Clonidine Hydrochloride

→ The top leadership at Lexicon Pharmaceuticals is still undergoing a major makeover as president and COO Jeff Wade heads for the exit on Sept. 30 after 25 years with the Texas-based biotech. Wade won’t be around for the outcome of Lexicon’s latest attempt at an FDA nod in type 1 diabetes for sotagliflozin , which has a Dec. 20 decision date. The drug has already been approved for heart failure and is marketed as Inpefa . Lexicon laid off about 75 people in its sales and commercial teams in August “to right-size our promotional efforts around Inpefa for heart failure and reallocate resources to prepare for our potential launch” in type 1 diabetes and chronic kidney disease, CEO Mike Exton said in a statement. Exton succeeded Lonnel Coats as CEO a month earlier. → Sarepta Therapeutics has appointed Genmab chair Deirdre Connelly to the board of directors. Connelly had a 26-year career with Eli Lilly before pivoting to GSK in 2009 as president, North America pharmaceuticals until her retirement in 2015. She’s also a board member at Macy’s and Lincoln Financial Group . Like Janet Woodcock before him, CBER Director Peter Marks helped Sarepta cross the goal line with a label expansion in June for its Duchenne muscular dystrophy treatment Elevidys — overruling his own staff and befuddling experts. → Mitchell Chan has replaced Vit Vasista as CFO of Regenxbio at a time when the company plans to start a pivotal study for its own Duchenne gene therapy. It’s another big change for Regenxbio after Ken Mills became executive chairman and Curran Simpson succeeded Mills as CEO on July 1. Chan spent a year with Catalio Capital Management as operating partner after he was elevated to the CFO post at Viela Bio. Vasista had been finance chief since 2009 and will be an advisor until Jan. 3. → Here’s a look at some CEO news that we covered earlier this week: Former Kite Pharma commercial chief Warner Biddle has succeeded Peter Maag as CEO of Kyverna Therapeutics , which also added ex-Kite CEO Christi Shaw to the board. Shares $KYTX of Kyverna have taken a precipitous fall since its $319 million IPO in February. Ochre Bio co-founder Jack O’Meara is calling it quits after five years as CEO, but he tells Lei Lei Wu that he has an Alzheimer’s startup in the works. In a LinkedIn post announcing his departure, O’Meara also said that former F-star CEO and current Levicept chief Eliot Forster will be interim executive chair. And Shilpa Sambashivan has been promoted to CEO of Nura Bio , which stapled an additional $68 million to its Series A — bringing its total to $141 million. Sambashivan was a project team lead at Amgen before becoming a founding member at Nura Bio. → Matt Kane is taking the top spot at Celyad Oncology , a Belgian biotech that has seen considerable turnover at the top. Celyad co-founder Michel Lussier filled in as interim CEO for a second time after Georges Rawadi ’s sudden exit last December; he also took over when Filippo Petti stepped down in 2022. Kane co-founded Precision BioSciences with Derek Jantz and Jeff Smith , leading the company until he joined epigenome editing startup Tune Therapeutics as CEO in 2021. Kane’s LinkedIn page indicates that he left Tune in April, a year after he hired Jantz as CSO. → Ex- Prometheus Biosciences chief Mark McKenna has found a CFO at his new I&I bet, Mirador Therapeutics . Gregg Gilbert had been a managing director in the healthcare investment banking group for Bank of America Securities since 2021 and worked at Bank of America Merrill Lynch for 15 years. After selling Prometheus and its inflammatory bowel disease drug to Merck for $10.8 billion , McKenna wasted little time getting Mirador off the ground with a $400 million Series A in March, one of the largest private financing rounds of 2024. → GLP-1 competitor Structure Therapeutics has promoted Blai Coll to CMO and welcomed Ashley Hall as chief development officer. Coll, who replaces Mark Boch , had led clinical development for Raymond Stevens ’ crew since May 2022 and was cardiovascular and metabolic platform lead, medical affairs at the end of a six-year stint with Amgen. Hall is an Amgen vet in her own right who held the role of development chief at Reneo Pharmaceuticals . Structure’s stock price soared in June $GPCR when it produced efficacy data with its oral GLP-1 GSBR-1290 that was on par with Eli Lilly’s obesity drug orforglipron . → Novartis and Gilead veteran Matthias Will has succeeded Edgar Braendle as chief development officer at Autolus Therapeutics . Will had led clinical development at CRISPR Therapeutics and CytomX before his most recent gig as CMO of Dren Bio . The FDA is expected to make a decision by Nov. 16 on Autolus’ CD19-directed CAR-T obecabtagene autoleucel , or obe-cel . → According to an SEC filing , Crinetics chief commercial officer Jim Hassard will pack his bags on Oct. 14 in what’s described as a “termination without cause.” The 17-year Amgen vet came to Crinetics from Arrowhead Pharmaceuticals in March 2022. Phase 3 successes with its acromegaly drug paltusotine have propelled Crinetics to an NDA filing “later this year” and “an expected market launch in 2025,” CEO Scott Struthers said in the San Diego biotech’s second-quarter report. CFO Mark Wilson cited personal reasons for his own upcoming departure, which was also included in Crinetics’ Q2 update. → Allosteric drug discovery shop HotSpot Therapeutics has introduced Alison O’Neill as CMO. O’Neill is the former medical chief at Surface Oncology , which was acquired by Coherus BioSciences last year for $65 million. She had a senior leadership role in the global oncology division at Sanofi and was VP of clinical development for Radius Health . → Ex- Icosavax CEO Adam Simpson has landed at Frazier Life Sciences as a venture partner on its company creation team and will work out of Frazier’s office in San Diego. Simpson sold the vaccine developer to AstraZeneca last December, and his previous company — celiac disease specialist PvP Biologics — was purchased by Takeda in 2020. → Ruben Tommasi has been named CSO of Entact Bio , a Watertown, MA-based biotech led by ex- Ribon Therapeutics CEO Victoria Richon that is developing enhancement-targeting chimeric molecules, or ENTACs. Tommasi has also held this role at Dunad Therapeutics and AstraZeneca spinout Entasis Therapeutics . Entact Bio has kept a relatively low profile since its $81 million Series A in 2022, but it did show up in Peer Review this spring when it recruited fellow Entasis vet Michael Gutch as COO. → Third Rock ’s Treg developer Abata Therapeutics has resurfaced in Peer Review for the first time in more than a year, naming Joanne Beck as chief technical officer. The Shire and Celgene alum is the ex-technical chief at Aerium Therapeutics , the Covid biotech led by Rajeev Venkayya that recently shut down because of a surge in new variants. In April 2023, Abata poached Sonoma Biotherapeutics CMO Leonard Dragone for the same role . → Led by ex- Teva R&D chief Michael Hayden , neuro biotech Prilenia has tapped Rob Lauzen as CFO and Jason Marks as chief legal officer. Lauzen was VP, investor relations and head of financial planning & analysis at Dyne Therapeutics , which made a CMO change and lost its COO and CBO earlier this month. Marks just had a short stint with Teva as a senior legal advisor to global transactions and global finance. The Bausch Health alum has also been chief legal and compliance officer at Amarin . → San Francisco eye disease biotech Ray Therapeutics has selected Christopher Whitmore as CFO. Whitmore had held the same position with Immune-Onc Therapeutics since July 2022, and he was VP, finance & administration for Harpoon Therapeutics from 2018-21. Ray Therapeutics, which hauled in a $100 million Series A last year, will test its gene therapy RTx-015 in patients with retinitis pigmentosa. → Tris Pharma has promoted VP of marketing Manesh Naidu to the role of chief commercial officer. Naidu joined the New Jersey-based company in 2023 after a stint as chief marketing officer of Althera Pharmaceutical . Naidu’s résumé also includes stints at McKinsey , Mallinckrodt , Novartis, Pfizer and Bellerophon Therapeutics . In May, Tris snagged the first liquid non-stimulant ADHD drug approval in the US. Onyda XR will be used to treat ADHD in children at least 6 years of age. → Backed by Jeff Bezos , Seattle-based Nautilus Biotechnology has named Kentaro Suzuki as chief marketing officer. Suzuki most recently served as VP and general manager of Agilent Technologies’ mass spectrometry division. Before that, he was with Takeda and Hewlett-Packard . → San Francisco-based 89bio has appointed Teresa Perney as chief regulatory and quality officer. Perney held this title at EQRx after five years with Myovant Sciences as SVP of regulatory and quality. Madrigal Pharmaceuticals finally broke through with an FDA approval for metabolic dysfunction-associated steatohepatitis (also referred to as NASH), and 89bio is enrolling patients for late-stage studies of pegozafermin — one for non-cirrhotic MASH and another for MASH with compensated cirrhosis. “I’m eager to leverage my experience to navigate the regulatory landscape and help bring this innovative therapy to patients worldwide,” Perney said in a statement . → There’s also a new regulatory chief at Disc Medicine : Steve Caffé spent five years with CRISPR Therapeutics as head of regulatory affairs until his departure last December. Earlier, he had numerous stops as a regulatory exec with such companies as Ra Pharmaceuticals , Sucampo Pharmaceuticals , AMAG Pharmaceuticals and MedImmune . Shares of Disc $IRON fell sharply in early April after mixed data with its drug for a rare blood disorder called erythropoietic protoporphyria, but have rebounded since. → Although AI-designed drugs haven’t exactly lived up to the hype, companies like BPGbio are trying to flip the narrative. This week, GSK alum John Beeler has joined the team as SVP of business development. Beeler comes to BPGbio from Bristol Myers Squibb , where he took on the role of business development search & evaluation lead. → Macrocycle startup Circle Pharma has brought in Marie Evangelista as SVP and head of cancer biology, and promoted Constantine Kreatsoulas to SVP and head of discovery technology sciences. Evangelista spent the last two years as VP of oncology at one of the most ambitious AI drug developers , Recursion . The longtime Genentech vet also had a brief tenure with Frontier Medicines as senior director of translation medicine. Kreatsoulas had a nine-year run at GSK and was the pharma giant’s interim head, computational toxicology before coming to Circle in 2021 as head of research and VP, research informatics and computational chemistry. Circle disclosed its $54 million Series D in an SEC filing this summer. → South San Francisco-based Ideaya Biosciences has recruited Douglas Snyder as general counsel. Snyder most recently served as chief legal officer and secretary of GW Pharmaceuticals . Earlier in his career, Snyder was general counsel and secretary at Actelion and Eisai . Back in July, Ideaya inked a $406.5 million deal with Biocytogen . → Deerfield Group has rolled out the welcome mat for two new execs: Guy Varady as EVP, integrated marketing and Bob Josefsberg as EVP, communications. Before this, Varady was SVP, management supervisor for DDB Health and held a number of roles at IPG Health and Omnicom Health Group . Meanwhile, Josefsberg was VP, corporate communications at Karuna Therapeutics and managing director at Chamberlain Healthcare Public Relations . → Tempest Therapeutics , which revealed early PFS and OS data for its liver cancer combo last October, has brought on Sheldon Mullins as VP, regulatory affairs. Mullins joins the team after gigs at Arrowhead Pharmaceuticals and Ardelyx . Earlier in his career, Mullins was with Anacor Pharmaceuticals , Onyx Pharmaceuticals and Genentech. In addition to Mullins’ appointment, Darrin Bomba and Henry Johnson have been promoted to the titles of VP, development operations and VP, CMC & medicinal chemistry, respectively. → Entrada Therapeutics CEO Dipal Doshi has picked up a seat on the board of directors at āshibio , a California biotech that will push its ex-Gilead drug andecaliximab into a Phase 2/3 study for patients with an ultra-rare bone disorder called fibrodysplasia ossificans progressiva (FOP). Āshibio came out of stealth mode this summer with a $40 million raise . → Akari Therapeutics , which is developing PAS-nomacopan for geographic atrophy, has elected former Epizyme CEO Rob Bazemore to the board of directors. Bazemore is also a board member at Ardelyx and Nuvation Bio .

- Top-line data with unexpected results requiring further clarification, did not meet primary endpoint as per protocol - Potent growth hormone release following macimorelin stimulation - Safety of macimorelin confirmed in the pediatric population TORONTO, ONTARIO, Aug. 27, 2024 (GLOBE NEWSWIRE) -- COSCIENS Biopharma Inc. (NASDAQ: CSCI) (TSX: CSCI) (“COSCIENS” or the “Company”), a specialty biopharmaceutical company developing and commercializing a diversified portfolio of pharmaceutical and diagnostic products and active ingredients for healthcare and cosmetics industries, today announced the top-line results of its Phase 3 safety and efficacy study AEZS-130-P02 (the "DETECT-trial") evaluating macimorelin for the diagnosis of Childhood Onset Growth Hormone Deficiency ("CGHD"). While top-line data show that macimorelin repeatedly demonstrated its capacity to stimulate growth hormone release as required to perform a growth hormone stimulation test, the primary efficacy endpoint for the DETECT-trial has not been met according to the definitions in the study protocol. In the study, the macimorelin test was compared to current standard growth hormone stimulation tests (arginine and clonidine). Although further analysis of the study results is required, initial review of the results indicates that the comparator tests may have led to a high false positive rate which impacted macimorelin’s ability to reach the primary efficacy endpoint. These unexpected results require further clarification and some re-analyses with the aim to consider the trial outcome and the strategy moving forward. The DETECT-trial was an international, multicenter safety and efficacy trial on macimorelin in a dose of 1.0 mg/kg body weight. The trial was conducted at clinical sites across the USA, Armenia, Georgia, Germany, Italy, Serbia, Slovakia, Poland, Romania, and Turkey. Following the screening period of Visit 1, the macimorelin test was conducted at Visit 2 and repeated at Visit 5, with blood samples being taken for Pharmacokinetic (PK) and Pharmacodynamic (PD) data at predefined times. At Visit 3 and Visit 4, an arginine test and a clonidine test were performed in a randomized order as standard growth hormone stimulation tests (GHSTs) to be compared with macimorelin. Visit 6 was a safety follow-up at study end. An independent external expert panel had been established to adjudicate the final growth hormone deficiency status of a child (i.e., child ill or child healthy) based on all medical data collected as well as the outcome of the two standard GHSTs. “The top-line results confirm the excellent safety of macimorelin, similar to what was demonstrated in our clinical and commercial experience with the use of macimorelin to diagnose adult growth hormone deficiency (AGHD). However, it was surprising to see that many subjects, who had been adjudicated as ill (growth hormone deficient) according to the comparator tests arginine and clonidine, showed high growth hormone stimulation following macimorelin. Our team will be further analyzing the detailed data from the DETECT-trial to gain insights into the reasons behind these unexpected results” commented Nicola Ammer, Chief Medical Officer of the Company. The completed study enrolled 102 subjects aged 3 to 17 years. The outcome of an independent adjudication of each subject was used to define the “true” GHD status (i.e., child ill or child healthy) at the end of the trial. Based on this, top-line data present an ‘optimal’ growth hormone (GH) cut-off point of 25.59 ng/mL, which is substantially higher than the standard cut-off points of 7-10 ng/mL as defined in current guidelines with existing tests. This surprisingly high cut-off point leads to a failure in the sensitivity and specificity assessments. Sensitivity as well as specificity are important parameters characterizing the performance of a diagnostic test, as they show a test’s capabilities to identify ill subjects as being ill (sensitivity) and to identify healthy subjects as being healthy (specificity), respectively. “While we expect the top-line data of our DETECT-trial to be further analyzed and discussed with health authorities, macimorelin is approved in the USA and continues to be marketed in Europe for AGHD. As we get the results of further analysis of the DETECT-trial we will be considering our action plan for macimorelin. In addition, we will continue our ongoing review and prioritization process for COSCIENS’ pipeline. Our goal remains to focus our resources and cash on those programs and products that we believe will propel the Company to its next phase of growth as a diversified biopharmaceutical company,” added Gilles Gagnon, Chief Executive Officer of the Company. “We would like to thank everyone involved in the DETECT-trial and especially the extraordinary engagement of the children enrolled in the study and their parents, as well as the excellent efforts of the clinical sites and our team members involved.” The DETECT-trial was the second study needed to evaluate the potential use of macimorelin as diagnostic test in children as agreed upon with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in the Company's Pediatric Investigation Plan. Full trial result reporting is expected to occur later this year. For more information about the DETECT-trial, please visit EU Clinical Trials Register and reference EudraCT #2018-001988-23 and clinicaltrials.gov identifier NCT04786873. About Macimorelin (Macrilen®; GHRYVELIN®) Macimorelin, an oral drug used for the diagnosis of adult growth hormone deficiency (AGHD) is approved for marketing under the brand name GHRYVELIN® in the European Economic Area and Macrilen® in the United States, South Korea and Israel. Macimorelin (Macrilen®; GHRYVELIN®), a ghrelin receptor agonist, is an orally administered peptidomimetic molecule that stimulates the secretion of growth hormone from the pituitary gland. Stimulated growth hormone levels are measured in blood samples taken after oral administration of macimorelin for the assessment of AGHD. Approval of macimorelin for