injectable rhGH, LUM-201 achieved similar AHV, illustrating the greater efficiency of LUM-201’s unique pulsatile mechanism of action OraGrowtH210 Trial met pre-specified primary endpoint of validation of Predictive Enrichment Marker (PEM) test and secondary endpoint demonstrating 100% reproducibility of PEM-Positive classification No safety signal to date for LUM-201 Pisit “Duke” Pitukcheewanont, MD Appointed Chief Medical Officer Dr. Duke as he is known was promoted to the position of CMO effective January 1, 2024. In this role, Dr. Duke will provide his leadership in Lumos Pharma’s efforts to hone its clinical and regulatory strategy and will continue to oversee medical affairs as the Company prepares to initiate a pivotal Phase 3 trial evaluating oral LUM-201 as a therapeutic for moderate PGHD. Financial Results for the Year Ended December 31, 2023 Cash Position – Lumos Pharma ended the year on December 31, 2023, with cash, cash equivalents, and short-term investments totaling $36.1 million compared to $67.4 million on December 31, 2022. Cash on hand as of December 31, 2023, is expected to support operations through the third quarter of 2024. R&D Expenses – Research and development expenses were $22.1 million, an increase of $4.2 million for the year ended December 31, 2023, compared to the same period in 2022, primarily due to increases of $3.3 million in clinical trial expenses, $0.9 million in contract manufacturing expenses, $0.2 million in consulting expenses and $0.2 million in other expenses, partially offset by a $0.4 million decrease in personnel-related expenses. G&A Expenses – General and administrative expenses were $16.6 million, an increase of $0.9 million for the year ended December 31, 2023, compared to the same period in 2022, primarily due to increases of $0.5 million in personnel-related expenses, $0.4 million in royalty expenses and $0.1 million in travel expenses, partially offset by a $0.1 million decrease in other expenses. Net Loss – The net loss for the year ended December 31, 2023, was $34.0 million compared to a net loss of $31.1 million for the same period in 2022. Lumos Pharma ended Q4 2023 with 8,102,555 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. Investors and the general public are invited to listen to the conference call. To access the call by phone, please click on this Registration Link, complete the form and you will be provided with dial-in details and a PIN. To avoid delays, we encourage participants to dial in to the conference call ten minutes ahead of the scheduled start time. The webcast may be accessed through this Webcast Link and may also be found in the “Investors & Media” section of the Lumos Pharma website, under “Events & Presentations.” A replay of the call will be available after the date of the call and may be accessed through the same link above or found on our website. About Lumos Pharma Lumos Pharma, Inc. is a clinical stage biopharmaceutical company focused on the development and commercialization of therapeutics for rare diseases. The Company was founded and is led by a management team with longstanding experience in rare disease drug development. Lumos Pharma’s lead therapeutic candidate, LUM-201, is a novel, oral growth hormone (GH) secretagogue, seeking to transform the ~$3.4B global GH market from injectable to oral therapy. LUM-201 is currently being evaluated in multiple Phase 2 clinical studies in Pediatric Growth Hormone Deficiency (PGHD) and has received Orphan Drug Designation in both the US and EU. For more information, please visit . Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements of Lumos Pharma, Inc. that involve substantial risks and uncertainties. All such statements contained in this press release are forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. A law that, in part, gives us the opportunity to share our outlook for the future without fear of litigation if it turns out our predictions were not correct. We are passionate about our business - including LUM-201 and the potential it may have to help patients in the clinic. This passion feeds our optimism that our efforts will be successful and bring about meaningful change for patients. Please keep in mind that actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements that we make. We have attempted to identify forward-looking statements by using words such as “projected,” "upcoming," "will," “would,” "plan," “intend,” "anticipate," "approximate," "expect," “potential,” “imminent,” and similar references to future periods or the negative of these terms. Not all forward-looking statements contain these identifying words. Examples of forward-looking statements include, among others, statements we make regarding the advancement of oral LUM-201 to Phase 3, the expected timing of trial data presentations and FDA meetings, substantial support for the advancement of LUM-201 toward a registrational Phase 3 trial in moderate PGHD, that we expect to present full twelve-month and longer-term data in a subset of patients from these trials at a medical meeting in the second quarter of this year, that next steps for this program are on track, that we expect to