Kringle Pharma（4884 TSE Growth）
＋English Summary on This Page
Biotech venture developing and productizing medicines for intractable diseases
Developer of Medicines for Intractable Diseases
Kringle Pharma is a biotech venture conducting research and development of medicines for intractable diseases. Intractable diseases refer to diseases that have a low number of cases with unknown etiology and no established treatment method, and those that would interfere with the daily life of the patients over the long term.
The company’s drug discovery seeds, which are the base of medicine development, are recombinant human HGF (hepatocyte growth factor) proteins. HGF was first discovered in Japan as a factor that facilitates the proliferation of liver cells, and later research reported cases that it affects functions of tissues such as cytoprotective actions in addition to cell proliferation. In addition, there are reports that it has therapeutic effects on cells of the kidneys, lungs, skin, etc. in addition to liver cells.
New Medicine under Development
There is currently no medicine that has been productized and four medicines have progressed to clinical trials. The target disease of the four medicines are the (1) acute phase of spinal cord injuries, (2) amyotrophic lateral sclerosis (ALS), (3) vocal fold scars, and (4) acute kidney injuries. As for medicines that are in the basic research stage, there are medicines for eye disease and medicines for other nervous system diseases.
Business Performance
The company discloses its business performance since the fiscal year ended September 2015. No net sales were recoded from the fiscal year ended September 2015 to the fiscal year ended September 2019. In the fiscal year ended September 2020, the company received two upfront payments and net sales were 467 million yen. In addition, other than the fiscal year ended September 2016, the company has been recording an ordinary loss.
For the fiscal year ending September 2021, the company expects net sales of 206 million yen and ordinary loss of 1,105 million yen. As for net sales, it expects sales from supplying active pharmaceutical ingredients for recombinant human HGF proteins to Claris Biotherapeutics and technology access fees from Claris Biotherapeutics for clinical trials that Claris Biotherapeutics conducts. In order to double research and development expenses, the company expects to largely increase its ordinary loss from 116 million yen in the fiscal year ended September 2020.
Developer of Medicines for Intractable Diseases
Kringle Pharma is a biotech venture conducting research and development of medicines for intractable diseases. Intractable diseases refer to diseases that have a low number of cases with unknown etiology and no established treatment method, and those that would interfere with the daily life of the patients over the long term.
The company’s drug discovery seeds, which are the base of medicine development, are recombinant human HGF (hepatocyte growth factor) proteins. HGF was first discovered in Japan as a factor that facilitates the proliferation of liver cells, and later research reported cases that it affects functions of tissues such as cytoprotective actions in addition to cell proliferation. In addition, there are reports that it has therapeutic effects on cells of the kidneys, lungs, skin, etc. in addition to liver cells.
New Medicine under Development
There is currently no medicine that has been productized and four medicines have progressed to clinical trials. The target disease of the four medicines are the (1) acute phase of spinal cord injuries, (2) amyotrophic lateral sclerosis (ALS), (3) vocal fold scars, and (4) acute kidney injuries. As for medicines that are in the basic research stage, there are medicines for eye disease and medicines for other nervous system diseases.
Business Performance
The company discloses its business performance since the fiscal year ended September 2015. No net sales were recoded from the fiscal year ended September 2015 to the fiscal year ended September 2019. In the fiscal year ended September 2020, the company received two upfront payments and net sales were 467 million yen. In addition, other than the fiscal year ended September 2016, the company has been recording an ordinary loss.
For the fiscal year ending September 2021, the company expects net sales of 206 million yen and ordinary loss of 1,105 million yen. As for net sales, it expects sales from supplying active pharmaceutical ingredients for recombinant human HGF proteins to Claris Biotherapeutics and technology access fees from Claris Biotherapeutics for clinical trials that Claris Biotherapeutics conducts. In order to double research and development expenses, the company expects to largely increase its ordinary loss from 116 million yen in the fiscal year ended September 2020.
概要（レポートから抜粋）
クリングルファーマ（以下、同社）は、難治性疾患に対する治療薬の研究開発を手掛ける大阪大学発のバイオベンチャーである。難治性疾患とは、症例数が少なく、原因不明で治療法が確立しておらず、患者の生活面への長期にわたる支障がある疾患を指している。治療薬の研究開発において、臨床試験まで進んでいるのは4件あり、製品化された薬剤はない。
同社の開発医薬品の元である創薬シーズは、組換えヒトHGF注1タンパク質である。HGFは当初、肝細胞の増殖を促進する因子として日本で発見されたが、その後の研究により、細胞増殖以外にも細胞保護等の組織の機能に作用する事例も報告されてきた。加えて対象となる細胞も肝臓だけでなく、腎臓、肺、皮膚等に対しても治療効果のあることも報告されている。
HGFは発見から長い年月が経過しているため、物質特許は既に失効している。同社は、組換えヒトHGFタンパク質を医薬品の製剤として設計・開発する過程で、製剤組成では2種類、用途特許でも1種類、都合3種類の特許を取得している（図表1）。
製剤組成では「HGF製剤」と「神経疾患の治療に適したHGF製剤」で、日本、米国等で特許を取得している。前者は長期的な保存に適するための安定性を目的とした凍結製剤である。後者は長期的な安定性に加え、神経疾患の治療に適用できるように組成を改良した凍結乾燥製剤である。用途特許はHGF製剤を脊髄損傷急性期に用いる特許である。
同社の開発医薬品の元である創薬シーズは、組換えヒトHGF注1タンパク質である。HGFは当初、肝細胞の増殖を促進する因子として日本で発見されたが、その後の研究により、細胞増殖以外にも細胞保護等の組織の機能に作用する事例も報告されてきた。加えて対象となる細胞も肝臓だけでなく、腎臓、肺、皮膚等に対しても治療効果のあることも報告されている。
HGFは発見から長い年月が経過しているため、物質特許は既に失効している。同社は、組換えヒトHGFタンパク質を医薬品の製剤として設計・開発する過程で、製剤組成では2種類、用途特許でも1種類、都合3種類の特許を取得している（図表1）。
製剤組成では「HGF製剤」と「神経疾患の治療に適したHGF製剤」で、日本、米国等で特許を取得している。前者は長期的な保存に適するための安定性を目的とした凍結製剤である。後者は長期的な安定性に加え、神経疾患の治療に適用できるように組成を改良した凍結乾燥製剤である。用途特許はHGF製剤を脊髄損傷急性期に用いる特許である。
