Ventus Therapeutics U.S., Inc.

Bree Harlin , who used to be director of clinical operations at Loxo Oncology , now Loxo@Lilly , is now in the C-suite for the first time. Harlin started at TC BioPharm on Wednesday as chief clinical officer, the biotech announced Friday — but her journey starts more than 20 years before then. After graduating from college, she moved to Boston, working in academia at some of the Harvard teaching hospitals in endocrinology and CNS. She later got convinced to interview with a company her neighbor founded, which was developing a gadolinium-based contrast agent used in diagnostic imaging. That’s where she got her start in the industry, moving from a clinical trial associate position to a clinical research associate role and got her first experience filing an NDA. While that NDA was rejected, it made an impact on Harlin. “It got me really hungry to learn more about how trials are run and more about the industry and in general,” Harlin added. That impact drove Harlin to other biotech and pharmaceutical companies, including Pfizer , Forest Pharmaceuticals (pre-buyout from Actavis / Allergan ) and Shire , with her latest stint being director of clinical operations at Loxo and helping to run Phase I to Phase IV trials. For Harlin, all of those jobs working with clinical trials and being a part of NDA and IND filings have conglomerated into a cornucopia of experience that, in her words, can be applied to her new job at TC BioPharm in order to “hit the ground running.” She’s already started meeting the team, hopping on meetings and instituting plans to get clinical trial sites on board in the US and outside the UK, where TC BioPharm is based. Currently the biotech is looking at expanding its cell therapy candidate outside the UK. Harlin’s goal is to utilize her experience to start recruiting trial sites in the US and in the EU, and navigate the clinical trial process to bring on board the right people. As far as the US goes, TC BioPharm is looking to file an IND with the FDA later this year in a bid to start up a Phase I trial, most likely in acute myeloid leukemia. CEO Bryan Kobel told Endpoints News that it was obvious Harlin was the right fit for the job, calling the entire process a matter of serendipity after the previous clinical director stepped aside to pursue a personal passion. “That put us in a really weird position where we had to find a chief clinical officer, and do it quickly,” Kobel added. After company leadership put together a sort of candidate attribute wishlist, Kobel said that he started cold-calling people — and Harlin was the first interview. After talking with eight to 10 more candidates, Harlin was still the top choice, and was brought on board. The chief executive added that the company looks for people who have experience working as part of a team and know how to communicate with them in high pressure situations without adding more stress or tension. While also noting the aspects of leading by example, “It’s hard to build a culture; it’s really easy to destroy it. So we need to be really, really prudent with who we add, how we add them, and the time we take to add them,” Kobel said. — Paul Schloesser → Although he “retired” after 14 years as CEO of Exelixis in 2010, and “retired” again from the top spot at Biogen in 2016, George Scangos assured Endpoints on Wednesday that Vir Biotechnology would be the last company he leads. We don’t know what the rest of 2023 holds in the industry, but we’ll be hard-pressed to find a bigger CEO transition this year than the 74-year-old Scangos passing the baton to Bayer ’s high-flying business leader Marianne De Backer on April 3. On De Backer’s watch, Bayer would swing deals like the acquisitions of AskBio and Vividion before either of them had the opportunity to go public. “I’m going to be called the IPO terminator,” De Backer said jokingly after the Vividion buyout . After turning Biogen’s fortunes around with the approval of MS drug Tecfidera in 2013, Scangos set off for Vir, where he put the biotech on the map by teaming up with GSK on sotrovimab for mild-to-moderate Covid-19. In an interview with John Carroll where he expressed his admiration for De Backer and a desire to travel with family, Scangos also discussed his risk-taking approach — invoking Kenny Rogers in the process. “I’ve always felt in my career that I would rather go down in flames by having failed big time than live my life in mediocrity because I was afraid to try,” Scangos said. “You can’t be in this industry if you’re not an optimist, but you have to be realistic and you have to know when to hold them and when to fold them.” → Kimball Hall has left the CDMO Abzena to become president and CEO of Irish biotech Innocoll , encompassing both Innocoll Biotherapeutics and the CDMO subsidiary Syntacoll . Hall, who rose to VP of manufacturing during her 14 years at Amgen , then moved on to Genentech as SVP of manufacturing biologics drug substance. She joined Abzena in 2019 as operations chief and was elevated to president a year later. → Launched in November and scouring the “secretome” for new therapies, Juvena Therapeutics has filled several leadership positions, with chief corporate development officer Neil Berkley leading off. A business development veteran with GSK and Cadence Pharmaceuticals (which was sold to Mallinckrodt in 2014), Berkley had a yearlong stay at AbCellera as CBO before leaving on June 29, according to an SEC filing . AbCellera CEO Carl Hansen has yet to name a replacement. Juvena has also welcomed the following: Amgen and Merck vet Gayathri Swaminath (SVP of discovery); Mo Tabrizi (VP of preclinical and nonclinical development), another Merck alum and the former senior director of pharmacology for Ascendis Pharma ; and Eddie Moler (VP of data science). Mubadala Capital and Horizons Ventures headlined Juvena’s $41 million Series A financing . → Jane Henderson has landed at immunology and inflammatory disease biotech Apogee Therapeutics , which emerged from stealth with a $149 million Series B and $169 million in total. The new CFO — no relation to Apogee’s CEO Michael Henderson — owned the same title at Invivyd , the Tillman Gerngross antibody developer that hit the scene with a lot of flash and a boatload of money as Adagio Therapeutics . But a substantial flop with its Covid-19 candidate led to a shakeup that included the departures of Gerngross , Henderson and the dismissal of an undisclosed number of staffers. She was previously the CFO of Voyager and Turnstone Biologics , and has board seats at Akero Therapeutics , Ventus Therapeutics and Iveric Bio . → The xenotransplantation experts at eGenesis have picked up Knut Niss as chief technology officer. Niss carried the same title at Mustang Bio , and he was part of the CMC leadership team at Novartis for Kymriah before it earned the distinction as the first approved gene therapy in the US. Last April, Michael Curtis replaced Paul Sekhri as CEO of eGenesis, the CRISPR upstart that’s trying to beat its xeno competitors to the clinic with its gene editing tech. Sekhri is currently the chief executive at vTv Therapeutics . → Thyroid eye disease biotech Sling Therapeutics , located south of the University of Michigan campus, has tapped Jeffrey Kent as CMO. Kent logged more than 10 years at Horizon and was EVP, medical affairs and outcomes research when Amgen struck M&A gold with a $28 billion deal in December. Sling’s lead asset linsitinib was in-licensed from Astellas , where Kent worked as an executive medical director before joining Horizon. We caught up with Gossamer Bio chief Faheem Hasnain when he was named chairman of Sling, which debuted with a $35 million Series A last June. → It’s been an eventful week for Pliant Therapeutics , raising $250 million after touting positive Phase IIa data with its idiopathic pulmonary fibrosis (IPF) drug bexotegrast . The Pliant news continues with the selection of Lily Cheung as chief human resources officer. Cheung brings HR experience from Actelion and Rigel Pharmaceuticals , and she had been VP of human resources for Synthekine since December 2021. → Right on Cue: Matteo Levisetti has been promoted to CMO at Cue Biopharma , setting the stage for acting medical chief Kenneth Pienta to step into a clinical advisory role. We had Levisetti in this space in February 2021 when he was named SVP of clinical development at the Boston biotech, whose pipeline is centered on IL-2. He’s been around the block in Big Pharma (Merck, Roche , Pfizer ) and spent a year as global clinical lead for sitravatinib at Mirati . → Sri Ryali has signed on as CFO of Codexis , the enzyme engineering outfit that sent 18% of its staff packing in November under first-year CEO Stephen Dilly . An Amgen finance alum, Ryali just completed four years as CFO of Eiger BioPharmaceuticals and was VP of finance at Aimmune from 2015-18. Earlier, he played a few quick measures at Jazz Pharmaceuticals as senior director, financial planning & analysis. → Targeting cancer and inflammatory bowel diseases like Crohn’s and ulcerative colitis with site specific immunomodulators (SSIs), Qu Biologics out of British Columbia has enlisted Matt Cahill as COO. Cahill was on the original crew at another Canadian company, Deep Genomics , as VP of finance & business operations, and he’s still on the board of directors with founder/CEO Brendan Frey , Nessan Bermingham and others. → Shawn Levy has been promoted to CSO and SVP of applications at DNA sequencing company Element Biosciences . Peer Review introduced you to Levy in February 2022 when he first came to Element as SVP of applications and scientific affairs. → Molecular diagnostics shop BillionToOne , which kept the cash flowing with a $125 million Series C in March 2022, has installed Amgen alum Gary Palmer as CMO, oncology. The ex-VP of medical affairs at Foundation Medicine and Genomic Health also had a three-year run as CMO of Tempus Labs . → Aurora, CO-based neuro biotech Cerebral Therapeutic s has appointed Julie Foster as COO. Foster put in 35 years at Medtronic , taking on such roles as VP of the cardiac and vascular businesses in the EMEA (Europe, Middle East and Africa) region and VP of customer experience, diabetes. → IO Biotech was one of the last trains out of the IPO station in late November 2021 before things went pear-shaped in the market, and this week the IDO/PD-L1 player has recruited Devin Smith as general counsel and chief compliance officer. Smith is an EMD Serono legal vet who was general counsel at Yumanity before it reverse merged with Kineta and shipped off Parkinson’s drug YTX-7739 — along with an assortment of other candidates — to J&J back in June. → CEPI’s mRNA vaccine partner SK bioscience has a pair of new execs : Richard Kensinger (VP of R&D project management) is an 18-year Sanofi veteran who comes from Affinivax , where he was executive director of process development and protein sciences. And Kenneth Lee (VP of the biologic business development department at SK’s R&D center in Pangyo, South Korea) led the commercial division in the Americas region for GenScript ProBio , the CDMO subsidiary of GenScript that racked up a $224 million Series C last week. → Speaking of CDMOs, Columbus, OH’s Forge Biologics has $90 million more in the bank and a couple more VPs on the team. Chris McPherson (VP of GMP manufacturing), a WuXi AppTec alum, just finished a four-and-a-half-year tenure at Samsung Biologics , and Taleen Barsoumian (VP of client development) is an ex-senior sales consultant at Novartis who was recently US director of sales and business development for cell and gene therapy at Barkey . Meanwhile, Adam Davis has been promoted to VP of analytical development. Davis ventured off to Forge Biologics in October 2020 after serving as director of manufacturing for Abeona Therapeutics . → Bayer-backed cryogenic electron microscopy (cryo-EM) specialist Gandeeva Therapeutics has elected Brenda Cooperstone to the board of directors. Cooperstone had held the roles of chief development officer, rare disease and Pennsylvania site head at Pfizer, but left the Big Pharma in December after 24 years overall. → Ex- Adicet Bio interim chief Donald Santel has been given a seat on the board of directors at Aerovate Therapeutics , chaired by Kala Pharmaceuticals CEO Mark Iwicki . Santel also helmed Hyperion Therapeutics and is chairman of Tentarix Biotherapeutics and Ocelot Bio .

Credit: GettyImages/ Bill Oxford After a tumultuous year that weathered the continued impact of a bear market , many in biotech are looking for signs of improvement in 2023. However, industry experts have buckled up in anticipation of continued challenges this year . While some say that the tide could turn in H2 2023, others are more apprehensive. Free Report What’s missing from your IPO industry assessment? IPO activity all but stopped in 2020, as the investment community grew wary of the effects of COVID-19 on economies. No matter how deserving a business was of flotation, momentum was halted by concerns of when a ‘new normal’ of working patterns and trade would set in. Recently, sentiment has changed. Flotations picked up again during the second half of 2021, and now in 2022 the mood is decidedly optimistic. Business leaders have their eyes on fast rebounding economies, buoyant market indices and the opportunity once again to take their businesses public. As a result, global IPOs are expected to hit back this year. With GlobalData’s new whitepaper, ‘IPOs in Consumer and Retail: 5 must-include elements for your prospectus industry report’, you can explore exactly what is needed in the essential literature. GlobalData’s focus lies in the critical areas to get right: Macroeconomic and demographic environment Consumer context Industry environment Competitive environment Route to market Interested to learn more about what to include in your IPO Industry Assessment report? Download our free whitepaper. By GlobalData Submit Country * United Kingdom United States Afghanistan Åland Islands Albania Algeria American Samoa Andorra Angola Anguilla Antarctica Antigua and Barbuda Argentina Armenia Aruba Australia Austria Azerbaijan Bahamas Bahrain Bangladesh Barbados Belarus Belgium Belize Benin Bermuda Bhutan Bolivia Bosnia and Herzegovina Botswana Bouvet Island Brazil British Indian Ocean Territory Brunei Darussalam Bulgaria Burkina Faso Burundi Cambodia Cameroon Canada Cape Verde Cayman Islands Central African Republic Chad Chile China Christmas Island Cocos (Keeling) Islands Colombia Comoros Congo Congo, The Democratic Republic of The Cook Islands Costa Rica Cote D"ivoire Croatia Cuba Cyprus Czech Republic Denmark Djibouti Dominica Dominican Republic Ecuador Egypt El Salvador Equatorial Guinea Eritrea Estonia Ethiopia Falkland Islands (Malvinas) Faroe Islands Fiji Finland France French Guiana French Polynesia French Southern Territories Gabon Gambia Georgia Germany Ghana Gibraltar Greece Greenland Grenada Guadeloupe Guam Guatemala Guernsey Guinea Guinea-bissau Guyana Haiti Heard Island and Mcdonald Islands Holy See (Vatican City State) Honduras Hong Kong Hungary Iceland India Indonesia Iran, Islamic Republic of Iraq Ireland Isle of Man Israel Italy Jamaica Japan Jersey Jordan Kazakhstan Kenya Kiribati Korea, Democratic People"s Republic of Korea, Republic of Kuwait Kyrgyzstan Lao People"s Democratic Republic Latvia Lebanon Lesotho Liberia Libyan Arab Jamahiriya Liechtenstein Lithuania Luxembourg Macao Macedonia, The Former Yugoslav Republic of Madagascar Malawi Malaysia Maldives Mali Malta Marshall Islands Martinique Mauritania Mauritius Mayotte Mexico Micronesia, Federated States of Moldova, Republic of Monaco Mongolia Montenegro Montserrat Morocco Mozambique Myanmar Namibia Nauru Nepal Netherlands Netherlands Antilles New Caledonia New Zealand Nicaragua Niger Nigeria Niue Norfolk Island Northern Mariana Islands Norway Oman Pakistan Palau Palestinian Territory, Occupied Panama Papua New Guinea Paraguay Peru Philippines Pitcairn Poland Portugal Puerto Rico Qatar Reunion Romania Russian Federation Rwanda Saint Helena Saint Kitts and Nevis Saint Lucia Saint Pierre and Miquelon Saint Vincent and The Grenadines Samoa San Marino Sao Tome and Principe Saudi Arabia Senegal Serbia Seychelles Sierra Leone Singapore Slovakia Slovenia Solomon Islands Somalia South Africa South Georgia and The South Sandwich Islands Spain Sri Lanka Sudan Suriname Svalbard and Jan Mayen Swaziland Sweden Switzerland Syrian Arab Republic Taiwan, Province of China Tajikistan Tanzania, United Republic of Thailand Timor-leste Togo Tokelau Tonga Trinidad and Tobago Tunisia Turkey Turkmenistan Turks and Caicos Islands Tuvalu Uganda Ukraine United Arab Emirates United States Minor Outlying Islands Uruguay Uzbekistan Vanuatu Venezuela Viet Nam Virgin Islands, British Virgin Islands, U.S. Wallis and Futuna Western Sahara Yemen Zambia Zimbabwe Submit By clicking the Download Free Report button, you accept the terms and conditions and acknowledge that your data will be used as described in the GlobalData privacy policy By downloading this Report, you acknowledge that we may share your information with our white paper partners/sponsors who may contact you directly with information on their products and services. Visit our privacy policy for more information about our services, how we may use, process and share your personal data, including information on your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address. Thank you. Please check your email to download the Report. News of layoffs and restructuring have evoked concerns that 2023 could be a year of attrition that will push companies to prioritise key candidates and hold onto their finances. While there will be some exceptions, fundraising and initial public offerings (IPOs) will be difficult for most. Instead, reverse mergers, like privately-held Elicio Therapeutics ’s plans to merge with the NASDAQ-listed company Angion Biomedica , might be a sign of things to come. Although