Hu­mani­gen, once led by the im­pris­oned Mar­tin Shkre­li, is find­ing it­self in a sim­i­lar­ly con­fined space.

On Thurs­day, the com­pa­ny said the FDA de­clined its re­quest for an EUA for its lead drug lenzilum­ab to treat new­ly hos­pi­tal­ized Covid-19 pa­tients, send­ing shares of the com­pa­ny’s stock $HGEN down by more than 50% pre-mar­ket.

Why ex­act­ly the FDA re­ject­ed the EUA re­mains large­ly un­known as the com­pa­ny of­fered few specifics.

“In its let­ter, FDA stat­ed that it was un­able to con­clude that the known and po­ten­tial ben­e­fits of lenzilum­ab out­weigh the known and po­ten­tial risks of its use as a treat­ment for COVID-19,” the com­pa­ny said in a state­ment, while not­ing that it can sub­mit ad­di­tion­al da­ta as it be­comes avail­able.

The NIH is still test­ing lenzilum­ab in com­bi­na­tion with Gilead’s remde­sivir in its AC­TIV-5/BET-B study, which Hu­mani­gen said could pro­vide fur­ther da­ta to sup­port a new EUA re­quest.

Those re­sults will be cru­cial for the com­pa­ny, which said that as of June 30, it had cash and cash equiv­a­lents of about $121 mil­lion. The long-suf­fer­ing biotech was a pen­ny stock play­er in the OTC mar­ket when Covid-19 came along and lift­ed its prospects, fu­el­ing a steep rise in share price and re­viv­ing its prospects with in­vestors. Hu­mani­gen burned through more than $100 mil­lion in cash in the first half of 2021, com­pared to about $20 mil­lion in the first half of 2020.

Hu­mani­gen CEO Cameron Dur­rant, who took the reins of then-bank­rupt Hu­mani­gen — known pre­vi­ous­ly as Kalo­Bios — from Shkre­li, said in a state­ment, “We be­lieve the on­go­ing AC­TIV-5/BET-B tri­al, which has been ad­vanced to en­roll up to 500 pa­tients, may pro­vide ad­di­tion­al safe­ty and ef­fi­ca­cy da­ta suf­fi­cient to sup­port our ef­forts to ob­tain an EUA to treat hos­pi­tal­ized COVID-19 pa­tients.”

The re­jec­tion is an about-face for the com­pa­ny, which in March tout­ed pos­i­tive re­sults from a 520-per­son, place­bo-con­trolled study that found lenzilum­ab im­proved the odds of ven­ti­la­tor-free sur­vival by 54%.

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting: