1.事實發生日:106/09/22
2.公司名稱:藥華醫藥股份有限公司
3.與公司關係(請輸入本公司或子公司):本公司
4.相互持股比例:不適用
5.發生緣由:
歐洲藥物管理局(European Medicines Agency, EMA)進行本公司藥品
Ropeginterferon alfa-2b（P1101）藥證核准前查廠，查廠結果無重
大缺失。
6.因應措施:
本公司歐洲夥伴AOP公司已於2017年2月23日向EMA提出適應症為治療真
性紅血球增生症(PV)第一線用藥之BESREMI (P1101）新藥上市許可申請，
審核程序目前正在進行中。EMA對生產Ropeginterferon alfa-2b（P1101）
的本公司台中廠實地查廠工作已於今天完成，查廠結果無重大缺失。
EMA將依其時程提供查廠報告，本公司在不影響Ropeginterferon alfa-2b
（P1101）藥證審核時程下，將於時限內完成相關更正作業。
7.其他應敘明事項:
新藥開發時程長、投入經費高且並未保證一定能成功， 此等可能使投資面
臨風險，投資人應審慎判斷謹慎投資。
To be or not to be.
日期：2017/9/25
【財訊快報／何美如報導】
藥華藥( 6446 )公告，EMA進行藥證核准前的實地查廠結果，無重大缺失，順利過關成為台灣第一家通過EMA查廠的生物藥新藥公司。藥華藥表示，EMA查廠小組將會依集中審核程序(CP)時間表期限內提交查廠報告，P1101治療真性紅血球增生症(PV)新藥的歐盟上市審核進度一如預期，目標明年上半年取得藥證，明年下半年進軍歐洲市場。
執行長林國鐘表示，本次查廠結果順利過關，再次證明公司同仁在製造P1101的各階段試驗都是按部就班進行，一旦獲EMA最終審查通過取得PV藥證，P1101將是全球第一個被核准用於治療PV病患的干擾素且為第一線用藥，屆時將大幅改變醫師的用藥方式，愛治膠囊(Hydroxyurea,HU)可能不再被使用做為治療PV藥物，這對P1101未來拓展歐美PV市場具相當大的指標意義，同時在使用傳統干擾素治療PV的中國市場更有激勵效果。
藥華藥指出，P1101在第一線用藥的方式下，隨著時間與劑量上的調整，呈現有效性與一致性，在治療第18個月時更可以明顯發現，使用P1101治療組別與HU對照組在完全血液反應率(CHR)指標上出現黃金交叉，P1101組別持續上升、HU對照組則迅速下滑，這表示P1101對PV病患的長期治療是有極大幫助的。
藥華藥解釋，這是因為P1101是新一代PEG長效型干擾素，除具低副作用、長效且可使用高劑量等特色外，更可以抵禦外來入侵的病毒、細菌達到調整病變基因，進行幹細胞修復，是具改善病程進展或延緩疾病進一步惡化的藥物(Disease-modifying drug)。目前Continuation-PV臨床試驗持續進行，合作夥伴AOP公司預計於12月初的美國血液學年會(ASH)中發表完整的臨床試驗成果。
歐洲夥伴AOP公司於今年2月23日向EMA提出適應症為治療PV第一線用藥之P1101(商品名BESREMI)新藥上市許可申請並完成確認，7月5日收到AOP轉來之歐盟人體用藥委員會(CHMP)依據EMA審核程序的第120天問題彙總清單(CHMP day 120 list of questions)，公司國內外團隊與AOP團隊將在期限內向CHMP提交相關資料，屆時EMA會再啟動第二階段審核並接續計時，目標期望明年6月前能取得EMA做出核發藥證的最終決定(Final Commission decision)。藥華藥指出，若一切順利，P1101治療PV新藥有機會於明年下半年正式於歐盟國家上市銷售、嘉惠病患。
藥華醫藥接獲美國肝病研究年會(AASLD)通知，藥華藥P1101對於未接受干擾素治療之慢性B型肝炎患者的抗病毒活性與安全性之開放性、隨機分配、有效藥對照、劑量探索的臨床I/II試驗(A12-201)的摘要已被接受並且被選為late breaking報告。(註：late breaking只有最新的研究才能投稿，因此競爭激烈)。AASLD年會將於2017年10月20日至24日於美國華盛頓特區舉行。黃奕文醫師(現為台北醫學大學醫學系內科部定助理教授，臺灣大學醫學系內科部定講師，國泰醫院胃腸肝膽科主治醫師，臺大醫院胃腸肝膽科兼任主治醫師)將會在2017年10月23日發表此臨床試驗詳細的療效及安全性數據。
另外...
公告本公司策略夥伴AOP公司將於ASH 2017發表新藥P1101
用於治療真性紅血球增生症(PV)之CONTIUNATION-PV臨床結果
5.發生緣由:
本公司策略夥伴AOP公司將於ASH 2017發表新藥P1101用於治療真性紅血球增生症
(PV)之CONTIUNATION-PV臨床結果
6.因應措施:
藥華醫藥接獲本公司策略夥伴AOP公司通知將於美國時間12月10日7:45AM在美國
亞特蘭大舉行的2017血液病醫學年會(ASH)，以口頭報告形式發表有關P1101用於
治療真性紅血球增生症(PV)之第三期人體臨床試驗PROUD-PV後繼續臨床試驗
CONTIUNATION-PV治療結果(含PROUD-PV共二年)。ASH年會將於2017年12月9日至
12日於美國亞特蘭大舉行。
公司說過，要在新藥上市前公開，來做一個大的宣傳......大家靜待吧!!
看來未來一年內都將佳音不斷!!
Ray Jo： My ET progressed to MF - confirmed by my BMB in April this year. I found out the results of my secondary mutations after my visit with Dr V. I did email him and he said it would not alter my current treatment plan with those results. His reasoning for not wanting to start interferon is due to clinical data only supports 5 year of it being effective. So he wants to wait. I’m intermediate +2. I’m not sure if I agree and will most likely seek a second opinion.
今年四月經由骨髓活檢(BMB), 證實我的紅血球增生症(ET) 已惡化到骨髓纖維化(MF)。在我見了Srdan醫生後得到了我二次突變的檢查結果。我透過e-mail聯繫Srdan醫生, 但他不打算針對我的檢驗數據改變目前的治療方式。他認為干擾素在臨床研究僅顯示5年數據是有效的, 所以他想等待。我目前診斷出在中間範圍, 我不確定我是否要尋求其他治療建議。
Francesca de Gasparis：
I would agree with you about seeking treatment now and another opinion. Interferon has had good results for early MF and can slow progression. Why wait?
我同意你現在就要求治療以及其他治療建議。干擾素在早期MF治療上有著良好成效且可減緩疾病惡化效果, 為何仍要等待？
Paul Cherubini：
Ray Jo in view of your transformation to early MF and secondary mutations the Danes, French and Dr. Silver would likely say you urgently need the most powerful progression stopping treatment available or at least interferon alone。
Ray, 針對你早期MF和二次突變的惡化, 包含丹麥、法國和Silver醫生治療上均會認為你至少需要強力控制疾病惡化的治療方式或至少使用干擾素進行治療。
Wim Louage：
Very strange, I saw a post last week from a younger woman who saw Dr V and he wanted to start with Interferon. I do believe he saw more results in the meantime from trials, he was also hoping that the new Ropeginterferon would come very fast to the US to start trials there ....
這很奇怪, 我上週看到一個年輕女性提到她見了Srdan 醫生, 而醫生希望她開始使用干擾素治療, 我相信醫生一定見到很多臨床成果。他也希望 P1101 (the new Ropeginterferon)能儘快到美國進行臨床....
Ray Jo：
Wim Louage, I saw him in June if this year
Wim Louage, 我是在今年六月見到他的
Wim Louage：
Ray Jo , yes I know you did, .... it is just not logic
Ray Jo, 我知道, 我只是覺得這邏輯上不合理。
Ray Jo：
Wim Louage, are you sure the patient wasn’t PV? I know with PV they start them on Peg
Wim Louage, 你確定你提的病患不是PV病患嗎? 我知道他們針對PV病患會使用長效型干擾素進行治療。
Wim Louage：
Ray Jo , no she morphed to MF
Ray Jo , 不, 她也惡化到MF。
Ray Jo：
Wim Louage yeah it doesn’t make sense. I have another follow up / lab draw in a few weeks with my local HemOnc. Going to see what the results look like and email Dr V again to see if he would reconsider
Wim Louage, 是的, 這不合理, 我會在近幾周內去見本地的血液學/腫瘤科醫生, 針對這個結果進行了解, 並且mail 給Srdan 醫生, 看他是否會重新考慮。
Wim Louage：
www.youtube.com/watch?v=NQBY1KyzcVQ
Ropeginterferon, welcome to the US In this presentation from the…
這是 "Ropeginterferon (P1101), 歡迎來到美國"的簡報影片, 請參考。
Ray Jo：
Wim Louage thanks for sharing - don’t know if his opinion for my treatment is different since it’s MF vs PV in the video
Wim Louage, 感謝分享, 我不確定是否因為我是MF 和這段影片說的是PV, 導致Srdan 醫生在我的治療方式上有所不同。
Wim Louage：
Ray Jo , he says in the beginning early MF
Ray, 他有提到早期MF。
Paul Cherubini：
Ray Jo , I hope you can track down the precise source of Dr. V’s assertion than Pegasys is effective for only 5 years. And determine what about it becomes ineffective after 5 years? Curiously Stanford’s Dr. Gotlib recently told a patient something similar - that if she started taking Pegasys for her PV that she could only take it for 5 years. The real interferon experts; i.e. Dr’s Silver, Hasselbalch, Kiladjian and Gisslinger have never mentioned anything about a time limitation on interferon. Indeed, there are sill some ET and PV patients alive today that started on interferon 25 years ago (and who never progressed to MF or AML)
Ray, 我希望你能去進一步去了解Srdan 醫生的結論來源, 他提到Pegasys 只有五年的效期, 那五年後就無效了嗎? 很詭異的是, 史丹佛的Gotlib醫生也和他的病人提到類似的觀點。而真正的干擾素權威如Silver 醫生、Hasselbalch、Kiladjian 和 Gisslinger (PROUD-PV 計畫主持人) 均未提及干擾素治療有時間上限制。事實上, 目前有一些ET和PV病患在25年前使用干擾素治療後至今仍活著, 且他們的疾病無惡化至骨髓纖維化(MF) or 急性骨髓型白血病(AML)。
Francesca de Gasparis：
There has been research on interferon efficacy in early MF (and later when used in conjunction with other treatments). If I were you, I would press for this treatment, unless there is an additional factor in your case that Dr V is concerned about, which he can then tell you.
有很多臨床研究均顯示干擾素在早期MF上的療效。如果我是你的話, 我會使用干擾素治療, 除非Srdan 醫生針對你的個別狀況有其他的考量。
癌症預防的追求：藥華醫藥 PharmaEssentia 創辦人兼醫學學者林國鐘
www.theceomagazine.com/business/ko-chung-lin/
Dr Ko-Chung Lin heads a cohort of scientists at PharmaEssentia that has made a major
breakthrough in the prevention of cancer.
林國鐘博士帶領藥華醫藥的一群科學家, 在癌症預防上有了重大突破。
Elite executives are often asked, ‘What’s the most rewarding aspect of your job?’ It’s a simple question, but due to the diverse nature of industries, it’s not totally unheard of to be met with dead-air. Of course, that wasn’t the case with Dr Ko-Chung Lin. How could it be, when the corporation he co-founded is on the verge of saving many, many lives?
傑出的管理者最常被問到的是：你的工作最有價值的部分為何? 這是個很簡單的問題, 但因為各行各業的領域不同, 導致有時會遇到氣氛很僵的時候。當然, 這件事並不會發生在林國鐘博士身上, 因為他創辦的公司正需要拯救許多、許多的生命。
PharmaEssentia was established in 2003 by Ko-Chung and a group of Taiwanese-American executives and scientists, aiming to develop effective, safe and cost-effective pharmaceutical products for the treatment of human diseases. Fourteen years and countless clinical tests later, and in cooperation with Austrian multinational pharmaceutical company AOP Orphan, the team is using a game-changing drug candidate called Ropeginterferon alfa-2b (P1101) to prevent cancer.