expert opinions differ on the future of special purpose acquisition companies (SPACs), some companies are continuing to explore their use. In December 2022, AEON Biopharma, a biopharmaceutical company, announced its plans to go public through a merger with the SPAC Priveterra Acquisition Corp. In such times, experts expect partnerships between biotechs and big pharma, like the Anima Biotech/ AbbVie deal announced earlier this month , to become even more popular. However, such partnerships depend on successful research and can also quickly fade. On January 13, the biotech Morphic Therapeutic lost its partnership with Johnson & Johnson’s subsidiary Janssen . In 2022, another partnership between AbbVie and Morphic was also dropped. Such challenges were a thematic mainstay of this year’s J.P. Morgan Healthcare Conference in San Francisco, which finished on January 12. Robert Lyne, CEO of the venture capital (VC) firm Arix Bioscience , says that the general sentiment at the conference points to expectations of a longer recovery period for public markets to become more open to activity and investment. A game of musical chairs Lyne compares the current situation to a game of musical chairs, where the status of each company essentially depends on where they were positioned when the market shifted. Consequently, companies were “unlucky” if they were short on funding when the market downturn started, he says. In 2020 and 2021, there was a boom in biotech companies going public. “We had over 100 companies go public in the sector in 2020 and 2021. Many of those companies were uncharacteristically early in development,” notes Barbara Ryan, senior advisor of Life Sciences at Ernst & Young. With the reopening of the economy months after the Covid-19 pandemic began, there was a V-shaped recovery, which gave way to increased interest rates and inflation , says Ryan. The IPO landscape seen in 2020 and 2021 should be viewed as a distortion rather than a norm, says Sameer Khambadkone, partner at ICR Westwicke, adding that this will likely not come back in 2024. One of the greatest challenges for biotechs is rising interest rates, which are inversely related to their valuations, explains Ryan. In 2022, the Federal Reserve raised the benchmark interest rate seven times, finishing in the 4.25–4.5% range in December 2022. On January 20, Christopher Waller, a member of the Board of Governors of the Federal Reserve, supported a 0.25% increase , which could be implemented at the end of January, marking a relative deceleration in interest rate increases. While the possibility of a recession lingers, some expect interest rates and inflation to peak in 2023, which could favour growth stocks and benefit biotech, adds Ryan. Inflation affects net present value (NPV) models used by biotech companies, given that they are a decade away from potential revenue, says Jack O’Meara, CEO of the biotech Ochre Bio . Pierre Socha, partner in the Early-Stage Fund of the VC firm Amadeus Capital Partners , says he views investments in blocks of 5–10 years. Companies at earlier stages of development have been hit the hardest, says Ryan. This is especially the case for public biotechs that are now having a difficult time securing money on the market, notes Ryan. Around 200 companies are now trading below their enterprise value, she says. While the stock market indices may be down by 40%, the market cap of some companies is down by 90%, leading to news of restructurings and closures, says Ryan. While it is only January, several companies have already announced cuts and layoffs this year. UK-based biotech ReNeuron announced plans to lay off 40% of its employees , and Cyteir Therapeutics will be firing 70% of its staff . Others, like Elevation Oncology and Fate Therapeutics , have announced similar plans. Alternate funding strategies The fiscally challenging environment means that smaller biotechs need to be especially careful when it comes to cash burn rates, as the nature of the market has changed, says Lyne. Since the 2008 financial crisis , the cost of capital was cheap, but that has now risen, he adds. While this is not unique to biotech, the sector is particularly exposed to these complications since the companies are loss-making during the development phase, he adds. Moreover, VC funds face challenges in not making returns on their portfolios, which means they will have to hold onto them longer, says Gabor Garai, co-chair of the Life Sciences Team at Foley & Lardner LLP, in an email to Pharmaceutical Technology . This will also make fundraising challenging and adversely impact peoples’ enthusiasm about life science companies, creating a vicious cycle, he adds. There is also the option of going the SPAC route . However, Garai describes SPACs as a fad and notes that eventually underperforming SPACs will be forgotten by investors. The degree of difficulty in completing these transactions has grown exponentially, says Khambadkone. In December 2022, AEON Biopharma announced plans to go public through a merger with the SPAC Priveterra Acquisition Corp. The current state of the market played a role in AEON’s decision to merge with a SPAC, says the company’s CEO Marc Forth, adding that IPOs are largely unavailable. The success of these mergers is very specific to the transaction itself, says Forth. IPOs will also likely not be an easy avenue to raise funds for the time being. Khambadkone says that the issue is not necessarily whether a company can go public, but how it will trade afterwards. While Garai says it will take time for the market to right itself, Lyne went further and said he does not expect to see a rush of successful IPOs in the coming months. Still, there could be more activity in the second half of the year compared to the first, says Chris Brinzey, partner at ICR Westwicke. Surviving a tough biotech market In the backdrop of these challenges, companies may prioritise licensing and partnerships with larger pharma earlier than usual, says Lyne. However, this will give even greater leverage to large players, allowing them to be more selective with partners, says Marcelo BIGAL , CEO of Ventus Therapeutics . In September 2022, Bigal’s Ventus Therapeutics partnered with the Danish giant Novo Nordisk and received a $70 million upfront payment with the possibility of milestone payments of up to $633 million. However, he emphasises the deal was not done for purely financial reasons, but rather as an opportunity to license treatments, which the company did not want to pursue. While it is hard to say if specific therapeutic areas will be particularly hit by these ongoing challenges, investors could favour those pursuing a well-understood disease profile and drug modality, says Lyne. But such areas are highly competitive. In contrast, having a successful product that addresses an unmet medical need would bring value and attract potential interest, says Robert Jacks, CEO of Sparrow Pharmaceuticals , a biotech that develops potential treatments in endocrinology and rheumatology. Most recently, Sparrow announced that the first patient was dosed on a Phase II trial of SPI-62, a potential Cushing’s Syndrome treatment. According to Bigal, there is a cycle of attractiveness in biotechs where certain indications or disease areas get increased attention at a point. Although the market is relatively hostile, the largest commercialised pharma companies have tremendous amounts of capital, says Ryan. Quoting EY’s annual Firepower Report , Ryan says pharma companies hold $1.4 trillion of dealmaking firepower. This capital could be spent on de-risked approaches, such as partnering, rather than acquisitions and mergers, she notes . In a nutshell, companies with enough funding and positive data could perform well in 2023, whereas those with significant capital constraints and uninspiring data will not, Ryan says. However, to Lyne, this is not necessarily a negative development. “As we come through this cycle, ensuring that the industry is more robust and [there is] a more judicious allocation of capital, [that] will make for a more sustainable industry in the medium to long term,” says Lyne. Free Report What’s missing from your IPO industry assessment? IPO activity all but stopped in 2020, as the investment community grew wary of the effects of COVID-19 on economies. No matter how deserving a business was of flotation, momentum was halted by concerns of when a ‘new normal’ of working patterns and trade would set in. Recently, sentiment has changed. Flotations picked up again during the second half of 2021, and now in 2022 the mood is decidedly optimistic. Business leaders have their eyes on fast rebounding economies, buoyant market indices and the opportunity once again to take their businesses public. As a result, global IPOs are expected to hit back this year. With GlobalData’s new whitepaper, ‘IPOs in Consumer and Retail: 5 must-include elements for your prospectus industry report’, you can explore exactly what is needed in the essential literature. GlobalData’s focus lies in the critical areas to get right: Macroeconomic and demographic environment Consumer context Industry environment Competitive environment Route to market Interested to learn more about what to include in your IPO Industry Assessment report? Download our free whitepaper. By GlobalData Submit Country * United Kingdom United States Afghanistan Åland Islands Albania Algeria American Samoa Andorra Angola Anguilla Antarctica Antigua and Barbuda Argentina Armenia Aruba Australia Austria Azerbaijan Bahamas Bahrain Bangladesh Barbados Belarus Belgium Belize Benin Bermuda Bhutan Bolivia Bosnia and Herzegovina Botswana Bouvet Island Brazil British Indian Ocean Territory Brunei Darussalam Bulgaria Burkina Faso Burundi Cambodia Cameroon Canada Cape Verde Cayman Islands Central African Republic Chad Chile China Christmas Island Cocos (Keeling) Islands Colombia Comoros Congo Congo, The Democratic Republic of The Cook Islands Costa Rica Cote D"ivoire Croatia Cuba Cyprus Czech Republic Denmark Djibouti Dominica Dominican Republic Ecuador Egypt El Salvador Equatorial Guinea Eritrea Estonia Ethiopia Falkland Islands (Malvinas) Faroe Islands Fiji Finland France French Guiana French Polynesia French Southern Territories Gabon Gambia Georgia Germany Ghana Gibraltar Greece Greenland Grenada Guadeloupe Guam Guatemala Guernsey Guinea Guinea-bissau Guyana Haiti Heard Island and Mcdonald Islands Holy See (Vatican City State) Honduras Hong Kong Hungary Iceland India Indonesia Iran, Islamic Republic of Iraq Ireland Isle of Man Israel Italy Jamaica Japan Jersey Jordan Kazakhstan Kenya Kiribati Korea, Democratic People"s Republic of Korea, Republic of Kuwait Kyrgyzstan Lao People"s Democratic Republic Latvia Lebanon Lesotho Liberia Libyan Arab Jamahiriya Liechtenstein Lithuania Luxembourg Macao Macedonia, The Former Yugoslav Republic of Madagascar Malawi Malaysia Maldives Mali Malta Marshall Islands Martinique Mauritania Mauritius Mayotte Mexico Micronesia, Federated States of Moldova, Republic of Monaco Mongolia Montenegro Montserrat Morocco Mozambique Myanmar Namibia Nauru Nepal Netherlands Netherlands Antilles New Caledonia New Zealand Nicaragua Niger Nigeria Niue Norfolk Island Northern Mariana Islands Norway Oman Pakistan Palau Palestinian Territory, Occupied Panama Papua New Guinea Paraguay Peru Philippines Pitcairn Poland Portugal Puerto Rico Qatar Reunion Romania Russian Federation Rwanda Saint Helena Saint Kitts and Nevis Saint Lucia Saint Pierre and Miquelon Saint Vincent and The Grenadines Samoa San Marino Sao Tome and Principe Saudi Arabia Senegal Serbia Seychelles Sierra Leone Singapore Slovakia Slovenia Solomon Islands Somalia South Africa South Georgia and The South Sandwich Islands Spain Sri Lanka Sudan Suriname Svalbard and Jan Mayen Swaziland Sweden Switzerland Syrian Arab Republic Taiwan, Province of China Tajikistan Tanzania, United Republic of Thailand Timor-leste Togo Tokelau Tonga Trinidad and Tobago Tunisia Turkey Turkmenistan Turks and Caicos Islands Tuvalu Uganda Ukraine United Arab Emirates United States Minor Outlying Islands Uruguay Uzbekistan Vanuatu Venezuela Viet Nam Virgin Islands, British Virgin Islands, U.S. Wallis and Futuna Western Sahara Yemen Zambia Zimbabwe Submit By clicking the Download Free Report button, you accept the terms and conditions and acknowledge that your data will be used as described in the GlobalData privacy policy By downloading this Report, you acknowledge that we may share your information with our white paper partners/sponsors who may contact you directly with information on their products and services. Visit our privacy policy for more information about our services, how we may use, process and share your personal data, including information on your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address. Thank you. Please check your email to download the Report.

