史上最大生物科技IPO來了！mRNA療法「獨角獸」美國交表 估值達到70億美元 華爾街見聞※來源：華爾街見聞2018/11/11 11 月 9 日周五，主打 mRNA（信使 RNA）療法的生物科技新銳公司 Moderna Therapeutics 已向美國證券交易委員會（SEC）遞交文件，申請在那斯達克上市，募資 5 億美元，股票代碼 mRNA。申報文件顯示，這一估值 70 億美元的生物科技獨角獸計劃將所募資金用於藥物研發和臨床開發，領頭承銷此次 IPO 的投行包括摩根士丹利、高盛和摩根大通，股東包括富達投資、紅杉資本等。此外，在截至9月30日的9個月中，Moderna 營收 1 億美元，虧損 2.43 億美元。去年同期，該公司則是營收 1.14億美元，凈虧損 2.18 億美元。根據官網的公開資訊，截至9月30日，Moderna資產負債表上共有現金 12.3 億美元，沒有負債。在此之前，該公司已經通過私人融資的方式籌集了 24 億美元資金，最新估值就是在今年 2 月的一輪 5 億美元融資中獲得的。這家成立於 2010 年的生物科技企業致力於研發傳染病、癌症免疫和罕見病相關療法。簡單來說，Moderna 計劃製造合成 mRNA，之後注射入患者體內，使其體內細胞自行產生藥物療法。這一療法廣受青睞，原因在於若能將 mRNA 導入體內的特定細胞或者組織中，那麼就可以利用細胞本身的核糖體表達出 mRNA 編碼的蛋白。因為 mRNA 可以表達的蛋白多種多樣，所以可以通過 mRNA 治療的疾病非常廣泛。與基因療法相比，mRNA 不會影響DNA 的結構，而且隨著時間的推移會被細胞分解，所以它的副作用能夠得到更精確的控制。因此，mRNA 療法在治療疾病方面有非常大的潛力。但值得注意的是，使用 mRNA 作為治療手段也需要克服兩個技術難關：一是體外合成的 mRNA會被人體的免疫系統識別成外來物，從而產生免疫反應。二是如何將mRNA導入到特定的細胞和組織中發揮作用。目前，該公司研發管線中有21個研發項目，其中 10 個已進入臨床開發階段，走得最遠的是代號為 AZD8601 的局部再生療法，現已進入第二階段。在 Moderna 之前，規模最大的生物科技 IPO是信達生物製藥在港上市，募資規模達 33 億港元，約合 4.2 億美元。10月31日，信達生物在港交所掛牌，以 13.98 港元的股價發售約 2.36 億股。作為中國生物製藥領域最具影響力的獨角獸之一，信達生物打破了港股新股破發潮，首日掛牌就取得開門紅，股價一度漲超 20%。『新聞來源／華爾街見聞』
Can Moderna Live up to the Hype? mRNA Company Increases IPO Goal to $600 Million Published: Nov 29, 2018 By Mark Terry It's very clear that Moderna Therapeutics can raise money. What is less clear is if the company can live up to the hype and enthusiasm of investors. The Cambridge, Mass.-based biotech company, which had proposed a $500 million initial public offering (IPO) earlier this month, came back Wednesday with a new goal of $600 million. Since its founding in 2010, Moderna has raised more than $2.6 billion in equity financing. But it still has no product on the market. As of September 30, the company had cash, cash equivalents, and investments of $1.2 billion. The company focuses on messenger RNA (mRNA) therapeutics. mRNA's job in the cells is to transport genetic information from DNA to the ribosome, providing the amino acid sequence of the eventual proteins the DNA is coded for. In Moderna's tech platform, the company engineers mRNA to then deliver whatever protein codes they want the cells to produce, turning the cells into vaccine or drug-manufacturing factories. There have been difficulties in getting the drugs into cells and there have been lawsuits over which delivery systems Moderna owns. However, in February, Moderna settled with Arbutus Biopharma and Acuitas Therapeutics over the delivery system technology. The company has a development pipeline of 21 programs. Ten of those are in the clinic and another three have open Investigational New Drug (INDs) submissions. Nine of those in the clinic are in Phase I and one is in Phase II, according to a July corporate update. The company has not provided any updates on any of its clinical programs. Most recently, on October 8, it published preclinical research in the journal Nature Medicine, demonstrating the utility of its RNA platform to express therapeutic levels of protein in liver tissue in preclinical models of acute intermittent porphyria (AIP), an inherited, rare metabolic disorder. There's never been any doubt about Moderna's ambition. The company's pipeline include therapies for infection disease vaccines, cancer vaccines, immuno-oncology, regenerative therapies, systemic secreted therapies for diseases like heart failure and Fabry disease, and systemic intraceullar therapeutics for rare metabolic disorders like propionic acidemia, methylmalonic acidemia and phenylketonuria. Funding has come from investors and grants from the Bill & Melinda Gates Foundation, the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS), and the Defense Advanced Research Projects Agency (DARPA).The filings with the U.S. Securities and Exchange Commission (SEC) not only updated the proposed IPO but disclosed the size of the company's largest investors' stakes. Noubar B. Afeyan, company chairman, holds 19.5 percent, or 58,882,696 shares. Flagship Pioneering holds 18 percent of Moderna shares. They helped launch the company. AstraZeneca, a collaboration partner, holds 8 percent. Harvard Medical School researcher Timothy Springer and affiliates with him hold 5 percent. Stephane Bancel, company chief executive officer, holds more than a 9 percent stake in the company. Slightly more than half of the expected proceeds of the IPO, about $315 million to $345 million, will be used to fund drug discovery and clinical development. About $75 million to $85 million will fund further development of the mRNA tech platform and creating new modalities. The rest, between $170 million and $210 million, will be working capital and other unspecified general corporate purposes. The company stated in its amended SEC filing, "We may also use a portion of the net proceeds to in-license, acquire, or invest in complementary businesses or technologies to continue to build our pipeline, research and development capabilities, and our intellectual property position, although we currently have no agreements, commitments, or understandings with respect to any such transaction."
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