藥華醫藥於 2003 年由林國鐘和一群台灣-美國的管理者和科學家所成立, 成立宗旨在於研發出有效、安全和具有成本效益的藥物以治療人類疾病。在 14 年間與奧地利的跨國製藥公司 AOP Orphan 合作進行無數的臨床試驗後，這個團隊研發出令人耳目一新的新藥稱作 Ropeginterferon alfa-2b (P1101)用來預防癌症。
Ko-Chung Lin is living a scientist's dream
林國鐘的科學夢
The diseases PharmaEssentia are targeting with P1101 are rare blood cancers, such as Polycythemia Vera. And after years of intensive trials, only a final sign-off from the governing medical bodies in Europe and the US stands between patients and the drug.
藥華藥所研發的 P1101 主要用來治療罕見的血癌疾病，如真性紅血球增多症(PV)。經過多年的密集的臨床臨床，現在只需要歐盟以及美國最終的藥證許可即可讓病患用藥。
“We have conceptualised an idea, and now we’re making it a reality through Ropeginterferon alfa-2b,” Ko-Chung smiles. “We found out it works in the treatment of Polycythemia Vera, and now we’re just waiting on the final approval to save patients’ lives. I’ve been a scientist for 40 years, and it’s the dream of any scientist to be in this position. It’s very rewarding.”
林國鐘笑著說 "我們藉由 Ropeginterferon alfa-2b 將原先的概念具體化, 我們發現 P1101 在 PV 的治療上很有效, 而我們現在在等待最後的批准即可用來拯救病患的性命。我擔任科學家已 40 年, 這是任何一個科學家在此夢寐以求的, 這一切是有意義的。
I’ve been a scientist for 40 years, and it’s the dream of any scientist to be in this position. It’s very rewarding. Testing has revealed that P1101 delays disease progression, has limited side effects, and has the potential to deliver a curative response. Patients can also self-administer at home, and on a monthly rather than fortnightly basis – a significant improvement in both convenience and comfort from current treatment options.
臨床試驗顯示出 P1101 可減緩疾病惡化、副作用極低，且有治癒的可能。病患可以在家自行施打, 周期為每個月一針，而非每兩週一針, 和現有治療方案相比, 這個顯著的改良不僅讓病患感到更便利且更舒適。
Most excitingly, PharmaEssentia’s potential is limitless. It has received another US$125 million in funding after being listed last year, has set up subsidiaries in the US, Japan and China, has more than 150 employees, and carried out its own manufacturing at the Central Taiwan Science Park.
最令人興奮的是，藥華醫藥 PharmaEssentia 的潛力無限。自去年掛牌上櫃後, 額外募集了 1.25 億美元的資金，藥華在美國、日本和中國設有子公司，擁有超過 150 名員工，並在台灣中部科技園區擁有自己的製造工廠。
“That funding allows us to maximise our current major candidates. So, we are currently running two phase-three trials simultaneously, and two more are scheduled to start at the end of this year. “Second, that funding allows us to build up our US operations, with our subsidiary in Boston opening three months ago.”
“這筆資金使我們的產品線最大化, 所以我們目前正同時進行兩個臨床三期試驗，而另外兩項是預計在今年年底啟動臨床三期。 “其次，這筆資金使我們能夠在三個月前在波士頓建構我們的美國營運團隊。
PharmaEssentia pushes the industry forward
藥華醫藥持續推動業務進展
While PharmaEssentia is well on its way to becoming a major international biopharmaceutical company, there was a time when it was purely a pipe dream. “An American scientist and friend of mine was informed that the Taiwan Government wanted to repeat the success story it had with semiconductors,” Ko-Chung reveals.
雖然藥華目前正朝著成為國際上主要的生物製藥公司, 但曾經這只是個白日夢。林國鐘說到 "一個美國科學家和我的一個朋友告知台灣政府想要複製半導體的成功經驗"。
“Dr Fu-Kuen Lin, the inventor of EPO at Amgen, saw an opportunity in the biotech industry, and that was almost 25 years ago. When we learned that, we invited him to come back to Taiwan to our locations to just talk to the people. He thought, if we could get sufficient funding and attract some of the world’s best scientists we would be challenged to help not only Taiwan but push the industry forward globally.”