be in position to initiate this registrational trial in the fourth quarter, that there is broad support among the pediatric endocrinology community for our novel approach to treating growth hormone disorders, that we remain confident in this asset’s potential to become the first oral therapeutic for the treatment of PGHD, that cash on hand as of December 31, 2023 is expected to support operations through the third quarter of 2024, and any other statements other than statements of historical fact. We wish we were able to predict the future with 100% accuracy, but that just is not possible. Our forward-looking statements are neither historical facts nor assurances of future performance. You should not rely on any of these forward-looking statements and, to help you make your own risk determinations, we have provided an extensive discussion of risks that could cause actual results to differ materially from our forward-looking statements including risks related to the continued analysis of data from our LUM-201 Trials, the timing and outcome of our future interactions with regulatory authorities including our end of Phase 2 meeting with the FDA, the timing and ability of Lumos to raise additional equity capital as needed to fund our Phase 3 Trial, our ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the ability to structure our Phase 3 trial in an effective and timely manner, the ability to successfully develop our product candidate, the effects of pandemics, other widespread health problems or military conflicts including the Ukraine-Russia conflict and the Middle East conflict and other risks could cause actual results to differ materially from those matters expressed in or implied by such forward-looking statements including information in the "Risk Factors" section and elsewhere in Lumos Pharma’s Annual Report on Form 10-K for the year ended December 31, 2022, as well as other reports filed with the SEC including our most recent Quarterly Report on Form 10-Q for the quarter ended September 30, 2023. All of these documents are available on our website. Before making any decisions concerning our stock, you should read and understand those documents. We anticipate that subsequent events and developments will cause our views to change. We may choose to update these forward-looking statements at some point in the future, however, we disclaim any obligation to do so. As a result, you should not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this press release. Investor & Media Contact: Lisa Miller Lumos Pharma Investor Relations 512-792-5454 ir@lumos-pharma.com Lumos Pharma, Inc. Consolidated Statements of Operations and Comprehensive Loss (unaudited) (In thousands, except share and per share amounts) Year Ended December 31, 2023 2022 Revenues: Royalty revenue $ 2,051 $ 1,523 Total revenues 2,051 1,523 Operating expenses: Research and development 22,096 17,857 General and administrative 16,569 15,706 Total operating expenses 38,665 33,563 Loss from operations (36,614 ) (32,040 ) Other income and expense: Other income, net 683 91 Interest income 1,868 874 Other income, net 2,551 965 Net loss before taxes (34,063 ) (31,075 ) Income tax benefit 29 13 Net loss $ (34,034 ) $ (31,062 ) Net loss per share of common stock Basic and diluted $ (4.18 ) $ (3.71 ) Weighted average number of common shares outstanding Basic and diluted 8,145,155 8,373,821 Other comprehensive income (loss): Unrealized loss on short-term investments 9 (9 ) Total comprehensive loss $ (34,025 ) $ (31,071 ) Lumos Pharma, Inc. Consolidated Balance Sheets (unaudited) (In thousands, except share and per share amounts) December 31, 2023 2022 Assets Current assets: Cash and cash equivalents $ 35,078 $ 56,007 Short-term investments 999 11,352 Prepaid expenses and other current assets 3,748 4,427 Other receivables 210 223 Total current assets 40,035 72,009 Non-current assets: Property and equipment, net — 53 Right-of-use asset 603 230 Total assets $ 40,638 $ 72,292 Liabilities and Stockholders' Equity Current liabilities: Accounts payable $ 890 $ 275 Accrued expenses 5,858 6,200 Current portion of lease liability 282 233 Total current liabilities 7,030 6,708 Long-term liabilities: Royalty obligation payable to Iowa Economic Development Authority 6,000 6,000 Lease liability 303 — Total liabilities 13,333 12,708 Commitments and contingencies: Stockholders' equity: Undesignated preferred stock, $0.01 par value: Authorized shares - 5,000,000 at December 31, 2023 and 2022; issued and outstanding shares - 0 at December 31, 2023 and 2022 — — Common stock, $0.01 par value: Authorized shares - 75,000,000 at December 31, 2023 and 2022; issued shares - 8,125,728 and 8,283,708 at December 31, 2023 and 2022, respectively, and outstanding shares - 8,102,555 and 8,267,968 at December 31, 2023 and 2022, respectively 81 82 Treasury stock, at cost, 23,173 and 15,740 shares held as of December 31, 2023 and 2022, respectively (196 ) (170 ) Additional paid-in capital 188,937 187,164 Accumulated deficit (161,517 ) (127,483 ) Accumulated other comprehensive loss — (9 ) Total stockholders' equity 27,305 59,584 Total liabilities and stockholders' equity $ 40,638 $ 72,292