use in adult was granted by the FDA in 2017 and by the EMEA in 2019, based on Phase III data showing that oral macimorelin provides accuracy comparable to that of standard insulin tolerance testing (ITT), but has a more favorable safety profile compared to ITT. Oral macimorelin also reduces false positive test results, helping to avoid unnecessary treatment of patients. About COSCIENS Biopharma Inc. COSCIENS is a specialty biopharmaceutical company engaged in the development and commercialization of a diverse portfolio of pharmaceutical and diagnostic products, including those focused on areas of significant unmet medical need. One of CONSCIENS’ lead products is macimorelin (Macrilen®; Ghryvelin®), the first and only U.S. FDA and European Medicines Agency approved oral test indicated for the diagnosis of adult growth hormone deficiency (AGHD). COSCIENS is also engaged in the development of therapeutic assets and proprietary extraction technology, which is applied to the production of active ingredients from renewable plant resources currently used in cosmeceutical products (i.e., oat beta glucan and avenanthramides which are found in leading skincare product brands like Aveeno and Burt’s Bees formulations) and being developed as potential nutraceuticals and/or pharmaceuticals. The company is listed on the NASDAQ Capital Market and the Toronto Stock Exchange, and trades on both exchanges under the ticker symbol “CSCI”. For more information, please visit COSCIENS’ website at www.cosciensbio.com. Forward-Looking Statements The information in this news release has been prepared as of August 27, 2024. Certain statements in this news release, referred to herein as "forward-looking statements", constitute "forward-looking statements" within the meaning of the United States Private Securities Litigation Reform Act of 1995, as amended, and "forward-looking information" under the provisions of Canadian securities laws. All statements, other than statements of historical fact, that address circumstances, events, activities, or developments that could or may or will occur are forward-looking statements. When used in this news release, words such as "anticipate", "assume", "believe", "could", "expect", "forecast", "future", "goal", "guidance", "intend", "likely", "may", "would" or the negative or comparable terminology as well as terms usually used in the future and the conditional are generally intended to identify forward-looking statements, although not all forward-looking statements include such words. Forward-looking statements in this news release include, but are not limited to, statements relating to: the timing for the release of the full results from the DETECT-trial; our consideration of our action plan for macimorelin; and our ongoing review and prioritization process for COSCIENS’ pipeline. Forward-looking statements are necessarily based upon a number of factors and assumptions that, while considered reasonable by the Company as of the date of such statements, are inherently subject to significant business, economic, operational and other risks, uncertainties, contingencies and other factors, including those described below, which could cause actual results, performance or achievements of the combined Company to be materially different from results, performance or achievements expressed or implied by such forward-looking statements and, as such, undue reliance must not be placed on them. Forward-looking statements involve known and unknown risks and uncertainties which include, among others: the combined Company’s present and future business strategies; operations and performance within expected ranges; anticipated future cash flows; local and global economic conditions and the environment in which the combined Company operates; anticipated capital and operating costs; uncertainty in our revenue generation from our marketed products, product development and related clinical trials and validation studies, including as a result of the DETECT-trial for Macrilen™ (macimorelin) not meeting its primary endpoint; results from our other products under development may not be successful or may not support advancing the product; our ability to raise capital and obtain financing to continue our currently planned operations; our now heavy dependence on sales by and revenue from our main distributor of our legacy Ceapro products (including AVA and OBG) and its customers; the continued availability of funds and resources to successfully commercialize products; the ability to secure strategic partners for late stage development, marketing, and distribution of our products, including our ability to enter into a new license agreement or similar arrangement following the termination of the license agreement with Novo Nordisk AG for rights to Macrilen™ in North America; our ability to enter into out-licensing, development, manufacturing, marketing and distribution agreements with other pharmaceutical companies and keep such agreements in effect; our ability to protect and enforce our patent portfolio and intellectual property; and our ability to continue to list our common shares on the NASDAQ Capital Market. Investors should consult our quarterly and annual filings with the Canadian and U.S. securities commissions for additional information on risks and uncertainties, including those discussed in our Annual Report on Form 20-F and MD&A filed under the Company’s profile on SEDAR+ at www.sedarplus.ca and on EDGAR at www.sec.gov. We disclaim any obligation to update any such risks or uncertainties or to publicly announce any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, unless required to do so by a governmental authority or applicable law. No securities regulatory authority has either approved or disapproved of the contents of this news release. The Toronto Stock Exchange accepts no responsibility for the adequacy or accuracy of this news release. Issuer: Gilles R. GagnonPresident & CEOggagnon@ceapro.com+1 (780) 421-4555 Investor Contact: Jenene ThomasJTC TeamT (US): +1 (833) 475-8247E: csci@jtcir.com