安進的 EPO(紅血球生長激素)發明人林福坤博士, 在 25 年前見到了生技業的機會。當我們知道時, 我們邀請他回國並且和大家說明。他想, 如果能更募集充足的資金且吸引全球最優秀的科學家前來, 我們將不僅僅只是立足台灣而是放眼全球。
In 2002, after two decades working as a scientist in the US with companies like Biogen, Ko-Chung returned to his native Taiwan to lobby for funding. Knowing, like his co-founder, that it would be the key ingredient to the success of their operation.
在 2002 年, 在美國如 Biogen 累積 20 年的科學家工作經歷後, 林國鐘回台灣開始募資。如他的共同創辦人, 他知道這將是未來公司營運成功的關鍵要素。
Securing funding
資金保證
Fortunately, he landed a big fish in the form of the Taiwan Government’s National Development Fund.“It was imperative that we got the government on board, because it gave an assurance to private investors. They associate government involvement with ongoing development and evaluation,” he says.
幸運的是，他取得台灣國發基金的資金。 “讓政府入股我們公司是必要的，因為這會給予一般投資者一個投資保障, 他們可以對於政府參與以及公司未來的發展做綜合性的評估，“他說。
“As a result, we raised another 60% of funding from six private investors. When we first started, we knew the funding wouldn’t be easy. Taiwan wasn’t very familiar with biotech success, and long-term investment with no guarantee of success is not an easy sell,” he explains.
“因此，我們從 6 位個人投資者募集了 60％的資金。我知道公司一開始成立時募集資金是不容易的事。台灣對生物技術產業的成功模式仍不熟悉，且無法保證長期投資的成功讓募資變成一件困難的事 “他解釋說。
“If you invest in other markets like manufacturing for instance, you can produce a result in three to five years. But in biotech, it will take 10 to 15 years at least before you can expect to see any sort of return. Our company is now in its fourteenth year, and only in the past 12 months have we had a product be reviewed by the European Medicines Agency (EMA).”
“如果投資者投資於像製造業這樣的產業，3-5 年即可產生結果。但是在生技產業，在獲得回報之前, 至少需要 10 到 15 年的時間。公司目前邁入第 14 年，但長達 14 年光景也只有在近 12 個月內，有一個產品進入歐洲藥品管理局（EMA）審查。“
Despite the fundraising challenge, Ko-Chung secured an initial sum of US$17 million, enough to set up a US-standard lab at Nankang Software Park. A second round of funding in 2006 almost doubled the previous total, and its entry into the Taipei Exchange has since guaranteed its long-term financial security.
儘管面臨募資方面的挑戰, 林國鐘仍募得初期的 1700 萬美金，這金額足以在南港軟體園區成立一個美國標準型的實驗室。2006 年的第二輪募資金額較前一輪翻倍，而掛牌進入台北金融交易也保證他們長期財務上的安全。
Determined to beat cancer
決心擊退癌症
While it’s full steam ahead with the P1101 phase-three trials, PharmaEssentia has also been granted the rights from business partner Kinex to sell Oraxol in certain Asian countries – an oral formulation of paclitaxel, a chemotherapy agent commonly used in first-line treatment for breast cancer.
雖然 P1101 臨床三期試驗已全面展開，帶藥華醫藥仍決定從合作夥伴 Kinex 中取得某些亞洲國家Oraxol 的銷售權, 這是一種用於口服的紫杉醇，主要用於乳癌一線用藥的化療藥物。
PharmaEssentia has found that Oraxol dramatically reduces the side effects experienced with paclitaxel, and Ko-Chung and his team are exploring the drug in relation to breast cancer – the leading cause of cancer death in women. As you can see, occupations don’t come much more gratifying than leading a biopharmaceutical company that is determined to beat cancer.
藥華醫藥發現 Oraxol 可以明顯降低紫杉醇的副作用，林國鐘和他的研發團隊也在探索這顆藥和乳癌的關聯性, 這在女性的癌症死亡率上排行領先。如您所見, 帶領一間決定擊敗癌症的生物製藥公司遠比這份工作更令人開心。
“We’ll be giving this company to the young people to actively get involved and to help to participate,” Ko-Chung continues. “I don’t want to say this is a legacy but it is such an important job, don’t you think?” Indeed, we do.
林國鐘持續說：「我們會讓這間公司給年輕人積極參與的機會，並扮演協助者的角色。」 “我不想被當成這僅是遺留下來的資產, 這一件很重要的工作，你不覺得嗎？”事實上，我們確實這樣做。
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