WALTHAM, MA, USA & MONTREAL, Canada I January 04, 2023 IVentus Therapeutics, Inc., a biopharmaceutical company utilizing structural biology and a proprietary computational chemistry platform to identify and develop differentiated small molecule therapeutics across a broad range of diseases, announced today the nomination of a potential first-in-class cGAS inhibitor, VENT-03, as the company’s first development candidate directed against cGAS. This represents the third development candidate nominated in the past 12 months to Ventus’ pipeline, following the nominations of VENT-02, a brain-penetrant NLRP3 inhibitor, and VENT-01, a peripherally restricted NLRP3 inhibitor that is partnered with Novo Nordisk. cGAS activation leads to a type I interferon response, which is implicated in a broad range of diseases, including cutaneous lupus erythematosus, systemic lupus erythematosus, systemic sclerosis, and dermatomyositis, as well as neuroinflammatory and neurodegenerative diseases. In addition, genetic diseases, such as severe infant onset Aicardi-Goutières Syndrome and genetic forms of chilblain lupus, are driven by genetic mutations that lead to the activation of the pathway in humans. In disease-relevant preclinical studies, VENT-03 demonstrated robust in vivo pharmacodynamic activity and efficacy. “cGAS is a highly coveted target for pro-inflammatory and neurodegenerative diseases for which no inhibitor has advanced into clinical development to date, despite significant efforts from the drug discovery industry over the past decade,” said Ventus’ President and CEO Marcelo Bigal, M.D., Ph.D. “We expect VENT-03 to be the first cGAS inhibitor to enter the clinic, with a clinical development plan tailored to provide multiple data readouts prior to Phase 2 trials.” In December 2022, Ventus scientists published a state-of-the-art review on the industry’s progress to develop inhibitors in the cGAS-STING pathway. In May 2022, Ventus announced that it had been awarded a Lupus Research Program Idea Award from the U.S. Department of Defense, and, along with it, a multi-year grant to study the role of cGAS in lupus and the potential for treatment of the disease using a cGAS inhibitor. “The nomination of VENT-03 as our first development candidate targeting cGAS was made possible by the unique insights provided by ReSOLVE, our proprietary drug discovery platform,” said Ventus’ Chief Scientific Officer Michael Crackower, Ph.D. “After identifying a high-resolution co-crystal structure of cGAS that was different from what had been previously published, we used ReSOLVE to determine characteristics of the binding pocket that are unique to only some of the target’s many conformations, a major breakthrough that transformed our ability to achieve high potency in our cGAS inhibitors. Given the highly validated role of the cGAS pathway as a driver of disease, we are now developing additional cGAS inhibitors to achieve the full potential of this attractive target in distinct therapeutic areas.” About cGAS cGAS is an intracellular pattern recognition receptor that is activated after binding to double-stranded DNA (dsDNA) in the cytoplasm. dsDNA in the cytoplasm is generally associated with cellular dysfunction, as seen in multiple diseases such as cutaneous lupus erythematosus (CLE), systemic lupus erythematosus (SLE) and dermatomyositis (DM). cGAS activation leads to cGAMP formation, activation of STING and downstream expression of type I interferons such as IFN-α, -ß and -κ, leading to pronounced inflammation and tissue damage. This pathway also leads to the expression of several other proinflammatory cytokines such as CXCL10. Genetic mutations that lead to the aberrant activation of cGAS/STING in humans are known to cause severe infant onset Aicardi-Goutières Syndrome (AGS) and genetic forms of chilblain lupus, demonstrating the relevance of the pathway in human diseases. Given that the pathophysiology and presentation of these genetic diseases have significant similarities with idiopathic complex diseases such as CLE, SLE and DM, these diseases represent highly compelling opportunities for treatment with a cGAS inhibitor. About Ventus Therapeutics Ventus Therapeutics is a biopharmaceutical company deploying leading-edge structural biology and computational chemistry tools to create novel small molecule medicines for challenging targets, with an initial focus on immunology, inflammation and neurology. The Company’s proprietary drug discovery platform ReSOLVE gives it the ability to discover and develop medicines through unique insights into protein targets and to create dynamic blueprints that can guide the development of differentiated small molecule medicines. The Company’s lead programs target key innate immune modulators, including NLRP3 and cGAS. The Company is using its ReSOLVE platform to improve the way medicines are discovered and developed, with the ultimate goal of restoring the health of people facing serious illnesses. For more information, please visit www.ventustx.com and engage with Ventus on LinkedIn. SOURCE: Ventus Therapeutics